Alnylam Pharmaceuticals, Inc.（ALNY）資訊與事件ALNYAlnylam Pharmaceuticals, Inc.

Alnylam Reports Initial Phase 1b Trial Results for ALN-HTT02

Alnylam reported initial Phase 1b clinical trial results for ALN-HTT02, an investigational RNAi therapeutic in development for the treatment of Huntington's disease, which will be developed under the International Nonproprietary Name, or INN, brezosiran. The data were presented in oral and poster presentations at the European Huntington's Disease Network, or EHDN, Clinical Research Congress 2026 in Krakow, Poland. In the ongoing Phase 1b study, participants on single ascending intrathecal doses of brezosiran experienced robust, dose-dependent reductions of mutant huntingtin protein in cerebrospinal fluid, or CSF, with target engagement demonstrated using two independent assays. Across the first five cohorts, dose-dependent reductions of CSF mHTT were observed, with a mean reduction from baseline of 60%-64% at month 3 in the highest dose group evaluated to date, with a maximum individual reduction of 83%. The observed durability of mHTT-lowering shows the potential for a twice-yearly dosing paradigm in downstream development. The initial data include individuals followed for up to 12 months, during which an encouraging safety and tolerability profile has been observed with no dose-dependent increases in CSF inflammatory markers, CSF neurofilament light chain or brain ventricular volume. All eligible Phase 1b study participants have the option to transition into a Ph1b open-label extension study, receiving brezosiran once every six months for up to 36 months to evaluate long-term safety, sustained target engagement, and exploratory measures of disease progression. Alnylam plans to initiate a placebo-controlled pivotal study in 2027, pending regulatory alignment, to evaluate the ability of brezosiran to slow disease progression in adults with Huntington's disease.

Alnylam Launches AMVUTTRA Educational Initiative

Alnylam Pharmaceuticals announced the launch of AMVUTTRA 'Play by Play,' a U.S. educational initiative featuring former professional athletes living with the cardiomyopathy of transthyretin-mediated amyloidosis and the care teams who support them. Through personal stories and expert perspectives, 'Play by Play' aims to help people recognize the symptoms of ATTR-CM earlier and encourage informed conversations with healthcare professionals to help patients advocate for their diagnosis and treatment. 'Play by Play' kicks off with Pete Shaw, a former professional football player living with ATTR-CM, alongside his wife and caregiver, Kari, and one of his cardiologists, Rajeev Mohan, M.D. Together, they share Pete's journey from experiencing symptoms that were mistakenly attributed to football or aging, to pursuing testing, receiving an ATTR-CM diagnosis, and ultimately starting treatment with AMVUTTRA, a transthyretin silencer, as part of his game plan for ATTR-CM management.

Alnylam Presents RNAi Data for Cardiovascular Disease

Alnylam Pharmaceuticals announced new data at the European Society of Cardiology, ESC, Congress 2026 demonstrating the strength of RNAi-powered silencing for cardiovascular disease. The findings further reinforce the clinical profile of AMVUTTRA across transthyretin amyloidosis, ATTR, patient populations, treatment settings, and manifestations of disease. Additionally, the data expand the potential application of RNAi to uncontrolled hypertension, the world's leading cause of cardiovascular disease. "With the power of our RNAi therapeutics platform, we have the potential to make a transformational impact on cardiovascular care," said Pushkal Garg, M.D., Chief Research and Development Officer at Alnylam. "The data presented at ESC demonstrate the consistency of clinical outcomes achieved by RNAi-powered TTR silencing, reinforcing our conviction in AMVUTTRA as a first-line treatment option for ATTR-CM. With zilebesiran, we have the potential to extend the precision and durability of RNAi to uncontrolled hypertension. Together, these programs reflect our ambition to change the course of cardiovascular disease for patients with high unmet need."