ALNY - Alnylam soars 42% as Phase 3 trial for ATTR amyloidosis therapy meets main goal

Alnylam Pharmaceuticals ( NASDAQ: ALNY ) added nearly 42% in the morning hours Wednesday after the RNAi therapeutics company said that its Phase 3 trial for patisiran reached the primary goal in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

According to topline data, the APOLLO-B trial involving 360 adult patients worldwide reached the primary endpoint of change from baseline in the 6-Minute Walk Test (6-MWT) at 12 months compared to placebo with statistical significance (p-value 0.0162).

In addition, the drug, also known as Onpattro, reached the first secondary endpoint: the change in the quality of life compared to placebo from baseline in terms of the Kansas City Cardiomyopathy Questionnaire (KCCQ) (p-value 0.0397).

Onpattro, already indicated in the U.S. for adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis, demonstrated an encouraging safety and tolerability profile, Alnylam ( ALNY ) said.

While five patients (2.8 percent) in the patisiran arm and 8 (4.5 percent) in the placebo arm died, the incidences of adverse events (AEs) and serious adverse events (SAEs) were found to be similar across both arms, the company added.

Alnylam ( ALNY ) intends to submit a Supplemental New Drug Application to the FDA in late 2022 and present APOLLO-B data at a medical conference in Heidelberg, Germany in September.

In June, Bank of America opened a 90-day catalyst watch on Alnylam ( ALNY ) ahead of the readout.

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