Investment aims to advance pancreatic insufficiency treatment
Blackstone commits $250M to Anagram’s replacement therapy
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Blackstone Life Sciences is investing $250 million in Anagram Therapeutics to advance ANG003, an oral enzyme replacement therapy (ERT) for exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).
ANG003 is designed to reduce the “enormous, disruptive” pill burden for CF patients with EPI, which can amount to 40 pills a day, Kiran Reddy, MD, senior managing director at Blackstone, said in a company press release. “We expect ANG003 to only require one tablet per meal which we believe will positively impact compliance and quality of life,” Reddy said.
The company said the treatment also has the potential to become the first ERT to contain no pig-derived enzymes.
The investment will support ANG003’s clinical development, including a Phase 2 clinical trial, potential approval, and an eventual commercial launch.
The announcement follows positive data from a Phase 1 clinical trial (NCT06052293), which was funded by the Cystic Fibrosis Foundation. The foundation has contributed more than $30 million to enable the company to develop the drug.
Transforming treatment
“We believe Anagram is well positioned to transform the treatment of pancreatic insufficiency, especially in patients of all ages who suffer from cystic fibrosis,” said Nicholas Galakatos, PhD, global head of Blackstone Life Sciences. “This is an excellent case study of our ownership strategy where we bring scale capital, deep domain expertise, and hands-on engagement to help address large unmet medical needs.”
Most people with CF develop pancreatic insufficiency, which occurs when thick, sticky mucus blocks the release of digestive enzymes from the pancreas into the gut. Without enough of these enzymes, the body has trouble breaking down and absorbing nutrients from food, particularly fats, proteins, and carbohydrates. This can lead to poor nutrition, digestive issues, and reduced quality of life.
ERT provides digestive enzymes to help people absorb nutrients from food. Several available ERT products contain pancrelipase, which is derived from pigs. Reddy said global supply issues for pig-derived products “continue to be a real problem.”
ANG003 contains lipase, protease, and amylase — enzymes that help digest fats, proteins, and carbohydrates, respectively. The company said the treatment is designed to be stable and immediately active in the digestive tract, to enhance digestion and nutrient absorption.
In the Phase 1 study, 51 adults with CF-related EPI were evaluated once without ERT and again after receiving a single dose of one of four ANG003 dosing combinations with a meal.
Results showed that ANG003 improved absorption of docosohexaenoic acid and eicosapentaenoic acid, two types of fatty acids, with statistically significant improvements at 80 mg and 120 mg lipase doses compared with 20 mg. Total fat absorption followed a similar pattern, and the highest protease dose also improved a marker of protein digestion. Adverse events were mild and transient, and no serious adverse events occurred.
“We believe the clinical data generated from the [Phase 1] study in patients with EPI due to CF is compelling and has the potential to be a transformational treatment for people living with EPI,” said Robert Gallotto, President and CEO of Anagram.
ANG003 is Anagram’s lead treatment candidate for EPI and malabsorption.
