Galapagos NV (GLPG, Financial), the Belgian clinical-stage biotechnology company that collaborated with AbbVie Inc. (ABBV, Financial) on a treatment for patients affected with cystic fibrosis, announced last week the “topline results from its SAPHIRA 1 Phase 2 study in cystic fibrosis patients with potentiator GLPG1837.”
The GLPG1837 is a molecule potentiator that researchers are experimenting in a triple combination therapy with another potentiator ,GLPG2451, and C1 corrector GLPG2222 in an effort to provide an effective therapy to all patients with cystic fibrosis.
A child born with cystic fibrosis possess two defective genes inherited from his or her parents, one from each parent, of a protein called CFTR (Cystic Fibrosis Transmembrane Conductance Regulator gene). The carrier frequency of cystic fibrosis genes in the western world is about one out of 25 people. People who carry only one abnormal version of the gene responsible for the disease do not have cystic fibrosis and do not show any symptoms. But when two carrier parents have a child, there is 25% chance that the child will be born with cystic fibrosis.
Cystic fibrosis is a genetic disorder that affects mostly the lungs, but can have an effect on other organs. It causes a broad range of gastrointestinal and respiratory problems. Although the degree of involvement differs greatly from person to person, persistent pulmonary infections cause progressive damage to the lung tissue and is a major cause of morbidity in cystic fibrosis patients.
Cystic fibrosis is 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.
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. Cystic fibrosis patients need to take numerous medications and are taken to the hospital frequently for assistance.
The SAPHIRA 1 study was conducted on 26 cystic fibrosis patients with the G551D mutation.
When the patients were screened, 25 of them were regularly following a therapy based on Kalydeco (ivacaftor), another drug used for treating patients with cystic fibrosis produced by Vertex Pharmaceuticals (VRTX, Financial). Before starting the new treatment with GLPG1837, these patients agreed to stop the therapy based on Kalydeco for one week to clean their system. In these patients, the reduced expiratory volume in one second (FEV1), a marker of cardiovascular mortality, fell 5.4% during the washout period but was restored to “the Kalydeco pre-washout levels” after the treatment with GLPG1837, the company said.
One patient had never undergone a Kalydeco treatment.
Increasing doses of the therapy based on GLPG1837 were administered to the patients in three subsequent periods. During the first period of 7 days, the patients took 125 milligrams of the drug two times per day. In the second period of another 7 days, the patients took 250 milligrams of the drug two times per day. During the last period, the patients took 500 milligrams of the drug two times per day.
Since cystic fibrosis causes an abnormal excretion of chlorine, which is usually connected with the protein encoded by the CFTR gene and is due to symptoms involving various organs, the progression is evaluated through clinical tests that measure the concentration of sweat chloride.
The significance of the treatment, from a statistical point of view, was assessed by observing the degree of the decline in the concentration of sweat chloride.
When patients took 500 milligrams of GLPG1837 two times per day, the sweat chloride concentration was observed to fall 32.7% from “a mean value of 98 mmol/L at baseline to 66 mmol/L (p <0.0001).” A 44.7% decline in the sweat chloride concentration was observed in “those patients exceeding the predicted target concentration,” the company said.
“Galapagos and AbbVie will further study the data before deciding which potentiator will be included in the triple combination," Dr. Piet Wigerinck, Galapagos’ chief scientific officer, said.
On Friday, Galapagos closed at $63.03, up $1.82 or 2.97%. The company has a market capitalization of $2.92 billion, has 46.17 million shares outstanding and 30.44 million shares available for trading on the stock market.
AbbVie closed at $62.34, up 68 cents or 1.10% from the previous trading day.
Vertex Pharmaceuticals closed at $74.31, up $2.29 or 3.18% from the previous trading day, but the stock is down $1.99 per share or 2.61% since Dec. 20.
Disclosure: I have no positions in any stock mentioned in this article.
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