Alnylam's Breakthrough Liver Cancer Study: A Promising Phase 1 Update

Alnylam Pharmaceuticals has announced an update on their ongoing Phase 1 study for ALN-BCAT in advanced liver cancer patients. The study aims to assess the safety, tolerability, and antitumor activity of ALN-BCAT, both as a standalone treatment and in combination with pembrolizumab. Positive results could boost investor confidence and position the company favorably in the oncology sector.

Regeneron Pharmaceuticals and Alnylam Pharmaceuticals have reported encouraging results from a late-stage study on cemdisiran, an RNA medicine developed to control symptoms of generalized myasthenia gravis in adults. The study, named NIMBLE, tested cemdisiran both as a standalone treatment and in combination with Regeneron's antibody drug, pozelimab.

In the trial, cemdisiran monotherapy reduced complement factor 5 (C5) activity by 74%, while the combination therapy achieved nearly 99% inhibition. However, cemdisiran alone demonstrated numerically greater treatment effects at 24 weeks on two scales assessing disease severity and its impact on daily life. This suggests that complete complement blockade may not be necessary for treating myasthenia gravis.

Regeneron plans to submit cemdisiran for U.S. approval in myasthenia gravis by the first quarter of next year. The company is also testing the drug in paroxysmal nocturnal hemoglobinuria and geographic atrophy associated with age-related macular degeneration. The study did not report any meningococcal infections, and no participants on cemdisiran monotherapy discontinued treatment due to side effects through week 24.

The most common adverse reactions included upper respiratory tract and urinary tract infections, common cold, and headache. Serious treatment-emergent adverse events occurred in 3% of patients given cemdisiran, 9% taking the combination, and 14% of those given placebo. Two deaths were reported during an extension period, both in patients receiving immuno-suppressive therapies.

Regeneron and Alnylam's collaboration on cemdisiran dates back to 2019, with Regeneron taking a global license to the drug last year. The companies plan to present full data from the trial at an upcoming medical meeting. Shares in both companies rose by about 1% on Tuesday following the announcement.

References:
[1] https://www.biopharmadive.com/news/regeneron-myasthenia-gravis-cemdisiran-alnylam-study-results/758607/