ALNY — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-07-30

• We are developing ALN-6400, a GalNAc-conjugated RNAi therapeutic that targets plasminogen, or PLG, for the treatment of a wide range of bleeding disorders. In preclinical studies, ALN-6400 demonstrated a greater than 90% reduction in circulating PLG in non-human primates with no evidence of increase…

• We are advancing ALN-HTT02, an investigational, intrathecally administered RNAi therapeutic targeting huntingtin, or HTT, that is in development in collaboration with Regeneron Pharmaceuticals, Inc., or Regeneron, for the treatment of Huntington’s Disease, or HD. ALN-HTT02 is designed to target a co…

• initiated a Phase 1b clinical trial of ALN-HTT02 in adult patients with HD and expect to present initial data from this trial in the second half of 2026.

• We have additional late-stage investigational programs advancing toward potential commercialization with collaborators, including cemdisiran for the treatment of complement-mediated diseases. Our collaborator, Regeneron, is advancing cemdisiran in combination with its anti-C5 monoclonal antibody, po…

• We achieved profitability for the first time in 2025. Nevertheless, we have incurred significant losses since inception, and as of June 30, 2026, we had an accumulated deficit of $6.33 billion. Historically, we generated losses primarily from costs associated with research and development activities…

Text removed vs the prior filing · source: 10-Q · 2026-04-30

• We have additional late-stage investigational programs advancing toward potential commercialization with collaborators, including cemdisiran for the treatment of complement-mediated diseases. Our collaborator, Regeneron Pharmaceuticals, Inc., or Regeneron, is advancing cemdisiran in combination with…

• We achieved profitability for the first time in 2025. Nevertheless, we have incurred significant losses since inception, and as of March 31, 2026, we had an accumulated deficit of $6.50 billion. Historically, we generated losses primarily from costs associated with research and development activitie…

• In September 2025, we issued $661.3 million aggregate principal amount of 0.00% Convertible Senior Notes due 2028, or the 2028 Notes. The 2028 Notes will mature on September 15, 2028, unless earlier converted, redeemed or repurchased. The 2028 Notes will not bear regular interest. Before June 15, 20…

• •Our partner, Regeneron, announced the submission of an NDA to the FDA for cemdisiran, an investigational RNAi therapeutic for adults with generalized myasthenia gravis. Additional global filings are planned for 2026.

• •Announced update to the TRITON-CM Phase 3 study of nucresiran, an investigational next-generation TTR silencer, in patients with ATTR-CM:

Text added vs the prior filing · source: 10-Q · 2026-07-30

• disclosure controls and procedures as of June 30, 2026, our Chief Executive Officer and executive vice president, Chief Financial Officer concluded that, as of such date, our disclosure controls and procedures were effective at the reasonable assurance level.

• There were no changes in our internal control over financial reporting during the quarter ended June 30, 2026 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

Text removed vs the prior filing · source: 10-Q · 2026-04-30

• There were no changes in our internal control over financial reporting during the quarter ended March 31, 2026 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

Text added vs the prior filing · source: 10-Q · 2026-07-30

• •the timing, receipt and amount of milestone, royalty, research and development funding and other payments, if any, from present and future collaborators, if any, including milestone, royalty and research and development funding payments from Roche with respect to the development and commercializati…

• We may not be successful in entering into future collaborations on terms favorable to us due to various factors, including our ability to demonstrate improved product profiles from our new technologies, our ability to successfully demonstrate proof-

• of-concept for our technology in humans in certain tissues or disease areas, our ability to demonstrate the safety and efficacy of our specific product candidates, our ability to manufacture or have third parties manufacture RNAi therapeutics, the strength of our intellectual property portfolio and/…

• Additionally, in December 2025, the BIOSECURE ACT was enacted into law as part of the National Defense Authorization Act for fiscal year 2026. The BIOSECURE ACT prohibits U.S. federal executive agencies from contracting with any entity where the biotechnology equipment or services of a “biotechnolog…

• We rely on independent clinical investigators, CROs, and other third-party service providers to assist us in managing, monitoring and otherwise carrying out our clinical trials. We have contracted with, and we plan to continue to contract with, certain third parties to provide certain services for o…

Text removed vs the prior filing · source: 10-Q · 2026-04-30

• AMVUTTRA will continue to account for a significant portion of our net product revenues in future years. As a result, our business is dependent upon our ability to sustain and grow revenues from sales of AMVUTTRA.

• •the timing, receipt and amount of milestone, royalty, research and development funding and other payments, if any, from present and future collaborators, if any, including milestone, royalty and research and development funding

• payments from Roche with respect to the development and commercialization of zilebesiran, as well as royalty payments from Novartis and Sanofi related to the commercialization of Leqvio and Qfitlia, respectively;

• been made and changes are likely to continue to occur in the future. Future changes in tax laws could have a material adverse effect on our business, prospects, operating results and financial condition.

• We may not be successful in entering into future collaborations on terms favorable to us due to various factors, including our ability to demonstrate improved product profiles from our new technologies, our ability to successfully demonstrate proof-of-concept for our technology in humans in certain …