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Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Get Free Report) CFO Howard Horn sold 7,066 shares of the company’s stock in a transaction dated Thursday, October 15th. The stock was sold at an average price of $15.27, for a total value of $107,897.82. Following the completion of the transaction, the chief financial officer owned 89,229 shares of the company’s stock, valued at $1,362,526.83. The trade was a 7.34% decrease in their ownership of the stock. The transaction was disclosed in a filing with the SEC, which is available at the SEC website. The sale was made to cover tax withholding obligations related to the vesting of equity awards.

Ultragenyx Pharmaceutical Trading Up 3.6% Shares of Ultragenyx Pharmaceutical stock opened at $15.07 on Tuesday. Ultragenyx Pharmaceutical Inc. has a 12 month low of $12.73 and a 12 month high of $39.89. The business’s 50-day moving average price is $18.59 and its 200-day moving average price is $23.54.

Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) last released its earnings results on Tuesday, August 4th. The biopharmaceutical company reported ($0.90) earnings per share for the quarter, topping analysts’ consensus estimates of ($1.22) by $0.32. The firm had revenue of $214.00 million during the quarter, compared to the consensus estimate of $183.08 million. The business’s quarterly revenue was up 28.5% compared to the same quarter last year. During the same quarter in the previous year, the firm earned ($1.17) EPS. As a group, sell-side analysts anticipate that Ultragenyx Pharmaceutical Inc. will post -3.97 earnings per share for the current fiscal year.

Institutional Investors Weigh In On Ultragenyx Pharmaceutical Several institutional investors have recently bought and sold shares of RARE. Versant Capital Management Inc increased its stake in Ultragenyx Pharmaceutical by 662.9% in the 2nd quarter. Versant Capital Management Inc now owns 1,152 shares of the biopharmaceutical company’s stock worth $38,000 after purchasing an additional 1,001 shares during the period. Quantbot Technologies LP bought a new stake in Ultragenyx Pharmaceutical in the second quarter valued at approximately $48,000. Harbor Investment Advisory LLC purchased a new stake in shares of Ultragenyx Pharmaceutical during the second quarter valued at approximately $52,000. Parallel Advisors LLC boosted its stake in shares of Ultragenyx Pharmaceutical by 778.4% during the first quarter. Parallel Advisors LLC now owns 1,625 shares of the biopharmaceutical company’s stock valued at $34,000 after purchasing an additional 1,440 shares during the period. Finally, Leonteq Securities AG boosted its stake in shares of Ultragenyx Pharmaceutical by 288.5% during the first quarter. Leonteq Securities AG now owns 1,795 shares of the biopharmaceutical company’s stock valued at $38,000 after purchasing an additional 1,333 shares during the period. 97.67% of the stock is currently owned by hedge funds and other institutional investors. Analysts Set New Price Targets RARE has been the topic of several research reports. JPMorgan Chase & Co. downgraded shares of Ultragenyx Pharmaceutical from an “overweight” rating to a “neutral” rating and decreased their price target for the stock from $80.00 to $36.00 in a research note on Thursday, September 3rd. Jefferies Financial Group cut their price objective on shares of Ultragenyx Pharmaceutical from $77.00 to $28.00 and set a “buy” rating for the company in a research note on Thursday, September 3rd. The Goldman Sachs Group reduced their target price on shares of Ultragenyx Pharmaceutical from $28.00 to $19.00 and set a “neutral” rating on the stock in a report on Friday, September 4th. Robert W. Baird cut shares of Ultragenyx Pharmaceutical from an “outperform” rating to a “neutral” rating and decreased their target price for the stock from $40.00 to $16.00 in a research report on Thursday, September 3rd. Finally, Guggenheim decreased their target price on shares of Ultragenyx Pharmaceutical from $43.00 to $35.00 and set a “buy” rating on the stock in a research report on Tuesday, August 4th. One equities research analyst has rated the stock with a Strong Buy rating, nine have assigned a Buy rating, eleven have given a Hold rating and one has assigned a Sell rating to the company’s stock. Based on data from MarketBeat.com, the company has a consensus rating of “Hold” and a consensus target price of $33.65.

View Our Latest Stock Report on Ultragenyx Pharmaceutical

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Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic diseases. The company targets conditions with limited or no treatment options, including metabolic disorders, skeletal diseases and other inherited conditions. Its work includes the development of medicines, biologic therapies and gene therapy candidates intended to address the underlying causes or significant symptoms of these disorders.

Ultragenyx’s commercial portfolio includes Crysvita (burosumab), a treatment for X-linked hypophosphatemia and related disorders; Dojolvi (triheptanoin), a medical food used to manage long-chain fatty acid oxidation disorders; and Mepsevii (vestronidase alfa), an enzyme replacement therapy for mucopolysaccharidosis VII.

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Key Takeaways Ultragenyx generated $350 million in first-half 2026 revenues, up 14%, led by Crysvita's $249 million.Evkeeza revenues surged 56% to $39 million, supported by new-country launches and early access.Genglycos and Fayuvi offer Ultragenyx opportunities to expand its revenue base and drive future growth. Ultragenyx Pharmaceutical (RARE - Free Report) is broadening its commercial portfolio of treatments for rare and ultra-rare diseases. The company generated $350 million in total revenues in the first half of 2026, up 14% year over year, supported by higher revenues across its marketed products. With two additional gene therapies now approved in the United States, Ultragenyx has more opportunities to expand its revenue base and reduce its dependence on established products.

Crysvita (burosumab) remains the company’s primary revenue driver. Approved for X-linked hypophosphatemia and tumor-induced osteomalacia, the drug generated $249 million in first-half 2026 revenues, up 11% year over year, accounting for roughly 71% of total revenue. Dojolvi (triheptanoin), approved for long-chain fatty acid oxidation disorders, generated $45 million, up 13%. Meanwhile, Mepsevii (vestronidase alfa), approved for mucopolysaccharidosis VII, generated $17 million, broadly unchanged from the prior-year period. These established therapies provide a commercial foundation, although Crysvita’s large contribution leaves overall growth exposed to its performance.

Evkeeza (evinacumab) provides another growth avenue. Ultragenyx commercializes the Regeneron-partnered drug outside the United States for homozygous familial hypercholesterolemia (HoFH). Evkeeza generated $39 million in first-half revenues, up 56% year over year, supported by new-country launches and early access. Its international expansion complements the company’s established rare-disease therapies and provides RARE with an incremental revenue stream.

Ultragenyx has also expanded its portfolio through two recent FDA approvals. Genglycos (pariglasgene brecaparvovec) received accelerated approval in August 2026 for adults and children aged eight years and older with glycogen storage disease type Ia. In September, Fayuvi (rebisufligene etisparvovec) received full approval as the first treatment for Sanfilippo syndrome type A in eligible pediatric patients. Ultragenyx also agreed to sell the priority review voucher awarded with Genglycos’ approval for $210 million. The transaction is expected to provide additional non-dilutive capital upon closing, although 20% of gross proceeds is payable to the NIH under an existing license agreement.

Overall, the expanding portfolio could drive RARE’s next growth phase, but the commercial impact of the new gene therapies will depend on treatment-center access, reimbursement and patient uptake. Ultragenyx’s 2026 revenue guidance of $730-$760 million excludes potential revenues from new product launches, making their contribution an additional opportunity rather than a guaranteed source of growth.

RARE Faces Competition Across Rare-Disease MarketsUltragenyx’s competitive landscape includes established biopharmaceutical company Amgen (AMGN - Free Report) and rare-disease-focused biotechnology company Denali Therapeutics (DNLI - Free Report) .

Amgen markets Repatha (evolocumab), an LDL-cholesterol-lowering treatment approved for adults and pediatric patients aged 10 years and older with HoFH. Repatha’s use in this patient population places it in the same treatment market as Evkeeza. Repatha generated $1.83 billion in first-half 2026 sales, up 35% year over year across all indications. Although Repatha and Evkeeza work through different mechanisms and can serve distinct roles in treatment, Amgen’s strong commercial presence creates a competitive consideration for Evkeeza.

Denali is advancing zafinofusp alfa (DNL126), an ETV-enabled investigational enzyme replacement therapy for Sanfilippo syndrome type A. Early-stage data showed substantial reductions in disease-related biomarkers upon treatment with the candidate. Based on such results, DNLI is preparing to initiate a global phase III confirmatory study of zafinofusp alfa for Sanfilippo syndrome type A. Denali expects a biologics license application submission and potential accelerated approval for zafinofusp alfa for Sanfilippo syndrome type A in 2027. Successful clinical progress could eventually introduce a competing treatment option for Fayuvi.

RARE’s Stock Price, Valuation & Estimate MovementsYear to date, RARE shares have lost 35.1% against the industry’s 1.8% growth. Ultragenyx has also underperformed the sector and the S&P 500 during the same time frame, as seen in the chart below.

RARE Stock Underperforms the Industry, Sector & the S&P 500Image Source: Zacks Investment Research

Ultragenyx is trading at a discount to the industry, as seen in the chart below. Going by the price/sales ratio, the company’s shares currently trade at 2.12, which is less than 2.39 for the industry. The stock is trading significantly below its five-year mean of 7.74.

RARE Stock ValuationImage Source: Zacks Investment Research

Loss estimates for 2026 have narrowed from $4.08 to $3.95 per share over the past 60 days. During the same time frame, RARE’s 2027 loss per share estimates have narrowed from 75 cents to 13 cents.

RARE Estimate MovementImage Source: Zacks Investment Research

Ultragenyx currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Atara Biotherapeutics (NASDAQ:ATRA – Get Free Report) and Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) are both small-cap healthcare companies, but which is the superior stock? We will compare the two companies based on the strength of their dividends, valuation, analyst recommendations, risk, institutional ownership, earnings and profitability.

Profitability This table compares Atara Biotherapeutics and Ultragenyx Pharmaceutical’s net margins, return on equity and return on assets.

Net Margins Return on Equity Return on Assets Atara Biotherapeutics -269.42% N/A -73.01% Ultragenyx Pharmaceutical -81.79% N/A -44.40% Insider and Institutional Ownership 70.9% of Atara Biotherapeutics shares are owned by institutional investors. Comparatively, 97.7% of Ultragenyx Pharmaceutical shares are owned by institutional investors. 41.2% of Atara Biotherapeutics shares are owned by company insiders. Comparatively, 5.2% of Ultragenyx Pharmaceutical shares are owned by company insiders. Strong institutional ownership is an indication that endowments, large money managers and hedge funds believe a stock is poised for long-term growth.

Volatility and Risk Atara Biotherapeutics has a beta of 0.21, indicating that its stock price is 79% less volatile than the S&P 500. Comparatively, Ultragenyx Pharmaceutical has a beta of 0.35, indicating that its stock price is 65% less volatile than the S&P 500. Analyst Ratings This is a breakdown of current ratings and recommmendations for Atara Biotherapeutics and Ultragenyx Pharmaceutical, as reported by MarketBeat.com.

Sell Ratings Hold Ratings Buy Ratings Strong Buy Ratings Rating Score Atara Biotherapeutics 1 1 1 1 2.50 Ultragenyx Pharmaceutical 1 10 9 1 2.48 Atara Biotherapeutics presently has a consensus price target of $9.50, suggesting a potential upside of 0.21%. Ultragenyx Pharmaceutical has a consensus price target of $34.58, suggesting a potential upside of 123.38%. Given Ultragenyx Pharmaceutical’s higher probable upside, analysts clearly believe Ultragenyx Pharmaceutical is more favorable than Atara Biotherapeutics.

Valuation and Earnings This table compares Atara Biotherapeutics and Ultragenyx Pharmaceutical”s gross revenue, earnings per share and valuation.

Gross Revenue Price/Sales Ratio Net Income Earnings Per Share Price/Earnings Ratio Atara Biotherapeutics $120.77 million 0.79 $32.69 million ($1.18) -8.03 Ultragenyx Pharmaceutical $717.00 million 2.13 -$575.00 million ($5.84) -2.65 Atara Biotherapeutics has higher earnings, but lower revenue than Ultragenyx Pharmaceutical. Atara Biotherapeutics is trading at a lower price-to-earnings ratio than Ultragenyx Pharmaceutical, indicating that it is currently the more affordable of the two stocks.

Summary Ultragenyx Pharmaceutical beats Atara Biotherapeutics on 9 of the 13 factors compared between the two stocks.

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Atara Biotherapeutics, Inc. engages in the development of transformative therapies for patients with solid tumors, hematologic cancers, and autoimmune diseases in the United States and the United Kingdom. Its lead product includes Tab-cel (tabelecleucel), a T-cell immunotherapy program that is in Phase 3 clinical trials for the treatment of epstein-barr virus (EBV) driven post-transplant lymphoproliferative disease, as well as nasopharyngeal carcinoma. Its CAR T immunotherapy pipeline products include ATA3219, currently in Phase 1 trials, as well as ATA3431, under preclinical trials for the treatment of B-cell malignancies and autoimmune diseases; and ATA188 that is in Phase 2 clinical trials to treat multiple sclerosis. The company has research collaboration agreements with Memorial Sloan Kettering Cancer Center, and Council of the Queensland Institute of Medical Research. Atara Biotherapeutics, Inc. was incorporated in 2012 and is headquartered in Thousand Oaks, California.

About Ultragenyx Pharmaceutical (Get Free Report)

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.

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Ultragenyx Pharmaceutical (NASDAQ:RARE) shares rose over 3.2% on Wednesday morning after the biopharmaceutical company agreed to sell a Rare Pediatric Disease Priority Review Voucher for $210 million.

The voucher was awarded to Ultragenyx after the US Food and Drug Administration approved GENGLYCOS, also known as DTX401, for glycogen storage disease (GSD) type Ia.

A voucher can be redeemed by its holder to obtain priority review of a subsequent marketing application for a different product, and sponsors can sell or transfer the voucher to another company.

Ultragenyx said the transaction will provide significant non-dilutive capital to support the development of therapies for rare and ultra-rare diseases and help advance its path to profitability.

The company said GENGLYCOS itself benefited from capital generated through a previous priority review voucher sale, highlighting the role of the FDA programme in funding rare disease drug development.

"Monetizing this PRV provides significant non-dilutive capital to advance our efforts to bring forward first-ever therapies for rare and ultra-rare diseases, and supports our path to profitability," said Howard Horn, chief financial officer and executive vice president, corporate strategy.

The sale remains subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.

GENGLYCOS is designed to address the underlying cause of GSDIa, a rare genetic disorder, and was the first treatment approved for the disease that targets its underlying cause.

The FDA’s Rare Pediatric Disease Priority Review Voucher programme is intended to encourage development of drugs and biological products for certain rare childhood diseases.

Ultragenyx, which develops treatments for serious rare and ultra-rare genetic diseases, said the proceeds will strengthen its financial position as it advances its pipeline.

The company appointed Jefferies as exclusive financial adviser and Gibson, Dunn and Crutcher as legal counsel for the transaction.

The agreement gives Ultragenyx a substantial source of non-dilutive funding while allowing the company to monetize an asset awarded through the FDA’s rare disease incentive programme.

NEW YORK, Oct. 06, 2026 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE).  Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

The investigation concerns whether Ultragenyx and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. 

[Click here for information about joining the class action]

On September 2, 2026, Ultragenyx issued a press release “announc[ing] results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome.”  Per the press release, “[t]he study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI).” 

On this news, Ultragenyx’s stock price fell $11.68 per share, or 44.03%, to close at $14.85 per share on September 3, 2026.

Pomerantz LLP, with offices in New York, Chicago, Los Angeles, London, Paris, and Tel Aviv, is acknowledged as one of the premier firms in the areas of corporate, securities, and antitrust class litigation. Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions. Today, more than 85 years later, Pomerantz continues in the tradition he established, fighting for the rights of the victims of securities fraud, breaches of fiduciary duty, and corporate misconduct. The Firm has recovered numerous multimillion-dollar damages awards on behalf of class members. See www.pomlaw.com.

Attorney advertising. Prior results do not guarantee similar outcomes.  

CONTACT:
Danielle Peyton
Pomerantz LLP
[email protected]
646-581-9980 ext. 7980

, /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") (NASDAQ: RARE).  Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

The investigation concerns whether Ultragenyx and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. 

[Click here for information about joining the class action]

On September 2, 2026, Ultragenyx issued a press release "announc[ing] results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome."  Per the press release, "[t]he study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI)." 

On this news, Ultragenyx's stock price fell $11.68 per share, or 44.03%, to close at $14.85 per share on September 3, 2026.

Pomerantz LLP, with offices in New York, Chicago, Los Angeles, London, Paris, and Tel Aviv, is acknowledged as one of the premier firms in the areas of corporate, securities, and antitrust class litigation. Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions. Today, more than 85 years later, Pomerantz continues in the tradition he established, fighting for the rights of the victims of securities fraud, breaches of fiduciary duty, and corporate misconduct. The Firm has recovered numerous multimillion-dollar damages awards on behalf of class members. See www.pomlaw.com.

Attorney advertising. Prior results do not guarantee similar outcomes. 

CONTACT:
Danielle Peyton
Pomerantz LLP
[email protected]
646-581-9980 ext. 7980

SOURCE Pomerantz LLP

Shares of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Get Free Report) have received an average rating of “Hold” from the twenty-one ratings firms that are currently covering the firm, MarketBeat.com reports. One investment analyst has rated the stock with a sell rating, ten have given a hold rating, nine have assigned a buy rating and one has assigned a strong buy rating to the company. The average 12 month target price among analysts that have covered the stock in the last year is $34.58.

Several equities research analysts recently commented on RARE shares. Wall Street Zen raised Ultragenyx Pharmaceutical from a “sell” rating to a “hold” rating in a research note on Saturday, August 8th. William Blair lowered shares of Ultragenyx Pharmaceutical from an “outperform” rating to a “market perform” rating in a research report on Thursday, September 3rd. Citigroup lifted their target price on shares of Ultragenyx Pharmaceutical from $31.00 to $32.00 and gave the company a “buy” rating in a research note on Friday, September 18th. Royal Bank Of Canada downgraded shares of Ultragenyx Pharmaceutical from a “moderate buy” rating to a “hold” rating in a research report on Thursday, September 3rd. Finally, Cantor Fitzgerald increased their price target on shares of Ultragenyx Pharmaceutical from $33.00 to $39.00 and gave the stock an “overweight” rating in a research note on Friday, September 18th.

View Our Latest Report on RARE

Ultragenyx Pharmaceutical Trading Down 1.8% Shares of NASDAQ:RARE opened at $14.50 on Friday. The firm has a market cap of $1.43 billion, a price-to-earnings ratio of -2.48 and a beta of 0.31. Ultragenyx Pharmaceutical has a 1 year low of $12.73 and a 1 year high of $39.89. The business has a fifty day simple moving average of $22.24 and a 200 day simple moving average of $24.18. Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) last issued its earnings results on Tuesday, August 4th. The biopharmaceutical company reported ($0.90) EPS for the quarter, beating analysts’ consensus estimates of ($1.22) by $0.32. Ultragenyx Pharmaceutical had a negative return on equity of 1,024.42% and a negative net margin of 81.79%.The firm had revenue of $214.00 million for the quarter, compared to the consensus estimate of $183.08 million. During the same quarter in the previous year, the company posted ($1.17) earnings per share. The business’s quarterly revenue was up 28.5% compared to the same quarter last year. On average, sell-side analysts forecast that Ultragenyx Pharmaceutical will post -4.29 EPS for the current year.

Hedge Funds Weigh In On Ultragenyx Pharmaceutical Several institutional investors have recently bought and sold shares of the stock. BlackRock Inc. bought a new position in shares of Ultragenyx Pharmaceutical in the second quarter valued at approximately $355,746,000. Connor Clark & Lunn Investment Management Ltd. acquired a new stake in shares of Ultragenyx Pharmaceutical in the second quarter worth $6,397,000. Deutsche Bank AG bought a new stake in shares of Ultragenyx Pharmaceutical during the 2nd quarter worth $5,503,000. Corient Private Wealth LP bought a new stake in shares of Ultragenyx Pharmaceutical during the 2nd quarter worth $4,644,000. Finally, Blue Owl Capital Holdings LP acquired a new position in Ultragenyx Pharmaceutical in the 2nd quarter valued at $6,098,000. 97.67% of the stock is currently owned by institutional investors.

Ultragenyx Pharmaceutical Company Profile (Get Free Report)

Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing treatments for rare and ultra-rare diseases. The company’s work includes therapies for genetic, metabolic and skeletal disorders, with an emphasis on conditions that have limited or no effective treatment options.

Ultragenyx’s marketed products include Crysvita (burosumab), used to treat certain forms of X-linked hypophosphatemia and tumor-induced osteomalacia; Dojolvi (triheptanoin), a medical food for long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab), a treatment for homozygous familial hypercholesterolemia.

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Selected retained-core intervals contain rare earth elements, including neodymium, praseodymium, terbium and dysprosium (magnet rare earths), together with uranium, vanadium and phosphate mineralization. 0.63 m @ 3,570 ppm TREO (0.357%) and 607 ppm MREO, the highest TREO value reported from the selected intervals in this resampling program. Additional selected intervals returned 1.53 m @ 2,267 ppm TREO and 329 ppm MREO and 0.50 m @ 2,532 ppm TREO and 414 ppm MREO, with reported Nd-Pr-Tb-Dy enrichment within the sampled U-V-phosphate horizon. Positioned as a potential Western Hemisphere critical minerals project and within the scope of the recent U.S. – Colombia Critical Minerals Cooperation Agreements , /PRNewswire/ -- Jaguar Uranium Corp. (NYSE American: JAGU) ("Jaguar" or the "Company") today announced assay results from its historical drill-core resampling program at the Berlin Uranium–Rare Earth Project in the Department of Caldas, Colombia. Results indicate a polymetallic system that includes rare earth elements with uranium, vanadium and phosphate mineralization in the selected intervals tested, Selected intervals returned up to 3,570 ppm TREO and 607 ppm MREO. The tested core was drilled between 2010 and 2012, and the resampling program was first announced on March 17, 20261.

Figure 1. Plan view location of the selected drill holes for the current resampling program. Location of cross-sections 1 and 2.

Figure 2. Core shack and boxes storing historical drill cores.

Figure 3. Cross-Sections showing the location of drill hole samples selected for reanalysis. Historical drill hole intercepts are shown in green on the left, and samples included in the current resampling program are shown in blue on the right. See Figure 1 for the cross-section location in plan view.

Figure 4. Oblique view of the 3D geological model of the mineralized domain. Historical drill hole intercepts are shown in green on the left, and samples included in the current resampling program are shown in blue on the right.

Table 2. Reported intervals and lithogeochemical results for TREO, MREO, U3O8, V2O5 and P2O5 obtained from the current resampling program.

Table 2. Reported intervals and lithogeochemical results for TREO, MREO, U3O8, V2O5 and P2O5 obtained from the current resampling program.

Table 2. Reported intervals and lithogeochemical results for TREO, MREO, U3O8, V2O5 and P2O5 obtained from the current resampling program. Rare earth elements (REE) comprise 17 metallic elements, 15 of which are reported. In this press release, analytical results are expressed as Total Rare Earth Oxides (TREO). Praseodymium, neodymium, terbium and dysprosium are reported collectively as Magnet Rare Earth Oxides (MREO) because these elements are used in permanent-magnet applications2.

The Company plans to evaluate whether the REO (Rare Earth Oxides) results warrant incorporation into future geological modelling and mineral resource work. Any such work would require additional drilling, continuity assessment, analytical review and other supporting technical studies.

These results constitute exploration results only and do not represent a mineral resource or mineral reserve. The economic viability of the reported mineralization has not been established.

Selected intervals from the resampling program returned the following rare earth element results:

DDB-041: 0.63 m grading 3,570 ppm TREO and 607 ppm MREO (from 170.57 m to 171.20 m). DDB-003: 1.53 m grading 2,267 ppm TREO and 329 ppm MREO (from 80.77 m to 82.30 m). DDB-015: 1.00 m grading 2,176 ppm TREO and 349 ppm MREO (from 162.86 m to 163.86 m). DDB-068: 0.50 m grading 2,532 ppm TREO and 414 ppm MREO (from 435.86 m to 436.36 m). The intervals were selected for the purposes described under "Resampling Results and Analytical Procedures" and are not necessarily representative of the mineralized horizon as a whole.

"The initial results indicate that rare earth elements occur with uranium, phosphate and vanadium mineralization in the selected Berlin core intervals. We plan to use these results, together with trenching, geophysics and drilling, to refine the geological model and evaluate the continuity of mineralization. This multi-commodity profile directly aligns with the accelerating strategic cooperation between Colombia and the United States on secure critical-mineral supply chains and nuclear energy," said Steven Gold, President & Chief Executive Officer.

___________________________
1 Caution: The company emphasizes that the data presented in this press release constitute "exploration results" under SEC Regulation S-K 1300. This technical information does not represent, nor does it assert, the discovery of a mineral resource or a mineral reserve. The economic viability or metallurgical recovery of these elements has not been established. The results identify additional elements of potential economic interest that are currently being evaluated.

2 TREO values were calculated by converting the reported concentrations of the analyzed rare earth elements to their stoichiometric oxide equivalents and summing those oxide values. MREO comprises the oxide equivalents of neodymium, praseodymium, terbium and dysprosium. The calculation does not represent a metallurgical recovery or a saleable product specification. Details in Table 1.

The Barranquilla Accords – U.S.-Colombia Framework on Critical Minerals

On September 8, 2026, the Barranquilla Accords were established as a U.S.-Colombia framework to mark a significant strengthening of U.S.–Colombia cooperation in critical minerals, rare earth elements and civil nuclear energy. The agreements establish a framework to advance secure supply chains, geological knowledge, investment and development of strategic mineral resources. With uranium, rare earth elements, vanadium and phosphate mineralization, Jaguar's Berlin Project is well positioned within this emerging strategic framework and Colombia's growing role in secure critical-mineral and nuclear supply chains.

Background and Historical Data

Berlin is an exploration-stage polymetallic project with historically reported uranium mineralization and associated rare earth elements and other commodities, including vanadium, phosphate, nickel, molybdenum, rhenium, yttrium and neodymium. More than 20,000 metres of historical drilling have been reported for the project.

The drill core subject to this resampling program was obtained during two historical exploration campaigns conducted by U3O8 Corp. The first campaign took place in 2010 and 2011, and the second campaign was conducted in 2012.

Storage Location: The drill core has been maintained since 2015 in a secure, locked core storage facility (Figure 2). Originally established by U3O8 Corp., the facility was acquired by Jaguar in 2024. It is situated directly within the project area on mining concession 664-17, where access remains strictly limited to authorized personnel to ensure continuous sample integrity. Physical Integrity: The sampled intervals showed no signs of degradation or mineral oxidation. Resampling Results and Analytical Procedures

The program selected and analyzed intervals from retained historical core to test REE occurrence and grade variation across the syncline, including samples from both flanks and the hinge and from positions above and below the mineralized horizon (Figure 3 and Figure 4). The Company selected the intervals from the available retained core; the tested samples do not constitute a systematic resampling of all historical mineralized intervals. The current results were not designed to verify any historical estimate. REE concentrations are reported as Total Rare Earth Oxides (TREO) and Magnet Rare Earth Oxides (MREO), and reported intervals are core lengths; true widths have not been determined3.

____________________________
3 The Total Rare Earth Oxide (TREO) calculation strictly corresponds to the stoichiometric sum of the oxides. The conversion factors used are: La2O3 (1.1728), CeO2 (1.2284), Pr6O11 (1.2082), Nd2O3 (1.1664), Sm2O3 (1.1596), Eu2O3 (1.1579), Gd2O3 (1.1526), Tb4O7 (1.1762), Dy2O3 (1.1477), Ho2O3 (1.1455), Er2O3 (1.1435), Tm2O3 (1.1421), Yb2O3 (1.1387), Lu2O3 (1.1371), and Y2O3 (1.2699). LREO represents Light Rare Earth Oxides, the sum of the oxide equivalents of Ce, La, Nd, Pr & Sm; HREO represents Heavy Rare Earth Oxides, the sum of the oxide equivalents of Eu, Gd, Tb, Dy, Ho, Er, Tm, Yb, Lu, & Y, and MREO represents Magnetic Rare Earth Oxides the sum of the oxide equivalents of Pr, Nd, Dy & Tb. Other conversion factors used are U3O8 (1.1792), P2O5 (2.2914) and V2O5 (1.7852).

Sampling and Analytical Procedures

Samples from 13 selected historical drill core intervals were collected, labelled, and submitted for laboratory analysis using procedures intended to preserve sample identity and chain of custody (Table 1). Sample lengths were selected to match the original sample lengths collected by U3O8 Corp. The batch consisted of 150 samples, comprising 136 drill core samples (108 from direct core sampling and 28 pulp samples from previous analyses) and 14 QA/QC inserts. The core samples consisted of quarter-cores cut from the remaining half-cores sampled at average 1-metre lengths (maximum 2.98 m and minimum 0.20 m). Reported intervals are core lengths. True widths have not been determined from the current resampling program. Note that a material proportion of these intervals came from DDB-035 and DDB-077, which were sampled for historical-geochemical verification only and are not representative of true thickness. Full results are provided in Table 2.

Sample preparation was performed at SGS Colombia S.A.S. in Medellín, Colombia (ISO 9001 accredited for sample preparation and management), and chemical analysis was conducted at SGS Callao, Peru (ISO/IEC 17025 for testing and calibration). Whole-rock geochemical analyses were conducted utilizing a dual-method approach to guarantee total element recovery. Samples underwent a four-acid digestion followed by ICP-MS analysis (SGS method ICM40B) for standard trace element characterization. For the accurate quantification of REE in resistate mineral phases, a sodium peroxide fusion with an ICP-MS finish (SGS method ICM90A) was systematically applied. This approach also accounted for any over-limit analyses encountered. Uranium was reported using the ICM40B method to account for over-limit results. Methods and detection limits are indicated on Table 2.

Quality Assurance and Quality Control (QA/QC)

The resampling program was executed under strict chain-of-custody protocols from the storage facilities in Colombia to the independent laboratories. As part of the QA/QC program, coarse blank material and certified reference materials (CRM) supplied by OREAS (ORE Research & Exploration Pty Ltd., Australia), an ISO 17034-accredited reference material producer, were inserted. OREAS 474 was used for rare earth control, featuring certified values for TREO and individual rare earth elements, as well as certified U and Th contents. For uranium control, OREAS 121 (~215 ppm U) was used as a low-grade standard and OREAS 124 (~1,845 ppm U) as a high-grade standard.

Of the 150 submitted samples, 14 were QA/QC inserts (9.33% of the total batch), consisting of: 7 coarse blanks (4.67%) and 7 CRMs (4.67%). The CRMs included 3 insertions of OREAS 474 (2.0%), 2 of OREAS 121 (1.33%), and 2 of OREAS 124 (1.33%). Controls were distributed throughout the sampling sequence to avoid clustering. The QP reviewed the blank and certified-reference-material results against the program's acceptance criteria and affirms that the data passed internal and external validation checks and is suitable for the purpose of this exploration disclosure.

Qualified Person Statement

Andrés Cáceres, an AIPG Certified Professional Geologist (CPG), meets the requirements of a Qualified Person under NI 43-101 and Subpart 1300 of Regulation S-K. Mr. Cáceres has reviewed the technical integrity of the data in this press release, evaluated the QA/QC results, and approves the publication of this technical disclosure. Mr. Cáceres is an independent contractor hired by Jaguar Uranium Corp. His experience includes eight years as Project Manager in Colombia with U3O8 Corp., where he led the Berlin Project; four years with the Colombian Geological Survey (SGC) as a Senior Consultant conducting research and exploration programs focused on uranium and rare earth element resources; and two years as Vice President of Exploration for Biondi Resources, leading uranium and rare earth element exploration activities in Brazil.

Andrés Cáceres, CPG, designed the resampling program, supervised data collection and interpreted the scientific and technical results. Because the program used retained historical core and historical pulps, the QP's verification was limited to the available material, storage and chain-of-custody records, resampling procedures, laboratory certificates and QA/QC results reviewed for this program. The QP was the project manager from 2007-2015.

About Jaguar Uranium Corp.

Jaguar Uranium Corp. is a South America-focused uranium exploration company advancing a portfolio of prospective brownfield projects across Colombia and Argentina. The Company's Berlin Project in Caldas Province, Colombia is a district-scale, sedimentary-hosted polymetallic system historically reported to host uranium vanadium, nickel, phosphate, molybdenum, rhenium and zinc. In Argentina, Jaguar is advancing the Laguna Salada Project in Chubut Province and the historic Huemul uranium mine in Mendoza Province. Anchored by a former producing mine, a historic uranium district and exploration projects supported by historical drilling, the Company is focused on advancing and expanding uranium opportunities across the region.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of applicable U.S. and Canadian securities laws. Forward-looking statements include, without limitation, statements regarding planned drilling, trenching and geophysical work; additional continuity testing; future geological modelling; the potential incorporation of REE data into future mineral resource work; the timing and results of future assays and technical studies; and the Company's expectations for the Berlin Project.

Forward-looking statements are based on material assumptions including the reliability of the historical records and retained core available to the Company; the accuracy of the current analytical results; the availability of financing, personnel, equipment and laboratory capacity; continued property access; receipt of required permits; and the Company's ability to complete its planned exploration and technical programs.

Forward-looking statements are subject to known and unknown risks, uncertainties and other factors that may cause actual results to differ materially. These include risks arising from incomplete or inaccurate historical records; limitations inherent in resampling retained core and historical pulps; the selective nature of the tested intervals; uncertainty regarding the continuity, geometry and true width of mineralization; analytical and QA/QC risks; the possibility that additional work will not support inclusion of REE data in a mineral resource estimate; commodity-price volatility; permitting, regulatory, operational and technical risks; and general economic, market and industry conditions.

Forward-looking statements speak only as of the date of this news release. Except as required by applicable securities laws, the Company undertakes no obligation to update or revise any forward-looking statements to reflect events or circumstances after the date of this release or to reflect the occurrence of unanticipated events.

Table 1. Sampled drillhole details.

Hole ID

Easting (m)

Northing (m)

Elevation (m)

Dip

Azimuth

EOH (m)

DDB-003

4782499.20

2174992.50

898.73

79

251

132.58

DDB-011

4781845.05

2176282.60

704.85

68

263

165.53

DDB-015

4781665.65

2176604.99

688.49

90

306

193.97

DDB-025

4781783.68

2176410.70

724.52

90

302

207.26

DDB-029

4781784.15

2176414.04

723.14

54

68

304.80

DDB-035

4782400.74

2175501.94

984.43

89

183

355.09

DDB-039

4782016.19

2176324.04

829.15

74

253

330.70

DDB-041

4782016.29

2176324.07

829.15

87

249

196.59

DDB-049

4781868.70

2176106.93

798.95

71

83

224.63

DDB-067

4782164.16

2175623.56

879.65

90

164

201.16

DDB-068

4781634.86

2177417.55

688.49

45

178

459.72

DDB-075

4781487.50

2177102.10

703.63

74

283

332.40

DDB-077

4782308.99

2175679.69

894.99

89

46

295.65

Note: Coordinates are reported in the CTM12 coordinate system.

Notes:

Reported intervals are core lengths; true widths have not been determined. The Total Rare Earth Oxide (TREO) calculation strictly corresponds to the stoichiometric sum of the 15 reported element oxides. Conversion factors used are: La2O3 (1.1728), CeO2 (1.2284), Pr6O11 (1.2082), Nd2O3 (1.1664), Sm2O3 (1.1596), Eu2O3 (1.1579), Gd2O3 (1.1526), Tb4O7 (1.1762), Dy2O3 (1.1477), Ho2O3 (1.1455), Er2O3 (1.1435), Tm2O3 (1.1421), Yb2O3 (1.1387), Lu2O3 (1.1371), and Y2O3 (1.2699). LREO represents Light Rare Earth Oxides, the sum of the oxide equivalents of Ce, La, Nd, Pr & Sm; HREO represents Heavy Rare Earth Oxides, the sum of the oxide equivalents of Eu, Gd, Tb, Dy, Ho, Er, Tm, Yb, Lu, & Y, and MREO represents Magnetic Rare Earth Oxides the sum of the oxide equivalents of Pr, Nd, Dy & Tb. Other conversion factors used are U3O8 (1.1792), P2O5 (2.2914) and V2O5 (1.7852). Whole-rock geochemical analyses were conducted utilizing a dual-method approach to guarantee total element recovery. Samples underwent a four-acid digestion followed by ICP-MS analysis (SGS method ICM40B) for standard trace element characterization. For the accurate quantification of resistate mineral phases, a sodium peroxide fusion with an ICP-MS finish (SGS method ICM90A) was systematically applied. SOURCE Jaguar Uranium

BICESTER, United Kingdom, Sept. 22, 2026 (GLOBE NEWSWIRE) -- Broad Arrow Auctions, driven by Hagerty (NYSE: HGTY), reveals an ultra-rare 1990 MCA Centenaire, the first of just five supercars produced by the Monaco-based manufacturer, for sale at the second annual Broad Arrow Zoute Concours Auction, taking place at the Approach Golf in Knokke-Heist, Belgium on 9 October in collaboration with Zoute Grand Prix Car Week.

1990 MCA Centenaire – Estimate: €900.000 - €1.200.000

Boutique Monaco manufacturer, Monte Carlo Automotive, was founded in 1985 by former Formula 2 and CART driver, Fulvio-Maria Ballabio, who hired an extraordinary collection of Italian motorsport talent to realise his vision. The operation was led by technical director Carlo Chiti, an engineer with ties to both Scuderia Ferrari and Autodelta, along with former Alfa Romeo Sporting Director, Perluigi Corbari, Formula 1 specialist, Marcel Zunnino, and former Formula 1 driver and constructor, Guglielmo Bellasi.

Unveiled in 1990, the MCA Centenaire was the world's first road-going supercar with a carbon fibre chassis, and was the first supercar built in Monaco. Coinciding with the centenary of the Automobile Club de Monaco, it was named the Centenaire and was presented to Prince Albert II, whose support helped establish the Centenaire as the Principality's homegrown supercar.

“It is an honour to present this piece of French supercar history,” says Jean-Louis Baldanza, Consignment Consultant at Broad Arrow Auctions. “Offered from the same collection as the Lamborghini Pregunta concept car sold at our 2025 Zoute Concours Auction, this car has been exceptionally well cared for by its knowledgeable and fastidious owner and is now ready to be shown to the world.” 

Only five Centenaires were produced between 1990 and 1992, each powered by a Lamborghini-supplied V12 as used in the Countach 5000 QV. The following year, two examples were further developed for trials for the 24 Hours of Le Mans, where they reportedly reached 354 km/h on the Mulsanne Straight.

This 1990 MCA Centenaire is the very first example produced and is thus the first road-going production car fitted with a carbon fibre chassis. This example was the sole Centenaire originally finished in Ivory White and was complemented by a stunning red leather interior, a combination crafted to represent the national colours of the Principality of Monaco. It is one of the original three examples that contains the robust 5.2-litre V12 from the Countach 5000 QV, producing 455 horsepower fed through a ZF six-speed manual transmission

An accompanying letter by MCA representative, Matteo Ramello, confirms that this car was the original chassis, explaining that the car was the first example produced and was initially used for development, testing and presentation purposes. Once these responsibilities were completed, the car was redesignated EL0002 and was held in Lamborghini's factory in Sant'Agata Bolognese for approximately ten years before it was gifted to company founder Fulvio Ballabio in the early 2000s

Refinished in blue, circa 2007, the car was notably displayed at Rétromobile that year alongside the Lamborghini Pregunta concept, another unique collaboration famously offered by Broad Arrow at their inaugural 2025 Zoute Concours Auction and graciously offered by the very same seller.

This example was acquired by the consignor in France in 2014. Approximately two years later, the Centenaire's bodywork was restored, whereupon it was refinished in correct Ivory White, with the car's red leather upholstery carefully preserved.

The 1990 MCA Centenaire is accompanied by a vast history file, including MCA correspondence and press documentation, period photos of the car in the Lamborghini factory and at Rétromobile, and even a copy of the contract between Automobili Lamborghini and Fulvio Ballabio for the supply of Lamborghini's engines.

“Today’s collector demands exclusivity and this car is unrivalled in that regard,” says Gregory Tuytens, Head of Consignments for Belgium and The Netherlands at Broad Arrow Auctions. “Our 2026 Zoute Concours Auction offers a fabulous selection of classic and contemporary collector cars, curated to delight those attending the Zoute Grand Prix Speed Week. Every lot is truly remarkable, but If you want to own a supercar that nobody else has, the MCA Centenaire is it.”

Additional information on Broad Arrow’s Zoute Concours Auction is available at broadarrowauctions.com. Collectors interested in registering to bid are invited to discuss their requirements with a Broad Arrow car specialist via [email protected] or at +44-2045-920-16.

Further details and images available on request.

Media accreditation for the 2026 Zoute Concours Auction is now open.

Ends.

Editor’s Notes

Photo Credit: All images Courtesy of Broad Arrow Auctions.

About Broad Arrow Auctions

Broad Arrow Auctions, driven by Hagerty (NYSE: HGTY), is a leading global collector car auction house founded in 2021 by industry veterans. As the fastest-growing auction house in its segment, Broad Arrow connects exceptional collector cars with enthusiasts worldwide through flagship events including The Broad Arrow Quail Auction (the official auction of The Quail by The Peninsula, A Motorsports Gathering), The Amelia Auction (the official auction of The Amelia Concours), The Porsche Auction in collaboration with Air | Water by Luftgekühlt, the Las Vegas Auction in partnership with Concours at Wynn Las Vegas, as well as international auctions held in partnership with Concorso d’Eleganza Villa d’Este, Zoute Grand Prix, and Auto Zürich.

Learn more at broadarrowauctions.com and follow us on Instagram, Facebook, LinkedIn, and Twitter.

About Hagerty, Inc. (NYSE: HGTY)

Hagerty is a company built by drivers for drivers, protecting 2.8 million vehicles in the United States, Canada and the UK. We make it easier and more enjoyable for enthusiasts to drive and celebrate the machines they love through innovative insurance products, live and digital auctions, engaging media and events, as well as the Hagerty Drivers Club, the world’s largest community of car lovers. For more information, please visit www.hagerty.com or www.newsroom.hagerty.com.

The Hagerty Automotive Intelligence team uses their collector car expertise to analyse Hagerty's massive trove of public auction results, private sales and insurance data, and buyer and seller behaviour. Learn more about how we collect our data at hagerty.com/valuation-tools.

Forward-Looking Statements - This press release contains statements that constitute “forward-looking statements” within the meaning of the federal securities laws. All statements provided, other than statements of historical fact, are forward-looking statements, including those regarding Hagerty’s future operating results and financial position, Hagerty’s business strategy and plans, products, services, and technology implementations, market conditions, growth and trends, expansion plans and opportunities, and Hagerty’s objectives for future operations. The words “anticipate,” “believe,” “envision,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” “ongoing,” “contemplate,” and similar expressions, and the negative of these expressions, are intended to identify forward-looking statements. 

Hagerty has based these forward-looking statements largely on current expectations about future events, which may not materialize. Actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. These factors include, among other things, Hagerty’s ability to: (i) compete effectively within our industry and attract and retain our insurance policyholders and paid Hagerty Drivers Club (“HDC”) subscribers; (ii) maintain key strategic relationships with our insurance distribution and underwriting carrier partners; (iii) prevent, monitor, and detect fraudulent activity; (iv) manage risks associated with disruptions, interruptions, outages or other issues with our technology platforms or our use of third-party services; (v) accelerate the adoption of our membership and marketplace products and services, as well as any new insurance programs and products we offer; (vi) manage the cyclical nature of the insurance business, including through any periods of recession, economic downturn or inflation; (vii) address unexpected increases in the frequency or severity of claims, and (viii) comply with the numerous laws and regulations applicable to our business, including state, federal and foreign laws relating to insurance and rate increases, privacy, the internet, and accounting matters. 

The forward-looking statements herein represent the judgment of Hagerty as of the date of this release and Hagerty disclaims any intent or obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments, or otherwise. This press release should be read in conjunction with the information included in Hagerty’s other press releases, reports and other filings with the Securities and Exchange Commission. Understanding the information contained in these filings is important in order to fully understand Hagerty’s reported financial results and its business outlook for future periods.

The incredibly rare 1990 Monte Carlo Automotive Centenaire to be offered at Broad Arrow's 2026 Zoute Concours Auction in Knokke-Heist, Belgium. The 1990 Monte Carlo Automotive Centenaire, Monaco's first supercar, set to be offered at Broad Arrow's 2026 Zoute Concours Auction.

The incredibly rare 1990 Monte Carlo Automotive Centenaire to be offered at Broad Arrow's 2026 Zoute... Credit - Sian Loyson/Courtesy of Broad Arrow Auctions The 1990 Monte Carlo Automotive Centenaire, Monaco's first supercar, set to be offered at Broad Arro... Credit - Sian Loyson/Courtesy of Broad Arrow Auctions

Ultragenyx (RARE) was a big mover last session on higher-than-average trading volume. The latest trend in earnings estimate revisions might help the stock continue moving higher in the near term.

Ultragenyx Pharmaceutical Inc. (RARE) Discusses FDA Approval of FAYUVI as First Treatment for Sanfilippo Syndrome Type A September 17, 2026 5:30 PM EDT

Company Participants

Joshua Higa - Director of Investor Relations & Corporate Communications
Emil Kakkis - Founder, President, CEO & Director
Eric Crombez - Chief Medical Officer & Executive VP
Erik Harris - Executive VP & Chief Commercial Officer
Howard Horn - Executive VP of Corporate Strategy & CFO

Conference Call Participants

Yigal Nochomovitz - Citigroup Inc., Research Division
Anupam Rama - JPMorgan Chase & Co, Research Division
Steven Ionov - TD Cowen, Research Division
Maurice Raycroft - Jefferies LLC, Research Division
Kristen Kluska - Cantor Fitzgerald & Co., Research Division
Lydia Erdman - Goldman Sachs Group, Inc., Research Division
Tejas Wein - Barclays Bank PLC, Research Division
Joseph Schwartz - Leerink Partners LLC, Research Division
Maxwell Skor - Morgan Stanley, Research Division
Laura Chico - Wedbush Securities Inc., Research Division

Presentation

Operator

Good afternoon, and welcome to the Ultragenyx Pharmaceutical conference call to discuss the U.S. Food and Drug Administration approval of the FAYUVI for the treatment of the MPS IIIA, also known as Sanfilippo Syndrome Type A. [Operator Instructions] It is now my pleasure to turn the call over to Joshua Higa, Chief of Staff and Vice President of Investor Relations. Please go ahead.

Joshua Higa
Director of Investor Relations & Corporate Communications

[indiscernible] the U.S. FDA approval of FAYUVI, the first-ever treatment for Sanfilippo syndrome Type A. We appreciate all of you making time to join us this afternoon to discuss this important milestone. A press release with details on this announcement is available on our website, along with an updated corporate presentation. Joining me today are Emil Kakkis, Chief Executive Officer and President; Eric Crombez, Chief Medical Officer; and Erik Harris, Chief Commercial Officer. Howard Horn, Chief Financial Officer, is also on the line for Q&A.

The U.S ​FDA said on Thursday ‌it has approved Ultragenyx's (RARE.O) gene therapy for the treatment of a ​rare, fatal disease.

The approval makes ​Fayuvi the first therapy for ⁠Sanfilippo syndrome Type A, ​caused by a missing enzyme that ​stops the body from breaking down a complex sugar molecule called heparan ​sulfate. Ultragenyx shares were ​up nearly 6% in afternoon trading.

The therapy ‌is ⁠designed to address the underlying sulfamidase (SGSH) enzyme deficiency responsible for abnormal accumulation of heparan sulfate ​in ​the brain ⁠that results in progressive cell damage and ​neurodegeneration.

The U.S. Food and ​Drug ⁠Administration had declined to approve the therapy last year citing ⁠manufacturing ​concerns.

NEW YORK, Sept. 15, 2026 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE).  Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

The investigation concerns whether Ultragenyx and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. 

[Click here for information about joining the class action]

On September 2, 2026, Ultragenyx issued a press release “announc[ing] results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome.”  Per the press release, “[t]he study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI).” 

On this news, Ultragenyx’s stock price fell $11.68 per share, or 44.03%, to close at $14.85 per share on September 3, 2026.

Pomerantz LLP, with offices in New York, Chicago, Los Angeles, London, Paris, and Tel Aviv, is acknowledged as one of the premier firms in the areas of corporate, securities, and antitrust class litigation. Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions. Today, more than 85 years later, Pomerantz continues in the tradition he established, fighting for the rights of the victims of securities fraud, breaches of fiduciary duty, and corporate misconduct. The Firm has recovered numerous multimillion-dollar damages awards on behalf of class members. See www.pomlaw.com.

Attorney advertising. Prior results do not guarantee similar outcomes.  

CONTACT:
Danielle Peyton
Pomerantz LLP
[email protected]
646-581-9980 ext. 7980

Martin Shkreli, known as Pharma Bro, said Ultragenyx Pharmaceutical (NASDAQ:RARE) is undervalued and a potential takeover target for rare-disease peers like BioMarin Pharmaceuticals (NASDAQ:BMRN) after a trial setback.

He said "$RARE is very cheap" and urged BioMarin and other rare-disease companies to explore buying the company, adding that "everyone and their brother with 3 months of med school/shkreli training knew this trial would fail."

Ultragenyx’s Cost-Cutting Measures Ultragenyx is planning significant expense reductions after its Phase 3 Aspire study for the Angelman syndrome therapy, apazunersen, failed to meet primary endpoints. The trial’s failure has prompted the company to review the program’s future and implement cost-cutting measures. Analyst Downgrades for Ultragenyx In a Thursday note, analysts downgraded Ultragenyx’s stock, with Baird’s Joel Beatty cutting the price target from $40 to $16.53. Evercore ISI Group’s Liisa Bayko also downgraded the stock and reduced the target price to $16.53, reflecting the impact of the trial results. These downgrades were part of a broader analyst review, which also included changes for Moderna, Inc. Technical AnalysisRARE’s RSI(14) is at 18.99, indicating an oversold condition. The stock trades 47.39% below its 50-day SMA of $27.99 and 43.54% below its 200-day SMA of $26.08. The 50-day SMA remains above the 200-day SMA.

RARE Stock Price Activity: Ultragenyx Pharmaceutical shares were down 43.95% at $14.87 on Thursday, according to Benzinga Pro data.

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It has been about a month since the last earnings report for Ultragenyx (RARE - Free Report) . Shares have added about 6.4% in that time frame, outperforming the S&P 500.

But investors have to be wondering, will the recent positive trend continue leading up to its next earnings release, or is Ultragenyx due for a pullback? Well, first let's take a quick look at its latest earnings report in order to get a better handle on the recent drivers for Ultragenyx Pharmaceutical Inc. before we dive into how investors and analysts have reacted as of late.

Ultragenyx's Q2 Earnings Beat Estimates, Revenues Jump Y/YUltragenyxreported second-quarter 2026 loss of 90 cents per share, which was narrower than the Zacks Consensus Estimate of a loss of $1.27. The company had incurred a loss of $1.17 per share in the year-ago quarter.

Total revenues in the second quarter were $214 million, which surged 28.1% year over year due to higher product sales. The top line also beat the Zacks Consensus Estimate of $181 million.

Management stated that second-quarter 2026 revenues were the highest quarterly revenues ever reported by the company.

RARE’s Q2 Results in DetailCrysvita’s total revenues were $156 million, up 28.9% year over year. Management noted that Crysvita sales were consistent with expected seasonality in the United States and Canada and ordering patterns in Latin America. Crysvita’s net product revenues in the second quarter of 2026 included $94 million from North America, $54 million from Latin America and Turkey, and $8 million from Europe.

Mepsevii product revenues increased 11.1% year over year to $10 million in the reported quarter. Dojolvi product revenues were $27 million, up 17.4%, driven by strong demand. Evkeeza recorded sales of $21 million in the second quarter, up 50%, driven by increased demand from new country launches and early access.

Operating expenses of $289 million in the quarter rose 5.1% year over year due to increased investments in multiple late-stage pipeline programs and marketing costs for approved drugs. Operating expenses included research and development (R&D) expenses of $167 million (up 1.2%), selling, general and administrative (SG&A) expenses of $88 million (up 1.1%) and cost of sales of $34 million (up 47.8%).

Cash, cash equivalents and marketable securities amounted to $436 million as of June 30, 2026, compared with $534 million as of March 31, 2026.

RARE Reiterates 2026 Financial GuidanceUltragenyx continues to expect total revenues in 2026, excluding potential revenues from new product launches, between $730 million and $760 million.

Crysvita revenues in 2026 are expected to be in the range of $500-$520 million, reflecting growing underlying global demand. Meanwhile, Dojolvi revenues are expected to be between $100 million and $110 million in 2026.

How Have Estimates Been Moving Since Then?It turns out, estimates revision have trended downward during the past month.

The consensus estimate has shifted 25.84% due to these changes.

VGM ScoresCurrently, Ultragenyx has a average Growth Score of C, however its Momentum Score is doing a lot better with an A. However, the stock was allocated a score of F on the value side, putting it in the lowest quintile for value investors.

Overall, the stock has an aggregate VGM Score of C. If you aren't focused on one strategy, this score is the one you should be interested in.

OutlookEstimates have been trending downward for the stock, and the magnitude of these revisions looks promising. Notably, Ultragenyx has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months.

Performance of an Industry PlayerUltragenyx belongs to the Zacks Medical - Biomedical and Genetics industry. Another stock from the same industry, CRISPR Therapeutics AG (CRSP - Free Report) , has gained 8.5% over the past month. More than a month has passed since the company reported results for the quarter ended June 2026.

CRISPR Therapeutics reported revenues of $10.18 million in the last reported quarter, representing a year-over-year change of +1043.8%. EPS of -$0.94 for the same period compares with -$1.29 a year ago.

CRISPR Therapeutics is expected to post a loss of $1.06 per share for the current quarter, representing a year-over-year change of +9.4%. Over the last 30 days, the Zacks Consensus Estimate has changed +5.9%.

CRISPR Therapeutics has a Zacks Rank #3 (Hold) based on the overall direction and magnitude of estimate revisions. Additionally, the stock has a VGM Score of F.

CompaniesSept 3 (Reuters) - Shares of Ultragenyx Pharmaceutical (RARE.O), opens new tab plunged more than 40% in premarket on Thursday after its neurodevelopmental disorder treatment failed a closely watched ​late-stage trial, dealing a major blow to one of ‌its biggest growth bets.

The drug, apazunersen, failed to meet both its main goal of improving cognitive skills and its secondary goals of overall patient response in ​participants with Angelman syndrome, a rare condition that affects the ​nervous system and impairs typical brain development in childhood.

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Ultragenyx ⁠said there were no differences between patients receiving the treatment and those ​on placebo, prompting a review of the program's future.

The trial outcome ​is a major blow to the company and the patient community, as there are currently no approved disease-modifying treatments for the condition.

It also marks Ultragenyx's second consecutive late-stage ​trial failure, a setback Jefferies analyst Maury Raycroft called "unambiguously negative" ​and likely to "further erode street confidence".

Following the trial results, at least five brokerages slashed ‌their ⁠price targets on the stock.

TD Cowen analysts said the failure "removes a key growth driver," while Cantor Fitzgerald analyst Kristen Kluska warned that Ultragenyx "truly has to lower its spend substantially for investors to want to ​even consider building ​a position".

Ultragenyx ⁠said it will implement "significant expense reductions" to manage its high operating costs.

Jefferies noted Ultragenyx's trial failure "raises risk ​for competitors" like Ionis Pharmaceuticals (IONS.O), opens new tab and Oak Hill Bio ​who ⁠are developing similar treatments.

"The investment case has changed shape," Leerink Partners analyst Joseph Schwartz said, arguing that Ultragenyx is now "a commercial and expense story ⁠rather ​than a pipeline execution story."

As of Wednesday's ​close, Ultragenyx stock was up 15.4% year-to-date, with a market value of $2.62 billion, according ​to LSEG data.

Reporting by Kamal Choudhury in Bengaluru; Editing by Vijay Kishore

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 | Source: Ultragenyx Pharmaceutical Inc.

Phase 3 Aspire did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI)

NOVATO, Calif., Sept. 02, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome. The study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI). The safety profile observed in Aspire was consistent with Phase 1/2.

“Based on everything we observed in the robust Phase 1/2 clinical development program and long-term extension study, we are disappointed by the Aspire result,” said Emil Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “Even more, we are disappointed for the global patient community who has invested so much in early-stage research, working to bring a first-ever treatment to their children.”

In Aspire, the randomized groups were comparable at baseline and consistent with the patients studied in Phase 2. There were no differences between the treated and control groups that could support efficacy in the Bayley Cognition raw scores nor in the MDRI when looking at net response or mean changes of the individual five endpoints included in the MDRI.

The Company will evaluate the apazunersen program in light of this outcome and make a decision on its disposition. The Company will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.

Dr. Kakkis continued: “We will maintain focus on our growing commercial business, which continues to create meaningful value, including new sources of revenue from the recent approval of GENGLYCOS for glycogen storage disease type Ia, the potential approval of UX111 for Sanfillipo syndrome, and the expansion of existing products to new territories. This strong commercial foundation will support our pipeline, while continuing toward profitability in 2027.”

About apazunersen (GTX-102)
Apazunersen (GTX-102) is an investigational antisense oligonucleotide (ASO) therapy delivered via intrathecal administration and designed to target and inhibit expression of the UBE3A-AS to prevent silencing of the paternally inherited allele of the UBE3A gene and reactivate expression of the deficient protein. Apazunersen has been granted Breakthrough Therapy Designation, Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the FDA and Orphan Designation and PRIME designation from the EMA.

About Angelman Syndrome
Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system (CNS), a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A-AS, the intended target of apazunersen. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is generally not inherited but instead occurs spontaneously. It is estimated to affect approximately 60,000 people in commercially accessible geographies.

Angelman syndrome is a lifelong neurodevelopmental disorder that causes cognitive impairment, motor impairment, balance issues and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. Although individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome, suggesting that improvement of symptoms can potentially be achieved at any age.

About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx’s plans to evaluate its operations and implement significant expense reductions, the Company’s expectations for profitability in 2027, the expected scope, timing, benefits and impact of those actions, its future operating results and financial performance, its business plans and objectives for GTX-102 following the Aspire results, the future development and regulatory path for GTX-102, the growth and importance of its commercial business, and the potential approval and commercialization of UX111 are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the company’s ability to accurately analyze and interpret the Aspire results and determine an appropriate path forward for GTX-102, the uncertainty of clinical drug development and the unpredictability and lengthy process for obtaining regulatory approvals, the risk that results from earlier studies may not be predictive of future study results, the company’s ability to define and implement expense reductions and realize anticipated savings and benefits, the risk that expense reductions may disrupt the company’s operations, adversely affect its workforce or impair its ability to execute its business plans, risks related to adverse side effects, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect the sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements

For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 5, 2026 and its subsequent periodic reports filed with the SEC. 

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).

Ultragenyx Contacts

Investors
Joshua Higa
[email protected]

Media
Jess Rowlands
[email protected]

At Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reduction of at least 50%

Complete elimination of nighttime cornstarch dosing observed in 33% of DTX401 treatment group and 42% of crossover group, while maintaining glycemic control

Analyses at Week 48 showed that 83% of DTX401-treated participants met or exceeded their own expectations for meaningful cornstarch reduction

NOVATO, Calif., Sept. 01, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the publication of 96-week data from its Phase 3 study of GENGLYCOS™ AAV gene therapy (pariglasgene brecaparvovec-opnr), also known as DTX401, for the treatment of glycogen storage disease type Ia (GSDIa) in The Journal of Inherited Metabolic Disease. GENGLYCOS was recently approved by the U.S. Food and Drug Administration (FDA) in patients ages eight and older with GSDIa.

“These results demonstrate the potential of gene therapy to provide greater stability in day-to-day life for patients with GSDIa and may help guard against the risk of severe hypoglycemia associated with missed doses of raw cornstarch," said Dr. John Mitchell, scientist in the Child Health and Human Development Program at the Research Institute of the McGill University Health Centre (The Institute), pediatric endocrinologist at the Montreal Children’s Hospital, lead author of the publication and an investigator on the study. “For me, the reduction in overnight cornstarch dosing will have the most meaningful impact by reducing sleep disruption, with patient-reported outcomes included in the publication underscoring the profound impact that cornstarch reductions may have on daily life. I view long-term outcomes from the 96-week period showing continued improvements as particularly important, offering valuable insight into how post-treatment management may continue to evolve and improve as clinical experience grows."

“The complete results from this Phase 3 study more fully capture the benefits of this gene therapy and the importance of providing patients the ability to breakdown glycogen to provide a source of glucose during fasting or times of increased metabolic demands,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “Most patients achieved cornstarch reductions that met or exceeded their own expectations, with substantially less overnight treatment burden and reduced dependence on the around-the-clock cornstarch need that defines life with this disease. Importantly, reducing cornstarch dependence while maintaining glycemic control indicates the establishment of the liver’s ability to regulate glucose production on its own, giving us confidence that the therapy is directly addressing the underlying cause of disease and offering protection from the risk of life-threatening hypoglycemia.”

Authors emphasize statistically significant and clinically meaningful reductions in cornstarch while maintaining glycemic control

As previously reported, the study met its primary endpoint with patients treated with DTX401 (n=20) experiencing a mean reduction in cornstarch of 41% at Week 48 compared to 10% reduction in the placebo group (n=24) (p < 0.0001). Data at Week 96 showed even greater improvements, with both the DTX401 group (n=20) and the crossover group (n=19) achieving a mean reduction in daily cornstarch intake of 61% from baseline. Participants in both groups also experienced statistically significant improvements in other cornstarch-related endpoints.

Additionally, 72% of participants in the crossover-DTX401 group and 67% of participants in the original DTX401 group achieved reductions of at least 50% in daily cornstarch intake by Week 96. In the manuscript, the authors noted that reductions within the crossover period are particularly meaningful, as that period more closely approximates anticipated patient management in a real-world setting.

Importantly, participants maintained low levels of hypoglycemia and improved levels in euglycemic range (70-120 mg/dL) throughout the second year of the study despite substantial reductions in daily cornstarch intake. Participants dosed with DTX401 also experienced improved normalized fasting tolerance in a controlled fasting challenge (CFC) through year 2 of the study, supporting the potential for protection from severe hypoglycemia (< 54 mg/dL).

Publication offers additional insights into nighttime treatment burden

The publication provides detailed analyses of nighttime cornstarch use, one of the most burdensome aspects of current GSDIa management.

Among participants requiring nighttime cornstarch at baseline:

At Week 48, 50% of DTX401-treated participants eliminated at least one nighttime cornstarch dose compared with 7% of placebo-treated participants (p=0.031).At Week 96, 67% of participants in both treatment groups eliminated at least one nighttime cornstarch dose.By Week 96, 33% of original DTX401 participants and 42% of crossover-DTX401 participants had completely eliminated nighttime cornstarch dosing. Despite substantial reductions in daily and nighttime cornstarch use, participants maintained glycemic control throughout the study, without inducing severe hypoglycemic episodes. These findings build upon previously reported reductions in nighttime cornstarch requirements and provide additional insight into the impact of DTX401 on overnight disease management.

Patient-reported outcomes support treatment effect as clinically meaningful

Authors detailed findings of a patient-centered analysis that showed the average reduction in daily cornstarch intake considered meaningful by participants at baseline was 45%. At Week 48, 83% of DTX401-treated participants met or exceeded their own baseline expectations for meaningful reduction in cornstarch use, with continued improvements through Week 96.

At Week 48, 79% of DTX401-treated participants reported improvement in GSDIa on the Patient Global Impression of Change compared with 52% of placebo-treated participants (p=0.131); at Week 96, improvement was reported by 95% of crossover-DTX401 participants and 83% of original DTX401 participants.

The publication further characterizes how reducing cornstarch requirements affected overall nutritional management. At baseline, cornstarch accounted for nearly 50% of study participants’ total caloric intake. Following treatment with DTX401, participants were able to transition to a more balanced, food-based diet closer to the U.S. Dietary Guidelines for the general population.

DTX401 was generally well tolerated with an acceptable safety profile

Consistent with previously reported findings, the authors concluded that DTX401 demonstrated an acceptable and manageable safety profile. The most common treatment-related adverse events were transient elevations in liver enzymes, which were generally nonserious and managed with prophylactic corticosteroids.

No AAV8 class effects of dorsal root ganglion toxicity, malignancy, or thrombotic microangiopathy were observed in the study through Week 96. Hypertriglyceridemia was observed in all study groups but more frequently following DTX401 treatment.

INDICATION

GENGLYCOS (pariglasgene brecaparvovec-opnr) is indicated to reduce daily cornstarch intake as an adjunct to nutritional management in adult and pediatric patients 8 years of age and older with glycogen storage disease type Ia (GSDIa).

This indication is approved under accelerated approval based on reduction in daily cornstarch intake. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS
GENGLYCOS is contraindicated in patients with known severe hepatic fibrosis or cirrhosis.

WARNINGS AND PRECAUTIONS

Hypersensitivity and Infusion Reactions (IRs)

Hypersensitivity reactions including anaphylaxis and IRs have occurred with GENGLYCOS treatment. Severe reactions have been reported. Monitor for signs and symptoms of hypersensitivity and IRs, including urticaria, flushing, hypotension, bronchospasm, dyspnea, chest tightness, nausea, vomiting, headache, abdominal pain, lightheadedness, flu-like symptoms, shivering, rash, and hypertension.Premedicate with acetaminophen and non-sedating antihistamines and administer GENGLYCOS according to recommended infusion rates. Monitor patients during and after completion of GENGLYCOS infusion as clinically indicated. If anaphylaxis or severe IR occurs, pause GENGLYCOS infusion immediately and initiate medical treatment as clinically indicated, monitoring as needed. For mild to moderate IRs, consider slowing or temporarily interrupting the infusion, and administer symptomatic treatment as clinically indicated. The infusion may be restarted at half the prior rate upon resolution of symptoms.Medical support measures, including cardiopulmonary resuscitation equipment and medications for the treatment of anaphylaxis (e.g., epinephrine, antihistamines, corticosteroids), should be available during GENGLYCOS administration. Hepatotoxicity

Immune-mediated hepatotoxicity, with elevated alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) levels, has occurred with GENGLYCOS. Avoid use in patients with preexisting hepatic impairment or acute hepatic viral infection.Prior to GENGLYCOS infusion, evaluate liver-related medical history and assess liver function by clinical examination and laboratory testing. Advise patients to immediately report signs and symptoms of hepatotoxicity, including fatigue, jaundice, dark urine, nausea, vomiting, and right upper quadrant pain. Administer corticosteroids to all patients after GENGLYCOS infusion in order to mitigate hepatic reactions. Elevated transaminases may require adjustment of the corticosteroid treatment regimen, including increased dose or prolongation of the corticosteroid taper.Monitor transaminase levels for the first 6 months after GENGLYCOS administration. Continue to monitor transaminases in all patients who develop transaminase elevations, until transaminases return to baseline or as clinically indicated. Adrenal Insufficiency

Adrenal insufficiency, including serious events, has been reported in patients receiving GENGLYCOS during corticosteroid use and tapering.Signs and symptoms of adrenal insufficiency include fatigue, weakness, anorexia, nausea, vomiting, hypotension, hyponatremia, and hypoglycemia. Adrenal crisis may present as severe hypotension, acute abdominal pain, or loss of consciousness.Monitor patients for signs and symptoms of adrenal insufficiency and adrenal crisis after GENGLYCOS administration during and after corticosteroid therapy and tapering. Taper corticosteroid therapy gradually. Do not abruptly discontinue corticosteroid therapy. AAV Vector Integration and Risk of Tumorigenicity

There is a theoretical risk of tumorigenicity due to integration of AAV vector DNA into the genome.GENGLYCOS is composed of a recombinant, non-replicating AAV8 vector whose DNA persists largely in episomal form. Random integration of recombinant AAV-vector DNA into human DNA has been reported with AAV gene therapies. The clinical relevance of individual integration events is unknown, but it is acknowledged that individual integration events could potentially contribute to a risk of tumorigenicity. If a tumor develops in a patient receiving GENGLYCOS, health care providers should contact and report the tumor to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657. Adverse Reactions

Seven serious adverse events were observed in the Primary Efficacy Analysis Period (PEAP) of Study 1 (Weeks 1-48), including anaphylaxis/infusion reaction (2), adrenal insufficiency (2), high lactate level (2) and hypoglycemia (1).The most common adverse reactions during the PEAP of Study 1 (occurring in ≥10% of patients) with higher frequency in GENGLYCOS compared to placebo were ALT/AST Enzyme elevated (71%), Nausea (38%), Headache (24%), Hypertriglyceridemia (29%), Adrenal Insufficiency (24%), Constipation (19%), Hyperglycemia (14%), Acne/Dermatitis Acneiform (19%), Cushingoid Features (14%), and Anaphylaxis (10%). DRUG INTERACTIONS

Vaccinations

Vaccine schedules may need to be adjusted for immunosuppressive therapy, and vaccines should be avoided 1 month prior to GENGLYCOS administration. USE IN SPECIFIC POPULATIONS

Pregnancy

GENGLYCOS should not be used during pregnancy. There are no data on the use of GENGLYCOS in pregnant women. It is unknown whether GENGLYCOS can cause fetal harm when administered to a pregnant woman or can affect reproductive capacity. Contraception

Women of childbearing potential should use effective contraception for at least 12 months after administration of GENGLYCOS.For 6 months after administration of GENGLYCOS, men must not donate semen, and men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception. ADDITIONAL PATIENT COUNSELING INFORMATION
Vector Shedding

Inform patients/caregivers that vector distribution in blood and vector shedding in urine, stool, and saliva can occur after GENGLYCOS infusion. Advise patients/caregivers on proper hygiene when handling patient body waste. These precautions should be followed for 3 months after GENGLYCOS infusion. Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657. 

Please see the full Prescribing Information for GENGLYCOS.

About the Phase 3 GlucoGene study
The 48-week randomized, double-blind, placebo-controlled study treated 46 participants aged eight years and older with DTX401 (1.0 x 10^13 GC/kg dose measured by ddPCR) or placebo. There were 44 participants in the modified intention-to-treat (mITT) population providing efficacy data within the Week 48 analysis period following treatment with DTX401 (n=20) or placebo (n=24). At Week 48, eligible participants crossed over and received the alternate treatment. After crossover, participants continued to be followed with analyses conducted at Week 96 and Week 144. After study completion, participants will be offered enrollment into the GSDIa Disease Monitoring Program (DMP) where they will be followed for 10 years post-DTX401 infusion.

About Glycogen Storage Disease Type Ia (GSDIa)
GSDIa is an ultra-rare, serious, and life-threatening disease due to an inborn error of carbohydrate metabolism caused by pathogenic variants of the G6PC gene, which encodes G6Pase, an enzyme that is critical for the release of glucose from glycogen and other metabolic sources. Deficiency of G6Pase activity results in severe hypoglycemia during periods of fasting between meals and during the night along with excess hepatic glycogen storage, metabolic derangements and other disease related complications. Cornstarch is critical in the management of GSDIa throughout the day and night in providing an exogenous source of glucose to help avoid sudden and severe drops in plasma glucose levels; however, current management strategies carry a significant burden to patients and families. GSDIa affects approximately 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide within commercially accessible geographies.

About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding the interpretation, significance and potential implications of the published 96-week Phase 3 data and analyses for GENGLYCOS (also known as DTX401); the clinical meaningfulness and durability of reductions in daily and nighttime cornstarch requirements; the ability of patients to maintain glycemic control and improve fasting tolerance following treatment; the potential for GENGLYCOS to protect against severe hypoglycemia, reduce treatment burden, improve nutritional management and provide other patient benefits; the safety and tolerability of GENGLYCOS; expectations regarding continued follow-up of study participants and the design, enrollment, timing, conduct and results of the GSDIa Disease Monitoring Program and other post-marketing requirements; Ultragenyx’s ability to confirm clinical benefit, satisfy FDA requirements and maintain accelerated approval for GENGLYCOS; and estimates of the prevalence of GSDIa and the potential patient population for GENGLYCOS, are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause actual results to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the risk that results from a limited number of study participants, including crossover and other analyses, may not be replicated or predictive of future or real-world results; the risk that longer-term follow-up may not demonstrate sustained efficacy, durability, safety or patient benefit; risks related to serious or undesirable side effects, including risks associated with AAV gene therapy; Ultragenyx’s ability to complete post-marketing requirements within required timeframes and confirm clinical benefit; the risk that the FDA may modify the approved indication or impose additional requirements, or may withdraw accelerated approval if clinical benefit is not confirmed or post-marketing requirements are not satisfied; and other matters that could affect the availability or commercial potential of Ultragenyx’s products and product candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.

For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 5, 2026, and its subsequent periodic reports filed with the SEC.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).

Ultragenyx Contacts

Investors
Joshua Higa
[email protected]

Media
Jess Rowlands
[email protected]

 | Source: Ultragenyx Pharmaceutical Inc.

NOVATO, Calif., Aug. 25, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 45,984 restricted stock units of the company’s common stock to 23 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of August 16, 2026, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

The restricted stock units vest over four years, with 25% of the underlying shares vesting on each anniversary of the grant date, subject to the employee being continuously employed by the company as of such vesting dates.

About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Contact Ultragenyx
Investors & Media
Joshua Higa
(415) 475-6370

Ultragenyx Pharmaceutical Inc. (RARE) Discusses FDA Approval of Gene Therapy for Glycogen Storage Disease Type 1a Transcript

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares are up during Thursday’s premarket session on the recent U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy, Genglycos (pariglasgene brecaparvovec-opnr, or DTX401).

Genglycos Approved For Ultra-Rare Genetic DisorderThis marks a significant milestone as it is the first-ever FDA-approved treatment for Glycogen Storage Disease Type Ia (GSDIa).

The approval of Genglycos represents Ultragenyx’s first gene therapy and fifth FDA approval.

GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream.

The deficiency reduces the liver’s ability to control glucose levels and is associated with potentially life-threatening hypoglycemia episodes and other serious complications.

GSDIa affects 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide.

Read Next

Phase 3 Data Supports FDA ApprovalThe approval of GENGLYCOS is based on data from the 48-week Phase 3 GlucoGene study, which treated 46 participants aged eight years and older with DTX401 (1.0 x 10^13 GC/kg dose) or placebo, showing a reduction in the cornstarch requirements in the treated group.

As part of accelerated approval, Ultragenyx has agreed to provide two years of safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patients through enhancement of its existing GSDIa Disease Monitoring Program (DMP).

The DMP will also evaluate previously treated clinical trial participants as well as these new commercial patients for a total of 10 years.  

Analyst BullishCiting a conference call, William Blair noted the wholesale acquisition cost for Genglycos will be $2.7 million per patient.

Analyst Sami Corwin said, “We are encouraged by the approval of Genglycos, which we believe is incrementally positive to Ultragenyx’s stock.”

Analyst Corwin resumed coverage of Ultragenyx with an Outperform rating and currently models $362 million in peak sales for Genglycos.

RARE Stock Price Activity: Ultragenyx Pharmaceutical shares were up 11.28% at $29.20 during premarket trading on Thursday, according to Benzinga Pro data.

Read Next

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Ultragenyx Pharmaceutical (RARE.O) said on Wednesday that its gene therapy became the ​first to receive the U.S. Food and Drug Administration's approval to ‌treat a rare metabolic disorder, sending its shares up 7.5% in after-hours trading.

The drug, branded as Genglycos, has been approved for glycogen storage disease type Ia (GSDIa), also known ​as Von Gierke disease, in adults and pediatric patients.

It is an ​ultra-rare genetic disorder that is caused due to deficiency of ⁠an enzyme that helps release glucose from the liver into the bloodstream. ​GSDIa affects 1,500 to 2,500 patients in the U.S., the company estimates.

The deficiency ​reduces the liver's ability to control glucose levels and is associated with potentially life-threatening low blood sugar levels.

Day-to-day management of the condition requires a relentless regimen of raw cornstarch ​as an oral glucose replacement therapy.

Ultragenyx's Genglycos addresses the underlying cause of ​the disorder by aiding the normal breakdown of glycogen to produce glucose, which helps ‌in regulating ⁠blood sugar levels and reducing reliance on cornstarch.

Genglycos will have a U.S. per-patient list price of $2.7 million and be available through qualified treatment centers within 30 to 60 days, the company said.

The regulator's authorization is based on ​a 48-week late-stage ​trial that found ⁠patients treated with Genglycos showed a reduction in the cornstarch requirements compared to placebo.

Reducing cornstarch intake is, however, the ​surrogate endpoint, and the manufacturer must complete additional clinical trials ​to ⁠confirm Genglycos's effectiveness, the FDA said.

The company said it has agreed to provide two years of safety and efficacy clinical data from patients.

Several experimental treatments for ⁠GSDIa are ​also in development, including those from Moderna (MRNA.O) ​and Beam Therapeutics (BEAM.O), though the candidates remain in early stages of clinical testing.

Posted by Defense World Staff on Aug 13th, 2026

Assenagon Asset Management S.A. lowered its stake in shares of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Free Report) by 94.6% during the 2nd quarter, according to the company in its most recent 13F filing with the SEC. The institutional investor owned 120,864 shares of the biopharmaceutical company’s stock after selling 2,136,807 shares during the quarter. Assenagon Asset Management S.A. owned 0.12% of Ultragenyx Pharmaceutical worth $4,036,000 at the end of the most recent quarter.

A number of other hedge funds also recently made changes to their positions in the stock. Parallel Advisors LLC increased its stake in Ultragenyx Pharmaceutical by 778.4% in the first quarter. Parallel Advisors LLC now owns 1,625 shares of the biopharmaceutical company’s stock valued at $34,000 after acquiring an additional 1,440 shares during the last quarter. Leonteq Securities AG lifted its stake in Ultragenyx Pharmaceutical by 288.5% during the first quarter. Leonteq Securities AG now owns 1,795 shares of the biopharmaceutical company’s stock worth $38,000 after purchasing an additional 1,333 shares during the last quarter. Versant Capital Management Inc lifted its stake in Ultragenyx Pharmaceutical by 662.9% during the second quarter. Versant Capital Management Inc now owns 1,152 shares of the biopharmaceutical company’s stock worth $38,000 after purchasing an additional 1,001 shares during the last quarter. Danske Bank A S bought a new stake in Ultragenyx Pharmaceutical during the third quarter worth about $39,000. Finally, EverSource Wealth Advisors LLC boosted its holdings in shares of Ultragenyx Pharmaceutical by 573.0% in the 1st quarter. EverSource Wealth Advisors LLC now owns 2,167 shares of the biopharmaceutical company’s stock worth $45,000 after purchasing an additional 1,845 shares in the last quarter. 97.67% of the stock is owned by institutional investors.

Insiders Place Their Bets In other Ultragenyx Pharmaceutical news, Director Corazon (Corsee) D. Sanders sold 2,000 shares of the stock in a transaction that occurred on Monday, June 15th. The stock was sold at an average price of $25.05, for a total value of $50,100.00. Following the completion of the sale, the director owned 21,095 shares in the company, valued at approximately $528,429.75. This trade represents a 8.66% decrease in their position. The transaction was disclosed in a filing with the SEC, which can be accessed through the SEC website. Also, EVP Karah Herdman Parschauer sold 1,899 shares of the firm’s stock in a transaction on Monday, June 15th. The stock was sold at an average price of $24.62, for a total value of $46,753.38. Following the completion of the sale, the executive vice president directly owned 94,462 shares of the company’s stock, valued at $2,325,654.44. The trade was a 1.97% decrease in their position. Additional details regarding this sale are available in the official SEC disclosure. Over the last 90 days, insiders sold 14,292 shares of company stock worth $351,644. 5.20% of the stock is currently owned by company insiders.

Ultragenyx Pharmaceutical Stock Down 0.4% Shares of Ultragenyx Pharmaceutical stock opened at $26.86 on Thursday. The firm has a market cap of $2.65 billion, a PE ratio of -4.60 and a beta of 0.35. Ultragenyx Pharmaceutical Inc. has a 12-month low of $18.29 and a 12-month high of $39.89. The company’s fifty day simple moving average is $28.16 and its two-hundred day simple moving average is $24.90.

Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) last issued its quarterly earnings data on Tuesday, August 4th. The biopharmaceutical company reported ($0.90) EPS for the quarter, beating the consensus estimate of ($1.22) by $0.32. The firm had revenue of $214.00 million during the quarter, compared to the consensus estimate of $183.08 million. Ultragenyx Pharmaceutical had a negative net margin of 81.79% and a negative return on equity of 1,024.42%. The company’s quarterly revenue was up 28.5% compared to the same quarter last year. During the same period in the prior year, the firm posted ($1.17) earnings per share. As a group, sell-side analysts expect that Ultragenyx Pharmaceutical Inc. will post -4.08 earnings per share for the current fiscal year.

Ultragenyx Pharmaceutical News Roundup Here are the key news stories impacting Ultragenyx Pharmaceutical this week:

Positive Sentiment: HC Wainwright sharply raised its Q3 2026 EPS estimate to $0.75 from a loss of $0.84 per share and increased its Q4 2026 forecast to $0.78 from a loss of $0.35. The changes suggest expectations for a faster-than-anticipated improvement in Ultragenyx’s profitability. Ultragenyx Pharmaceutical Earns Buy Rating from HC Wainwright Neutral Sentiment: HC Wainwright maintained its Buy rating and $50 price target, well above the stock’s recent trading level. Separately, a brokerage consensus cited a price target of approximately $57.82, indicating substantial potential upside if the company executes on its pipeline and reaches profitability. Brokerages Set Ultragenyx Pharmaceutical Price Target Neutral Sentiment: Ultragenyx and Baylor Research Institute reportedly settled a patent lawsuit involving generic competition to DOJOLVI. The settlement removes some litigation uncertainty, but the financial and commercial terms were not provided, limiting the immediate valuation impact. Ultragenyx and Baylor Settle DOJOLVI Patent Lawsuit Negative Sentiment: HC Wainwright lowered its 2027 EPS estimates across the board: Q1 to $0.06 from $0.27, Q2 to $0.91 from $1.18, Q3 to $1.25 from $1.54, Q4 to $2.27 from $2.63, and full-year 2027 to $4.50 from $5.61. These reductions weigh on longer-term earnings expectations, even though the analyst remains bullish. Wall Street Analyst Weigh In RARE has been the topic of a number of research reports. Barclays decreased their price objective on Ultragenyx Pharmaceutical from $44.00 to $43.00 and set an “overweight” rating on the stock in a report on Wednesday, April 29th. Guggenheim cut their target price on Ultragenyx Pharmaceutical from $43.00 to $35.00 and set a “buy” rating for the company in a research note on Tuesday, August 4th. Wells Fargo & Company reduced their price target on shares of Ultragenyx Pharmaceutical from $47.00 to $45.00 and set an “overweight” rating on the stock in a research report on Wednesday, August 5th. Royal Bank Of Canada lifted their price target on shares of Ultragenyx Pharmaceutical from $35.00 to $40.00 and gave the stock an “outperform” rating in a research note on Tuesday, July 7th. Finally, Wall Street Zen upgraded shares of Ultragenyx Pharmaceutical from a “sell” rating to a “hold” rating in a report on Saturday, August 8th. One investment analyst has rated the stock with a Strong Buy rating, fourteen have issued a Buy rating, two have assigned a Hold rating and one has given a Sell rating to the company’s stock. According to MarketBeat.com, the stock presently has a consensus rating of “Moderate Buy” and an average price target of $57.82.

View Our Latest Analysis on Ultragenyx Pharmaceutical

About Ultragenyx Pharmaceutical (Free Report)

Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic disorders. Since its founding in 2010 and headquarters in Novato, California, the company has built expertise in protein replacement therapies, small molecules and gene therapy approaches to address high-unmet medical needs. Ultragenyx applies a precision medicine model, leveraging both in-house research and strategic collaborations to advance its product pipeline from discovery through regulatory approval.

The company’s commercial portfolio includes Crysvita (burosumab-tmyl) for X-linked hypophosphatemia, Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis VII and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders.

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Key Takeaways RARE beat Q2 earnings estimates as revenues rose 28% year over year to a record quarterly high.Crysvita, Dojolvi and Evkeeza sales grew on demand, supporting higher product revenues.Ultragenyx reaffirmed 2026 revenue guidance of $730M-$760M, excluding new product launches. Ultragenyx Pharmaceutical (RARE - Free Report) reported second-quarter 2026 loss of 90 cents per share, which was narrower than the Zacks Consensus Estimate of a loss of $1.27. The company had incurred a loss of $1.17 per share in the year-ago quarter.

Total revenues in the second quarter were $214 million, which surged 28.1% year over year due to higher product sales. The top line also beat the Zacks Consensus Estimate of $181 million.

Management stated that second-quarter 2026 revenues were the highest quarterly revenues ever reported by the company.

Ultragenyx markets four drugs, namely Crysvita, Mepsevii, Dojolvi and Evkeeza. Crysvita is approved for treating X-linked hypophosphatemia, an inherited disorder and tumor-induced osteomalacia, an ultra-rare disease. Mepsevii is approved to treat Mucopolysaccharidosis VII, also known as Sly syndrome. Dojolvi is approved for treating all forms of long-chain fatty acid oxidation disorders. Evkeeza is indicated for homozygous familial hypercholesterolemia (HoFH).

In 2022, Ultragenyx announced a license and collaboration agreement with Regeneron Pharmaceuticals (REGN - Free Report) for Evkeeza, which is approved in multiple geographies as a first-in-class therapy for use together with diet and other low-density lipoprotein-cholesterol-lowering therapies to treat adults and adolescents aged 12 years and older with HoFH.

Per the deal, RARE has obtained the rights to develop, commercialize and distribute Evkeeza outside the United States. The regions include the European Economic Area. The collaboration with Regeneron for Evkeeza gives Ultragenyx a fourth approved product that adds to the top line. However, REGN solely commercializes Evkeeza in the United States.

Year to date, shares of Ultragenyx have gained 12.2% compared with the industry’s 1.6% rise.

Image Source: Zacks Investment Research

RARE’s Q2 Results in DetailCrysvita’s total revenues were $156 million, up 28.9% year over year. Management noted that Crysvita sales were consistent with expected seasonality in the United States and Canada and ordering patterns in Latin America. Crysvita’s net product revenues in the second quarter of 2026 included $94 million from North America, $54 million from Latin America and Turkey, and $8 million from Europe.

Mepsevii product revenues increased 11.1% year over year to $10 million in the reported quarter. Dojolvi product revenues were $27 million, up 17.4%, driven by strong demand. Evkeeza recorded sales of $21 million in the second quarter, up 50%, driven by increased demand from new country launches and early access.

Operating expenses of $289 million in the quarter rose 5.1% year over year due to increased investments in multiple late-stage pipeline programs and marketing costs for approved drugs. Operating expenses included research and development (R&D) expenses of $167 million (up 1.2%), selling, general and administrative (SG&A) expenses of $88 million (up 1.1%) and cost of sales of $34 million (up 47.8%).

Cash, cash equivalents and marketable securities amounted to $436 million as of June 30, 2026, compared with $534 million as of March 31, 2026.

RARE Reiterates 2026 Financial GuidanceUltragenyx continues to expect total revenues in 2026, excluding potential revenues from new product launches, between $730 million and $760 million.

Crysvita revenues in 2026 are expected to be in the range of $500-$520 million, reflecting growing underlying global demand. Meanwhile, Dojolvi revenues are expected to be between $100 million and $110 million in 2026.

RARE’s Key Pipeline UpdatesIn April 2026, the FDA accepted the resubmitted biologics license application (BLA) seeking accelerated approval of UX111 for the treatment of MPS IIIA. The application included extensive long-term data with follow-up of up to eight years. The data showed sustained clinical benefits compared with the decline seen in natural history studies, along with durable treatment effects across multiple clinical measures and biomarkers, while maintaining an acceptable safety profile. A final decision from the regulatory body is expected on Sept. 19, 2026.

The FDA has also accepted for review Ultragenyx’s BLA for its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia. A final decision from the regulatory agency is expected on Aug. 23, 2026.

Ultragenyx is also evaluating UX701, an investigational AAV9 gene therapy, in a phase I/II/III Cyprus2+ study to treat Wilson disease and expects to share top-line data in the fourth quarter of 2026.

Ultragenyx’s GTX-102, an investigational antisense oligonucleotide, is being developed in the pivotal phase III Aspire study for treating Angelman syndrome (AS) patients with a genetically confirmed diagnosis of UBE3A deletion. Top-line data are expected in September or October 2026. Meanwhile, enrollment in the phase II/III Aurora study is currently ongoing to evaluate the safety and efficacy of GTX-102 for treating other AS genotypes in other patient age groups. This additional study aims to enable treatment for a broader range of AS patients. The study is expected to complete enrollment in the second half of 2026.

RARE’s Zacks Rank & Other Stocks to ConsiderUltragenyx currently carries a Zacks Rank #2 (Buy).

Some other top-ranked stocks in the biotech sector are Repligen (RGEN - Free Report) and Liquidia Corporation (LQDA - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for Repligen’s 2026 earnings per share have risen from $1.99 to $2.06, while estimates for 2027 have increased from $2.57 to $2.62 during the same time. RGEN shares have declined 8.6% year to date.

Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.

Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $2.97 to $3.02, while estimates for 2027 have increased from $4.81 to $5.31 during the same time. LQDA shares have surged 156.3% year to date.

Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.

Ultragenyx (RARE - Free Report) came out with a quarterly loss of $0.9 per share versus the Zacks Consensus Estimate of a loss of $1.27. This compares to a loss of $1.17 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of +29.13%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.55 per share when it actually produced a loss of $1.84, delivering a surprise of -18.71%.

Over the last four quarters, the company has surpassed consensus EPS estimates just once.

Ultragenyx, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $214 million for the quarter ended June 2026, surpassing the Zacks Consensus Estimate by 18.23%. This compares to year-ago revenues of $166.5 million. The company has topped consensus revenue estimates two times over the last four quarters.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

Ultragenyx shares have added about 8.8% since the beginning of the year versus the S&P 500's gain of 11%.

What's Next for Ultragenyx?While Ultragenyx has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for Ultragenyx was favorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #2 (Buy) for the stock. So, the shares are expected to outperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is -$0.85 on $189.98 million in revenues for the coming quarter and -$4.53 on $746.71 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 42% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

One other stock from the same industry, Zealand Pharma A/S (ZLDPF - Free Report) , is yet to report results for the quarter ended June 2026. The results are expected to be released on August 13.

This company is expected to post quarterly loss of $0.42 per share in its upcoming report, which represents a year-over-year change of -102.6%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days.

Zealand Pharma A/S's revenues are expected to be $81.55 million, down 94.1% from the year-ago quarter.