Alnylam Pharmaceuticals, Inc. - ALNY STOCK NEWS

Alnylam Pharmaceuticals announced FDA approval for AMVUTTRA™ (vutrisiran), the first RNAi therapeutic for hereditary transthyretin-mediated (hATTR) amyloidosis, administered subcutaneously every three months. The approval follows the HELIOS-A Phase 3 study, where over 50% of patients showed improvement or reversal in neuropathy symptoms. AMVUTTRA met all primary and secondary endpoints with significant enhancements in quality of life and gait speed. Launch is anticipated in early July, supported by value-based agreements.

Alnylam Pharmaceuticals (Nasdaq: ALNY) recently reported topline results from a Phase 2 study of cemdisiran, an investigational RNAi therapeutic for IgA Nephropathy (IgAN). The treatment led to a 37% mean reduction in the 24-hour urine protein to creatinine ratio, a key indicator for disease progression. The study demonstrated an acceptable safety profile, with no significant drug-related safety signals. Alnylam plans to advance cemdisiran into Phase 3 clinical development to address the unmet needs in IgAN.

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.