Agile Sciences lands $1.5M NIH grant for cystic fribosis therapy

Raleigh biotechnology company Agile Sciences has landed a $1.5 million National Institutes of Health grant to help develop its cystic fibrosis therapy derived from sea sponges.

Agile was spun out of North Carolina State University with the help of a $30,000 startup loan from Continue reading Agile Sciences lands $1.5M NIH grant for cystic fribosis therapy

Costly Vertex Drug Is Denied, and Medicaid Patients Sue

Kalydeco, a $300,000-a-Year Cystic Fibrosis Treatment, Sparks Legal Battle in Arkansas and Shows Dilemma States Face

LITTLE ROCK, Ark.— Vertex Pharmaceuticals Inc. VRTX -0.72% ‘s $300,000-a-year cystic-fibrosis drug has sparked a legal battle here, where the state’s Medicaid program is restricting Continue reading Costly Vertex Drug Is Denied, and Medicaid Patients Sue

Cystic Fibrosis Trust and NovaBiotics Enter Partnership for Phase IIa Clinical Trial

What happens when a cystic fibrosis charity and a biotechnology company team up through a partnership? The answer is a Phase IIa clinical trial in the United Kingdom put together by Cystic Fibrosis Trust and NovaBiotics. Also included in the effort are the University of Aberdeen, Health Continue reading Cystic Fibrosis Trust and NovaBiotics Enter Partnership for Phase IIa Clinical Trial

PTC Therapeutics Initiates Confirmatory Phase 3 Clinical Trial of Translarna™ (ataluren) in Patients with Nonsense Mutation Cystic Fibrosis (nmCF)

SOUTH PLAINFIELD, N.J., June 30, 2014 /PRNewswire/ — PTC Therapeutics, Inc. PTCT +0.02% today announced the initiation of a global confirmatory Phase 3 clinical trial of Translarna™ (ataluren), an investigational new drug, in patients with nonsense mutation cystic Continue reading PTC Therapeutics Initiates Confirmatory Phase 3 Clinical Trial of Translarna™ (ataluren) in Patients with Nonsense Mutation Cystic Fibrosis (nmCF)

Art-175 Is a Highly Efficient Antibacterial against Multidrug-Resistant Strains and Persisters of Pseudomonas aeruginosa.

Artilysins constitute a novel class of efficient enzyme-based antibacterials. Specifically, they covalently combine a bacteriophage-encoded endolysin, which degrades the peptidoglycan, with a targeting peptide that transports the endolysin through the outer membrane of Gram-negative Continue reading Art-175 Is a Highly Efficient Antibacterial against Multidrug-Resistant Strains and Persisters of Pseudomonas aeruginosa.

Vertex’s Two-Drug Cystic Fibrosis Treatment Shows Promise in Clinical Trials

Vertex Pharmaceuticals said on Tuesday that a combination of two of its drugs had successfully treated cystic fibrosis in closely watched clinical trials, potentially clearing the way for approval of a new option for nearly half the patients with the genetic disease. Continue reading Vertex’s Two-Drug Cystic Fibrosis Treatment Shows Promise in Clinical Trials

Gilead’s AZLI Therapy For Treatment Of PALS In Cystic Fibrosis Deemed Safe, Effective In Recent Study

Following the recent success of a phase 3b clinical trial for Aztreonam in cystic fibrosis (CF) patients with Burkholderia infections, Gilead Sciences, Inc., a research-based biopharmaceutical company engaged in CF drug discovery and development, completed another phase 3 clinical trial Continue reading Gilead’s AZLI Therapy For Treatment Of PALS In Cystic Fibrosis Deemed Safe, Effective In Recent Study

Clinical Trials for Kalydeco Mutations G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, and G1349D

The new cystic fibrosis medication, ivacaftor (Kalydeco), has been shown to drastically change the course of cystic fibrosis lung disease for some people. Currently it is approved only for use in CF patients with the following Class II mutations: G551D, G178R, S549N, S549R, G551S, G1244E, Continue reading Clinical Trials for Kalydeco Mutations G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, and G1349D

Cystic Fibrosis: Once-A-Day Antibiotic Durable

A once-daily inhaled antibiotic appeared to offer long-term benefits in cystic fibrosis (CF) patients with Pseudomonas aeruginosa infection, researchers said here.

In an extension of a trial that compared investigational liposomal amikacin for inhalation (Arikace) Continue reading Cystic Fibrosis: Once-A-Day Antibiotic Durable

Ataluren Phase 3 Trial Results in Nonsense Mutation Cystic Fibrosis Published in The Lancet Respiratory Medicine

– Data Demonstrate Positive Trends in Lung Function and Pulmonary Exacerbations –

SOUTH PLAINFIELD, N.J., May 16, 2014 /PRNewswire/ — PTC Therapeutics, Inc. PTCT -2.85% today announced that the results of a Phase 3 study of ataluren in patients with nonsense mutation Continue reading Ataluren Phase 3 Trial Results in Nonsense Mutation Cystic Fibrosis Published in The Lancet Respiratory Medicine