Benzinga | Jun 7, 2021 07:33AM EDT

Alnylam Highlights Data From ATTR Amyloidosis Programs At Peripheral Nerve Society's 2021 Annual Meeting

? Phase 3b Open-Label Study Showed Treatment with Patisiran Achieved Rapid and Sustained Reduction in Serum TTR Levels in hATTR Amyloidosis Patients with Polyneuropathy Progression Following Orthotopic Liver Transplant --

? Additional Results from Pre-specified Patient Subgroups Analysis Included with Encore Presentation of HELIOS-A Phase 3 Study of Investigational Vutrisiran ?

Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced positive results from a Phase 3b open-label study conducted to evaluate the safety, efficacy and pharmacokinetics (PK) of patisiran in hereditary ATTR (hATTR) amyloidosis patients with polyneuropathy progression after receiving an orthotopic liver transplant (OLT). In patients treated with patisiran, the median reduction in serum TTR levels compared to baseline was 91 percent, measured as an average of the month six and month 12 reduction. In addition, the safety profile of patisiran was consistent with the previously reported safety results observed in the APOLLO Phase 3 study. Patisiran is the established name for ONPATTRO(r), which is approved in the United States, Canada and Japan for the treatment of the polyneuropathy of hATTR amyloidosis in adults, and in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy. These data will form the basis of post-approval supplements which have the potential to change labeling for ONPATTRO where approved, including in the European Union.

In addition, positive results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, were presented today with additional data from pre-specified patient subgroups. Improvement in the modified Neuropathy Impairment Score (mNIS+7) and Norfolk Quality of Life-Diabetic Neuropathy score (Norfolk QOL-DN) from vutrisiran treatment was consistently observed across all pre-specified patient subgroups, including age, sex, race, geographic region, baseline neuropathy impairment, genotype, prior TTR stabilizer use, baseline Familial Amyloid Polyneuropathy (FAP) stage, and in a pre-specified cardiac subpopulation. These data were presented as posters during the 2021 Peripheral Nerve Society's Annual Meeting, and the HELIOS-A results will also be featured during the Richard A.C. Hughes - Clinical Science Highlights Presentation on Sunday, June 27th.

"We are excited to share new data from our TTR clinical program at this year's PNS virtual conference, which help demonstrate the potential of patisiran and vutrisiran for a broad group of patients with hATTR amyloidosis with polyneuropathy. Patients with hATTR amyloidosis who experience polyneuropathy progression post-OLT have a significant treatment need and the results of the Phase 3b study of patisiran demonstrated robust TTR knockdown, improved neuropathy, and quality of life after 12 months of treatment," said John Vest, M.D., Vice President of Clinical Research at Alnylam. "In addition, results from our HELIOS-A study of investigational vutrisiran underscore the potential of vutrisiran as an attractive potential new treatment option for hATTR amyloidosis patients with polyneuropathy with subcutaneous administration and quarterly dosing."