Alnylam Pharmaceuticals, Inc. - ALNY STOCK NEWS

Alnylam Pharmaceuticals (ALNY) announced FDA acceptance of their supplemental New Drug Application (sNDA) for vutrisiran to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM). The FDA set a PDUFA date of March 23, 2025. The application is based on positive results from the HELIOS-B Phase 3 study, which showed favorable effects on cardiovascular outcomes, survival, and quality of life in ATTR-CM patients. If approved, vutrisiran would become the first treatment approved in the U.S. for both polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis.

Alnylam Pharmaceuticals presented new Phase 1 study results for nucresiran, a next-generation RNAi therapeutic for ATTR amyloidosis treatment. A single dose of ≥300mg achieved rapid TTR knockdown of >90% by Day 15, sustained through Day 180. Peak reductions of >96% were reached by Day 29. At 300mg, TTR reduction remained >70% at Day 360. The drug showed encouraging safety profile with low inter-patient variability. The 600mg dose achieved 95.0% TTR reduction at Day 15 and 96.0% at Day 180, while the 900mg dose showed 91.7% reduction at Day 15 and 94.2% at Day 180. The data supports potential biannual or annual dosing regimens.

Alnylam Pharmaceuticals (Nasdaq: ALNY) has announced its upcoming presentations at two major investor conferences in November 2024. The company will present at the UBS Global Healthcare Conference on November 12 at 8:45 am PT in Ranchos Palos Verdes, CA, and at the Jefferies London Healthcare Conference on November 20 at 9:30 am GMT in London.

Live audio webcasts will be accessible through the Investors section of Alnylam's website, with replays available within 48 hours after each presentation.

Alnylam Pharmaceuticals (ALNY) reported strong Q3 2024 financial results with global net product revenues of $420 million, representing 34% year-over-year growth. The growth was primarily driven by their TTR business, with AMVUTTRA generating $259 million and ONPATTRO $50 million in revenues. The company submitted regulatory applications in U.S. and EU for AMVUTTRA for ATTR-CM treatment. Cash position stood at $2.78 billion as of September 30, 2024. The company reiterated its 2024 financial guidance, expecting combined net product revenues of $1,575-$1,650 million.

Alnylam Pharmaceuticals (Nasdaq: ALNY) will present data from its ATTR amyloidosis and hypertension programs at the AHA Scientific Sessions 2024. The presentations include new analysis from the HELIOS-B Phase 3 study of vutrisiran for ATTR amyloidosis with cardiomyopathy, interim results from a Phase 1 study of ALN-TTRsc04, and new analysis of the KARDIA-1 Phase 2 study of zilebesiran for hypertension. The HELIOS-B study, published in The New England Journal of Medicine, achieved all 10 pre-specified primary and secondary endpoints.

Alnylam Pharmaceuticals (Nasdaq: ALNY), a leading RNAi therapeutics company, has announced that it will report its financial results for the third quarter of 2024 on Thursday, October 31, 2024, before the U.S. financial markets open. The company's management will provide an update and discuss the Q3 2024 results, along with future expectations, via a conference call at 8:30 am ET on the same day.

A live audio webcast of the call will be available on the Investors section of Alnylam's website at www.alnylam.com/events. An archived version of the webcast will be accessible approximately two hours after the event.

Alnylam Pharmaceuticals (Nasdaq: ALNY) has submitted a Type II Variation to the European Medicines Agency (EMA) for vutrisiran, an investigational RNAi therapeutic for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). This submission is based on positive results from the HELIOS-B Phase 3 study, which met all 10 primary and secondary endpoints with statistical significance.

The study demonstrated vutrisiran's effects on reducing mortality and cardiovascular events, as well as improving functional capacity, quality of life, and heart failure symptoms in ATTR-CM patients. Vutrisiran's safety profile was consistent with its established profile for hATTR amyloidosis with polyneuropathy.

Alnylam has also submitted a supplemental New Drug Application (sNDA) to the U.S. FDA for vutrisiran in ATTR-CM treatment, with additional global regulatory submissions planned.

Alnylam Pharmaceuticals (Nasdaq: ALNY) has submitted a supplemental New Drug Application (sNDA) to the FDA for vutrisiran, an investigational RNAi therapeutic for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). The submission is based on positive results from the HELIOS-B Phase 3 study, which demonstrated favorable effects on outcomes of death and cardiovascular events, functional capacity, and quality of life in ATTR-CM patients.

Alnylam utilized a Priority Review Voucher to accelerate the FDA review timeline. Vutrisiran, currently approved as AMVUTTRA® for treating polyneuropathy of hereditary ATTR amyloidosis in adults, showed a safety profile consistent with its established profile in the HELIOS-B study. The company believes vutrisiran has the potential to become a first-line therapy for ATTR-CM, pending regulatory approval.

Alnylam Pharmaceuticals (Nasdaq: ALNY) presented new data from the HELIOS-B Phase 3 study of vutrisiran for ATTR amyloidosis with cardiomyopathy (ATTR-CM) at the HFSA Annual Scientific Meeting 2024. The data showed that vutrisiran significantly improved echocardiographic assessments of cardiac structure, systolic function, and diastolic function compared to placebo over 30 months. Vutrisiran also demonstrated relative stability of cardiac biomarkers NT-proBNP and Troponin-I.

Key findings include:

• Improvements in cardiac wall structure, diastolic function, and systolic function
• 32% relative reduction in NT-proBNP and troponin I in the overall population
• Larger treatment effects observed in the monotherapy population

Alnylam plans to complete multiple global regulatory submissions for vutrisiran in ATTR-CM by the end of the year.