BETHESDA, Md. — The Cystic Fibrosis Foundation recently agreed to provide up to $15.5 million to Anagram Therapeutics (formerly known as Synspira Therapeutics) to conduct early-stage clinical trials of a novel enzyme replacement therapy.
If the therapy is successful, people with CF would be able to decrease the number of enzyme pills they must take to digest food properly from a handful of pills to only one per meal. In addition, the therapy would be available as either a tablet that can be dissolved in water, or granules that could be mixed with soft food. The drug is non-porcine, which means it contains non-animal derived enzymes, and is designed to break down fats, proteins, and carbohydrates.
“This potential therapy would be more convenient and provide another option to current therapies for people with CF who struggle with digestion,” said JP Clancy, MD, senior vice president of clinical research for the Foundation. “We are striving to develop better therapies for CF complications, such as poor digestion, to improve the everyday lives of people with CF.”
Anagram is planning to start a Phase 1 clinical trial of the enzyme therapy over the summer.
The Foundation previously provided $20.4 million in funding to Synspira Therapeutics, Anagram’s predecessor, bringing the total commitment to $35.9 million. The ongoing support has been essential in preparing this therapy for testing in clinical trials and is an example of the Foundation’s venture philanthropy model through which it provides funding to companies to de-risk CF drug discovery and development.
Complications in CF
Between 85-90% of people with CF suffer from pancreatic insufficiency and require enzymes before eating most foods. Pancreatic insufficiency occurs when thick, sticky pancreatic secretions block the release of enzymes needed to break down food for digestion.
With no enzymes to break down food, much of the protein, fat, and carbohydrates in food are not absorbed. This can result in poor growth, gastrointestinal symptoms such as gas and bloating, and malnutrition.
The CF Foundation is focused on advancing the next generation of transformative therapies to address complications, treat the underlying cause of CF in every individual with CF, and find a cure. Innovators who are interested in pursuing programs in cystic fibrosis can learn about specific funding opportunities.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.