ANG-003

Blackstone makes $250M bet on digestive enzyme therapyFDA rebukes Alnylam for Amvuttra marketingUK scientists push hantavirus vaccine toward clinicBlackstone makes $250M bet on digestive enzyme therapyBlackstone Life Sciences is investing $250 million to help Anagram Therapeutics advance an experimental oral enzyme replacement therapy for people with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).Current pancreatic enzyme replacement therapies are derived from pig pancreas glands and can require patients to take as many as 40 pills a day. Anagram says its lead candidate, ANG003, could lower that burden to a single tablet while also becoming the first non-porcine therapy in the category."The large unmet need in EPI is clear as gastrointestinal (GI) symptoms and global supply issues for existing porcine derived products continue to be a real problem," said Kiran Reddy, senior managing director at Blackstone. Anagram's recombinant enzyme therapy was engineered to remain stable and immediately active in the GI tract to improve digestion and nutrient absorption. It contains lipase for fat malabsorption, protease for protein malabsorption, and amylase for carbohydrate malabsorption.The investment builds on more than $30 million in prior backing from the Cystic Fibrosis Foundation, and comes as Anagram prepares to launch an international Phase II trial of the therapy after presenting positive data from an earlier clinical study.FDA rebukes Alnylam for Amvuttra marketingThe FDA has accused Alnylam Pharmaceuticals of making misleading promotional claims about the survival benefit of its ATTR amyloidosis drug Amvuttra (vutrisiran). Initially approved for transthyretin amyloidosis with polyneuropathy in 2022, the gene-silencing treatment saw its label expanded in the US last year to include the cardiomyopathy form of the disease (ATTR-CM).However, in a recent letter to Jennifer Backo, Alnylam's senior director of regulatory affairs, the FDA challenged statements on the company's consumer website that claim the therapy was "proven to help people with ATTR-CM live longer." According to the agency, claims on the site created a "misleading impression" that Amvuttra demonstrated a "definitive and quantified benefit, with specific established levels of efficacy, on mortality over 3.5 years, when this is not the case."The FDA said the claims relied on data from the open-label, extension portion of the three-year Phase III HELIOS-B study, after placebo patients had already crossed over to receive Amvuttra. Because no true placebo comparator group remained during the six-month extension period, regulators argued the study design could not support "conclusory representations" about the impact of Amvuttra on all-cause mortality over 3.5 years.The agency asked that Alnylam immediately cease or revise promotional materials containing similar representations and submit a written response within 15 working days. Amvuttra has rapidly become a major growth driver for Alnylam, generating first-quarter sales of $890 million.UK scientists push hantavirus vaccine toward clinicA new vaccine against hantavirus is under development at the University of Bath as global health officials respond to a suspected outbreak on a cruise ship that has led to the deaths of several passengers. There is currently no vaccine against the virus, which originates and is transmitted by rodents. The vaccine candidate has shown "excellent immune response" in the lab and in animal models, according to researcher Asel Sartbaeva, a co-founder of university spinout company Ensilitech. The team expects to proceed to Phase I testing in the near future, according to a report in the Wiltshire Times.The vaccine uses a combination of mRNA technology and a proprietary stabilisation method known as Ensilication developed by Sartbaeva. The approach encases proteins in protective silica structures, designed to keep them stable at ambient temperatures and reduce reliance on cold-chain refrigeration.