- A Look At Alnylam Pharmaceuticals (ALNY) Valuation After Recent Share Price Weakness
May 8, 2026
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Alnylam Pharmaceuticals (ALNY) has drawn fresh attention after recent share price weakness. The stock is down over the past week, month, and past 3 months, prompting investors to reassess its current valuation.
See our latest analysis for Alnylam Pharmaceuticals.
That pullback sits against a weaker short term picture, with the share price down 26.05% year to date, but a stronger long term record shown by a 122.62% five year total shareholder return. Recent moves suggest momentum has been fading as investors reassess risk and growth expectations around the current US$295.91 share price.
If this kind of volatility has you looking wider across healthcare, it could be a good moment to scan other opportunities in AI driven drug development and treatment using the 35 healthcare AI stocks
So with Alnylam shares sitting at US$295.91, a 26.05% year to date decline and a five year total return of 122.62%, is this recent weakness a chance to buy in, or is the market already pricing in future growth?
Most Popular Narrative: 39.8% Undervalued
Simply Wall St's most followed narrative puts Alnylam's fair value at $491.92, well above the recent $295.91 close, framing the current pullback as a valuation gap to scrutinize.
Advancements in Alnylam's pipeline (e.g., fast-tracked nucresiran for ATTR-CM, mivelsiran in Alzheimer's, new programs in diabetes and bleeding disorders) demonstrate increasing R&D efficiency and accelerate time-to-market, potentially driving future step-function increases in both revenue and operational leverage as more assets advance or launch.
Read the complete narrative.
Curious what kind of revenue run rate, margin profile, and future earnings multiple are baked into that fair value, and how much hinges on AMVUTTRA versus the broader RNAi pipeline? The full narrative lays out the numbers, the timing, and the tension between growth ambitions and analyst disagreement in a way the share price alone does not.
Result: Fair Value of $491.92 (UNDERVALUED)
Have a read of the narrative in full and understand what's behind the forecasts.
However, this hinges on AMVUTTRA and the wider TTR franchise holding up, while pricing pressure and higher royalty obligations could squeeze margins and reset growth expectations.
Find out about the key risks to this Alnylam Pharmaceuticals narrative.
Another Angle On Value
On simple P/E math, Alnylam looks expensive. The stock trades around 73.4x earnings, compared with a fair ratio estimate of 29.8x, the US Biotechs average of 17.8x and a 30.4x peer average. That gap suggests investors are paying up today for strong growth expectations and taking on valuation risk if those expectations shift.
Story Continues
See what the numbers say about this price — find out in our valuation breakdown.NasdaqGS:ALNY P/E Ratio as at May 2026
Next Steps
With mixed signals on valuation, growth, and risk, it makes sense to review the full data set, pressure test your assumptions, and weigh both sides through the 3 key rewards and 1 important warning sign.
Looking for more investment ideas?
If Alnylam has sharpened your focus, do not stop here. The market is full of other stocks with different risk, income, and value profiles worth your attention.
Spot potential bargain opportunities before they get crowded by checking companies on the 51 high quality undervalued stocks. Strengthen your income stream by reviewing stocks in the 12 dividend fortresses. Prioritize resilience by focusing on companies highlighted in the 72 resilient stocks with low risk scores.
This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.
Companies discussed in this article include ALNY.
Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team@simplywallst.com
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- ClearBridge Large Cap Growth adds, and exits multiple positions in Q1
May 7, 2026
* ClearBridge’s Large Cap Growth ESG Strategy outperformed its Russell 1000 Growth Index benchmark in Q1 2026, as the broader market sell-off driven by Middle East tensions pushed the S&P 500 down 4.3% and the Russell 1000 Growth Index lower by 9.8%.
* The fund initiated new positions in Texas Instruments (NASDAQ: TXN [https://seekingalpha.com/symbol/TXN]), Roche (RHHBY [https://seekingalpha.com/symbol/RHHBY]), Alnylam Pharmaceuticals (NASDAQ: ALNY [https://seekingalpha.com/symbol/ALNY]), and Blackstone (NYSE: BX [https://seekingalpha.com/symbol/BX]) during Q1 2026.
* In Q1 2026, the fund exited PayPal (NASDAQ: PYPL [https://seekingalpha.com/symbol/PYPL]), Equinix (NASDAQ: EQIX [https://seekingalpha.com/symbol/EQIX]), and Marvell Technology (NASDAQ: MRVL [https://seekingalpha.com/symbol/MRVL]).
* Source [https://seekingalpha.com/article/4899307-clearbridge-large-cap-growth-esg-strategy-q1-2026-commentary].
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- Alnylam to Webcast Presentations at Upcoming Investor Conferences
May 7, 2026
CAMBRIDGE, Mass., May 07, 2026--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the following upcoming investor conferences:
BofA Securities Healthcare Conference on Wednesday, May 13, 2026, at 10:40 am PT (1:40 pm ET) in Las Vegas, Nevada Goldman Sachs Annual Healthcare Conference on Tuesday, June 9, 2026, at 11:20 am ET in Miami, Florida.
A live audio webcast of each presentation will be available on the Investors section of the Company’s website, www.alnylam.com/events. Replays will be available on the Alnylam website within 48 hours after each event.
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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260507963806/en/
Contacts
Alnylam Pharmaceuticals, Inc.
Christine Akinc
(Investors and Media)
617-682-4340
Josh Brodsky
(Investors)
617-551-8276
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- MBX Biosciences Announces Appointment of Mark Soued as Chief Commercial Officer and Inducement Grant Award
May 7, 2026
MBX Biosciences, Inc.
CARMEL, Ind. and BURLINGTON, Mass., May 07, 2026 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc. (Nasdaq: MBX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel precision peptide therapies for the treatment of endocrine and metabolic disorders, today announced the appointment of Mark R. Soued, MBA, as Chief Commercial Officer (CCO). Mr. Soued brings more than two decades of commercial leadership across global biopharmaceutical organizations, with deep expertise in product launches, market access, and building high-performing commercial teams.2026-05-07 -- MBX -- Mark Soued New CCO -- Headshot
"Mark is an exceptional commercial leader with a demonstrated ability to build and scale commercial organizations and bring products to market," said Kent Hawryluk, President and Chief Executive Officer of MBX Biosciences. "His track record of delivering landmark product launches and generating significant revenue growth at organizations like Alnylam and Pfizer makes him uniquely positioned to lead MBX’s commercialization efforts as we continue to advance our pipeline of clinically validated, proprietary Precision Endocrine Peptides™."
"I am excited to join MBX Biosciences at such a pivotal moment in the company's development," said Mr. Soued. "The team has built a differentiated pipeline and world-class team with the potential to meaningfully address unmet needs in endocrine and metabolic diseases. I look forward to helping shape and execute a commercial strategy that brings these therapies to the patients who need them most."
Mark Soued is a seasoned commercial biopharma executive with extensive experience building and growing global commercial organizations overseeing multiple product launches, sales and marketing, market access, and lifecycle management. Most recently, Mr. Soued served as Senior Vice President, Head of US at Alnylam Pharmaceuticals, where he led the Company’s US amyloidosis business, including the category-defining launch of AMVUTTRA® in ATTR cardiomyopathy. Prior to that, he served as Senior Vice President, Head of Global Commercial at Alnylam, where he built a global commercial organization spanning six functions and four inline products. Earlier in his career, Mr. Soued held a series of senior commercial leader roles at Pfizer, including Regional President for North America's Hospital Business Unit, Vice President and Global Rare Cardiology Franchise Head, and multiple international leadership positions across Asia Pacific and Northwestern Europe. He led the successful US launch of VYNDAQEL®/VYNDAMAX® and negotiated a multi-billion US government contract for PAXLOVID®, among other milestones. Mr. Soued holds an MBA from Northwestern University's Kellogg School of Management and a B.S. in Finance from the University of Colorado. He is fluent in English and French.
Story Continues
Inducement Grant
In connection with the appointment of Mr. Soued as Chief Commercial Officer, on May 1, 2026, MBX granted Mr. Soued an inducement equity award, which was approved by the Company’s board of directors in accordance with Nasdaq Listing Rule 5635(c)(4). The inducement award consisted of non-qualified stock options to purchase an aggregate of 160,000 shares of the company’s common stock with an exercise price of $29.78 per share, which is equal to the closing price of the company’s common stock as reported by Nasdaq on May 1, 2026. The option has a 10-year term and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of April 29, 2026, and the remainder vesting in 36 equal monthly installments for the three years thereafter, subject to Mr. Soued’s continued service. The award is subject to the terms and conditions of the company’s 2026 Inducement Plan and the terms and conditions of the stock option agreement covering the grant.
AboutMBX Biosciences
MBX Biosciences is a biopharmaceutical company focused on the discovery, development and commercialization of novel precision peptide therapies based on its proprietary PEP™ platform, for the treatment of endocrine and metabolic disorders. The Company is advancing a pipeline of novel candidates for endocrine and metabolic disorders with clinically validated targets, established endpoints for regulatory approval, significant unmet medical needs and large potential market opportunities. The Company’s pipeline includes canvuparatide (MBX 2109) for the treatment of chronic hypoparathyroidism (HP) preparing for Phase 3 development; an obesity portfolio that includes MBX 4291 in Phase 1 development, as well as multiple discovery and pre-clinical obesity candidates; and imapextide (MBX 1416) for the treatment of post-bariatric hypoglycemia (PBH) in Phase 2 development. The Company is based in Carmel, Indiana. To learn more, please visit the Company website at www.mbxbio.com and follow it on LinkedIn.
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, each as amended. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, but are not limited to, express or implied statements regarding: MBX Biosciences’ expectations regarding Mr. Soued’s ability to help advance commercialization, MBX Biosciences’ expectations regarding the further advancement of its pipeline of programs in endocrine and metabolic disorders; and MBX Biosciences’ plans for delivery of differentiated endocrine and metabolic compounds to underserved patients.
Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect MBX Biosciences’ business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to the Company’s research and development activities; uncertainties relating to preclinical and clinical development activities; uncertainties relating to preclinical and clinical development activities; the risk that preliminary results may not be indicative of later results; MBX Biosciences’ ability to attract, integrate and retain key personnel; as well as other risks described in “Risk Factors,” in MBX Biosciences’ Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC), as well as subsequent filings with the SEC. MBX Biosciences expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.
MBX uses and intends to continue to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company's Investor Relations website, in addition to following the Company's press releases, SEC filings, public conference calls, presentations, and webcasts.
Media Contact:
George Shea
We. Communications
gshea@wecommunications.com
Investor Contact:
Jim DeNike
MBX Biosciences
jdenike@mbxbio.com
A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/b8b07cf9-9fda-4eb3-8581-f664b7c317b1
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- Alnylam to Webcast Presentations at Upcoming Investor Conferences
May 7, 2026 · businesswire.com
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the following upcoming investor conferences: BofA Securities Healthcare Conference on Wednesday, May 13, 2026, at 10:40 am PT (1:40 pm ET) in Las Vegas, Nevada Goldman Sachs Annual Healthcare Conference on Tuesday, June 9, 2026, at 11:20 am ET in Miami, Florida. A live audio webcast of each presentation will be.
- ALNYLAM TO WEBCAST PRESENTATIONS AT UPCOMING INVESTOR CONFERENCES
May 7, 2026
CAMBRIDGE, MASS.--(BUSINESS WIRE)--ALNYLAM PHARMACEUTICALS, INC. (NASDAQ: ALNY), THE LEADING RNAI THERAPEUTICS COMPANY, ANNOUNCED TODAY THAT MANAGEMENT WILL PRESENT COMPANY OVERVIEWS AT THE FOLLOWING UPCOMING INVESTOR CONFERENCES: BOFA SECURITIES HEALTHCARE CONFERENCE ON WEDNESDAY, MAY 13, 2026, AT 10:40 AM PT (1:40 PM ET) IN LAS VEGAS, NEVADA GOLDMAN SACHS ANNUAL HEALTHCARE CONFERENCE ON TUESDAY, JUNE 9, 2026, AT 11:20 AM ET IN MIAMI, FLORIDA. A LIVE AUDIO WEBCAST OF EACH PRESENTATION WILL BE.
- Tenaya Therapeutics Reports First Quarter 2026 Financial Results and Provides Business Update
May 6, 2026
Tenaya Therapeutics, Inc.
One-Year Cohort 1 Data and Initial Cohort 2 Data from RIDGE™-1 Phase 1b/2 Trial of TN-401 for PKP2-Associated ARVC to be Presented at ASGCT 2026
New Data from Both Cohorts of the MyPEAK™-1 Phase 1b/2 Trial of TN-201 for Adults with MYBPC3-Associated HCM Expected in the Second Quarter 2026
Preclinical Data at MDA 2026 Highlighted TN-301’s Activity in Duchenne Muscular Dystrophy Disease Models; Distinct Mechanism of HDAC6 Inhibition Supportive of TN-301’s Potential in Multiple Indications
Entered Research Collaboration with Alnylam to Identify and Validate Genetic Targets for Cardiovascular Conditions
SOUTH SAN FRANCISCO, Calif., May 06, 2026 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced financial results for the first quarter ended March 31, 2026, and provided a corporate update.
“We are entering a catalyst-rich period for Tenaya, with multiple clinical milestones expected across our lead gene therapy programs throughout 2026. Building on the encouraging initial readouts we reported in 2025, we believe the additional data expected this year from both TN-201 and TN-401 may support alignment on registrational pathways for these novel gene therapies,” said Faraz Ali, Chief Executive Officer of Tenaya.
Mr. Ali continued, “While our focus remains on the advancement of TN-201 and TN-401 for patients suffering from these genetic cardiomyopathies, we also announced meaningful steps in the direction of our next horizon of opportunities to address unmet patient needs and to create value for stockholders. We presented new preclinical data for TN-301, our clinical-stage small molecule candidate, in Duchenne muscular dystrophy adding to the body of compelling preclinical evidence for the broad clinical utility of this molecule in multiple prevalent and rare cardiac and cardiac-adjacent indications. Advancing TN-301 toward a trial in patients reflects our commitment to building a diversified portfolio grounded in mechanistic insight and translational rigor. The recently announced collaboration with Alnylam also reinforces the strength of Tenaya's innovation engine and expands the reach and impact of our modality-agnostic research capabilities.”
Business and Program Updates
TN-201 – Gene Therapy for MYBPC3-Associated Hypertrophic Cardiomyopathy (HCM)
On May 9, 2026, at the upcoming European Society of Cardiology (ESC) Heart Failure Conference, in Barcelona, Spain, Milind Desai, M.D., Director of the Hypertrophic Cardiomyopathy Center, Vice Chair of the Heart, Vascular & Thoracic Institute at Cleveland Clinic, and principal investigator for the MyPEAK-1 clinical trial, will present insights that emerged early in the trial that enabled reductions in the cumulative dose and duration of immune suppressive medications, even when TN-201 is administered at the higher dose.
Per the MyPEAK-1 protocol, sirolimus and prednisone are administered prophylactically in patients receiving TN-201 gene therapy, accompanied by post dose tapering in conjunction with monitoring of liver enzyme levels – an early indicator of potential complement system activation. Investigators found that minor adjustments, including administering sirolimus earlier, reducing the starting dose of prednisone and monitoring patients weekly, led to faster tapering and an overall decrease in the burden of immunosuppression. The ESC-HF presentation offers more detail on the immunosuppressive regimen results previously reported in November 2025 and includes safety data for the first seven patients enrolled in MyPEAK-1. The optimized regimen and monitoring protocols that were successfully deployed in MyPEAK-1 are also being utilized in the RIDGE-1 clinical trial of TN-401. Tenaya expects to report interim MyPEAK-1 data for Cohort 2 (6E13 vg/kg) and updates from Cohort 1 (3E13 vg/kg) in the second quarter of 2026. In January, Tenaya resumed enrollment and screening in MyPEAK-1 following implementation of modest protocol amendments in alignment with U.S. Food and Drug Administration (FDA) input. At the American Society of Gene and Cell Therapies (ASGCT) Annual Meeting, taking place May 11-15, 2026, in Boston, MA, Tenaya will present results from a survey exploring parental perceptions of gene therapy treatment for children with cardiomyopathies. This work was conducted by Tenaya in partnership with DDC Clinic and Children’s Cardiomyopathy Foundation. The poster presentation is scheduled for May 12, 2026.
Story Continues
TN-401 – Gene Therapy for PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Data from the ongoing RIDGE-1 Phase 1b/2 clinical trial of TN-401 in adults with ARVC due to variants in the PKP2 gene have been accepted as a late-breaking presentation at the upcoming ASGCT Annual Meeting. The presentation, scheduled for Friday, May 15, is expected to include one-year data for Cohort 1 (3E13 vg/kg) and initial Cohort 2 (6E13 vg/kg).
Tenaya management plans to conduct a webcast conference call on Friday, May 15, 2026, at 10:30 a.m. EDT / 7:30 am PDT following the Late Breaker Session. The webcast conference call, including an accompanying slide presentation, will be accessible from the Investor section of the Tenaya website at www.tenayatherapeutics.com. In January, the RIDGE-1 data and safety monitoring board (DSMB) reviewed all available data for the six patients that have received TN-401 gene therapy. The DSMB determined that TN-401 had an acceptable safety profile and endorsed continued enrollment of patients in RIDGE-1 expansion cohorts at either dose.
TN-301 – Small Molecule HDAC6 Inhibitor for the Potential Treatment of Heart Failure with Preserved Ejection Fraction (HFpEF) and Related Cardiac, Metabolic, or Muscular Diseases
In March 2026, Tenaya presented encouraging preclinical data comparing TN-301, the company’s highly selective HDAC6 inhibitor, with givinostat, an approved pan-HDAC inhibitor, in well-established preclinical models of Duchenne muscular dystrophy (DMD) at the Muscular Dystrophy Association’s 2026 Clinical and Scientific Congress. Results of the study showed that:
TN-301 treatment at doses as low as 3 mg/kg improved grip strength to wild-type levels within five weeks, whereas mdx mice treated with givinostat (10 mg/kg, approximating clinical exposures) failed to reach wild-type performance. TN-301-mediated functional improvements were accompanied by reductions in circulating creatine kinase and favorable changes in gene expression, indicating reduced muscle cell injury. In cardiomyocytes derived from human DMD-induced pluripotent stem cells, TN-301 corrected calcium handling abnormalities and mitochondrial dysfunction, while givinostat exacerbated these established drivers of DMD cardiomyopathy. TN-301 was granted both Rare Pediatric Disease Designation and Orphan Drug Designation for the treatment of DMD from U.S. Food and Drug Administration. In 2026, Tenaya plans to advance TN-301 toward clinical trials in patients in order to generate proof-of-activity data, with HFpEF and DMD being among the most promising potential indications identified to date.
Research
In March 2026, Tenaya entered into a multi-target research collaboration with Alnylam Pharmaceuticals to identify and validate novel genetic targets aimed at treating cardiovascular disease.
Under the terms of the collaboration agreement, in April 2026, Tenaya received an upfront payment of $10.0M and may be eligible for future development, regulatory and sales-based milestones totaling up to $1.1 billion, in addition to reimbursement of associated research costs. Updated results of preclinical studies characterizing TN-501, a gene editing therapeutic candidate intended for the treatment of PLN-R14del-associated dilated cardiomyopathy (DCM) will be presented at ASGCT on Thursday, May 14, 2026. TN-501 is designed to specifically inactivate the pathogenic phospholamban (PLN) R14del allele while preserving healthy function.
First Quarter 2026 Financial Highlights
Cash: As of March 31, 2026, cash and cash equivalents were $80.9 million. Tenaya expects that such resources, along with the $10.0 million upfront payment from the Alnylam collaboration, will be sufficient to fund planned operations into the second half of 2027. Research & Development (R&D) Expenses: R&D expenses were $14.8 million for the first quarter of 2026, compared to $21.1 million for the same period in 2025. Non-cash stock-based compensation included in R&D expense was $1.2 million for the first quarter of 2026 compared to $2.0 million for the same period in 2025. General & Administrative (G&A) Expenses: G&A expenses were $5.4 million for the first quarter of 2026 compared to $6.5 million for the same period in 2025. Non-cash stock-based compensation included in G&A expense was $1.0 million for the first quarter of 2026 and $1.7 million for the same period in 2025. Net Loss: Net loss was $19.3 million, or $0.09 loss per share, for the first quarter ended March 31, 2026, compared to a net loss of $26.9 million, or $0.24 per share, for the same period in 2025.
About Tenaya Therapeutics
Tenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Tenaya’s pipeline includes clinical-stage candidates TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM); TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC); and TN-301, a highly specific small molecule HDAC6 inhibitor with broad potential clinical utility in cardiac, metabolic and muscular conditions, including heart failure with preserved ejection fraction (HFpEF) and Duchenne muscular dystrophy (DMD). Tenaya has employed a suite of integrated internal capabilities including modality agnostic target discovery and validation, to generate a portfolio of novel medicines based on genetic insights, aimed at the treatment of both rare genetic disorders and more prevalent heart conditions. For more information, visit www.tenayatherapeutics.com.
Forward Looking Statements
This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as “expected,” “believe,” “may,” “focused,” “commitment,” “will,” “plans,” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, planned timing for sharing data from RIDGE-1 and MyPEAK-1 and the expected content of such data releases; the potential for additional data from Tenaya’s TN-201 and TN-401 programs to support regulatory alignment on registrational pathways; Tenaya’s focus on the advancement of TN-201 and TN-401; Tenaya’s commitment to the building a diversified portfolio and advance TN-301 toward clinical trials; the potential for Tenaya to receive development, regulatory and sales-based milestone payments, as well as research reimbursement under the collaboration with Alnylam; planned presentation for TN-501; the sufficiency of Tenaya’s cash resources to fund the company into the second half of 2027; and statements made by Tenaya’s chief executive officer. The forward-looking statements contained herein are based upon Tenaya’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: availability of data at the referenced times; the timing and progress of Tenaya’s clinical trials; unexpected concerns that may arise as a result of the occurrence of adverse safety events in Tenaya’s clinical trials; the potential failure of Tenaya’s product candidates to demonstrate safety and/or efficacy in clinical testing; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; the potential for the FDA to conclude at any time that Tenaya’s clinical programs may not have an appropriate risk/benefit profile; Tenaya’s ability to enroll and maintain patients in clinical trials; risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; Tenaya’s ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; Tenaya’s ability to achieve the expected benefits from the collaboration with Alnylam; the occurrence of any event, change or other circumstance that could give rise to the termination of the collaboration with Alnylam; Tenaya’s continuing compliance with applicable legal and regulatory requirements; regulatory developments in the United States and foreign countries; Tenaya’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; Tenaya’s reliance on third parties; Tenaya’s manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya’s ability to comply with specified operating covenants and restrictions in its loan agreement; Tenaya’s ability to obtain and maintain intellectual property protection for its product candidates and platform technology; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Tenaya Contacts
Michelle Corral
VP, Corporate Communications and Investor Relations
IR@tenayathera.com
Investors
Anne-Marie Fields
Precision AQ
annemarie.fields@precisionaq.com
Media
Wendy Ryan
Ten Bridge Communications
wendy@tenbridgecommunications.com
TENAYA THERAPEUTICS, INC.
Condensed Statements of Operations
(In thousands, except share and per share data)
(Unaudited) Three Months Ended March 31, 2026 2025 Revenue Collaboration revenue $ 225 $ — Operating expenses: Research and development 14,843 21,076 General and administrative 5,447 6,462 Total operating expenses 20,290 27,538 Loss from operations (20,065 ) (27,538 ) Other income, net: Interest income 793 635 Other income, net — 39 Total other income, net 793 674 Net loss before income tax expense (19,272 ) (26,864 ) Income tax expense — — Net loss $ (19,272 ) $ (26,864 ) Net loss per share, basic and diluted $ (0.09 ) $ (0.24 ) Weighted-average shares used in computing net loss per share, basic and diluted 216,883,164 109,869,278
Condensed Balance Sheet Data
(In thousands)
(Unaudited) March 31, December 31, 2026 2025 Cash and cash equivalents $ 80,887 $ 100,547 Total assets $ 135,070 $ 146,921 Total liabilities $ 28,881 $ 23,656 Total liabilities and stockholders’ equity $ 135,070 $ 146,921
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- Alnylam to Present New Analyses at Heart Failure 2026 Underscoring Consistent Clinical Profile of Vutrisiran in ATTR-CM
May 4, 2026
CAMBRIDGE, Mass., May 04, 2026--(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
Story Continues
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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260504658209/en/
Contacts
Alnylam Pharmaceuticals, Inc.
Christine Akinc
(Investors and Media)
+1-617-682-4340
Josh Brodsky
(Investors)
+1-617-551-8276
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- Alnylam to Present New Analyses at Heart Failure 2026 Underscoring Consistent Clinical Profile of Vutrisiran in ATTR-CM
May 4, 2026 · businesswire.com
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.
- ALNYLAM TO PRESENT NEW ANALYSES AT HEART FAILURE 2026 UNDERSCORING CONSISTENT CLINICAL PROFILE OF VUTRISIRAN IN ATTR-CM
May 4, 2026
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.
