Blackstone Life Sciences Invests $250M in Anagram Therapeutics

Blackstone Life Sciences (BXLS) announces a $250 million investment in Anagram Therapeutics, a clinical-stage biopharmaceutical company focused on developing innovative oral enzyme replacement therapies. The funding will accelerate the development, regulatory approval, and commercialization of ANG003, a novel orally delivered recombinant enzyme therapy designed to treat exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF) and related disorders.

Patients with EPI currently face a significant treatment burden, often requiring up to 40 pills daily to manage digestive enzyme deficiencies. ANG003 aims to reduce this burden substantially by requiring only one tablet per meal. Additionally, ANG003 is the first non-porcine extract enzyme replacement therapy, addressing supply shortages and patient concerns associated with pig-derived products.

Anagram Therapeutics, headquartered in Natick, Massachusetts, specializes in orally delivered enzyme therapeutics targeting malabsorption syndromes and nutrient metabolism disorders. Its lead product, ANG003, contains a broad spectrum of digestive enzymes—lipase, protease, and amylase—engineered for stability and immediate activity in the gastrointestinal tract to maximize nutrient absorption.

Dr. Nicholas Galakatos, Global Head of Blackstone Life Sciences, states, “We believe Anagram is well positioned to transform the treatment of pancreatic insufficiency, especially in patients of all ages who suffer from cystic fibrosis. This investment exemplifies our strategy of combining capital, expertise, and operational support to address large unmet medical needs.”

Robert Gallotto, President and CEO of Anagram Therapeutics, adds, “The clinical data from our ANG003-22-101 study in patients with EPI due to CF is compelling. We are grateful for Blackstone Life Sciences’ support as we advance ANG003 and other oral enzyme therapies for rare diseases. We also acknowledge the Cystic Fibrosis Foundation for their early funding and ongoing partnership.”

The investment follows over $30 million in prior funding from the Cystic Fibrosis Foundation, which supported early clinical development. Anagram is now initiating an international Phase 2 study to further evaluate ANG003’s efficacy and safety.

The current U.S. market for pancreatic enzyme replacement therapies (PERT) is approximately $2 billion annually. Existing treatments rely on porcine-derived enzymes, which face global supply constraints and require extensive pill regimens due to protective coatings needed to survive stomach acid. ANG003’s recombinant, non-porcine formulation offers a potential breakthrough by simplifying dosing and improving patient compliance.

Blackstone Life Sciences manages $17 billion in assets and invests across the life cycle of companies and products in the life sciences sector. Its hands-on approach combines capital with operational leadership to bring innovative medicines to market.

Looking ahead, Blackstone and Anagram plan to leverage their combined expertise to navigate clinical development risks, regulatory approvals, and manufacturing scale-up challenges. The partnership aims to establish ANG003 as a leading oral enzyme replacement therapy, expanding treatment options for patients with EPI beyond cystic fibrosis to include pancreatic cancer and other disorders.

As the market evolves, competitors offering porcine-based therapies may face pressure to innovate or adjust pricing strategies in response to ANG003’s novel profile and reduced pill burden. Successful commercialization could significantly improve quality of life for patients and capture a meaningful share of the growing rare disease treatment market.