Alnylam Pharmaceuticals announced promising results from a study evaluating its drug Vutrisiran, marketed as Amvuttra, for the treatment of ATTR amyloidosis with cardiomyopathy, a serious heart condition. The study aimed to reduce death and hospitalizations among patients already using Pfizer Inc.’s approved drug for the condition, as well as those not using it.
The study, involving 655 adults, demonstrated significant benefits with Vutrisiran. It showed a 28% reduction in deaths and a 33% decrease in heart-related hospitalizations among patients in the two respective groups compared to those on a placebo.
Alnylam highlighted the drug’s “encouraging” safety profile and plans to proceed with regulatory approval filing later this year, anticipating a new standard of care in this field.
The positive results sparked a substantial market response, with Alnylam shares surging up to 33% upon the news. This marked a significant recovery for the stock, which had previously seen a decline of 13% since the beginning of the year.
ATTR amyloidosis with cardiomyopathy, once considered rare, is increasingly diagnosed due to advances in testing methods. Alnylam estimates over 300,000 patients globally suffer from this condition, making it a focal point for pharmaceutical research and development.
Other companies like BridgeBio Pharma Inc., Ionis Pharmaceuticals Inc., and Intellia Therapeutics Inc. are also pursuing treatments for this disease, with market reactions varying based on comparative study results.
Analysts, such as Myles Minter from William Blair, hailed Alnylam’s findings as a “clear home run,” indicating potential positive implications for other companies pursuing similar therapeutic approaches. The news buoyed shares of Ionis and Intellia, demonstrating market optimism for innovative treatments in this competitive landscape.
In response to Alnylam’s breakthrough, competitors like BridgeBio Pharma experienced a notable decline in their stock value as investor expectations adjusted following the heightened standards set by Alnylam’s results.
BridgeBio had previously reported promising late-stage study results for its own drug targeting the same condition, and it is awaiting regulatory decisions by late November.
Looking ahead, Alnylam aims to expand Amvuttra’s usage beyond its current approval for treating related disorders involving abnormal proteins damaging multiple organs. CEO Yvonne Greenstreet expressed confidence in the drug’s revenue potential, projecting it as a multibillion-dollar opportunity.
In conclusion, Alnylam’s study results for vutrisiran represent a significant advancement in the treatment of ATTR amyloidosis with cardiomyopathy, reshaping expectations in the pharmaceutical industry and positioning the company for substantial growth pending regulatory approvals.
