Alnylam (ALNY) Reports Phase III ENVISION Data on Givosiran
Alnylam Pharmaceuticals, Inc. ALNY announced positive complete results from the ENVISION phase III study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP).
In the study, givosiran met the primary efficacy endpoint, with a 74% mean reduction relative to placebo in the annualized rate of composite porphyria attacks. There was a corresponding 90% median reduction in composite annualized attack rate (AAR), with a median AAR of 1.0 in givosiran patients compared with a median AAR of 10.7 in placebo patients. In the trial, 50% of givosiran-treated patients were attack-free during the six-month treatment period as compared to 16.3% of placebo-treated patients.
Givosiran also achieved statistically significant positive results for five out of nine secondary endpoints, with an overall safety and tolerability profile. Adverse events (AEs) were reported in 89.6% of givosiran patients and 80.4% of placebo patients, while serious adverse events (SAEs) were reported in 20.8% of givosiran patients and 8.7% of placebo patients.
Shares of Alnylam have rallied 23.9% in the past year compared with the industry’s growth of 8.8%.
In March 2019, the company released ENVISION study data, where givosiran met the main goal of reducing the yearly number of attacks in patients with acute intermittent porphyria (AIP).
In October 2018, Alnylam announced positive early results from the interim analysis of the ENVISION phase III study. The results showed that treatment with givosiran led to a statistically significant reduction in urinary aminolevulinic acid (ALA) levels in AIP patients compared to placebo. The study also showed SAEs in 22% of givosiran patients and 10% of placebo patients in the interim analysis cohort of 43 patients, with one patient (4%) on givosiran discontinuing the treatment due to an increase in liver transaminase that was resolved.
The open-label extension (OLE) period of the study has already enrolled 99% of patients. Based on the ENVISION results, the company plans to complete its rolling submission of a new drug application (NDA) and file a Marketing Authorization Application (MAA) in mid-2019.
We remind investors that the company’s only approved drug, Onpattro, is a first-of-its-kind RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The drug is the first and only FDA-approved treatment for this indication.
Alnylam also has other candidates in its pipeline. The company along with partner The Medicine Company MDCO is evaluating inclisiran in phase III ORION studies for hypercholesterolemia.
Alnylam’s expertise in RNAi therapeutics and broad intellectual property estate have allowed it to enter collaborations with leading pharmaceutical and life sciences companies, including Ionis Pharmaceuticals, Novartis NVS, Roche, Takeda, Merck, The Medicines Company and Sanofi’s specialty care global business unit, Genzyme, among others.
Recently, Alnylam and Regeneron Pharmaceuticals REGN extended their collaboration agreement. Both the companies will work together to discover, develop and commercialize new RNAi therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and central nervous system (CNS), in addition to a select number of targets expressed in the liver.
Alnylam Pharmaceuticals, Inc. Price
Alnylam currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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