CALL TO ACTION – The Ensuring Access to Clinical Trials Act (EACT)

Hello All,
The Ensuring Access to Clinical Trials Act (EACT) makes permanent a law that will expire on OCTOBER 5th if we do not take action now. EACT will permanently allow people with rare diseases, like CF, to participate in clinical trials without the risk of losing Supplemental Security Income and Medicaid benefits. Continue reading CALL TO ACTION – The Ensuring Access to Clinical Trials Act (EACT)

Scientists use gene editing to correct mutation in cystic fibrosis

Yale researchers successfully corrected the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder.

The study was published April 27 in Nature Communications. Continue reading Scientists use gene editing to correct mutation in cystic fibrosis

Clinical Trial Alert Vertex Pharmaceuticals

Phase 3 Trial with VX-661 and VX-770 in CF Patients Homozygous DeltaF508 Now Enrolling!

CFF info about this study: http://www.cff.org/Display/dsp_ClinicalResearchHTML.cfm?id=402&CTSubId=79 Continue reading Clinical Trial Alert Vertex Pharmaceuticals

Cystic Fibrosis Clinical Trial Alert:

PTC Therapeutics Phase 3 Clinical Trial of Ataluren (PTC124) in CF Patients with “Nonsense” CF Mutations:

Locations Currently Enrolling: United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain and United Continue reading Cystic Fibrosis Clinical Trial Alert:

Pharmaxis Osmotherapy Clinical Trial for CF Now Recruiting

Australian specialist pharmaceutical company Pharmaxis has just announced it has successfully enrolled the first participant in an international Phase 3 clinical trial designed to evaluate their lead pipeline product for cystic fibrosis, Bronchitol® (mannitol). Pharmaxis Continue reading Pharmaxis Osmotherapy Clinical Trial for CF Now Recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of QBW251 in Healthy Subjects and Cystic Fibrosis Patients

Sponsor: Novartis Pharmaceuticals

Purpose: This study is designed to assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics (proof of concept) of QBW251 in healthy subjects and Continue reading Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of QBW251 in Healthy Subjects and Cystic Fibrosis Patients

Clinical Trial Alert: Prevention of CF Related Diabetes Using Sitagliptin

Currently Enrolling (According to clinicaltrials.gov)
Locations: Georgia, Tennessee, Ohio and Quebec Canada
http://clinicaltrials.gov/ct2/show/NCT00967798?term=cystic+fibrosis&recr=Open&rank=12

Acute systemic hyperglycemia causes oxidative stress and a pro-inflammatory response. The pro-inflammatory cytokines induced by hyperglycemia are toxic to islet insulin producing cells, and Continue reading Clinical Trial Alert: Prevention of CF Related Diabetes Using Sitagliptin

Savara Pharmaceuticals Raises $10 Million to Advance AeroVanc for MRSA Lung Infection in People with Cystic Fibrosis

Company Completes Enrollment in Phase 2 Clinical Trial

Austin, TX – October 6, 2014 – Savara Pharmaceuticals announced today that it closed a $10 million bridge financing round to support the  development of AeroVanc, the first inhaled antibiotic Continue reading Savara Pharmaceuticals Raises $10 Million to Advance AeroVanc for MRSA Lung Infection in People with Cystic Fibrosis

Clinical Trial Recruiting

A Prospective, Multicenter, Collaborative Study to Determine the Prevalence of Nontuberculous Mycobacteria (NTM) in Pediatric Patients With Cystic Fibrosis in Florida

Link for more info: http://www.clinicaltrials.gov/ct2/show/NCT02198079?term=cystic+fibrosis&rank=39 Continue reading Clinical Trial Recruiting

Galapagos cystic fibrosis program progresses towards therapy for largest patient group

Regulated information 17 June 2014

• Galapagos cystic fibrosis program progresses towards therapy for largest patient group

• Review of CF and other highlighted programs in today’s R&D update

• GLPG0634 DARWIN 1 study To deliver 12week top line data in Q12015

• GLPG0974 shows biomarker effect & good safety in ulcerative colitis patients, but lacks clinical improvement in 4 week Proof-of-Concept study

• GLPG1492 mode of action scalable to ESKAPE and other pathogens
Multiple Phase 2 readouts with novel modes-of-action in coming two years

Live webcast presentation today at 8:00 am ET/
2:00 pm CET on

www.glpg.com, call number +322 404 0660, confirmation code 2952966 Mechelen, Belgium; 17, June 2014–Galapagos NV (Euronext: GLPG) will give an R&D Update today in New York City, highlighting the company strategy, progress and plans for its portfolio of more than 35 R&D programs. R&D strategy Galapagos selects diseases with large, unmet medical need and discovers novel mode-of-action medicines to address these diseases. The Company’s R&D focus on inflammation, orphan, anti-infectives, and fibrosis has yielded a substantial pipeline with multiple Phase 2 readouts the coming two years. Galapagos seeks to partner programs at an optimal stage, with the ambition to ring fence certain proprietary programs

GLPG0634 Selective JAK1 inhibitor GLPG0634 has shown a best-in-class profile in two rheumatoid arthritis Phase 2 studies. GLPG0634 is currently in a global Phase 2b program (DARWIN) in 875 rheumatoid arthritis patients and a Phase 2 study in 180 patients with Crohn’s disease. Due to longer than anticipated approval rounds with national regulators, topline 12 week results for DARWIN 1 (595 patients, methotrexate add-on) are expected in Q1 2015, DARWIN 2 (280 patients, monotherapy) topline 12 week results are expectedin Q22015, with complete 24 week data package expected in Q3 2015. Topline data from the Phase 2 study in Crohn’s disease remains on track for disclosure in Q2 2015. AbbVie will base its licensing decision on the complete 24 week DARWIN data package from GLPG0634.GLPG0974 topline results GLPG0974 is the first selective antagonist of FFA2 to be tested in the clinic. GLPG0974 showed good results in Phase 1 studies and recently completed a 4-week Phase 2 proof-of-concept study in 45 ulcerative colitis (UC) patients in 16 centers in 4 European countries. Patients received 200 mg of GLPG0974 twice-daily for 4 weeks. Patients on treatment tolerated it well and showed a decrease in fecal calprotectin, a byproduct of neutrophil breakdownin the gut, as well as a decrease in the number of infiltrating neutrophils. These biomarker reductions are evidence for the novel mode-of-action directed toward neutrophil migration. Reduction in neutrophil influx did not translate to improvement in signs and symptoms during this four week study. Galapagos is performing subgroup analyses, exploring additional indications, and discussing further development of GLPG0974 with potential partners