Alnylam Pharma (NASDAQ:ALNY) shares tumbled 11.5% Friday following the release of results from its HELIOS-B Phase 3 study of vutrisiran, despite the study meeting all primary and secondary endpoints.
The investigational RNAi therapeutic, aimed at treating ATTR amyloidosis with cardiomyopathy (ATTR-CM), was shown to reduce the risk of death and cardiovascular events by 28% compared to placebo in the overall population.
However, the market reacted negatively to the results.
In contrast, BridgeBio Pharma Inc . (NASDAQ:BBIO) Pharma saw its shares rise by 12%, as investors may have perceived the results as strengthening the competitive landscape for ATTR-CM treatments, where BridgeBio is also a key player.
Alnylam highlighted the potential of vutrisiran to become a new standard of care, especially for patients in the earlier stages of ATTR-CM, where early treatment can better preserve functional capacity and quality of life.
While Alnylam’s Chief Medical Officer, Pushkal Garg, emphasized the significant advances in survival and reduced cardiovascular hospitalizations, the market's response suggests caution regarding the drug's future impact in a highly competitive field.
"With this study, we have demonstrated that the rapid knockdown of toxic TTR seen with vutrisiran improves survival, and reduces cardiovascular hospitalizations and disease progression versus placebo, with benefits consistently observed across populations and regardless of background stabilizer use,” said Pushkal Garg, Chief Medical Officer of Alnylam.
“While the results have not yet been reviewed by a regulatory authority, the data we have shared today suggest that vutrisiran has the potential to become a new standard of care treatment for ATTR-CM, a progressive and ultimately fatal disease with limited treatment options."