Alnylam Pharmaceuticals Inc Stock Price, News & Analysis

Alnylam Pharmaceuticals (Nasdaq: ALNY) will report its financial results for Q4 and year-end 2021 on February 10, 2022, before U.S. market opening. A conference call will be held at 8:30 am ET the same day to discuss results and future expectations. Notable RNAi therapeutics by Alnylam include ONPATTRO, GIVLAARI, OXLUMO, and Leqvio, with a robust pipeline including six late-stage candidates. The company aims to deliver innovative treatments for rare and prevalent diseases, ensuring strong financial performance.

Alnylam Pharmaceuticals announced that its investigational drug vutrisiran met all secondary endpoints of the HELIOS-A Phase 3 study at 18 months. The study demonstrated statistically significant improvements in neuropathy impairment, quality of life, gait speed, nutritional status, and overall disability compared to placebo. Additionally, vutrisiran showed potential for cardiac benefits, including reduced technetium uptake, suggesting possible amyloid regression. The drug is under regulatory review globally, with a potential FDA action date of April 14, 2022.

Ceptur Therapeutics, a biotechnology company focused on U1 Adaptor technology, has successfully completed a $75M Series A financing round. The round was co-led by venBio Partners and Qiming Venture Partners USA, with participation from various investors including Bristol Myers Squibb and Janus Henderson Investors. The funds will be used to advance their genetic medicine pipeline. Ceptur's U1 Adaptors aim to regulate gene expression at the pre-mRNA level, offering innovative therapeutic applications for genetic diseases.

Alnylam Pharmaceuticals has partnered with n-Lorem Foundation to support nano-rare disease patients through innovative therapies. The collaboration aims to utilize Alnylam's RNA interference technology alongside n-Lorem's experimental antisense oligonucleotide medicines. This partnership is designed to identify treatment opportunities for rare diseases affecting 1 to 30 patients globally, addressing significant healthcare challenges.

Alnylam Pharmaceuticals announced the upcoming presentation of full 18-month results from the HELIOS-A Phase 3 study of vutrisiran, aimed at treating hereditary transthyretin-mediated (hATTR) amyloidosis. This presentation will occur on January 21, 2022, at the Société Francophone du Nerf Périphérique Annual Meeting in Paris. Vutrisiran is under review by multiple regulatory agencies, including the FDA and EMA, with a PDUFA action date set for April 14, 2022. The study involved 164 patients and sought to evaluate the drug's efficacy and safety.

Soleo Health has announced FDA-approved access to OXLUMO™ (lumasiran) for treating primary hyperoxaluria type 1 (PH1) in patients as young as three months. This marks a significant advancement in specialty pharmacy services for an ultra-rare genetic disorder characterized by excessive oxalate production, leading to kidney issues. Soleo Health will administer OXLUMO injections in its Ambulatory Infusion Centers, supported by trained clinical teams. The initiative aligns with Soleo's commitment to innovative pharmacy services and patient-centered care.

Alnylam Pharmaceuticals reported preliminary global net product revenues of $662 million for 2021, marking an 83% growth compared to 2020. The company also maintained a strong balance sheet with approximately $2.4 billion in cash and investments. Key revenue contributions came from ONPATTRO, GIVLAARI, and OXLUMO, with ONPATTRO alone generating around $475 million for the full year. Alnylam anticipates significant improvement in its non-GAAP operating loss for 2021 and highlighted its successful launch of Leqvio in partnership with Novartis.

Alnylam Pharmaceuticals has entered a collaboration with Novartis to develop a siRNA-based therapy aimed at restoring functional liver cells for patients with end-stage liver disease (ESLD). This partnership seeks to create a non-invasive alternative to liver transplantation. A major challenge in ESLD is the limited availability of donor organs, impacting over one million deaths annually. The three-year agreement allows Alnylam to leverage Novartis' proprietary assays to identify therapeutic candidates, with further development transitioning to Novartis.

Alnylam Pharmaceuticals has appointed Akshay Vaishnaw, M.D., Ph.D., as President, enhancing its commitment to scientific leadership and sustainable innovation. Dr. Vaishnaw has been with the company since 2006, previously serving as President of R&D. His promotion reflects Alnylam's strategy to advance RNAi therapeutics, which are poised to transform treatment options for various diseases. CEO Yvonne Greenstreet emphasized the importance of this leadership change in driving long-term value through innovative medicines.