Blackstone invests $250M in Anagram to reduce burden of cystic fibrosis complication

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.