CF Foundation investing up to $15.5M in potential enzyme therapy

Therapy being developed by Anagram contains no animal-derived enzymes

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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The Cystic Fibrosis Foundation (CFF) is investing up to $15.5 million in Anagram Therapeutics to conduct early-stage trials of a new oral enzyme replacement therapy.

The company is planning to start a Phase 1 clinical trial this summer. The therapy would let people with cystic fibrosis (CF) take only one enzyme pill per meal to digest food properly, according to CFF.

“This potential therapy would be more convenient and provide another option to current therapies for people with CF who struggle with digestion,” JP Clancy, MD, senior vice president of clinical research at the CFF, said in a press release.

Most people with CF develop pancreatic insufficiency caused by the buildup of thick and sticky mucus that blocks the release of enzymes needed to digest food, particularly fats, proteins, and starch. Without those enzymes in the digestive tract, patients have difficulty breaking down food and absorbing it properly.

This can result in inadequate nutrition, as well as poor growth and weight gain in children, and complications such as gas and bloating.

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Pancreatic enzyme replacement therapy (PERT) provides the missing enzymes through supplements to help break down food. These products contain a combination of three types of digestive enzymes: lipase, to break down fats; protease, to break down proteins; and amylase, to break down carbohydrates. Several enzyme supplements contain pancrelipase, which is derived from pigs.

Anagram (formerly known as Synspira Therapeutics) is developing an oral enzyme therapy that would be available either as a tablet or as granules which can be mixed with soft food. It doesn’t contain animal-derived enzymes and is intended to help digest fats, proteins, and carbohydrates.

The ongoing CFF funding has been essential in developing the therapy for testing in clinical trials, the foundation noted. It’s an example of CFF’s venture philanthropy model, a strategy through which a nonprofit provides funding to a company to de-risk an investment. The CFF previously granted $20.4 million to Synspira.

“We are striving to develop better therapies for CF complications, such as poor digestion, to improve the everyday lives of people with CF,” Clancy said.