Alnylam Pharmaceuticals Inc Stock Price, News & Analysis

Alnylam Pharmaceuticals (Nasdaq: ALNY) will present at major healthcare conferences, including the Jefferies Healthcare Conference on June 9, 2022, and the Goldman Sachs 43rd Annual Global Healthcare Conference on June 13, 2022. Live audio webcasts of the presentations will be accessible on the Investors section of their website, with replays available within 48 hours. Alnylam is a leader in RNA interference therapeutics, offering innovative treatments for rare diseases, with a deep pipeline and a commitment to delivering transformative medicines globally.

Alnylam Pharmaceuticals has published promising preclinical data in Nature Biotechnology showing that 2'-O-hexadecyl (C16)-conjugated siRNA can effectively silence target genes outside the liver, particularly in the CNS, eye, and lung. In rodent and non-human primate models, C16-siRNA targeting amyloid beta precursor protein showed significant reductions in target levels and improved behavioral outcomes in an Alzheimer's disease model. The findings support infrequent dosing and have led to the initiation of a Phase 1 study for ALN-APP, targeting early-onset Alzheimer's.

Alnylam Pharmaceuticals announced positive results from the ILLUMINATE-C Phase 3 study of lumasiran, an RNAi therapeutic for advanced primary hyperoxaluria type 1 (PH1). The study demonstrated significant reductions in plasma oxalate levels in patients with severe renal impairment and improvements in exploratory endpoints such as cardiac measures, nephrocalcinosis, and kidney stone events. Patient symptoms including fatigue and bone pain showed notable improvements. Future analyses will continue over a 54-month extension period to evaluate long-term clinical outcomes.

Alnylam Pharmaceuticals (Nasdaq: ALNY) reported positive 18-month findings from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic for hereditary ATTR amyloidosis. The analysis indicated significant improvements in NT-proBNP levels, a key cardiac stress marker, in patients treated with vutrisiran compared to an external placebo. Cardiac echocardiographic parameters also showed favorable trends. Vutrisiran treatment reduced cardiac technetium uptake, suggesting benefits for patients with high amyloid burden. The drug has Orphan Drug Designation and is undergoing review by regulatory authorities, with a PDUFA date of July 14, 2022.

Alnylam Pharmaceuticals (NASDAQ: ALNY) announced key presentations at three upcoming conferences. Management will provide company overviews at the BofA Securities 2022 Healthcare Conference on May 11, 2022, at 11:20 am PT in Las Vegas, at the H.C. Wainwright Global Investment Conference on May 24, 2022, at 7:00 am ET virtually, and at the UBS Global Healthcare Conference on May 25, 2022, at 10:00 am ET in New York. Live audio webcasts will be available on the company’s website, with replays accessible within 48 hours.

Alnylam Pharmaceuticals reported Q1 2022 global net product revenues of $187 million, driven by ONPATTRO, GIVLAARI, and OXLUMO. Positive results from the HELIOS-A Phase 3 study of vutrisiran were also announced, indicating improvements in neuropathy impairment and quality of life for patients. However, the company has lowered its 2022 revenue guidance from $900-$1 billion to $870-$930 million. The FDA extended the review period for vutrisiran due to pending inspection issues, now targeting a July 14, 2022 decision.

Alnylam Pharmaceuticals (NASDAQ: ALNY) is set to report its first-quarter financial results for 2022 on April 28, 2022, before U.S. markets open. The company will also host a conference call at 8:30 AM ET to discuss these results and future expectations. Investors can access the live audio webcast via Alnylam's website. Alnylam is recognized for its innovative RNA interference (RNAi) therapies and has a robust pipeline with six candidates in late-stage development.

Alnylam Pharmaceuticals (Nasdaq: ALNY) announced the retirement of Steven Paul, M.D. from its Board of Directors effective April 16, 2022. Dr. Paul has served on the board since September 2010, contributing over 25 years of management experience in the pharmaceutical sector. His leadership has been crucial in guiding Alnylam's growth, including the launch of three products in recent years. CEO Yvonne Greenstreet acknowledged his significant contributions and wished him well in his future endeavors.

Alnylam Pharmaceuticals announced an extension of the New Drug Application (NDA) review for vutrisiran, an investigational treatment for transthyretin-mediated amyloidosis (ATTR). The new Prescription Drug User Fee Act (PDUFA) goal date is July 14, 2022, due to regulatory amendments concerning a third-party packaging facility. No FDA-requested clinical data is pending, and a new facility has been identified. Vutrisiran is also under review by the European Medicines Agency, the Brazilian Health Regulatory Agency, and the Japanese Pharmaceuticals and Medical Devices Agency.