Anagram Therapeutics, Inc.

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.

Massachusetts-based Anagram Therapeutics has closed a USD 250 million strategic investment from Blackstone Life Sciences to advance ANG003, a recombinant, non-porcine oral enzyme replacement therapy for exocrine pancreatic insufficiency caused by cystic firbrosis. A Phase II trial in cystic fibrosis patients is now initiating. Blackstone Life Sciences, the life sciences investment arm of Blackstone with approximately USD 17 billion in assets under management, is the sole investor in the transaction. The capital is intended to fund ANG003’s continued development, regulatory approval, and commercial launch. Prior to this deal, Anagram had received more than USD 30 million in cumulative funding from the Cystic Fibrosis Foundation, which supported the clinical and preclinical work underpinning ANG003’s development. Earlier government grants, including tranches from the US Department of Health and Human Services, contributed to the company’s early-stage funding. ANG003 is a broad-spectrum recombinant enzyme replacement therapy combining lipase, protease, and amylase — the three enzyme classes required to digest fats, proteins, and carbohydrates, respectively. The therapy is engineered for intrinsic stability and immediate activity in the gastrointestinal tract, which the company said allows it to function without the enteric coating required by current porcine-derived pancreatic enzyme replacement therapies such as Creon. Patients with exocrine pancreatic insufficiency currently take up to 40 capsules per day; Anagram said ANG003 is expected to require only one tablet per meal. The existing standard of care — porcine-derived pancreatic enzyme replacement — faces ongoing supply constraints and carries batch-to-batch variability inherent to animal-sourced biologics. ANG003’s recombinant, non-porcine origin positions it as a potential alternative that addresses both supply reliability and the formulation limitations of enteric-coated products. The US pancreatic enzyme replacement market is estimated at approximately USD 2 billion annually. Anagram is now initiating a Phase II multicenter, randomized, active-controlled study evaluating ANG003 at two dose levels in patients with exocrine pancreatic insufficiency due to cystic fibrosis, with Creon as the active comparator. The trial follows a previously completed Phase I/II study, referenced internally as ANG003-22-101, in which the company said ANG003 generated positive clinical data in this population. The company’s enzyme engineering capabilities are supported by a collaboration with ATUM, a bioengineering firm whose ProteinGPS platform applies machine learning to navigate protein sequence space and optimize candidates across multiple fitness dimensions — including catalytic activity, acid stability, and protease resistance — simultaneously. That partnership was extended in 2023, coinciding with ANG003’s entry into the clinic. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Privacy Terms About Us Copyright © 2026. AllSci Corp. All rights reserved.

Current pancreatic enzyme replacement therapies for exocrine pancreatic insufficiency carry a high treatment burden and are associated with global shortages and side effects such as weight loss and abdominal pain.\n For people living with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF), the only treatment option is a regimen that can include up to 40 pills a day. With a $250 million investment from Blackstone Life Sciences, Anagram Therapeutics hopes to reduce that pill burden to just three pills daily.The investment is intended to support the development, approval and launch of ANG003, an oral recombinant enzyme replacement therapy for pancreatic insufficiency, according to a May 7 release.BLXS’ move follows more than $30 million in funding from the Cystic Fibrosis Foundation, which has supported the clinical and development work for ANG003. The drug is designed for patients with CF and other conditions associated with EPI, who do not produce enough pancreatic enzymes to break down food and absorb nutrients. This deficiency can lead to malnutrition, gastrointestinal symptoms and other complications.\"We believe Anagram is well positioned to transform the treatment of pancreatic insufficiency, especially in patients of all ages who suffer from cystic fibrosis,\" Nicholas Galakatos, Ph.D., global head of BLXS, said in a statement.Anagram specializes in therapies targeting the CF complication EPI, pancreatic cancer and related disorders, with ANG003 serving as its lead product. Patients with EPI are currently treated with pancreatic enzyme replacement therapies (PERT) derived from pig pancreas glands, which carry a high treatment burden and are associated with global shortages and side effects such as weight loss and abdominal pain. ANG003’s phase 2 study for patients with EPI due to CF is not yet recruiting, but it is intended to evaluate the drug’s safety and compare it with AbbVie’s Creon, an approved PERT.BLXS launched its sixth fund in March with $6.3 billion in capital, 40% larger than its predecessor. The fund is part of the $15 billion in assets managed by BLXS, which has contributed to 34 regulatory approvals for medicines and devices. BLXS’ investment in Anagram follows the $925 million acquisition of Anthos by Novartis after Blackstone and Novartis founded Anthos in 2019.