Alice Melão, MSc,  —

Clinical trials on all three Proteostasis Therapeutics’ investigational cystic fibrosis modulators — PTI-428, PTI-801, and PTI-808 — have demonstrated they are safe and have the potential to improve the effectiveness of Orkambi in patients with cystic fibrosis (CF). The positive clinical data supports the initiation of new clinical trials evaluating the…

AIT Therapeutics’ inhaled nitric oxide formulation improved the breathing and physical functioning of cystic fibrosis patients with a lung infection, a Phase 2 clinical trial shows. The preliminary results applied to the formulation’s use against nontuberculous mycobacteria, which are collectively known as the mycobacterium abscessus complex. AIT said the NO-NTM…

Researchers at the University of Alabama at Birmingham (UAB) and the University of North Carolina at Chapel Hill (UNC) have developed a new method that can quickly and easily predict which treatment is more suited for each cystic fibrosis (CF) patient. Their study, “Nasospheroids permit…

Proteostasis Therapeutics is expecting clinical trial results on three of its cystic fibrosis therapies in the next few months, and plans to start additional trials in 2018. CF is a genetic disease caused by mutations in the sequence of the CFTR gene, which leads to impaired activity of the…

Two preclinical studies support the ability of Synspira’s lead candidate SNSP113 to prevent bacteria from forming treatment-resistant layers (biofilms), and in reducing mucus viscosity and easing clearance, new data show. These findings, presented at the 31st Annual North American Cystic Fibrosis Conference (NACFC) taking place in Indianapolis through…

Orkambi (lumacaftor/ivacaftor) reduced levels of the main biomarker of the lung disease cystic fibrosis and improved the nutritional status of children with the condition, according to a Phase 3 clinical trial. The results were part of a recent update that Vertex Pharmaceuticals provided on Orkambi and three of its…

AzurRx BioPharma is planning to expand the clinical development of its investigative therapy MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) caused by chronic inflammation of the pancreas and cystic fibrosis (CF) into Europe. The company has submitted an Investigational Medicinal Product Dossier (IMPD) — similar to an…

The Institute for Clinical and Economic Review (ICER) is planning to evaluate and compare the clinical effectiveness and value of cystic fibrosis treatments now available or awaiting approval, with a tentative focus on several by Vertex Pharmaceuticals. According to information provided in an ICER press release, the institute intends…

A research team at Imperial College London (ICL) has developed a quick, cheap method to detect the presence of Pseudomonas aeruginosa in sputum samples collected from patients with cystic fibrosis (CF). Using small engineered DNA circuits called cell-free biosensors, the method reveals molecules specifically produced by P. aeruginosa. When…

Interim data from an ongoing Phase 2a clinical trial indicate that the investigational therapy MS1819-SD may provide therapeutic benefits for patients with cystic fibrosis (CF) who have exocrine pancreatic insufficiency (EPI), a digestive disorder. The trial (ACTRN12616000962437) is being conducted in patients with chronic pancreatitis (CP) who have EPI, but the drug is being…