Overshadowing the strong sales growth revealed in its fourth quarter 2023 earnings call, Alnylam Pharmaceuticals Inc. worried investors on 15 February with a late change to the endpoints and expected topline readout for the Phase III HELIOS-B study that it hopes will support expanding Amvuttra’s label into transthyretin amyloid cardiomyopathy (ATTR-CM), which would substantially increase the drug’s addressable population.
Alnylam Surprises Investors With Last-Minute HELIOS-B Revisions
Alnylam pushed back the study’s top-line readout to June or July. But analysts said the changes increase the odds of demonstrating monotherapy benefit with Amvuttra in ATTR-CM.
More from Clinical Trials
• By
Investing heavily in antibody-drug conjugates, AbbVie is pioneering a dual STEAP1xPSMA-targeting approach in prostate cancer, as other candidates advance.
• By
Strong sales growth for the German group’s SGLT2 inhibitor in 2024
• By
After a more than three-year hiatus, China's Hengrui has signalled its return to multiregional Phase III trials as it looks to globalize its innovative pipeline. Meanwhile, a number of other Chinese players have announced plans to kick off Phase III trials this year and beyond.
• By
After failing a Phase II monotherapy study in early Parkinson’s, Cerevance will focus on adjunctive therapy without abandoning the monotherapy concept.
More from R&D
• By
The firm has lofty ambitions for the aldosterone synthase inhibitor to treat hypertension and kidney disease.
• By
The ZENITH study is a landmark for Merck’s first-in-class activin signalling inhibitor and pulmonary arterial hypertension treatment.
• By
Seeking cell therapy approaches to cure type 1 diabetes, Vertex abandons a candidate encapsulated to avoid immune system detection but hopes to file another candidate for approval in 2026.