Alnylam posts Q3 2026 results, widens nucresiran trial, advances Huntington's program

Alnylam Pharmaceuticals reported third quarter 2026 results on October 29, 2026, for the period ended September 30, 2026. The company achieved global net product revenues for AMVUTTRA and ONPATTRO for the third quarter of $1,130 million and $20 million, respectively, together representing $1,150 million in total TTR net product revenues and 59% total TTR growth compared to Q3 2025.¹

On the pipeline side, Alnylam announced it is expanding target enrollment from 1,750 to approximately 2,250 patients¹ in the TRITON-CM Phase 3 study of nucresiran in ATTR-CM. The company said this expansion is enriched for patients with early disease who are expected to experience greatest benefit and ensures adequate study power under conservative estimates of outcome event rates.¹ Alnylam said that given the rapid pace of enrollment and the event-driven nature of the study's primary endpoint, it still expects to launch nucresiran, assuming positive data and regulatory approval, in ATTR-CM by 2030.¹

For brezosiran (ALN-HTT02), an RNAi candidate for Huntington's disease developed with Regeneron, Alnylam reported that patients treated with single ascending intrathecal doses experienced robust, dose-dependent reductions of mutant huntingtin protein in cerebrospinal fluid, with a mean reduction of 60-64% from baseline observed at month 3 in the highest dose group evaluated to date, and a maximum individual reduction of 83%.¹ The company noted an encouraging safety and tolerability profile has been observed to date, with no dose-dependent increases in CSF inflammatory markers, CSF neurofilament light chain or brain ventricular volume.¹ Alnylam plans to initiate a placebo-controlled pivotal study in 2027, pending regulatory alignment.¹

The company also disclosed that the PDUFA target action date for cemdisiran, an investigational RNAi therapeutic for the treatment of adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor antibody-positive, is in November 2026.¹ Cemdisiran is being advanced by Alnylam's collaborator Regeneron, and Alnylam is eligible to receive tiered, double-digit royalties of up to 15 percent on global net sales if approved.¹

On cash position, Alnylam reported that cash, cash equivalents and marketable securities were $3.6 billion as of September 30, 2026, compared to $2.9 billion as of December 31, 2025.¹ The company said full-year 2026 financial guidance was reiterated.