Track market-moving stories before they get noisy

Posted by Defense World Staff on Jul 21st, 2026

Andra AP fonden increased its stake in Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY – Free Report) by 78.6% in the 1st quarter, according to the company in its most recent Form 13F filing with the Securities and Exchange Commission. The institutional investor owned 86,697 shares of the biopharmaceutical company’s stock after acquiring an additional 38,157 shares during the period. Andra AP fonden owned approximately 0.06% of Alnylam Pharmaceuticals worth $28,685,000 as of its most recent SEC filing.

Several other hedge funds and other institutional investors have also made changes to their positions in the company. MV Capital Management Inc. purchased a new stake in shares of Alnylam Pharmaceuticals in the 4th quarter worth $25,000. Caitong International Asset Management Co. Ltd purchased a new stake in shares of Alnylam Pharmaceuticals during the 3rd quarter valued at $29,000. HM Payson & Co. acquired a new stake in Alnylam Pharmaceuticals in the fourth quarter worth about $27,000. Golden State Wealth Management LLC increased its stake in Alnylam Pharmaceuticals by 100.0% during the 4th quarter. Golden State Wealth Management LLC now owns 72 shares of the biopharmaceutical company’s stock valued at $29,000 after purchasing an additional 36 shares in the last quarter. Finally, Manchester Capital Management LLC increased its holdings in shares of Alnylam Pharmaceuticals by 600.0% in the fourth quarter. Manchester Capital Management LLC now owns 84 shares of the biopharmaceutical company’s stock valued at $33,000 after purchasing an additional 72 shares during the period. Hedge funds and other institutional investors own 92.97% of the company’s stock.

Analyst Upgrades and Downgrades Several equities analysts have recently issued reports on the company. Truist Financial decreased their price objective on Alnylam Pharmaceuticals from $505.00 to $410.00 and set a “buy” rating for the company in a report on Monday, July 13th. Weiss Ratings upgraded shares of Alnylam Pharmaceuticals from a “sell (d+)” rating to a “hold (c-)” rating in a research report on Monday, May 4th. Wells Fargo & Company upped their target price on Alnylam Pharmaceuticals from $376.00 to $377.00 and gave the company an “equal weight” rating in a report on Friday, May 1st. Citigroup assumed coverage on shares of Alnylam Pharmaceuticals in a research report on Tuesday, May 19th. They issued a “buy” rating and a $380.00 price target for the company. Finally, Bank of America cut their price target on shares of Alnylam Pharmaceuticals from $460.00 to $410.00 and set a “buy” rating on the stock in a research note on Tuesday, July 14th. Two equities research analysts have rated the stock with a Strong Buy rating, eighteen have issued a Buy rating and six have issued a Hold rating to the stock. According to MarketBeat, Alnylam Pharmaceuticals currently has a consensus rating of “Moderate Buy” and a consensus target price of $455.31.

Check Out Our Latest Research Report on ALNY

Insider Activity at Alnylam Pharmaceuticals In other Alnylam Pharmaceuticals news, Director David E. I. Pyott sold 3,830 shares of the company’s stock in a transaction on Monday, June 1st. The stock was sold at an average price of $299.18, for a total transaction of $1,145,859.40. Following the transaction, the director owned 1,582 shares of the company’s stock, valued at approximately $473,302.76. This represents a 70.77% decrease in their position. The transaction was disclosed in a filing with the Securities & Exchange Commission, which can be accessed through the SEC website. The transaction was executed under a pre-arranged Rule 10b5-1 trading plan. 0.86% of the stock is owned by company insiders.

Alnylam Pharmaceuticals Stock Performance Shares of NASDAQ ALNY opened at $272.67 on Tuesday. Alnylam Pharmaceuticals, Inc. has a 12-month low of $266.27 and a 12-month high of $495.55. The company has a market cap of $36.40 billion, a PE ratio of 74.50 and a beta of 0.27. The stock has a 50 day simple moving average of $293.92 and a 200-day simple moving average of $319.69. The company has a current ratio of 3.13, a quick ratio of 3.06 and a debt-to-equity ratio of 0.94.

Alnylam Pharmaceuticals (NASDAQ:ALNY – Get Free Report) last released its earnings results on Thursday, April 30th. The biopharmaceutical company reported $1.99 earnings per share (EPS) for the quarter, beating analysts’ consensus estimates of $0.87 by $1.12. Alnylam Pharmaceuticals had a return on equity of 85.76% and a net margin of 11.72%.The company had revenue of $1.17 billion during the quarter, compared to analyst estimates of $1.12 billion. During the same quarter in the prior year, the company earned ($0.44) earnings per share. The company’s revenue for the quarter was up 96.4% on a year-over-year basis. On average, equities research analysts forecast that Alnylam Pharmaceuticals, Inc. will post 7.26 EPS for the current year.

About Alnylam Pharmaceuticals (Free Report)

Alnylam Pharmaceuticals, Inc (NASDAQ: ALNY) is a biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Founded to translate the scientific discovery of RNAi into new medicines, Alnylam applies small interfering RNA (siRNA) technology to silence disease-causing genes. The company develops therapies designed to provide durable disease modification by targeting underlying genetic drivers across a range of rare and more prevalent conditions.

Alnylam has advanced multiple siRNA-based products into commercialization, initially using lipid nanoparticle delivery and more recently employing GalNAc-conjugate chemistry to enable targeted delivery to the liver with subcutaneous dosing.

See Also Five stocks we like better than Alnylam Pharmaceuticals The Ugliest Stocks in the Market Just Got a Very Expensive Vote of Confidence Is Domino’s Stock Serving Up a Buying Opportunity? A $1T Black Hole: SpaceX Eyes Pentagon AI to Break Free Why Gold Miners Could Be the Market’s Biggest Comeback Story Want to see what other hedge funds are holding ALNY? Visit HoldingsChannel.com to get the latest 13F filings and insider trades for Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY – Free Report).

Receive News & Ratings for Alnylam Pharmaceuticals Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Alnylam Pharmaceuticals and related companies with MarketBeat.com's FREE daily email newsletter.

« PREVIOUS HEADLINEAndra AP fonden Grows Stake in McDonald’s Corporation $MCD

NEXT HEADLINE »Andra AP fonden Has $25.74 Million Holdings in AppLovin Corporation $APP

Posted by Defense World Staff on Jul 18th, 2026

Allspring Global Investments Holdings LLC grew its stake in Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY – Free Report) by 1.1% in the 1st quarter, according to its most recent filing with the SEC. The firm owned 281,793 shares of the biopharmaceutical company’s stock after buying an additional 3,136 shares during the quarter. Allspring Global Investments Holdings LLC owned 0.21% of Alnylam Pharmaceuticals worth $92,634,000 as of its most recent filing with the SEC.

Other large investors have also recently made changes to their positions in the company. Norges Bank acquired a new position in Alnylam Pharmaceuticals during the 4th quarter worth approximately $895,816,000. Qube Research & Technologies Ltd purchased a new stake in Alnylam Pharmaceuticals during the 3rd quarter worth about $369,943,000. AQR Capital Management LLC lifted its holdings in Alnylam Pharmaceuticals by 295.2% during the 3rd quarter. AQR Capital Management LLC now owns 744,008 shares of the biopharmaceutical company’s stock worth $336,009,000 after buying an additional 555,759 shares during the last quarter. Orbis Allan Gray Ltd boosted its position in shares of Alnylam Pharmaceuticals by 29.8% during the 2nd quarter. Orbis Allan Gray Ltd now owns 2,121,412 shares of the biopharmaceutical company’s stock worth $691,771,000 after acquiring an additional 486,489 shares in the last quarter. Finally, Jacobs Levy Equity Management Inc. boosted its position in shares of Alnylam Pharmaceuticals by 2,221.7% during the 3rd quarter. Jacobs Levy Equity Management Inc. now owns 410,342 shares of the biopharmaceutical company’s stock worth $187,116,000 after acquiring an additional 392,668 shares in the last quarter. 92.97% of the stock is currently owned by hedge funds and other institutional investors.

Insider Activity at Alnylam Pharmaceuticals In other Alnylam Pharmaceuticals news, Director David E. I. Pyott sold 3,830 shares of the stock in a transaction on Monday, June 1st. The stock was sold at an average price of $299.18, for a total transaction of $1,145,859.40. Following the sale, the director owned 1,582 shares in the company, valued at approximately $473,302.76. This represents a 70.77% decrease in their position. The sale was disclosed in a filing with the Securities & Exchange Commission, which can be accessed through this link. The transaction was executed under a pre-arranged Rule 10b5-1 trading plan. 0.86% of the stock is owned by corporate insiders.

Alnylam Pharmaceuticals Trading Down 5.5% Shares of ALNY opened at $267.47 on Friday. The firm’s 50-day moving average is $294.16 and its two-hundred day moving average is $321.11. The firm has a market capitalization of $35.71 billion, a P/E ratio of 73.08 and a beta of 0.27. The company has a current ratio of 3.13, a quick ratio of 3.06 and a debt-to-equity ratio of 0.94. Alnylam Pharmaceuticals, Inc. has a twelve month low of $266.43 and a twelve month high of $495.55.

Alnylam Pharmaceuticals (NASDAQ:ALNY – Get Free Report) last released its quarterly earnings data on Thursday, April 30th. The biopharmaceutical company reported $1.99 earnings per share for the quarter, beating the consensus estimate of $0.87 by $1.12. The firm had revenue of $1.17 billion during the quarter, compared to the consensus estimate of $1.12 billion. Alnylam Pharmaceuticals had a return on equity of 85.76% and a net margin of 11.72%.Alnylam Pharmaceuticals’s quarterly revenue was up 96.4% on a year-over-year basis. During the same period last year, the business posted ($0.44) EPS. On average, research analysts predict that Alnylam Pharmaceuticals, Inc. will post 7.26 earnings per share for the current fiscal year.

Analyst Upgrades and Downgrades Several equities research analysts recently weighed in on the company. Weiss Ratings upgraded Alnylam Pharmaceuticals from a “sell (d+)” rating to a “hold (c-)” rating in a research note on Monday, May 4th. Citigroup initiated coverage on shares of Alnylam Pharmaceuticals in a research note on Tuesday, May 19th. They issued a “buy” rating and a $380.00 price target on the stock. Morgan Stanley raised their price objective on shares of Alnylam Pharmaceuticals from $370.00 to $400.00 and gave the stock an “equal weight” rating in a report on Friday, July 10th. Truist Financial reduced their price objective on shares of Alnylam Pharmaceuticals from $505.00 to $410.00 and set a “buy” rating for the company in a research report on Monday. Finally, Wall Street Zen cut shares of Alnylam Pharmaceuticals from a “strong-buy” rating to a “buy” rating in a report on Tuesday, July 7th. Two analysts have rated the stock with a Strong Buy rating, eighteen have given a Buy rating and six have given a Hold rating to the company. According to MarketBeat.com, Alnylam Pharmaceuticals has an average rating of “Moderate Buy” and an average price target of $455.31.

Get Our Latest Report on Alnylam Pharmaceuticals

Alnylam Pharmaceuticals Company Profile (Free Report)

Alnylam Pharmaceuticals, Inc (NASDAQ: ALNY) is a biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Founded to translate the scientific discovery of RNAi into new medicines, Alnylam applies small interfering RNA (siRNA) technology to silence disease-causing genes. The company develops therapies designed to provide durable disease modification by targeting underlying genetic drivers across a range of rare and more prevalent conditions.

Alnylam has advanced multiple siRNA-based products into commercialization, initially using lipid nanoparticle delivery and more recently employing GalNAc-conjugate chemistry to enable targeted delivery to the liver with subcutaneous dosing.

Featured Articles Five stocks we like better than Alnylam Pharmaceuticals AST SpaceMobile Stock Sinks as SpaceX Fallout Rattles Space Sector Aehr Test Systems Stock Soars on Earnings, Eyes Over 150% Revenue Growth TSMC Just Gave AI Chip Bulls Another Reason to Stay Confident GE Aerospace Faces a Prove-It Moment in Q2 Earnings

Receive News & Ratings for Alnylam Pharmaceuticals Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Alnylam Pharmaceuticals and related companies with MarketBeat.com's FREE daily email newsletter.

« PREVIOUS HEADLINEBurlington Stores, Inc. $BURL Shares Sold by Allspring Global Investments Holdings LLC

NEXT HEADLINE »Allspring Global Investments Holdings LLC Lowers Stock Holdings in RTX Corporation $RTX

I initiate coverage of Alnylam Pharmaceuticals with a non-consensus BUY rating, citing the recent pullback as an overreaction. AMVUTTRA now holds a near-term commercial monopoly in TTR silencing, with strong clinical data and $890 million in Q1 2026 revenue. ALNY's robust cash position ($3.0 billion) and GAAP profitability materially reduce investment risk and support pipeline development.

AstraZeneca said Wainua “did not provide a statistically significant benefit” over a placebo in preventing deaths in patients with transthyretin amyloid cardiomyopathy. (Mikael Sjoberg/Bloomberg)

AstraZeneca shares slumped after the British-Swedish drugmaker announced a late-stage trial failure, denting the commercial prospects for its gene silencing drug. Analysts were seeing an overreaction.

Shares of Alnylam Pharmaceuticals (ALNY +0.44%) have dropped 24% so far this year (through June 30). The biotech company is still a darling of Wall Street analysts, with 14 of 29 analysts following it listing the stock as a buy and seven listing it as a strong buy as I write this. The average price target is $436, about 45% above the June 30 closing price.

Since 2018, the company has brought to market six RNA interference (RNAi) therapeutics, genetic medicines that use RNA interference to inhibit specific disease-associated genes. Here's why things are looking good for the stock, and one note of caution. 

Image source: Getty Images.

Alnylam is showing explosive revenue growth In the first quarter, product revenue surged 121% year over year to $1.04 billion, fueled primarily by Alnylam's transthyretin amyloidosis (ATTR) franchise, which grew 153% to $910 million. The driver for that growth was Amvuttra, an injectable therapy used to treat polyneuropathy (damage of multiple nerves throughout the body) in adults with hereditary transthyretin-mediated amyloidosis (hATTR).

Alnylam reported a huge jump in profitability, with earnings per share (EPS) of $1.51, compared to a loss per share of $0.14 in the same period a year ago.

The company's full-year 2026 guidance calls for combined net product revenue of between $4.9 billion and $5.3 billion, up 71% year over year at the midpoint. Alnylam is rapidly transitioning from a high-burn clinical biotech into a highly profitable, self-sustaining commercial powerhouse.

Today's Change

(

0.44

%) $

1.33

Current Price

$

302.37

Its products are expanding into new indications While Alnylam has done well in treating rare orphan diseases, its pipeline is on the cusp of penetrating mainstream, high-volume therapeutic markets. New data reinforces Amvuttra's profile as a robust, first-line treatment for cardiomyopathy, setting up a massive commercial launch. Cardiomyopathy affects 0.2% of the U.S. population, and in 40% of cases, leads to heart failure.

Partnering with Roche Holding (RHHBY 2.01%), Alnylam has also developed zilebesiran to treat hypertension, which impacts nearly half of the adults in the U.S.

Zilebesiran and nucresiran are in phase 3 trials -- the first to treat hypertension and the second to treat ATTR. Zilebesiran is unique in that it treats a common condition but in a different manner, as an RNAi therapeutic targeting liver-expressed angiotensinogen and requiring dosing only a few times a year. Nucresiran is in phase 3 trials both to treat hATTR with polyneuropathy, and to treat ATTR-CM.

Another therapy, cemdisiran, is licensed to Regeneron Pharmaceuticals. Among its phase 3 trials are one to treat the autoimmune disorder myasthenia gravis and another to treat the rare blood disease paroxysmal nocturnal hemoglobinuria.

Unlike traditional small molecules or biologics that face immediate patent cliffs, Alnylam's RNAi delivery platforms form a deep technological moat. And its RNAi approach allows it to quickly replicate success from one liver-targeted disease to another with highly predictable clinical translation.

It is aggressively maintaining this edge by deploying artificial intelligence (AI), notably via a strategic AI collaboration with private biotech company Inceptive Nucleics, to accelerate the discovery of next-generation RNAi structures.

A note of caution Even with its tumble this year, the stock is trading at 75 times trailing earnings. That's high for a biotech, particularly one that isn't consistently profitable. Much of the share price is already factoring in the continued commercial uptake of Amvuttra. However, aggressive pricing pressure from competitors such as Pfizer or BridgeBio Pharma could trigger a sharp drop in the price.

The experts are right In the long run, this is a solid stock, even though it trades at a relatively high valuation. The company is already profitable, is growing revenue and earnings, and has a few new therapies on the cusp of commercialization. It has a unique delivery system that will help it retain patent protection. And it is branching out beyond rare diseases into areas such as heart disease and high blood pressure, which have larger patient populations.

Going on company guidance, its forward price-to-earnings (P/E) ratio is just below 30, meaning the stock isn't that expensive given its potential.

NEW YORK & SAN FRANCISCO--(BUSINESS WIRE)-- #AI--Komodo Health®, the leader in AI-powered healthcare intelligence, today announced an expanded strategic partnership with Alnylam Pharmaceuticals (Nasdaq: ALNY), the leading RNAi therapeutics company to scale Marmot™, Komodo's analytics AI platform, across key enterprise functions at Alnylam. Built on the Healthcare Map®, the industry's most comprehensive view of more than 330 million de-identified patient journeys, Marmot helps organizations move beyon.

Taking full advantage of the stock market and investing with confidence are common goals for new and old investors, and Zacks Premium offers many different ways to do both.

Featuring daily updates of the Zacks Rank and Zacks Industry Rank, full access to the Zacks #1 Rank List, Equity Research reports, and Premium stock screens, the research service can help you become a smarter, more self-assured investor.

Zacks Premium also includes the Zacks Style Scores.

What are the Zacks Style Scores? The Zacks Style Scores, developed alongside the Zacks Rank, are complementary indicators that rate stocks based on three widely-followed investing methodologies; they also help investors pick stocks with the best chances of beating the market over the next 30 days.

Each stock is assigned a rating of A, B, C, D, or F based on their value, growth, and momentum characteristics. Just like in school, an A is better than a B, a B is better than a C, and so on -- that means the better the score, the better chance the stock will outperform.

The Style Scores are broken down into four categories:

Value ScoreValue investors love finding good stocks at good prices, especially before the broader market catches on to a stock's true value. Utilizing ratios like P/E, PEG, Price/Sales, Price/Cash Flow, and many other multiples, the Value Style Score identifies the most attractive and most discounted stocks.

Growth ScoreGrowth investors are more concerned with a stock's future prospects, and the overall financial health and strength of a company. Thus, the Growth Style Score analyzes characteristics like projected and historic earnings, sales, and cash flow to find stocks that will see sustainable growth over time.

Momentum ScoreMomentum trading is all about taking advantage of upward or downward trends in a stock's price or earnings outlook, and these investors live by the saying "the trend is your friend." The Momentum Style Score can pinpoint good times to build a position in a stock, using factors like one-week price change and the monthly percentage change in earnings estimates.

VGM ScoreIf you want a combination of all three Style Scores, then the VGM Score will be your friend. It rates each stock on their combined weighted styles, helping you find the companies with the most attractive value, best growth forecast, and most promising momentum. It's also one of the best indicators to use with the Zacks Rank.

How Style Scores Work with the Zacks Rank A proprietary stock-rating model, the Zacks Rank utilizes the power of earnings estimate revisions, or changes to a company's earnings outlook, to help investors create a successful portfolio.

#1 (Strong Buy) stocks have produced an unmatched +24% average annual return since 1988, which is more than double the S&P 500's performance over the same time frame. However, the Zacks Rank examines a ton of stocks, and there can be more than 200 companies with a Strong Buy rank, and another 600 with a #2 (Buy) rank, on any given day.

With more than 800 top-rated stocks to choose from, it can certainly feel overwhelming to pick the ones that are right for you and your investing journey.

That's where the Style Scores come in.

To maximize your returns, you want to buy stocks with the highest probability of success. This means picking stocks with a Zacks Rank #1 or #2 that also have Style Scores of A or B. If you find yourself looking at stocks with a #3 (Hold) rank, make sure they have Scores of A or B as well to ensure as much upside potential as possible.

As mentioned above, the Scores are designed to work with the Zacks Rank, so any change to a company's earnings outlook should be a deciding factor when picking which stocks to buy.

A stock with a #4 (Sell) or #5 (Strong Sell) rating, for instance, even one with Scores of A and B, will still have a declining earnings forecast, and a greater chance its share price will fall too.

Thus, the more stocks you own with a #1 or #2 Rank and Scores of A or B, the better.

Stock to Watch: Alnylam Pharmaceuticals (ALNY - Free Report) Cambridge, MA-based Alnylam Pharmaceuticals Inc. is a development-stage biopharmaceutical company focused on the development of novel therapeutics based on RNA interference (RNAi). The company’s pipeline of experimental RNAi therapeutics is focused across three strategic therapeutic areas – genetic medicines, cardio-metabolic disease, and hepatic infectious disease. In 2018, Onpattro (patisiran) received regulatory approvals in the United States and Europe for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. In 2019, the FDA approved Givlaari (givosiran) for acute hepatic porphyria (AHP). In 2020, the FDA approved Oxlumo (lumasiran) injection for subcutaneous use to treat primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. In 2022, the FDA approved Amvuttra (vutrisiran) for the treatment of adult patients with polyneuropathy of hATTR amyloidosis. Amvuttra is also approved by the European Commission (EC) for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. Its label has also been expanded to treat the cardiomyopathy indication.

ALNY is a #3 (Hold) on the Zacks Rank, with a VGM Score of A.

Momentum investors should take note of this Medical stock. ALNY has a Momentum Style Score of A, and shares are up 0.5% over the past four weeks.

For fiscal 2026, three analysts revised their earnings estimate upwards in the last 60 days, and the Zacks Consensus Estimate has increased $0.09 to $9.22 per share. ALNY boasts an average earnings surprise of +321.8%.

With a solid Zacks Rank and top-tier Momentum and VGM Style Scores, ALNY should be on investors' short list.

Alnylam Pharmaceuticals remains well-positioned in the TTR amyloidosis market, with a robust franchise and reliable clinical asset generation. Q1 2026 results showed 150% year-over-year TTR franchise growth despite insurance and pricing headwinds; Amvuttra continues to gain front-line share. ALNY maintains full-year TTR revenue guidance of $4.4B–$4.7B, with ongoing investments in earlier diagnosis and care coordination to expand the addressable market.

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced it will present new data analyses for vutrisiran in patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) at Heart Failure 2026, a scientific congress of the European Society of Cardiology, taking place May 9-12, 2026, in Barcelona, Spain.

ATTR-CM is a heterogeneous and rapidly progressive disease, and patients often present with complex clinical profiles. At Heart Failure 2026, Alnylam will present multiple new analyses from the Phase 3 HELIOS-B study of vutrisiran, which continue to support its use as a first-line treatment for patients with ATTR-CM, and further characterize its clinical profile across patients with a high disease burden, and in the context of concomitant therapies. Vutrisiran is the first and only transthyretin (TTR) silencer for ATTR-CM that delivers rapid knockdown of TTR at the source.

Additional analyses being presented at the congress include a pharmacodynamic analysis of TTR knockdown with vutrisiran across subgroups, a pooled safety analysis evaluating the incidence of TTR lowering with vitamin-A-related outcomes across clinical trial and real-world datasets, as well as the design of DemonsTTRate, a global, long-term observational study evaluating real-world use in patients with ATTR-CM.

Presentation Details

Design and Rationale of DemonsTTRate: A Global, Long-Term Observational Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
Session: Evolving management of cardiomyopathies: early detection, precision diagnostics, and emerging treatments (Moderated ePosters 3)
Sunday, May 10, 08:30 – 09:15 CEST, 2:30 – 3:15 A.M. EST
Presenting Author: Pablo Garcia-Pavia, Spain

Influence of Vutrisiran on Systolic Blood Pressure in ATTR-CM: Insights From HELIOS-B
Session: Cardiac amyloidosis: therapeutic strategies and clinical outcomes (Moderated ePosters 4)
Sunday, May 10, 15:30 – 16:15 CEST, 9:30 – 10:15 A.M. EST
Presenting Author: Awais Sheikh, United Kingdom

Effect of Vutrisiran in Patients with and without Atrial Fibrillation or Flutter: Analysis from HELIOS-B
Session: Advances in amyloidosis and hypertrophic cardiomyopathy: from diagnosis to treatment (Moderated ePosters 2)
Monday, May 11, 10:30 – 11:15 CEST, 4:30 – 5:15 A.M. EST
Presenting Author: Xiaowen Wang, United States of America

Treatment with Transthyretin-Lowering RNA Interference Therapeutics is Not Associated with Ocular or Other Clinical Events Due to Vitamin A Reduction: Pooled Analysis of Vutrisiran and Patisiran Data
Session: Advances in amyloidosis and hypertrophic cardiomyopathy: from diagnosis to treatment (Moderated ePosters 2)
Monday, May 11, 10:30 – 11:15 CEST, 4:30 – 5:15 A.M. EST
Presenting Author: Mathew Maurer, United States of America

Vutrisiran-Mediated Knockdown of Transthyretin in Patients with Transthyretin Amyloidosis
Session: Transthyretin amyloidosis: long-term outcomes and emerging therapies (Rapid Fire Abstracts)
Monday, May 11, 16:45 – 17:45 CEST, 10:45 – 11:45 A.M. EST
Presenting Author: Vincent Algalarrondo, France

Influence of Disease-Modifying Therapy on the Effectiveness of Vutrisiran in Transthyretin Cardiac Amyloidosis
Session: Transthyretin amyloidosis: long-term outcomes and emerging therapies (Rapid Fire Abstracts)
Monday, May 11, 16:45 – 17:45 CEST, 10:45 – 11:45 A.M. EST
Presenting Author: Arielle Abovich, United States of America

Consistent Efficacy of Vutrisiran Across Sexes in ATTR-CM, HELIOS-B Trial –Prespecified Sex Analysis
Session: Transthyretin amyloidosis: long-term outcomes and emerging therapies (Rapid Fire Abstracts)
Monday, May 11, 16:45 – 17:45 CEST, 10:45 – 11:45 A.M. EST
Presenting Author: Josephine Mansell, United Kingdom

Comorbidity Burden in Transthyretin Amyloidosis With Cardiomyopathy: Insights From the HELIOS-B Trial
Session: Risk factors and comorbidities (Moderated ePosters 3)
Tuesday, May 12, 08:30 to 09:15 CEST, 2:30 – 3:15 A.M. EST
Presenting Author: Sarah Birkhoelzer, United Kingdom

AMVUTTRA® (vutrisiran) INDICATIONS AND IMPORTANT SAFETY INFORMATION

Indications
In the EU, AMVUTTRA® (vutrisiran) is indicated for the treatment of:

hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Availability across the EU is subject to local reimbursement timelines.

Important Safety Information
Reduced Serum Vitamin A Levels and Recommended Supplementation
Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions
Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase.

For additional information about vutrisiran, please see the full Summary of Product Characteristics.

About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) is a transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source to address the underlying cause of transthyretin amyloidosis (ATTR). In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. It is approved as a treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in various countries, globally. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only silencer approved for the treatment of ATTR-CM and hATTR-PN.

About Transthyretin Amyloidosis (ATTR)
Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating, and fatal disease caused by pathogenic transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant. It is estimated that more than 500,000 people worldwide live with ATTR.

About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health.

Alnylam Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding the potential for AMVUTTRA to be used as a first-line treatment for ATTR-CM; and Alnylam’s ability to execute on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health, should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its “Alnylam 2030” strategy; Alnylam’s ability to successfully launch, market and sell Alnylam’s approved products globally, including AMVUTTRA; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam’s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

More News From Alnylam Pharmaceuticals, Inc.

− Reductions in All-Cause Mortality and Recurrent Cardiovascular Events Maintained Across Key Patient Subgroups, Including Patients Taking a Broad Range of Heart Failure Therapies –

− Pooled Analysis of Over 25,000 Patient-Years of Experience with TTR-Silencing RNAi Therapies Shows a Consistent Safety Profile, Including No Clinically Meaningful Ocular Effects of Vitamin A Lowering –

− DemonsTTRate Study Designed to Generate Long-Term Real-World Evidence in More Than 2,000 Patients with ATTR-CM −

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced new analyses from the HELIOS-B Phase 3 study of vutrisiran in patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), adding to the growing body of evidence supporting vutrisiran and reinforcing the durability of transthyretin (TTR) knockdown and its well-characterized safety profile. Vutrisiran is the first and only TTR silencer approved for ATTR-CM that is designed to deliver rapid knockdown of TTR at the source. The data presented at Heart Failure 2026, the annual congress of the Heart Failure Association of the European Society of Cardiology, show consistent clinical benefit across patient populations commonly encountered in clinical practice, including those with a high disease burden, supporting its use as a first-line treatment option for this rapidly progressive and life-threatening disease.

“The analyses presented at Heart Failure 2026 provide important insights into how vutrisiran performs across the patients we see in clinical practice, including those who present with features such as atrial fibrillation, low systolic blood pressure and a high comorbidity burden,” said Scott Solomon, M.D., Professor of Medicine at Harvard Medical School and cardiologist at Brigham and Women’s Hospital. “These new HELIOS-B analyses show that the clinical benefits of vutrisiran were maintained across these clinically complex patient groups, as well as in patients receiving background therapies, including TTR stabilizers and disease-modifying heart failure therapies, reinforcing both the consistency of the treatment effect and its relevance in real-world clinical practice. Taken together, these findings support the use of vutrisiran as a first-line treatment option for ATTR-CM across a broad range of patient populations.”

In patients with atrial fibrillation, representing approximately 65% of the HELIOS-B study population and associated with more advanced disease, vutrisiran significantly reduced the risk of all-cause mortality and recurrent cardiovascular (CV) events compared with placebo. Treatment effects were also maintained in patients with low systolic blood pressure (SBP), a higher-risk phenotype, with vutrisiran slowing the progressive decline in SBP observed over time. Clinical benefits were similarly consistent regardless of comorbidity burden or concomitant use of disease-modifying therapies, including tafamidis and heart failure medications such as SGLT2 inhibitors, MRAs, ß-blockers and ACEi/ARB/ARNI. Consistent effects were also observed in women, a historically underrepresented population in ATTR-CM trials.

A separate pooled analysis of clinical trial and post-marketing safety data evaluated the relationship between transthyretin-lowering RNAi therapies and vitamin A deficiency-related adverse events. Patients treated with vutrisiran and patisiran are suggested to take the recommended daily allowance of vitamin A. The analysis included more than 25,000 patient-years of treatment exposure across vutrisiran and patisiran programs. Rates of ocular adverse events potentially associated with vitamin A deficiency were low and comparable to placebo. No cases of clinically meaningful vitamin A deficiency were observed.

“Vitamin A plays an essential role in vision and other key physiological functions. While transthyretin contributes to its transport, multiple pathways support its delivery throughout the body,” said William S. Blaner, Ph.D., Professor of Nutritional Medicine at Columbia University and expert in vitamin A metabolism and transport. “The low and comparable to placebo rates of vitamin A deficiency-related adverse events observed in this large analysis provide strong reassurance that lowering transthyretin does not meaningfully increase these events in patients with ATTR amyloidosis.”

Alnylam also presented the design and rationale of the DemonsTTRate study, a global, prospective, observational study evaluating real-world outcomes in patients with ATTR-CM. The study is expected to enroll more than 2,000 patients and follow them for up to five years, generating longitudinal data on clinical outcomes, treatment patterns and healthcare utilization across routine clinical practice.

Across ATTR-CM and hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN), worldwide experience with vutrisiran to date exceeds 13,000 patient-years, reflecting a robust and expanding body of clinical evidence across both manifestations of the disease. To view Alnylam’s Heart Failure 2026 presentations please visit Capella.

AMVUTTRA® (vutrisiran) INDICATIONS AND IMPORTANT SAFETY INFORMATION

Indications

In the EU, AMVUTTRA® (vutrisiran) is indicated for the treatment of:

hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Availability across the EU is subject to local reimbursement timelines.

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation

Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions

Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase.

For additional information about vutrisiran, please see the full Summary of Product Characteristics.

About AMVUTTRA® (vutrisiran)

AMVUTTRA® (vutrisiran) is a transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source to address the underlying cause of transthyretin amyloidosis (ATTR). In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. It is approved as a treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in various countries, globally. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only silencer approved for the treatment of ATTR-CM and hATTR-PN.

About Transthyretin Amyloidosis (ATTR)

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating, and fatal disease caused by pathogenic transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant. It is estimated that more than 500,000 people worldwide live with ATTR.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health.

Alnylam Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding the potential for vutrisiran to be used as a first-line treatment for ATTR-CM; the potential efficacy of vutrisiran in patients who present with features such as atrial fibrillation, low systolic blood pressure and a high comorbidity burden; the number of patients who will be enrolled in the DemonsTTRate study, the duration of the follow-up period for those patients, and the data the study will generate; and Alnylam’s ability to execute on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health, should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its “Alnylam 2030” strategy; Alnylam’s ability to successfully launch, market and sell Alnylam’s approved products globally, including AMVUTTRA; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam’s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

More News From Alnylam Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc. (ALNY) Presents at Bank of America Global Healthcare Conference 2026 Transcript

-

− Collaboration is valued at up to $2B with upfront consideration of $30M −

− Alliance pairs Alnylam’s RNAi platform and 20+ years of proprietary data with Inceptive’s foundation models and AI expertise to catalyze progress beyond rational drug design –

− Partnership designed to accelerate timelines and unlock innovative oligonucleotide designs −

CAMBRIDGE, Mass. & PALO ALTO, Calif.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Inceptive Nucleics, Inc., which builds foundation models of life, today announced a strategic collaboration agreement to increase the pace of therapeutic innovation. The collaboration is valued at up to $2B with upfront consideration of $30M, including cash and the purchase of Inceptive equity. Inceptive is eligible to receive additional payments based on the achievement of preclinical, regulatory, and commercial sales milestones.

Alnylam and Inceptive Form Strategic AI Collaboration to Accelerate the Discovery of RNAi Therapeutics

Share By integrating Inceptive’s generative AI models with Alnylam’s R&D engine, Alnylam aims to accelerate the discovery of novel RNAi therapeutics as it advances ambitious pipeline expansion goals as part of its Alnylam 2030 strategy.

“We’re thrilled to partner with Inceptive to push the boundaries of what is possible in the discovery of RNAi medicines,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “Inceptive stands apart as one of the most visionary companies working at the intersection of AI and biology. It is led by pioneers of the AI revolution and driven by an ambitious mission to fundamentally reinvent how RNA medicines are designed. Together, we have an extraordinary opportunity to accelerate the creation of transformative medicines with a speed, ingenuity, and sophistication that simply has not been possible before.”

Inceptive’s foundation model learns the patterns underlying biology and hence can adapt to diverse therapeutic modalities without retraining. In joint exploratory work, the model achieved exceptional performance within weeks, uncovering meaningful biological insights from relatively small datasets to characterize siRNA molecules, the active ingredient in RNAi therapeutics.

"Most drug design still works through a process of trial and error, testing thousands of molecules and hoping something sticks,” said Jakob Uszkoreit, Inceptive co-founder and CEO. “Inceptive was built on a different premise: that life follows rules of such complexity that only AI can learn them. Alnylam’s breakthrough platform and scientific vision are an ideal match for AI. Together, we’re not just accelerating drug discovery; we’re changing the way we understand and improve life.”

Generalizable Platforms Open New Therapeutic Design Spaces

The collaboration pairs Alnylam’s RNAi leadership with Inceptive’s foundation models and AI expertise to catalyze and accelerate progress in nucleic-acid based drug design. Inceptive focuses on developing models for sequence-based medicines such as RNAi therapeutics, which were pioneered by Alnylam.

Alnylam’s platform: An R&D engine that has produced six approved drugs, backed by 20+ years of proprietary siRNA data. Inceptive’s foundation models of life: AI models for sequence-based medicines that generalize across programs and continuously improve. The collaboration seeks to advance siRNA design, by modeling target mRNAs and jointly exploring sequence space and novel chemical modifications to enhance potency and efficacy, and by predicting top-performing therapeutic candidates in preclinical models for further development by Alnylam. The goal is to help Alnylam prioritize the most promising molecules and improve experimental productivity.

By combining Alnylam’s deep biological expertise with Inceptive’s frontier models, the alliance advances Alnylam’s ambition to unlock new therapeutic innovation through frontier AI. The collaboration gives Alnylam access to Inceptive’s AI expertise and talent, including CEO Jakob Uszkoreit, co-inventor of the Transformer architecture (i.e., the “T” in ChatGPT), and pioneers of scalable, AI-enabled wet-lab training data generation methods.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. For more information, please visit www.alnylam.com or follow Alnylam on X, LinkedIn, Facebook, Instagram, or YouTube.

About Inceptive

Inceptive builds AI foundation models of life that extrapolate from data to design breakthrough biological medicines beyond the reach of nature and traditional drug discovery. The company’s antedisciplinary team of AI researchers, biochemists, and engineers trains models on diverse biological data and designs experiments to generate missing training data at unprecedented scale. Inceptive partners with leading drugmakers to customize molecule-design models for discovery of sequence-based medicines such as siRNA, ASOs, peptides, and mRNA for applications such as in-vivo cell therapies. Founded in 2021 and backed by a16z, NVIDIA, S32 and Obvious, Inceptive is headquartered in Palo Alto with offices in Berlin and Zurich. Learn more at inceptive.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding the potential for Alnylam’s collaboration with Inceptive to achieve the goals for which it was established, including to increase the pace of therapeutic innovation, to catalyze and accelerate progress in nucleic-acid based drug design, accelerate timelines and unlock innovative oligonucleotide designs, and to accelerate the creation of transformative medicines with a speed, ingenuity and sophistication that has not been possible before; Alnylam’s ability to accelerate the discovery of novel RNAi therapeutics and to push the boundaries of what is possible in the discovery of RNAi medicines; and Alnylam’s ability to achieve the goals in its Alnylam 2030 strategy should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its Alnylam 2030 strategy; Alnylam’s ability to successfully launch, market and sell Alnylam’s approved products globally, including AMVUTTRA; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam’s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products, including Roche, Novartis, Sanofi, and Regeneron; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

More News From Alnylam Pharmaceuticals, Inc. and Inceptive

Back to Newsroom

The partnership aims to enhance the discovery of RNA interference (RNAi) therapeutics by integrating Alnylam’s RNAi platform with Inceptive’s AI expertise, potentially accelerating timelines for innovative drug designs.

Alnylam aims to accelerate the discovery of novel RNAi therapeutics as it advances ambitious pipeline expansion goals as part of its Alnylam 2030 strategy.

Alnylam and Inceptive Nucleics CollaborationThe collaboration is valued at up to $2 billion, with an upfront consideration of $30 million, including cash and equity.

By combining their strengths, Alnylam and Inceptive aim to unlock new therapeutic designs and improve the productivity of experimental processes.

ALNY Technical Analysis: Trend, RSI And Key SupportCurrently, Alnylam’s stock price is $296.00, which places it about 0.5% above its 20-day simple moving average (SMA) of $294.61.

The stock is trading 3.8% below its 50-day SMA of $307.82, indicating a bearish trend as the 20-day SMA is below the 50-day SMA, suggesting potential resistance at higher levels.

The Relative Strength Index (RSI) is at 46.01, indicating neutral momentum, suggesting that the stock is neither overbought nor oversold at this time.

This neutral reading implies that there may be room for movement in either direction, depending on upcoming catalysts or market conditions.

Key Resistance: $317.50 — a nearby level where rebounds can stall, closely tied to recent pivot zones. Key Support: $284.50 — a level where buyers previously stepped in, aligning with the 52-week low zone. Analyst Consensus & Recent Actions: The stock carries a Buy rating with an average price target of $438.47. Recent analyst moves include:

Citigroup: Initiated with Buy (Target $380.00) (May 19) HC Wainwright & Co.: Buy (Maintains Target to $510.00) (May 5) RBC Capital: Outperform (Lowers Target to $445.00) (May 1) How Alnylam (ALNY) Ranks On Value And MomentumBelow is the Benzinga Edge scorecard for Alnylam Pharmaceuticals, highlighting its strengths and weaknesses compared to the broader market:

Value: 6.83 — The stock is trading at a premium relative to peers. Momentum: 10.33 — Indicates strong upward price movement in recent sessions. The Verdict: Alnylam Pharmaceuticals’s Benzinga Edge signal reveals a growth-heavy profile, with strong momentum but a premium valuation. This suggests that while the stock is performing well, it may be priced for continued success in the market.

ALNY Top ETF Holdings And Passive Flow ExposureALNY Stock Price Activity: Alnylam Pharmaceuticals shares were up 1.79% at $297.81 at the time of publication on Thursday, according to Benzinga Pro data.

Photo via Shutterstock

Market News and Data brought to you by Benzinga APIs

© 2026 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

To add Benzinga News as your preferred source on Google, click here.

Alnylam Stock Soars 65%: Find Out What’s Behind the GainsAlnylam Pharmaceuticals NASDAQ: ALNY executives outlined the company’s 2030 financial targets, TTR franchise strategy and near-term pipeline priorities during a Goldman Sachs event hosted by analyst Salveen Richter.

Jeff Poulton, Alnylam’s chief financial officer, said the company’s “Alnylam 2030” plan is its fourth set of five-year goals and is intended to clarify priorities for both investors and employees. The plan calls for a 25% compound annual growth rate in total revenue, including product sales, collaboration revenue and royalty revenue, as well as a 30% non-GAAP operating margin across the period.

Get ALNY alerts:

3 biotech powerhouses poised to thrive amid sector reboundPoulton said Alnylam is defining leadership in TTR by both peak franchise revenue in 2030 and cumulative revenue over the five-year period. He said AMVUTTRA is expected to be the primary TTR revenue driver through 2030, given the timing of nucresiran’s phase 3 studies. Poulton said Alnylam expects the nucresiran polyneuropathy study could support market entry around 2028, while cardiomyopathy is more likely around 2030.

“For 2030, that’s primarily going to be AMVUTTRA,” Poulton said, referring to the company’s TTR revenue contribution. He added that nucresiran could become a more important growth and margin driver after 2030 if it achieves the profile Alnylam expects.

Margin Targets Reflect Royalty Burden and R&D Spending Argenx's 28% Surge & Promising Product Propel Investor ConfidencePoulton said the company’s 30% non-GAAP operating margin target was below where market expectations had been, noting that consensus had been closer to 50% operating margins in 2030. He said the company’s gross margin profile is affected by the AMVUTTRA royalty burden, with market consensus around 75% gross margins.

Alnylam also plans to invest heavily in research and development to diversify beyond TTR. Poulton said the company is targeting R&D reinvestment of about 30% of revenue across the period, including capacity for business development.

Pushkal Garg, Alnylam’s head of development, said the company’s primary focus will remain its internal pipeline, which includes about 25 drugs in development and an expected three or four new investigational new drug applications each year. He said Alnylam will consider external opportunities, but only with a “very high bar,” emphasizing early-stage assets that advance delivery objectives or fit with the company’s commercial footprint.

AMVUTTRA Launch and TTR Market Expansion Poulton said Alnylam’s revenue guidance for the year is $4.4 billion to $4.7 billion. He said first-quarter performance was affected by phasing issues in both the U.S. and international markets, including insurance reauthorization in January, fewer shipping Wednesdays compared with the fourth quarter and a German pricing adjustment tied to cardiomyopathy launch dynamics. He said those factors are not expected to affect the rest of the year.

In the U.S., Poulton said Alnylam has achieved more than 90% first-line patient access to AMVUTTRA from a payer perspective. He said the company is focused on expanding the prescriber base and improving diagnosis, including through a partnership with Viz.ai to use an artificial intelligence algorithm connected to electronic health records to review echocardiogram tests in five health networks.

Poulton said Alnylam estimates the TTR opportunity at 200,000 or more patients in the U.S. and 500,000 or more globally, with about 20% of the prevalent population currently treated. Garg said he expects the silencer class to become foundational therapy, beginning with AMVUTTRA and potentially followed by nucresiran, which he described as a twice-yearly drug targeting 95% knockdown.

Nucresiran and Combination Therapy On nucresiran’s cardiomyopathy study, Garg said it is largely designed as a combination therapy trial, with many patients expected to be on background stabilizer therapy. He said Alnylam used patient-level data from HELIOS-B to understand event rates and trial design. Garg also said enrollment has been ahead of schedule and that patients appear somewhat milder than those in HELIOS-B, prompting the company to use a predefined option to increase sample size by 500 patients.

Poulton said Alnylam has seen some AMVUTTRA and tafamidis combination use, primarily in the Medicare fee-for-service market, where payer management is less restrictive. He said combination use is generally restricted in the commercial and Medicare Advantage segments. Poulton added that a larger opportunity for combination therapy could emerge when tafamidis becomes generic, which he said is expected in the middle of 2031 based on Pfizer’s recently announced settlements.

Near-Term Pipeline Readouts Garg highlighted several programs expected to generate data in the near term. In metabolic disease, Alnylam’s ACVR1C, or ALK7, program targets adipose tissue and is intended to reduce visceral fat while preserving muscle mass. Garg said the obesity and overweight market is likely to segment over time, creating possible roles for the drug as monotherapy or in combination with other agents.

In Huntington’s disease, Garg said Alnylam’s program targets both full-length mutant huntingtin and the exon 1 fragment, which he said is viewed by many academics as important in the propagation of huntingtin aggregates. He said the company hopes to show high levels of knockdown, infrequent dosing and favorable safety data later this year, potentially supporting movement into pivotal trials.

Garg also discussed a plasminogen-targeting program that could act as a universal hemostatic agent by stabilizing clots without increasing thrombosis risk, based on genetic and preclinical data. Alnylam’s first indication is hereditary hemorrhagic telangiectasia, with bleed data expected later this year. The company has also initiated work in von Willebrand disease and heavy menstrual bleeding.

In central nervous system disease, Garg said mivelsiran targets amyloid precursor protein and is being studied in early-onset Alzheimer’s disease and cerebral amyloid angiopathy. He said enrollment completion in the CAA study is targeted for the second quarter, with data expected in late 2027 or early 2028. Alnylam is also advancing a tau-targeting siRNA in phase 1 development.

About Alnylam Pharmaceuticals NASDAQ: ALNYAlnylam Pharmaceuticals, Inc NASDAQ: ALNY is a biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Founded to translate the scientific discovery of RNAi into new medicines, Alnylam applies small interfering RNA (siRNA) technology to silence disease-causing genes. The company develops therapies designed to provide durable disease modification by targeting underlying genetic drivers across a range of rare and more prevalent conditions.

Alnylam has advanced multiple siRNA-based products into commercialization, initially using lipid nanoparticle delivery and more recently employing GalNAc-conjugate chemistry to enable targeted delivery to the liver with subcutaneous dosing.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected].

Should You Invest $1,000 in Alnylam Pharmaceuticals Right Now?Before you consider Alnylam Pharmaceuticals, you'll want to hear this.

MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and Alnylam Pharmaceuticals wasn't on the list.

While Alnylam Pharmaceuticals currently has a Moderate Buy rating among analysts, top-rated analysts believe these five stocks are better buys.

View The Five Stocks Here

Just getting into the stock market? These 10 simple stocks can help beginning investors build long-term wealth without knowing options, technicals, or other advanced strategies.

Get This Free Report

ATHENS, Greece--(BUSINESS WIRE)--GENESIS Pharma, a leading regional biopharma company focused on the commercialization of innovative medicines in Europe, today announced the expansion of its longstanding commercial partnership with Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY), the leading RNAi therapeutics company. The extended agreement broadens the geographical reach of the collaboration to include four Nordic markets -Denmark, Finland, Norway, and Sweden- alongside the thirteen markets in Southeast Europe already within the scope of the partnership. GENESIS Pharma shall commercialize a portfolio of RNAi therapeutics for serious cardiomyopathy conditions and rare genetic diseases across this expanded territory.

The partnership, established in 2019 for Southeast Europe, has progressively evolved to encompass a broader portfolio and geographic footprint, covering Greece, Cyprus, Bulgaria, Romania, Slovenia, Croatia, Serbia, Bosnia and Herzegovina, Albania, the Republic of North Macedonia, Montenegro, Malta, and Kosovo. This latest strategic expansion reinforces both companies’ shared commitment to improving patient access to innovative therapies across Europe, particularly in regions where unmet medical needs remain significant.

Norton Oliveira, Senior Vice-President and Head of Partner and Emerging Markets at Alnylam Pharmaceuticals stated: “We are proud of our strong and established partnership with GENESIS Pharma and are delighted to broaden this across the Nordic region. Our commitment is to deliver transformational impact for patients across the world. By working alongside GENESIS Pharma, we can continue to address the needs of even more patients and their families, enabling them to benefit from Alnylam’s innovative RNAi therapeutics.”

Constantinos Evripides, Managing Director of GENESIS Pharma stated: “Since 2019, we have been working closely with Alnylam to ensure patient access across Southeast Europe. The expansion in the Nordic countries marks a significant milestone in our partnership and reflects our ongoing efforts to strengthen our European footprint, building on our three-decade legacy and commitment to biotechnology. By combining Alnylam’s pioneering science with our strong regional expertise in bringing innovation closer to patients, we continue to expand our reach and enhance the value we deliver across healthcare systems. We are honored by the trust Alnylam has placed in our company and our people.”

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today.1 Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.2 By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made.1 This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About GENESIS Pharma

GENESIS Pharma is a European biopharma company focused on the commercialization of innovative biopharmaceutical products targeting severe and rare diseases, currently covering 24 countries in Europe. Established in 1997, GENESIS Pharma was among the first pharmaceutical companies in the region to specialize in the marketing, sales and distribution of biopharmaceutical products. GENESIS Pharma maintains a strong portfolio in therapeutic areas with high unmet medical need through long standing strategic alliances with some of the leading global biopharma companies. For more information, please visit www.genesispharma.com and follow us on LinkedIn.

REFERENCES

1 Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498.

2 Zamore P. Cell. 2006;127(5):1083-1086.

Key Takeaways ALNY's Amvuttra generated $889.9M in Q1 2026 sales, up 187% year over year, comprising 76% of total revenues.ALNY won U.S. and EU approval to expand Amvuttra into ATTR-CM, broadening eligible patients.ALNY faces ATTR-CM competition from Pfizer's Vyndaqel/Vyndamax and BridgeBio's Attruby. Alnylam Pharmaceuticals (ALNY - Free Report) is a commercial-stage company that currently markets four drugs. The company first received regulatory approval in 2018 for Onpattro to treat adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis. Subsequently, ALNY received approval for Givlaari (acute hepatic porphyria), Oxlumo (primary hyperoxaluria type 1) and Amvuttra (polyneuropathy of hATTR amyloidosis).

In 2025, Alnylam received approval in the United States and the EU for the label expansion of Amvuttra to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. Following the label expansion, Amvuttra became the first and only RNAi therapeutic approved in the United States and the EU for both ATTR-CM and hATTR-PN in adults, marking a significant milestone.

Currently, Amvuttra (vutrisiran), its newest drug, is Alnylam’s primary top-line driver. The drug generated $889.9 million in global sales in the first quarter of 2026, representing 187% year-over-year growth. The figure accounted for 76% of Alnylam’s total revenues generated in the quarter. The drug’s solid uptake has been driven by increased patient demand, mainly in ATTR-CM patients in the United States, as well as several patients switching from Onpattro.

This label expansion is expected to continue boosting Amvuttra sales in the quarters ahead. Alnylam also expects approvals for Amvuttra for the ATTR-CM indication in other geographies this year. This will expand the eligible patient population for the drug, driving substantial growth for the company in the future.

ALNY’s Competition in the Market for ATTR TreatmentsAlnylam’s Amvuttra faces notable competition in the ATTR-CM market from Pfizer’s (PFE - Free Report) Vyndaqel/Vyndamax (tafamidis) and BridgeBio’s (BBIO - Free Report) Attruby (acoramidis), both of which are already approved for this indication. While Amvuttra is positioned with a differentiated clinical profile, Pfizer’s and BridgeBio’s therapies carry the advantage of oral administration and comparatively lower list prices in the United States. These factors could influence prescribing decisions and pose challenges to Alnylam’s competitive positioning in this space.

Vyndaqel is one of the key in-line products that has driven improvement in Pfizer’s revenues in the first quarter of 2026. Global Vyndaqel family revenues of $1.6 billion rose 8% year over year in the quarter, primarily driven by international growth on the back of higher demand due to increases in diagnosis and treatment rates. Pfizer’s Vyndaqel family includes global revenues from Vyndaqel as well as revenues for Vyndamax in the United States and Vynmac in Japan.

Approved in late 2024, Attruby is BridgeBio’s only marketed product. The drug generated sales worth $180.6 million in the first quarter of 2026, up significantly year over year, driven by solid uptake. BridgeBio is also currently evaluating acoramidis for the prevention of early-stage variant transthyretin amyloidosis in a late-stage study.

ALNY’s Stock Price, Valuation and EstimatesShares of Alnylam have plunged 27.3% so far this year compared with the industry’s 4% decline. The stock has also underperformed the sector and the S&P 500 index during the same time frame, as seen in the chart below.

ALNY Stock Price MovementImage Source: Zacks Investment Research

From a valuation standpoint, Alnylam stock is expensive. Going by the price/sales ratio, the company’s shares currently trade at 9.33 trailing 12-month sales per share, higher than 2.25 for the industry. However, the stock is also trading much below its five-year mean of 18.40.

ALNY Stock ValuationImage Source: Zacks Investment Research

Estimates for Alnylam’s 2026 earnings have improved from $9.13 to $9.22 per share in the past 60 days, while the estimates for 2027 earnings have deteriorated from $14.59 to $13.68 over the same timeframe.

ALNY Estimate MovementImage Source: Zacks Investment Research

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