Alnylam Adds Amyloidosis Candidate to Growing List of Wins
Alnylam CEO Yvonne Greenstreet, Courtesy of Alnylam Pharmaceuticals
RNAi-focused biopharma company Alnylam Pharmaceuticals revealed topline results from the Phase III APOLLO-B study, showing that its candidate patisiran improves both functional capacity and quality of life in patients with transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.
With 69 sites across 21 countries, APOLLO-B is a randomized, double-blinded and placebo-controlled trial that seeks to evaluate patisiran in 360 ATTR amyloidosis patients with cardiomyopathy. The study showed that 12 months of treatment with Alnylam’s candidate led to significantly better improvements in the 6-minute walk test compared to placebo.
Patisiran-treated patients scored higher on the Kansas City Cardiomyopathy Questionnaire than placebo counterparts, indicating a much better quality of life.
Aside from being effective, the drug also demonstrated an encouraging safety profile. Over 12 months of treatment, fewer patients in the patisiran arm died as opposed to placebo. Adverse events of all severities also arose at similar frequencies between treatment groups. Common side effects that could be related to patisiran include infusion-related reactions, arthralgia, and muscle spasms.
“We are thrilled that APOLLO-B successfully met all its major objectives, which we believe for the first time validates the hypothesis that TTR silencing by an RNAi therapeutic can be an effective approach for treating the cardiomyopathy of ATTR amyloidosis,” Pushkal Garg, M.D., chief medical officer of Alnylam, said in a statement.
The company is expecting full results by September and is looking to file a New Drug Application for patisiran later this year.
Patisiran’s promising Phase III performance is but the latest win for Alnylam. On Tuesday, the Cambridge, Massachusetts-based firm won first place on Fast Company’s annual list of Best Workplaces for Innovators, besting hundreds of companies across many other industries.
Just a few days earlier, Alnylam’s big scientific discovery made it into a peer-reviewed publication. The paper, published in Nature Communications, identified rare mutations that deactivated the INHBE gene and showed how these could exert a protective effect against abdominal obesity. Carriers of this mutation also had better metabolic profiles—low triglyceride and fasting glucose levels and elevated concentrations of good cholesterol.
After this discovery, Alnylam has expressed its intent to develop a candidate targeting INHBE and its protein product Activin E.
June was also a stellar month for Alnylam, opening with another peer-reviewed paper, showing promising preclinical evidence that its delivery platform for short interfering RNAs could be leveraged to treat central nervous system diseases. Shortly after, the company announced that cemdisiran, its RNAi candidate for immunoglobulin A nephropathy, aced Phase II assessments based on early data and is likely to qualify for a Phase III trial.
Continuing the company’s midyear rally is Amvuttra (vutrisiran), which earned an early FDA approval in June for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults.
Alnylam’s science is based on the young field of RNA interference, which uses small bits of stray RNA to bind protein-coding mRNA transcripts, thereby stopping them from being translated into proteins. This cutting-edge approach has driven the company’s strong growth.
According to its Q2 financial report, Alnylam’s lead assets, Onpattro, Givlaari and Oxlumo, earned the company some $214 million, up 33% from the same period last year. The company’s future seems secure, too, with its R&D projects progressing well and several of its candidates expected to move forward in their respective trials.
“We believe the first half of 2022 has been one of steady progress and commercial execution, and we look forward to the many important milestones through the end of the year,” Yvonne Greenstreet, MBChB, chief executive officer of Alnylam, said in a release accompanying its Q2 results.