Story Stocks^®

Alnylam Pharmaceuticals (ALNY) is soaring 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 by about 18% on a year-to-date basis prior to today's surge, ALNY was in need of a spark and this development is lighting a fire under the stock as it has the potential to be a game-changer for the company.

• Marketed under the Amvuttra brand name, Vutrisiran is already an FDA approved and commercially available product for a related disease called hereditary transthyretin-mediated amyloidosis (hATTR). In Q4, Amvuttra generated global revenue of $175 mln, making it the company's top-selling drug, but its total addressable market may be set to expand significantly if it receives regulatory approval for ATTR-CM.
• While ATTR-CM, a debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, is still considered to be a rare condition, an increasing number of people are being diagnosed with the disorder due to advancements in medical technology and improved testing and screening. According to ALNY, approximately 250,000-350,000 people are affected by the two different kinds of ATTR.

The results from this Phase 3 trial suggest that Vutrisiran has the potential to become the standard of care for ATTR-CM.

• In the study, a cohort of the 655 adults took Vutrisiran, while another cohort took the placebo subcutaneously once every three months for up to 36 months. Additionally, a portion of the study population was also already taking a Pfizer (PFE) approved drug for this condition, called Vyndamax, while another subset was not. 
• ALNY's goal was to see if the drug reduced death and hospitalizations in both the Pfizer and non-Pfizer populations and the results were quite positive. Specifically, Vutrisiran achieved a 28% and 33% reduction in death and heart-related hospitalizations in those groups, respectively, meeting the study's primary endpoint.
• Furthermore, the study also showed statistically significant improvements across all secondary endpoints in both populations, while the drug demonstrated a safety and tolerability profile that was consistent with its established profile.
• Looking ahead, the next step for ALNY will be to file a New Drug Applications (NDA) with the FDA, which should happen later this year. If the FDA votes to approve Vutrisiran, then ALNY could potentially begin manufacturing the drug in mid-2025.

From a financial standpoint, an approval would represent a major turning point for the company. Currently, analysts are expecting ALNY to generate a little less than $2.0 bln in revenue this year. For some context, PFE's Vyndamax is expected to achieve revenue of at least $4.0 bln this year. ALNY is far from being profitable right now, but that could change quickly if it receives a favorable FDA decision in the coming months.