The U.S. Food and Drug Administration has granted Fast Track Designation to Proteostasis Therapeutics’ triple combo treatment program for cystic fibrosis.
The program includes the company’s amplifier, corrector, and potentiator of the CFTR gene — a combination designed to overcome the impact of the mutations that cause CF. The therapeutic agents are known as PTI-428, PTI-801, and PTI-808.
Top CF advocacy groups in the U.S. and Europe have already endorsed a planned Phase 1 clinical trial of the combo. They include the Cystic Fibrosis Foundation’s and European CF Society’s clinical trial arms, the Therapeutics Development Network and the Clinical Trial Network.
Fast Track status is expected to expedite the development and regulatory review of the combo. It gives Proteostasis the chance to have more frequent communications with the regulatory agency. The combo will be eligible for priority regulatory review if certain criteria are met.
“Fast Track designation represents another positive step for the development of our triple combination therapy and underscores the serious unmet need that remains for the vast majority of CF patients,” Meenu Chhabra, the president and CEO of Proteostasis Therapeutics, said in a press release.
“We believe this designation, together with other recent designations from regulators and CF organizations following review of our results with PTI-428, PTI-801 and PTI-808, is recognition of the potential of these programs in this disease,” Chhabra added.
Previous results of several clinical trials assessing the safety and effectiveness of each of the three CFTR modulators, alone or in combination, have demonstrated their potential to restore proper CFTR activity, Proteostatis said. The therapies improved CF patients’ lung function and helped them with long-term symptom management.
Also, data from a clinical trial in healthy volunteers demonstrated that the triple combination was safe and that the participants tolerated it well.
The company plans to start a new Phase 1 trial in the first half of 2018, and to have initial results in the second half of the year.
“We believe combinations of CFTR modulators hold the promise of improving treatment efficacy for different segments of the CF population, including F508del heterozygotes and F508del homozygotes, where patients have limited access to treatment, are underserved by existing treatments, or see a declining benefit in lung function over time,” Chhabra concluded.
The F508del mutation of the CFTR gene is the most common cause of CF.