Eloxx Pharmaceuticals will explore the potential of its lead investigational drug candidate, ELX-02, as a treatment for patients with cystic fibrosis (CF) caused by rare nonsense genetic mutations, in new trials integrated in the European HIT-CF project. “We are extremely excited to be participating in the HIT-CF research…

After 37 hours of travel, I landed in Belgrade, Serbia. I hopped in a cab and rolled past rows of rusted relics — airplanes of various centuries, including the rotting wreckages of NATO planes that once carried disastrous payloads during the ruinous Yugoslav Wars of the 1990s.

The most influential cystic fibrosis advocacy groups in the United States and Europe have endorsed the idea of a Phase 1 clinical trial to test a triple-combo therapy that Proteostasis Therapeutics has developed, the company announced. Proteostatis said the endorsements came from the Cystic Fibrosis Foundation’s and European CF Society’s clinical…

The first patient has been enrolled and dosed in the HOPE-1 Phase 2 clinical trial evaluating Spyryx Biosciences‘ investigational drug SPX-101 as a treatment for cystic fibrosis (CF). The HOPE-1 study (NCT03229252) will take place in Alberta and Ontario in Canada, the United Kingdom, France, and Portugal. Clinical…

An influential group of European cystic fibrosis experts has signed off on the design of a Phase 3 clinical trial of Sollpura (lipromatase) as a treatment for a CF-related digestive disorder known as exocrine pancreatic insufficiency. The European Cystic Fibrosis Society Clinical Trial Network’s approval will speed up the recruitment of…

Vertex Pharmaceuticals recently revealed new clinical data on real-world long-term outcomes in cystic fibrosis (CF) patients treated with Kalydeco (ivacaftor). The company also presented data from a Phase 3 study evaluating Orkambi (lumacaftor/ivacaftor) for children with CF ages 6-11 years. The results were available June 11-14 at the 39th European Cystic Fibrosis Society (ECFS)…

Vertex Pharmaceuticals Incorporated recently announced in a press release new data on its long-term PROGRESS study testing the investigational regimen ORKAMBI (lumacaftor/ivacaftor) for cystic fibrosis (CF). The data was presented at the 38th European Cystic Fibrosis Society (ECFS) Conference, June 10-13, 2015 held in Brussels, Belgium in…

European Cystic Fibrosis Society (ECFS) president and Cystic Fibrosis Trust professor Stuart Elborn was one of the contributors of the latest breakthrough in cystic fibrosis (CF) research, and he believes that the positive results of two phase 3 studies of the drugs ivacaftor (Kalydeco) and lumacaftor may…