Alnylam Pharma Shares Dip Despite Positive Phase 3 HELIOS-B Heart Disease Results

Investor sentiment took a hit as Alnylam Pharmaceuticals, Inc. (ALNY) recently saw its shares fall over 8.5%. The decline followed the company’s release of detailed data from the Phase III HELIOS-B study. This trial aimed to expand the use of Amvuttra (vutrisiran) for treating transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM). Despite the study meeting its primary endpoint by showing a significant reduction in mortality and recurrent cardiovascular events, vutrisiran’s impact on mortality in the monotherapy group was deemed non-significant. This may have contributed to the negative market reaction.

30% Reduction in Mortality Non-Significant, Impacting Alnylam Stock

Shares of Alnylam declined following the Phase III HELIOS-B study results. In this instance this study assessed vutrisiran, an investigational RNAi therapeutic, for an expanded indication. The study reported a 30% reduction in mortality for vutrisiran in the monotherapy group during the double-blind period. However, with a nominal p-value of 0.1179, this reduction was not statistically significant, indicating that the observed effect could be due to chance rather than a definitive impact of the drug.

Despite this, the HELIOS-B study achieved its primary endpoint, showing a statistically significant reduction in both all-cause mortality and recurrent cardiovascular events among all participants, including those receiving Amvuttra alone. In short, vutrisiran significantly reduced the risk of death and cardiovascular events compared to placebo. Overall, the drug lowered all-cause mortality and cardiovascular events by 28%, with mortality reduced by 31% during the double-blind period and by 36% up to 42 months. In the monotherapy group, vutrisiran reduced mortality by 35% up to 42 months and overall mortality and cardiovascular events by 33%.

ALNY Stock Outperforming Industry; Vutrisiran Updates

In spite of this notable drop, overall shares of Alnylam have surged 37.3% year-to-date, significantly outperforming the industry’s modest growth of 0.6%. This strong performance contrasts with recent challenges, highlighting the company’s robust market position.

The initial positive data of the HELIOS-B trial reported in June 2024, resulted in a notable increase in Alnylam’s shares. Vutrisiran is currently approved in the United States for treating adult patients with polyneuropathy of hereditary ATTR amyloidosis. It also has approval in the European Union for the same indication in adults with stage 1 or stage 2 polyneuropathy. The drug is marketed as Amvuttra in both regions. Currently, Alnylam is preparing for global regulatory submissions. The company plans to file a supplemental new drug application for vutrisiran with the FDA later in 2024, using a Priority Review Voucher. This step marks a significant move towards expanding the drug’s market presence.

Amvuttra Drives Significant Revenue for Alnylam

Amvuttra is currently Alnylam’s top revenue driver, generating $425.4 million in sales during the first half of 2024. This represents an 82% increase year over year on a reported basis. The strong uptake of Amvuttra results from both new patients starting treatment and existing patients switching from Alnylam’s Onpattro (patisiran).

Onpattro, which is also approved in the United States and the European Union, treats ATTR amyloidosis in adults. The continued success of Amvuttra reflects its growing role in the company’s portfolio and its competitive position in the market.

If Alnylam secures approval for the new indication, vutrisiran could become the new standard of care for ATTR amyloidosis with cardiomyopathy. This potential approval is expected to further boost sales and expand the patient population eligible for the drug in future quarters.

More About Alnylam Pharmaceuticals: Major Partnerships with Novartis, Roche, and Regeneron Drive Drug Approvals and Revenue Growth

Cambridge, MA-based Alnylam Pharmaceuticals Inc. is a biopharmaceutical company specializing in RNA interference (RNAi) therapeutics. The company focuses on three strategic therapeutic areas: genetic medicines, cardio-metabolic disease, and hepatic infectious disease. Its pipeline includes experimental RNAi therapies targeting these areas.

In August 2018, Alnylam’s lead drug, Onpattro (patisiran), received regulatory approval in the United States and Europe for hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Onpattro is the first FDA-approved treatment for this condition. Subsequently, the FDA approved Givlaari (givosiran) for acute hepatic porphyria (AHP) in November 2019. In November 2020, Oxlumo (lumasiran) received FDA approval for primary hyperoxaluria type 1 (PH1). Most recently, in June 2022, the FDA and European Commission approved Amvuttra (vutrisiran) for polyneuropathy of hATTR amyloidosis.

Additionally, Alnylam has developed inclisiran for hypercholesterolemia in collaboration with Novartis. Novartis received marketing authorization for Leqvio (inclisiran) from the European Commission in December 2020, followed by FDA approval in December 2021. Alnylam is also co-developing zilebesiran with Roche for hypertension. Furthermore, Alnylam’s RNAi expertise has led to collaborations with major companies such as Regeneron, Novartis, Roche, and Genzyme. For 2023, Alnylam reported revenues of $1.83 billion, up from $1.04 billion in 2022.

Author

Bernice Lottering