Payout From Drug Raises Questions for Cystic Fibrosis Group

About 15 years ago, the Cystic Fibrosis Foundation made what was considered a risky foray for a nonprofit organization into the world of business. It began giving money to a small biotechnology company to entice it to develop drugs for the deadly lung disease. Continue reading Payout From Drug Raises Questions for Cystic Fibrosis Group

Deal with CF Foundation raises cash & concern

The Cystic Fibrosis Foundation will sell royalties it gets from Vertex Pharmaceuticals Inc. (VRTX)for $3.3 billion to Royalty Pharma, taking a profit from charitable investments made to fight the fatal lung disease.

Continue reading Deal with CF Foundation raises cash & concern

New Cystic Fibrosis drug treatment may be ‘potential game-changer’

Scientists from the University of Dundee have, as part of an international collaboration, made a potential breakthrough in the treatment of Cystic Fibrosis (CF).

The study of ten patients found that a combination of two drugs would offer hope to patients suffering from the debilitating and life-shortening inherited disease. Continue reading New Cystic Fibrosis drug treatment may be ‘potential game-changer’

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

Cystic Fibrosis Therapy Lynovex Granted Orphan Drug Status

Drug development companies such as NovaBiotics that are devoted to designing and developing treatments for patients with difficult-to-treat, medically unmet diseases are often faced with the hurdle of recruiting enough patients to conduct the clinical trials necessary to approve new Continue reading Cystic Fibrosis Therapy Lynovex Granted Orphan Drug Status

Is the influenza vaccine safe for cystic fibrosis patients?

Cystic fibrosis (CF) patients are particularly susceptible to complications if they acquire a respiratory infection. The live-attenuated influenza vaccine can significantly reduce the risk of infection; however, the question arose as to whether it was safe for CF patients. Therefore, Canadian researchers conducted a study to evaluate the safety of the vaccine in these individuals. They published their findings online on September 15 in the journal Pediatrics. Continue reading Is the influenza vaccine safe for cystic fibrosis patients?

Sinus Solutions? By Andrea Eisenman

What can be done for sinuses that seem to get infected quite frequently? Oral antibiotics, IV antibiotics, nasal lavage, inhalations and lions, tigers and bears, oh my.

But seriously, aside from the easiest, oral antibiotics, which can wreak havoc on the rest of our Continue reading Sinus Solutions? By Andrea Eisenman

Travel Checklist – Guest Blog by Mark Levine

When I travel, I always seem to forget at least one thing. I’m telling you, it’s always something. Every time I travel. I’m not saying I have a poor memory or that I am starting to experience early senility in my mid-forties. Truth is there’s a lot to remember. Continue reading Travel Checklist – Guest Blog by Mark Levine

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