Alnylam Pharmaceuticals develops and commercializes RNA interference (RNAi) therapeutics, with news centered on its approved medicines, TTR franchise, clinical pipeline and financial performance. Company updates commonly address AMVUTTRA/vutrisiran and ONPATTRO/patisiran for transthyretin-mediated amyloidosis, GIVLAARI/givosiran for acute hepatic porphyria, OXLUMO/lumasiran for primary hyperoxaluria type 1, and partner-commercialized RNAi medicines.
Recurring developments include quarterly product revenue reports, guidance, clinical and real-world data for ATTR-CM and other cardiovascular programs, pipeline progress across cardio-metabolic disease, neuroscience and hematology, disease-awareness collaborations, and investor conference presentations. Alnylam also reports on its Alnylam 2030 strategy and commercialization of RNAi medicines in global markets.
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.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has announced plans to webcast its TTR Investor Day event on October 9, 2024, at 8:30 am ET. The event will be accessible through the Investors section of the company's website, with a replay available within 48 hours.
The TTR Investor Day will feature presentations from Alnylam's management team, including senior commercial leaders who will discuss launch preparation and market leadership potential in ATTR amyloidosis with cardiomyopathy (ATTR-CM). Dr. Ahmad Masri, Associate Professor of Medicine at Oregon Health & Science University, will join as a leading expert in ATTR amyloidosis to share insights on clinical management of ATTR-CM and unmet medical needs.
Alnylam Pharmaceuticals (ALNY) announced positive results from the HELIOS-B Phase 3 study of vutrisiran for ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study met all primary and secondary endpoints, showing significant benefits in reducing mortality and cardiovascular events.
Key findings include:
- 28% reduction in primary composite of all-cause mortality and recurrent cardiovascular events in the overall population
- 36% reduction in all-cause mortality up to 42 months in the overall population
- 33% reduction in primary composite endpoint in the monotherapy population
- Significant improvements in disease progression measures and cardiac biomarkers
Vutrisiran demonstrated an encouraging safety profile consistent with previous studies. Alnylam plans to submit regulatory filings globally, starting later this year.
Alnylam Pharmaceuticals (Nasdaq: ALNY), a leader in RNAi therapeutics, has announced its participation in two major investor conferences in September 2024. The company will present overviews at the Wells Fargo 2024 Healthcare Conference in Boston on September 4 at 9:30 am ET, and the Morgan Stanley 22nd Annual Global Healthcare Conference in New York City on the same day at 1:05 pm ET.
Investors and interested parties can access live audio webcasts of both presentations through the Investors section of Alnylam's website at www.alnylam.com/events. Replays will be made available within 48 hours after each event, allowing those unable to attend live to catch up on the company's latest updates and strategies.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present detailed results from the HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy at the European Society of Cardiology (ESC) Congress 2024. The study met its primary endpoint, showing a significant reduction in all-cause mortality and recurrent cardiovascular events. Vutrisiran also demonstrated an acceptable safety profile.
The company will host an Investor Webcast on August 30, 2024, to discuss the results. Additionally, Alnylam will present findings from the KARDIA-2 Phase 2 study of zilebesiran for hypertension and new data from post-hoc analyses of the APOLLO-B Phase 3 and APOLLO-OLE studies of patisiran in ATTR amyloidosis with cardiomyopathy patients.