Cystic Fibrosis

Here's a roundup of top developments in the biotech space over the last 24 hours. Stocks In Focus Ampio Pharma Shares Fall As FDA Rejects Proposed Change In Knee Osteoarthritis Trial Ampio Pharmaceuticals Inc (NYSE: AMPE) received written responses from the FDA under a Type C meeting...

Eloxx Pharmaceuticals Inc (NASDAQ: ELOX) posted topline results from the monotherapy arms of its Phase 2 trial of ELX-02 in Class 1 cystic fibrosis patients with at least one G542X nonsense allele mutation.  Eloxx delivered one-week ELX-02 data on 12 people with a G542X...

Calithera Biosciences Inc (NASDAQ: CALA) shared interim safety and efficacy results from a Phase 1b trial evaluating CB-280, the company's investigational arginase inhibitor, in adults with cystic fibrosis (CF).  The data were shared at the North American Cystic Fibrosis...

The FDA has granted Fast Track designation Eloxx Pharmaceuticals Inc's (NASDAQ: ELOX) ELX-02, a drug candidate intended to treat cystic fibrosis patients with nonsense mutations. ELX-02 is currently in Phase 2 clinical trials. It expects to present data from the first four...

AzurRx BioPharma Inc (NASDAQ: AZRX) has announced topline results from its Phase 2 Combination Trial evaluating MS1819 with porcine-derived pancreatic enzyme replacement therapy (PERT) for severe exocrine pancreatic insufficiency in cystic fibrosis (CF) patients. Data collected from...

Eloxx Pharmaceuticals Inc (NASDAQ: ELOX) provided an enrollment update for the ongoing Phase 2 trial of ELX-02 to treat cystic fibrosis (CF) in patients with at least one G542X allele.  Based on enrollment to date, Eloxx expects to present data from the first four treatment...

Arrowhead Pharmaceuticals Inc (NASDAQ: ARWR) has voluntarily paused the AROENaC1001 Phase 1/2 study evaluating ARO-ENaC for cystic fibrosis. ARO-ENaC is the company's investigational RNA interference (RNAi) therapeutic. The decision comes after receiving a preliminary update...

The FDA has approved Vertex Pharmaceuticals Incorporated's (NASDAQ: VRTX) expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years. The approval covers children who have at least one F508del...

Concert Pharmaceuticals Inc (NASDAQ: CNCE) has sold potential future milestones relating to VX-561 (deutivacaftor) under the 2017 asset purchase agreement to Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) for $32 million. "This transaction provided an opportunity...

AzurRx BioPharma Inc (NASDAQ: AZRX) announced positive interim data from the first 18 out of 20 patients from its Phase 2 trial. The study was evaluating MS1819 in combination with the current standard of care, porcine-derived pancreatic enzyme replacement therapy (PERT),...

AzurRx BioPharma Inc (NASDAQ: AZRX) reported topline results from its Phase 2b OPTION 2 trial evaluating MS1819 in cystic fibrosis patients with exocrine pancreatic insufficiency. MS1819 demonstrated to be safe and well-tolerated. However, the study did not consistently meet...

Micro-cap biotech Proteostasis Therapeutics Inc (NASDAQ: PTI) is strongly Tuesday morning. What Happened Proteostasis, which focuses on therapies for dysfunctional protein processing, specifically for cystic fibrosis, announced Tuesday its PTI-428 has been granted orphan drug designation, or ODD,...

Shares of Proteostasis Therapeutics Inc (NASDAQ: PTI) were moving higher Monday after the clinical stage biopharmaceutical company announced a worldwide exclusive license agreement with Roche Group's Genentech unit. What Happened Proteostasis Therapeutics develops therapies for the...