Anagram wins $15.5m boost to develop enzyme therapy for cystic fibrosis

2023-04-05

基因疗法

Anagram wins $15.5m boost to develop enzyme therapy for cystic fibrosis

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来源: Pharmaceutical Technology

Anagram Therapeutics will be awarded up to $15.5m from the Cystic Fibrosis Foundation to conduct early-stage clinical trials of a novel enzyme replacement therapy (ERT) for patients with cystic fibrosis (CF).

On April 4, the Framingham, Massachusetts-based company, previously known as Synspira Therapeutics, launched a rebrand with a focus on its enzyme research platform. Using previously gained research expertise, Anagram plans to develop orally administered enzymes that can treat malabsorption syndromes and nutrient metabolism disorders. On the same day, the Cystic Fibrosis Foundation announced the funding for Anagram under its venture philanthropy model designed to provide financial support to “de-risk CF drug discovery and development”. Earlier this year, the Foundation had announced an investment of upto $15m in ReCode Therapeutics for the development of an mRNA therapy for CF.

Anagram’s lead candidate, ANG003 is meant to treat exocrine pancreatic insufficiency, a condition with reduced or improper secretion of certain pancreatic enzymes, which hampers digestion. The condition is often observed in 80%-90% of patients with cystic fibrosis. If trials are successful, Anagram’s therapy will reduce the number of enzyme pills patients need to take with meals to help digest food.

“This potential therapy would be more convenient and provide another option to current therapies for people with CF who struggle with digestion,” said Dr. JP Clancy, senior vice president of clinical research for the Cystic Fibrosis Foundation.

The financial aid brings the Foundation’s total commitment to $35.9m, with $20.4 previously given to Anagram’s predecessor – Synspira Therapeutics. Anagram is planning to start a Phase I clinical trial of the enzyme therapy in summer of 2023.

The current CF therapeutic landscape is dominated by targeted therapies developed by Vertex Pharmaceuticals. With limited number of pipeline agents in late-stage development, the focus is now shifting to therapies that will be accessible to all CF patients regardless of the type of mutation driving their disease.

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机构

Anagram