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Alnylam Reports Positive Topline Results from ILLUMINATE-A Phase 3 Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1
− Lumasiran Met Primary and All Tested Secondary Endpoints, with Significant Reduction in Urinary Oxalate Levels Relative to Placebo – − Alnylam Intends to
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n− Lumasiran Met Primary and All Tested Secondary Endpoints, with Significant Reduction in Urinary Oxalate Levels Relative to Placebo –\n\n\n− Alnylam Intends to File New Drug Application (NDA) and Marketing Authorisation Application (MAA) in Early 2020 –\n\n\n− Full Results to be Presented at OxalEurope International Congress in March 2020 –\n\n\n− Alnylam to Host Conference Call Today at 8:00 am ET –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the ILLUMINATE-A Phase 3 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1), met its primary efficacy endpoint and all tested secondary endpoints. Specifically, lumasiran met the primary efficacy endpoint of percent change from baseline, relative to placebo, in 24-hour urinary oxalate excretion averaged across months 3 to 6 (p less than 0.0001). The study also achieved statistically significant results for all six tested secondary endpoints (p less than or equal to 0.001). Lumasiran also demonstrated an encouraging safety and tolerability profile. Based on these results, the Company plans to submit a New Drug Application (NDA) and file a Marketing Authorisation Application (MAA) for lumasiran in early 2020.\n\n\n“We are very pleased to report positive topline Phase 3 results for lumasiran, our third wholly owned investigational RNAi therapeutic. Patients living with PH1 and their families are faced with the burden of recurrent and painful stone events and a progressive and unpredictable decline in kidney function that ultimately results in end-stage renal disease and the need for intensive dialysis as a bridge to dual liver/kidney transplantation. The results from ILLUMINATE-A demonstrate that lumasiran can significantly reduce the hepatic production of oxalate, which we believe can thereby address the underlying pathophysiology of PH1,” said Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam. “Further, we are encouraged by the safety and tolerability profile of lumasiran and believe this investigational medicine has the potential to have a meaningful clinical impact on patients living with PH1. We look forward to submitting regulatory filings in early 2020 and advancing ...