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Pulmonary Diseases

Vertex’s Cystic Fibrosis Combo Meets Primary Endpoints in Two Late-Stage Studies

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced results from two Phase 3 studies of the tezacaftor (VX-661) / ivacaftor combination treatment that showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in people with cystic fibrosis (CF) ages 12 and older …

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Genetics Reveal Mysteries of Hard-to-Treat Bacterial Infection in Cystic Fibrosis

New UBC research on bacteria that cause major problems for those with cystic fibrosis reveals clues as to how it proliferates for so long in the lungs and offers new ideas for treatments to explore. “Someone with cystic fibrosis has about one less teaspoon of water in the mucous in …

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Vertex Acquires Cystic Fibrosis Candidate from Concert Pharmaceuticals in Potential $250 Million Deal

BOSTON–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it has signed a definitive asset purchase agreement to acquire CTP-656 from Concert Pharmaceuticals (Nasdaq: CNCE). CTP-656 is an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has the potential to be used as part of future once-daily combination …

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PTC Therapeutics Discontinues Ataluren Program After Failed Phase 3 Trial in Nonsense Mutation Cystic Fibrosis

SOUTH PLAINFIELD, N.J., March 2, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced that the Ataluren Confirmatory Trial (ACT CF) in nonsense mutation cystic fibrosis (nmCF) did not achieve its primary or secondary endpoints. Ataluren was generally well tolerated and ACT CF confirmed a favorable safety profile for ataluren, …

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Concert Pharmaceuticals’ Cystic Fibrosis Candidate Receives Orphan Drug Status from the FDA

LEXINGTON, Mass.–(BUSINESS WIRE)– Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for CTP-656, Concert’s next generation CFTR potentiator being developed for the treatment of cystic fibrosis. In December 2016, Concert initiated a Phase 2 trial in the U.S. …

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RaNA Therapeutics Acquires Shire’s Messenger RNA Platform

CAMBRIDGE, Mass.–(BUSINESS WIRE)–RaNA Therapeutics, a leader in the discovery and development of a new class of medicines that target RNA, today announced the acquisition of the MRT platform, an mRNA therapy platform, developed by subsidiaries of Shire plc. In addition, Shire’s former MRT employees, the group focused on the development …

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Galapagos Reports Positive Results From Its Phase 2 Study of Its Cystic Fibrosis Drug

Mechelen, Belgium; 20 December 2016 – Galapagos NV (Euronext & NASDAQ: GLPG) reports topline results from its SAPHIRA 1 Phase 2 study in cystic fibrosis patients with potentiator GLPG1837. The SAPHIRA 1 trial included 26 patients with the G551D mutation in CFTR each receiving three sequential doses of GLPG1837. Of …

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Vertex’s Orkambi and Kalydeco Show the Potential to Modify the Progression of Cystic Fibrosis

ORLANDO, Fla.–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the presentation of long-term data demonstrating that ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) show the potential to modify the progression of cystic fibrosis (CF). The presentations given at the 30th Annual North American Cystic Fibrosis Conference (NACFC) include final data from the …

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Gilead Discontinues PAH and DKD Tests After Mid-Stage Failure; Advances NASH Treatment into Phase III Trials

FOSTER CITY, Calif.–(BUSINESS WIRE)–Oct. 20, 2016– Gilead Sciences, Inc.(Nasdaq:GILD) today announced the top-line results from three Phase 2 studies of GS-4997 (selonsertib), an investigational inhibitor of apoptosis signal-regulating kinase 1 (ASK1), in nonalcoholic steatohepatitis (NASH), pulmonary arterial hypertension (PAH) and diabetic kidney disease (DKD). GS-4997 demonstrated anti-fibrotic activity in an …

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AstraZeneca Licenses Pulmonary Drug to Insmed in $150 Million+ Deal

BRIDGEWATER, N.J., Oct. 05, 2016 (GLOBE NEWSWIRE) — Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, today announced a licensing agreement with AstraZeneca (NYSE:AZN) for global exclusive rights to AZD7986, a novel oral inhibitor of dipeptidyl peptidase I (DPP1, also known …

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