Cystic Fibrosis Rescued by Reprogramming the Cell’s Secretory Machinery
BETHESDA, Md.--(BUSINESS WIRE)--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. nn“We are striving to develop better therapies for CF complications, such as poor digestion, to improve the everyday lives of people with CF.” nnTweet this nIf the therapy is successful, most 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. nn“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.” nnAnagram is planning to start a Phase 1 clinical trial of the enzyme therapy over the summer. nnThe 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. nnComplications in CF nnBetween 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. nnWith 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. nnThe 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.
Cystic Fibrosis Foundation Announces Kathryn Brown as Next Chief Communications and Marketing Officer
Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global, late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases, today announced that it will release its financial results for the quarter ended June 30, 2022 after the market close on Tuesday, August 9 and will also host a conference call and webcast at 4:30 pm Eastern Daylight Time on August 9, 2022.
The biotech's decision to pause testing of a cystic fibrosis drug is the latest reminder of the difficulty of delivering RNA medicines into the lungs.