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.