Investors wary after Alnylam delays readout of key heart drug trial

15 Feb 2024
firstwordpharma.com
Drug ApprovalPhase 3AHA
Shares in Alnylam Pharmaceuticals dipped over 10% on Thursday after the company said it was modifying the statistical plan for a key heart drug trial that was due to read out in early 2024. As a result of the changes, Alnylam says the Phase III HELIOS-B trial of its RNAi therapeutic Amvuttra (vutrisiran) in transthyretin-mediated amyloidosistransthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) are now expected in late June or early July.
The changes in study design at this late stage may be perceived as a "lack of management's confidence in HELIOS-B outcome," remarked BMO Capital Markets analyst Kostas Biliouris.
The FDA approved Amvuttra in 2022 to treat nerve damage in adult patients with hereditary ATTR amyloidosis, but AlnylamAlnylam is hoping to broaden the label to encompass the more lucrative ATTR-CM market. It estimates that a label expansion could boost the potential market for the drug by about 10% to over 300,000 patients globally, according to the company's quarterly earnings presentation Thursday.
An approval would also help the company bounce back from a setback involving its older treatment Onpattro (patisiran). That drug was approved in 2018 for patients with polyneuropathy due to hereditary ATTR, but Alnylam scrapped plans late last year to seek an expanded label that would include ATTR-CM after the FDA rejected its application.
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