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

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

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)

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

Vertex says cystic fibrosis drugs shown to boost lung function

(Reuters) – Vertex Pharmaceuticals Inc on Thursday said a combination of its cystic fibrosis drug Kalydeco and an experimental compound was shown to improve lung function in a mid-stage trial, Continue reading Vertex says cystic fibrosis drugs shown to boost lung function