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	Saved 2026-09-02 21:34 7d ago Published 2026-09-02 16:01 7d ago	Ultragenyx: studie Aspire u Angelmanova syndromu selhala Ultragenyx oznámila, že studie fáze 3 Aspire s apazunersenem u Angelmanova syndromu nesplnila primární ani klíčový sekundární cíl. Bezpečnostní profil byl v souladu s fází 1/2. RARE Ultragenyx	FMP Stock News	88
Original source text \| 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]
	Saved 2026-09-01 21:11 8d ago Published 2026-09-01 16:05 8d ago	Ultragenyx hlásí 61% pokles dávky kukuřičného škrobu u GSDIa Ultragenyx zveřejnila 96týdenní data ze studie GENGLYCOS/DTX401 u GSDIa: průměrná denní dávka kukuřičného škrobu klesla o 61 % při zachování glykemické kontroly. U 33 % původní skupiny a 42 % crossover skupiny byla noční dávka zcela ukončena. RARE Ultragenyx	FMP Stock News	88
Original source text 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]
	Saved 2026-08-19 22:57 21d ago Published 2026-08-19 17:21 21d ago	Ultragenyx získal zrychlené schválení FDA pro GENGLYCOS Ultragenyx získal od FDA zrychlené schválení GENGLYCOS pro dospělé i pediatrické pacienty od 8 let s GSDIa. Jde o první genovou terapii firmy a pátou léčbu schválenou FDA pro společnost, zároveň první léčbu zaměřenou na příčinu této nemoci. RARE Ultragenyx	FMP Stock News	92
Original source text GENGLYCOS is the first gene therapy approval, and fifth FDA approval overall, for the company Approval provides a long-awaited first-ever option to reduce the burden of care associated with GSDIa Ultragenyx received a Priority Review Voucher upon approval Ultragenyx to host conference call on 8/19/26 at 6:00 p.m. Eastern Time NOVATO, Calif., Aug. 19, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval for GENGLYCOS™ (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). “The approval of GENGLYCOS fulfills our commitment to provide the first therapy that directly targets the root cause of GSDIa. The reduced reliance on cornstarch, experienced by patients in our clinical studies, demonstrates this gene therapy’s ability to establish the normal breakdown of glycogen to produce glucose during fasting or episodes of metabolic stress. This ability to regulate glucose has alleviated the disease burden and has the potential to mitigate the risk of severe or life-threatening hypoglycemia for these patients,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “As our first gene therapy approval, GENGLYCOS represents an important achievement for our company and the realization of the promise of a powerful new tool to deliver transformative medicines for people living with rare diseases.” 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, requiring rigorous nutritional management that involves a burdensome, around-the-clock regimen of raw cornstarch intake as an oral glucose replacement therapy. Glucose control with cornstarch is crude with large swings in glucose, and patients instead end up spending a large fraction of their day significantly hyperglycemic to avoid hypoglycemic episodes. GSDIa affects 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide within commercially accessible geographies. “Day-to-day management of GSDIa requires a relentless regimen of raw cornstarch and strict dietary management that can be extraordinarily demanding for patients and families. Even with meticulous adherence to this regimen, patients must be perfect. Any missed cornstarch puts patients at risk of severe hypoglycemia, seizures, and even death,” said David Weinstein, M.D., MMSc, one of the world's leading GSDIa experts. “The approval of GENGLYCOS represents a major step forward for the GSDIa community and reflects almost 30 years of work and scientific progress aimed at improving safety and the quality of life of people living with this disease.” “For families affected by GSDIa, every day revolves around strict schedules, overnight vigilance, and the constant worry that a missed meal or dose of cornstarch could trigger life-threatening hypoglycemia,” said David and Wendy Feldman, co-founders and current Board members at The Children’s Fund for Glycogen Storage Disease Research. “This approval is an incredibly meaningful milestone for a community that has spent decades hoping, advocating, and helping advance the research for new treatment options that could ease the burdens of this disease.” Clinical Program and Post-Marketing Study Requirements Supporting Accelerated Approval of GENGLYCOS The approval of GENGLYCOS is based on positive data from the 48-week randomized, double-blind, placebo-controlled 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 (p<0.001). 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. 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 control group will consist of patients who sought commercial treatment but cannot be treated with GENGLYCOS due to the presence of anti-AAV8 antibodies. The study will provide more data to support the reduction in cornstarch clinical burden, fasting tolerance, and other measures in a post-marketing setting where patients can know their immediate glucose levels, and their cornstarch and diet can be managed more promptly by their physician. The DMP will also evaluate previously treated clinical trial participants as well as these new commercial patients for a total of 10 years. Enabling Access for Eligible Patients Ultragenyx will provide support to help enrolled patients and caregivers navigate access to treatment through its UltraCare® program, which now includes specially trained UltraCare® Gene Therapy Guides to help understand insurance coverage, assist in obtaining treatment support, and answer questions about the treatment process. Dedicated in-house UltraCare Gene Therapy Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657. More information is available at www.ultracaresupport.com. GENGLYCOS will be available through a national network of Qualified Treatment Centers (QTCs) with specialized expertise and training to safely administer gene therapy. GENGLYCOS is manufactured entirely at Ultragenyx’s Gene Therapy Manufacturing Facility (GTMF) in Bedford, Mass., strengthening the Company’s ability to scale production of high-quality gene therapy products and deliver them to patients as efficiently as possible. More information will be available at www.genglycos.com. This approval reflects the work of a remarkable team spanning many years and organizations. Ultragenyx is deeply grateful to the patients, families, and clinical investigators who made our clinical studies possible; Dr. David Weinstein, whose scientific and clinical leadership laid critical groundwork across early research and clinical trials; Dr. Janice Chou for her early work at NIH, and the team from Dimension Therapeutics. Their collective commitment and perseverance have transformed a scientific vision into a new therapeutic option for patients. Investor Conference Call and Webcast Information Ultragenyx will host a conference call today at 6:00 p.m. Eastern Time / 3:00 p.m. Pacific Time to discuss the approval of GENGLYCOS. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. 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 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 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 therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, 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 commercial launch, availability and market acceptance of GENGLYCOS; Ultragenyx's ability to manufacture GENGLYCOS at its gene therapy manufacturing facility, scale production and supply GENGLYCOS to Qualified Treatment Centers; patient access to GENGLYCOS, including insurance coverage and reimbursement; the safety, efficacy, durability and potential benefits of GENGLYCOS; estimates of the number of patients with GSDIa and the potential commercial opportunity for GENGLYCOS; the design, enrollment, timing, conduct and results of the post-marketing Disease Monitoring Program and other post-marketing requirements; and Ultragenyx's ability to satisfy FDA requirements and maintain accelerated approval 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, risks and uncertainties related to the commercial launch and market acceptance of GENGLYCOS; the ability to identify eligible patients and establish and support a network of Qualified Treatment Centers; uncertainty related to insurance coverage and reimbursement; risks related to serious or undesirable side effects, including risks associated with AAV gene therapy; manufacturing risks, including Ultragenyx's limited experience operating its own manufacturing facility and the ability to manufacture and supply GENGLYCOS in sufficient quantities and in compliance with regulatory requirements; Ultragenyx's ability to complete the post-marketing Disease Monitoring Program and other post-marketing requirements within required timeframes and to 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; smaller than anticipated market opportunities; competition from other therapies or products; product liability; regulatory scrutiny; 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]
	Saved 2026-08-05 02:23 1mo ago Published 2026-08-04 20:02 1mo ago	Ultragenyx snížil ztrátu a překonal odhad tržeb Ultragenyx vykázal ve čtvrtletí končícím v červnu 2026 ztrátu 0,9 USD na akcii, méně než očekávaných 1,27 USD. Tržby dosáhly 214 milionů USD a překonaly odhad o 18,23 %. RARE Ultragenyx	FMP Stock News	78
Original source text 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.