Alnylam Pharmaceuticals Soars on Positive Phase 3 Study Results for Vutrisiran

Alnylam Pharmaceuticals (ALNY) is surging to its highest levels since mid-February after reporting encouraging results from its Phase 3 study of Vutrisiran, an RNAi treatment for ATTR amyloidosis with cardiomyopathy (ATTR-CM). With shares down about 18% year-to-date before today’s surge, this development is a potential game-changer for the company.

• Marketed under the Amvuttra brand name, Vutrisiran is already FDA approved for hereditary transthyretin-mediated amyloidosis (hATTR). In Q4, Amvuttra generated global revenue of $175 million, making it ALNY’s top-selling drug. The total addressable market could expand significantly if it receives regulatory approval for ATTR-CM.
• ATTR-CM, caused by misfolded transthyretin (TTR) proteins, is a rare but increasingly diagnosed condition due to advancements in medical technology. Approximately 250,000-350,000 people are affected by the two kinds of ATTR, according to ALNY.

The Phase 3 trial results suggest that Vutrisiran could become the standard of care for ATTR-CM.

• In the study, 655 adults were divided into cohorts; one took Vutrisiran, and the other took a placebo subcutaneously every three months for up to 36 months. Some participants were also taking Pfizer’s (PFE) approved drug, Vyndamax, while others were not.
• ALNY aimed to see if the drug reduced death and hospitalizations in both the Pfizer and non-Pfizer groups. Vutrisiran achieved a 28% and 33% reduction in death and heart-related hospitalizations, respectively, meeting the study’s primary endpoint.
• The study also showed statistically significant improvements across all secondary endpoints in both populations. The drug demonstrated a safety and tolerability profile consistent with its established profile.
• Next, ALNY plans to file a New Drug Application (NDA) with the FDA later this year. If approved, manufacturing could begin by mid-2025.

Financially, an approval would be a major turning point for ALNY. Analysts expect ALNY to generate just under $2.0 billion in revenue this year. For comparison, PFE’s Vyndamax is expected to achieve at least $4.0 billion in revenue this year. While ALNY is not yet profitable, this could change quickly with a favorable FDA decision.

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