Galapagos NV (GLPG, Financial), the Belgian clinical-stage biotechnology company that collaborates with AbbVie Inc. (ABBV, Financial) on a treatment for patients affected with cystic fibrosis, announced Nov. 28 through Globe Newswire that it has started “a randomized, double-blind, placebo-controlled, single-center phase 1 study in at least 64 healthy Dutch volunteers with GLPG2737.”
The Belgian company says that, with the commencement of this study, it will receive a payment of $10 million from AbbVie.
GLPG2737 is an innovative C2 corrector of the gene mutation for the CFTR protein responsible for cystic fibrosis, one of the most common rare genetic diseases that “affects approximately 80,000 patients worldwide and approximately 30,000 patients in the U.S.,” according to Galapagos NV.
There is no definitive cure for cystic fibrosis, but the use of some therapeutic approaches is extending and improving patients’ quality of life and ensuring them high standards of living. CF patients need to take numerous medications and be taken to the hospital frequently for assistance.
Cystic fibrosis is a disease that affects several internal organs of the body, but the digestive tract and the lungs have an augmented susceptibility to the disease.
Although the degree with which the disease affects the various organs of the body greatly differs from person to person, the persistence of infection in the lungs that causes progressive damage to the lung tissue is the major cause of morbidity in CF patients.
The company says that “the aim of the Phase 1 study is to evaluate the safety, tolerability and pharmacokinetics of oral single and multiple ascending doses of GLPG2737.”
The study is in two parts. In the beginning researchers will evaluate the treatment consisting of rising doses; after that they will evaluate the therapy based on rising daily doses of GLPG2737 for a period of two weeks. The company estimates that the results of this study will be released sometime in the second quarter of 2017.
Galapagos NV says that it is closely working with AbbVie to advance, within its portfolio of candidates for a possible CF therapy, a first triple combo in which the GLPG2737, the first of multiple C2 correctors under development, is its final element.
Researchers are already experimenting with the potentiators GLPG1837 and GLPG2451 and C1 corrector GLPG2222 in the clinic.
The company says that in nonclinical studies, “Triple combinations of CF compounds in the portfolio have consistently shown restoration of healthy activity levels in in vitro assays with human bronchial epithelial (HBE) cells of patients with the F508del mutation. These combinations result in an increase in chloride transport compared to Orkambi in HBE cells with the homozygous F508del mutation.”
Vertex Pharmaceuticals' (VRTX, Financial) Orkambi® is the drug used as therapy for CF patients who are older than 11 years of age and whose CFTR gene shows two copies of the F508del mutation.
Galapagos is trading at $59.86, up 95 cents (1.61%) on Nasdaq. The company has market capitalization of $2.72 billion and 46.17 million of shares outstanding and 30.44 million of shares available for trading on the stock market.
AbbVie is trading at $61.79, up $2.33 (or 3.92%) from the previous trading day.
Disclosure: I have no positions in any stock mentioned in this article.
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