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Alnylam Pharma (ALNY) Announces New Advances in ATTR Amyloidosis Program

May 11, 2021 7:06 AM EDT

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has started a clinical study of a biannual dosing regimen of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. The study is being conducted during a randomized treatment extension (RTE) period in the HELIOS-A Phase 3 study, and will characterize the safety, efficacy, and TTR reduction of a 50 mg biannual dosing regimen of subcutaneously administered vutrisiran in patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy. In addition, the Company announces today advancement of a new pre-clinical ATTR amyloidosis program aimed at achieving highly potent and reversible TTR reduction of over 90 percent with an annual subcutaneous dosing regimen.

“Alnylam has a long-standing commitment to continuous innovation aimed at providing breakthrough treatment options for patients with ATTR amyloidosis. We’re pleased to have now initiated a clinical study of a biannual dosing regimen for vutrisiran. If shown to be safe and efficacious, the new dosing regimen may provide some patients with a treatment option that neatly aligns the frequency of administration with biannual visits to their doctor,” said Rena Denoncourt, Vice President, Vutrisiran Program Leader. “In addition, based on continued discoveries from our RNAi therapeutics platform, we’re also advancing a new pre-clinical program that could enable highly potent and reversible TTR reduction with an annual dosing regimen. We believe that once yearly dosing could provide a vaccine-like dosing schedule strategy for management of ATTR amyloidosis, providing meaningful life-cycle management for our ATTR franchise.”

As reported previously, positive results were achieved in HELIOS-A with a 25 mg quarterly dosing regimen of subcutaneously administered vutrisiran; a New Drug Application (NDA) for the approval of vutrisiran was filed with the U.S. Food and Drug Administration (FDA) in April 2021 for the treatment of the polyneuropathy of hATTR amyloidosis in adults1. The vutrisiran biannual dosing study is being conducted as part of the HELIOS-A randomized, open-label study. During the RTE period, which begins following completion of the 18-month HELIOS-A treatment period, patients are randomized 1:1 to receive 25 mg of vutrisiran quarterly or 50 mg of vutrisiran biannually for the remainder of the study. Patients will undergo periodic assessments for safety and TTR reduction, and efficacy assessments at months nine and 18 of the RTE. As previously disclosed in mid-2020, clinical pharmacology data indicate that a 50 mg biannual dose of vutrisiran is expected to achieve comparable TTR reduction – over 80 percent – to that demonstrated with the 25 mg quarterly dosing regimen of vutrisiran. Alnylam expects results from the RTE study in 2022; if positive, data would support a supplemental NDA (sNDA) submission to the FDA.

The new ATTR program announced today stems from continued innovation from Alnylam’s RNAi therapeutics platform efforts. Specifically, Alnylam scientists have developed an extended duration platform, called IKARIA™, that is aimed at highly potent (>90 percent) target mRNA silencing with an annual dosing regimen. Targeting mRNA with the IKARIA platform is expected to yield small interfering RNA (siRNA) with potentially long-acting and reversible effects and a proven RNAi mechanism of action. This new platform has yielded ALN-TTRsc04, which is planned to enter clinical development at or around year-end 2022 with an investigational new drug (IND) application filing. If successfully developed, ALN-TTRsc04 is expected to have minimal, if any, third-party royalty obligations. Alnylam plans to present data from its IKARIA platform and ALN-TTRsc04 program at a scientific meeting in mid-2021.

“Our platform team continues to discover innovations for RNAi therapeutics that we believe can deliver meaningful advances for patients. Our new IKARIA platform represents an excellent example,” said Kevin Fitzgerald, Ph.D., Senior Vice President, Chief Scientific Officer. “With IKARIA, we are confident we can design long-acting siRNA with all the proven pharmacological advantages and established human experience of RNAi therapeutics, including predictable dose-dependence and onset/offset kinetics, without permanent effects on the target or cell.”



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