Alnylam Pharmaceuticals, Inc. ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) is a cardio-renal-metabolic drug developer. Its lead program, nucresiran, is in Phase 3 for ATTR amyloidosis (polyneuropathy and cardiomyopathy). Its next catalyst on record is cemdisiran monotherapy for myasthenia gravis: FDA action date (November 2026).
Upcoming catalysts
- November 2026cemdisiran monotherapy for myasthenia gravis — FDA action date
NDA ... accepted for review in June 2026 with a target action date of November 2026
Next further out: ALN-6400 — Phase 1 additional data (Q4 2026). 7 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| AMVUTTRA (vutrisiran) | hATTR amyloidosis with polyneuropathy and ATTR-CM | RNA (siRNA) | Approved |
| nucresiran | hATTR-PN | RNA (siRNA) | Phase 3 |
| nucresiran | ATTR-CM | RNA (siRNA) | Phase 3 |
| zilebesiran | uncontrolled hypertension | RNA (siRNA) | Phase 3 |
| cemdisiran | generalized myasthenia gravis | RNA (siRNA) | Phase 3 |
| cemdisiran + pozelimab | paroxysmal nocturnal hemoglobinuria | RNA (siRNA) | Phase 3 |
| cemdisiran | geographic atrophy | RNA (siRNA) | Phase 3 |
| mivelsiran | cerebral amyloid angiopathy (CAA) | RNA (siRNA) | Phase 2 |
| mivelsiran | Down syndrome-associated Alzheimer's disease | RNA (siRNA) | Phase 2 |
| ALN-6400 | Hereditary Hemorrhagic Telangiectasia (HHT) | RNA (siRNA) | Phase 2 |
| ALN-6400 | von Willebrand disease | RNA (siRNA) | Phase 2 |
| brezosiran | Huntington's disease | RNA (siRNA) | Phase 1 |
Recent results and decisions
- Oct 29, 2026positive Alnylam presented positive Phase 1 data for brezosiran demonstrating robust target engagement and encouraging safety and tolerability in patients with Huntington's disease.
Presented Positive Phase 1 Data for Brezosiran (ALN-HTT02) Demonstrating Robust Target Engagement and Encouraging Safety and Tolerability Profile in Patients with Huntington's Disease
Filing
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 cardio-renal-metabolic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.