cystic fibrosis

Proteostasis Therapeutics is eliminating 13 research positions in order to save $3 million as it “readjusts priorities to focus resources on researching and developing” its cystic fibrosis (CF) programs, including clinical trials for PTI-428, PTI-801 and PTI-808 small molecules. The R&D shift also includes its discovery-stage program in…

Vertex Pharmaceuticals recently presented data from several clinical trials of cystic fibrosis treatments showing positive outcomes for CF patients. Data focused on the approved therapies Orkambi and Kalydeco, and investigative treatments like tezacaftor/ivacaftor and several triple regimens. These findings were presented at the North American Cystic Fibrosis Conference (NACFC) that ran Nov.

A long-term clinical study showed that RELiZORB improves fatty acid absorption in patients suffering from fat malabsorption or pancreatic deficiencies, which is good news for cystic fibrosis (CF) patients suffering from insufficient lipase production. Alcresta Therapeutics, RELiZORB’s developer, presented data from its ASSURE study at the North American Cystic Fibrosis…

Vertex Pharmaceuticals, a clinical-stage biopharmaceutical company, recently announced that two Phase 3 studies evaluating its tezacaftor/ivacaftor combination therapy have shown statistically significant and clinically meaningful improvements in lung function in patients with certain mutations associated with cystic fibrosis (CF). The first study, called EVOLVE (NCT02347657), was conducted to…

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…

Corbus Pharmaceuticals recently announced that the U.S. Patent and Trademark Office has issued a patent for the use of anabasum, the company’s lead therapy candidate for the treatment of inflammatory and fibrotic diseases. Anabasum is a synthetic oral endocannabinoid-mimetic drug that binds to a receptor called cannabinoid receptor…

Pulmatrix recently announced that it is presenting positive preclinical data on its antifungal therapy Pulmazole at the North American Cystic Fibrosis Conference (NACFC) (Nov. 2-4, 2017). Pulmazole (PUR1900) is based on itraconazole, an antifungal drug, combined with an innovative technology developed by Pulmatrix called iSPERSE, which is a dry powder…

Vertex Pharmaceuticals announced a $750,000 addition to its cystic fibrosis (CF) research innovation program that supports the work of young scientists aiming to establish themselves as independent CF researchers. The initiative expands the company’s CF Research Innovation Awards (RIA) program furthering post-doctoral research that aims to advance the understanding…

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…

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…