Alnylam Presents New Long-Term Results from Global Open-Label Extension Study of ONPATTRO (patisiran) at the European Academy of Neurology Virtual Congress 2020

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today presented new results from the Global Open-Label Extension (OLE) study of ONPATTRO (patisiran), an RNAi therapeutic for the treatment of the polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults, at the European Academy of Neurology (EAN) Virtual Congress. In addition, interim results were presented from a Phase 3b trial evaluating treatment with patisiran in patients with hATTR amyloidosis with disease progression after receiving an orthotopic liver transplant (post-OLT).

“In collaboration with our clinical investigators, we are pleased to have the opportunity to share data from our patisiran clinical program in two abstracts at this year’s EAN virtual conference. In particular, results from our Global OLE study demonstrate sustained benefit for patients with hATTR amyloidosis with polyneuropathy treated with ONPATTRO, with maintained improvement in polyneuropathy symptoms and quality of life after an additional 24 months of treatment. As of March 2020, thirteen patients who rolled onto the Global OLE study have received ONPATTRO for more than six years, the longest-running clinical experience with an RNAi therapeutic,” said Eric Green, SVP and General Manager of the TTR Program. “In addition, the interim post-OLT data demonstrated robust TTR knockdown in this very high unmet need population and point to the breadth of patients that may benefit from treatment with ONPATTRO.”

Updated Results from Global OLE Study

24-month interim results were presented from the ongoing Global OLE study of patisiran evaluating the drug’s long-term efficacy and safety in eligible patients (N=211) who completed the Phase 2 OLE (N=25) and APOLLO Phase 3 (N=186) studies. The data shared include 178 patients who had 24 months or greater of exposure as of an October 7, 2019 cutoff date. Reductions in serum TTR levels were maintained in patisiran-treated patients with continued dosing in the Global OLE study. Patients on treatment for 42 months demonstrated sustained improvement in neuropathy impairment and quality of life relative to APOLLO study baseline, as shown by mean negative changes in modified Neuropathy Impairment Score + 7 (mNIS+7) and Norfolk Quality of Life – Diabetic Neuropathy (QOL-DN) scores. Patients on treatment from the Phase 2 OLE population also demonstrated an improvement in mNIS+7 score over 48 months.