Alnylam to Present HELIOS-B Phase 3 Vutrisiran Results at ESC Congress 2024

Alnylam Pharmaceuticals, Inc., a leading company in RNA interference (RNAi) therapeutics, recently announced that it will be presenting results from its HELIOS-B Phase 3 study involving vutrisiran, a treatment for ATTR amyloidosis with cardiomyopathy, at the upcoming European Society of Cardiology (ESC) Congress 2024 in London. The event is scheduled to take place from August 30 to September 2, 2024. The company has already shared positive topline results from the study, which met its primary endpoint by demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events. Additionally, the study also achieved all secondary endpoints in both the overall and monotherapy populations, with an acceptable safety profile.

Vutrisiran is currently being developed to treat ATTR amyloidosis with cardiomyopathy, a condition that can be debilitating. Alnylam Pharmaceuticals will also host an Investor Webcast on August 30, 2024, to discuss these results further.

The ESC Congress 2024 will also feature findings from other studies on RNAi therapeutics by Alnylam. These include a subgroup analysis of the KARDIA-2 Phase 2 study on zilebesiran, which is an investigational treatment for hypertension. Zilebesiran is designed to target angiotensinogen (AGT) to aid in blood pressure regulation, utilizing Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology. This technology allows for infrequent subcutaneous dosing, aiming to provide consistent and durable blood pressure reduction.

Moreover, the ESC Congress will showcase new data from post-hoc analyses of the APOLLO-B Phase 3 and APOLLO-OLE studies involving patisiran, another RNAi therapeutic. Patisiran aims to silence TTR messenger RNA, thereby reducing the production of TTR protein in the liver. It has been approved for treating the polyneuropathy of hereditary transthyretin amyloidosis (hATTR-PN) in adults in about 40 countries.

ATTR amyloidosis is an underdiagnosed and rapidly progressing disease caused by misfolded transthyretin (TTR) proteins that accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. It is categorized into two types: hereditary ATTR (hATTR), caused by a TTR gene variant, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant.

The company will detail the results of the HELIOS-B study, which will be presented by Dr. Marianna Fontana on August 30, 2024. Additionally, Dr. Manish Saxena will present findings from the KARDIA-2 study focusing on the impact of demographic and baseline disease characteristics on zilebesiran response in hypertensive patients. Dr. Olivier Lairez will discuss long-term effects of patisiran on survival and cardiac parameters in patients with transthyretin-mediated cardiac amyloidosis on September 1, 2024.

Alnylam Pharmaceuticals has been at the forefront of RNA interference technology, translating this scientific breakthrough into a new class of therapeutics. Since its inception in 2002, the company has been committed to developing RNAi-based treatments for various diseases, contributing significantly to the field of gene silencing and therapeutic development.

RNA interference is a natural cellular process of gene silencing that has opened new avenues in biology and drug development. This technology uses small interfering RNA (siRNA) molecules to silence specific genes, preventing the production of disease-causing proteins. Alnylam's RNAi therapeutic platform uses these molecules to target and silence messenger RNA (mRNA), which encodes for these harmful proteins.

Alnylam continues to develop its pipeline of investigational medicines, aiming to address both rare and common diseases with significant unmet medical needs. The company's efforts are part of their broader "Alnylam P5x25" strategy to deliver transformative medicines and achieve sustainable innovation and financial performance.

In summary, Alnylam’s upcoming presentations at the ESC Congress 2024 highlight the company's commitment to advancing RNAi therapeutics. The results from the HELIOS-B Phase 3 study of vutrisiran and other investigational studies on zilebesiran and patisiran are expected to provide valuable insights into the treatment of complex diseases like ATTR amyloidosis and hypertension, thereby pushing the boundaries of current medical science and patient care.