The drug development arms of two leading cystic fibrosis (CF) organizations, one in the U.S. and one in Europe, have endorsed Proteostasis Therapeutics’ coming trial of a triple combination therapy for CF.
The study also received a high strategic fit score from these two organizations — the Therapeutics Development Network (TDN) of the Cystic Fibrosis Foundation and the Clinical Trial Network (CTN) of the European CF Society (ECFS).
“We are humbled that the two preeminent global CF patient advocacy organizations share our view on the positive potential clinical impact of our CFTR modulators,” Meenu Chhabra, Proteostasis Therapeutics’ president and CEO, said in a press release.
The trial will test a combination of the three CFTR modulators, PTI-428, PTI-801 and PTI-808, developed by Proteostasis. CFTR is the faulty ion channel that causes CF.
Since this will be the first study of the combo, the trial will mainly focus on the safety and tolerability of the treatments. Researchers will also study the combination’s pharmacokinetics — assessing how the drugs are taken up and processed within the body.
The team will also examine participants’ lung function by measuring forced expiratory volume in one second (FEV1).
The two organizations evaluate clinical studies based on their scientific merit, study design, feasibility, and overall clinical research priorities of the CF community. A group made up of CF physicians, researchers, research coordinators, biostatisticians, patients, and other specialists are responsible for reviewing study protocols.
A trial protocol endorsement is not only a symbolic statement. TDN can provide access to 89 accredited CF care centers that hold expertise in clinical research. The organization also supports the recruitment of study participants and aids in study execution.
In Europe, CTN has 43 CF centers tied to its organization. The centers are located in 15 different countries across the continent.
Both organizations work together on studies undertaken in the U.S. and Europe.
“We are experiencing an exciting time in CF drug development and research, with an unprecedented number of ongoing clinical trials,” said Patrick A. Flume, MD, a professor of Medicine and Pediatrics and director of the Cystic Fibrosis Center at the Medical University of South Carolina.
“At the same time, our CF patient population is a valuable and limited resource. The TDN’s protocol review and scoring process creates a framework that helps the investigator and patient community maintain alignment on clinical research priorities,” added Flume, who also is the lead principal investigator for Proteostasis’ triple combination trial.
Chhabra said the trial will start dosing patients in the first half of 2018, with initial results expected in the second half of the year.
“We believe that PTI-428, PTI-801 and PTI-808 have the potential to be a novel, best in class treatment for CF patients,” Chhabra concluded.