Alnylam Pharmaceuticals Clinical Trial Pipeline: 26 Active Studies

Phases, active trials: Phase 1: 7, Phase 1/2: 3, Phase 2: 5, Phase 3: 5, No phase listed: 6

Alnylam Pharmaceuticals pioneered RNA interference (RNAi) as a therapeutic modality and has translated that platform into five approved medicines and one of the most productive rare disease pipelines in the industry. Their GalNAc-conjugated siRNA platform enables precise, durable gene silencing in the liver, enabling twice-yearly or even once-yearly dosing for previously untreatable conditions. Amvuttra (vutrisiran) and Onpattro (patisiran) have transformed the treatment of hereditary transthyretin-mediated amyloidosis (hATTR), a fatal rare disease with limited options. Leqvio (inclisiran), partnered with Novartis, extends RNAi into mainstream cardiovascular disease with twice-yearly PCSK9 silencing for LDL reduction. Beyond approved medicines, Alnylam is systematically expanding into ATTR cardiomyopathy (a much larger population than hATTR polyneuropathy), hypertension (zilebesiran targeting angiotensinogen), and metabolic disease including obesity (ALN-2232). Their pipeline also includes programs in Huntington's disease (ALN-HTT02) and central nervous system applications.

• ATTR amyloidosis (hereditary and wild-type, polyneuropathy and cardiomyopathy)
• Cardiovascular disease (PCSK9, angiotensinogen, hypertension)
• Rare liver disease (acute hepatic porphyria, primary hyperoxaluria)
• Metabolic disease (obesity, diabetes)
• Neurodegenerative disease (Huntington's)
• Complement-mediated diseases

• Amvuttra (vutrisiran): siRNA, hATTR-PN + ATTR-CM (cardiomyopathy)
• Onpattro (patisiran): first-approved siRNA, hATTR-PN
• Leqvio (inclisiran, Novartis): PCSK9 siRNA, LDL reduction
• Givlaari (givosiran): ALAS1 siRNA, acute hepatic porphyria
• Zilebesiran (ALN-AGT): angiotensinogen siRNA, hypertension
• ALN-2232: siRNA for obesity (undisclosed target)

Alnylam's expansion of Amvuttra into ATTR cardiomyopathy is one of the most important rare disease label expansions: the cardiomyopathy population is 10-50x larger than the polyneuropathy population it was originally approved in. Their zilebesiran hypertension program (partnered with Roche) could be the first RNAi drug in a mainstream CV indication. Each new Alnylam trial registration signals potential expansion of the RNAi modality into new therapeutic areas.

DataLookout monitors ClinicalTrials.gov daily and surfaces changes to Alnylam Pharmaceuticals's registered studies: new trial registrations, status transitions (for example "Not yet recruiting" to "Recruiting"), site additions, and protocol amendments. This is the intelligence layer that bridges the gap between quarterly investor calls and day-to-day pipeline developments.

• Sponsor normalization: Alnylam Pharmaceuticals may register trials under multiple legal entity names. DataLookout normalizes all related entities to a single canonical sponsor for complete pipeline coverage.
• Daily updates: ClinicalTrials.gov is queried each morning. New registrations and updates appear in that day's digest.
• Field-level change detection: Status changes, enrollment updates, and site additions are detected and surfaced, not just new registrations.
• Phase filtering: Focus on Phase 3 pivotal trials, Phase 2 proof-of-concept studies, or all phases for comprehensive coverage.
• Indication filtering: Combine sponsor monitoring with indication keywords to focus on the therapeutic areas relevant to your business.