Date: 2016-02-16
Type of information: Treatment of the first patient
phase: 2
Announcement: treatment of the first patient
Company: Galapagos (Belgium)
Product: GLPG1837
Action
mechanism: CFTR potentiator. GLPG1837 is a candidate CFTR potentiator drug.
Disease: cystic fibrosis with the S1251N mutation
Therapeutic area: Rare diseases - Genetic diseases
Country: Belgium, The Netherlands
Trial
details: The SAPHIRA Phase 2 program will explore the safety, tolerability and efficacy properties of GLP1837 in CF patients with a G551D (SAPHIRA 1) or S1251N (SAPHIRA 2) Class III mutation in six EU countries and Australia. Primary objectives are to evaluate the safety and tolerability; secondary objectives are to assess changes in sweat chloride from baseline as the biomarker of cystic fibrosis transmembrane conductance regulator (CFTR) ion channel function and to explore the changes in pulmonary function (forced expiratory volume in 1 second [FEV1]) from baseline. Both studies will include subjects treated with Kalydeco® as well as those who are naive to this drug. In each study, different doses of GLPG1837 tablets will be administered twice daily for a total duration of four weeks. The SAPHIRA 1 phase 2 study is an open-label study of three doses of GLPG1837 in at least 12 patients with the G551D mutation, is expected to begin dosing soon. The SAPHIRA 2 Phase IIa, open-label study is evaluating two doses of GLPG1837 in subjects with cystic fibrosis and the S1251N mutation. At least 6 cystic fibrosis patients with the S1251N mutation will be treated for 4 weeks, consisting of two consecutive treatment periods of two weeks evaluating one dose of GLPG1837 each. After the treatment period, there is a 7-10 days follow-up period. During the course of the study, subjects will be examined for any side effects that may occur (safety and tolerability). Changes in sweat chloride will be assessed as biomarker from baseline onwards, and changes in pulmonary function (efficacy) will be explored throughout the study. The amount of GLPG1837 present in the blood (pharmacokinetics) will also be determined. (NCT02690519)
Latest
news: * On February 16, 2016, Galapagos announced the first dosing in its Phase 2 exploratory program of GLPG1837 in patients with cystic fibrosis. The SAPHIRA Phase 2 program will explore the safety, tolerability and efficacy properties of GLP1837 in CF patients with a G551D (SAPHIRA 1) or S1251N (SAPHIRA 2) Class III mutation. Topline results from the SAPHIRA Phase 2 program are expected in Q4 2016. SAPHIRA 2, an open-label study of two doses of GLPG1837 in at least six CF patients with the S1251N mutation, was first dosed in a patient last week. SAPHIRA 1, an open-label study of three doses of GLPG1837 in at least 12 patients with the G551D mutation, is expected to begin dosing soon. In September 2013 Galapagos and AbbVie entered into a global collaboration agreement focused on the discovery and worldwide development and commercialization of potentiator and corrector molecules in a potential triple combination therapy for the treatment of CF. Under the terms of the agreement, AbbVie made an upfront payment of $45 million to Galapagos. Upon successful completion by Galapagos of clinical development through to completion of Phase 2, AbbVie will be responsible for Phase 3, with financial contribution by Galapagos. Galapagos has earned $20 million in milestone payments to date and is eligible to receive up to $340 million in total additional payments for developmental and regulatory milestones, sales milestones upon the achievement of minimum annual net sales thresholds and additional tiered royalty payments on net sales, ranging from mid-teens to 20%.
