The first stage of Phase 1/2 clinical trials using the experimental drug called BL-7010 has just been successfully completed. This part of the ongoing testing process was designed to evaluate safety by looking for any adverse effects which would limit the proposed dosing of the drug. This stage had a secondary goal of seeing if any of the drug was absorbed into the body of test subjects.
BL-7010 is a substance that attaches to gliadins, the parts of gluten that are problematic for people with celiac disease. When taken orally, it sticks to the gliadins and holds them together. Gliadins in clumps along with the drug cannot be broken down further by enzymes in the intestine, so they are not converted into the even smaller pieces that stimulate the immune system and lead to intestinal damage in people with CD. The clumping is described as effectively masking gliadins from the intestinal cells and immune systems of celiac patients.
BL-7010 is taken orally, and passes out of the body through the intestinal tract along with the gliadins. It is expected that significant amounts of the drug will not get into the circulation of study participants.
This first stage involved giving six single, different doses of BL-7010 or placebo to 24 patients with well-controlled celiac disease in a double blind study where neither the researchers nor patients knew what they were taking. All participants had celiac disease proven by biopsy, had been on a gluten-free diet for at least 6 months, and had negative antibody tests, meaning they had neither IgA to tissue transglutaminase (tTG) nor IgA endomysial antibodies (EMA).
The study was able to reach the highest dose planned, which is more than what will probably be needed for treatment. Participants were checked for adverse events for up to 7 weeks after their dose of BL-7010. Vital signs such as blood pressure and pulse were followed, as were EKGs (measurements of the heart’s electronic activity) as well as laboratory tests. There were no serious side effects and no reason not to proceed from one dose to the next higher dose. This was the so-called “safety and tolerability” phase. All of the participants finished this safely.
While the company released information about the success to date, there was no specific reporting about whether or not any of the BL-7010 was found in the blood of study participants. This will continue to be evaluated, and when all the results are formally presented, information should be available.
The success of the first stage means that the second stage is now underway with results expected later in 2014. This is called the “repeated administration phase” in which participants will receive either BL-7010 or placebo three times a day for 14 days. This is the Phase 2 part of the study.
Success of Phase 2 would mean the drug could go into Phase 3 clinical testing, which is randomized, controlled efficacy studies in patients with celiac disease. The company is hoping to begin this phase by the end of 2014. If Phase 3 is successfully completed, meaning that safety and effectiveness have been proven in participants, the FDA could then approve the medication for treatment.
BioLineRx is the company testing the drug, which is taking place at Tampere Hospital in Finland. Phase 2 is still recruiting participants (see trial description at http://clinicaltrials.gov/ct2/show/NCT01990885?term=BL-7010&rank=1).
In the press release which contained the results of the first stage, Dr. Kinneret Savitsky, Chief Executive Officer of BioLineRx said, “BL-7010 for the treatment of celiac disease is one of our lead development programs, and we are very happy to see that it is safe and well tolerated – even at very high doses that are significantly above the expected clinical efficacious dose.”
He added, “BL-7010 is a unique and very promising product, with excellent pre-clinical efficacy and safety results. We expect to report the full results of the current Phase 1/2 study in mid-2014 and assuming they are successful, we expect to commence a randomized, controlled efficacy study in celiac patients by the end of this year.”
When Phase 3 begins, recruitment will also be advertised on the Clinical Trials website. Whether or not participants will be included outside of Finland is not known.
There are a number of potential treatments for celiac disease going through these phases of study, but none are near FDA approval at the current time.
Clinical Trials website listing of the study:http://clinicaltrials.gov/ct2/show/NCT01990885?term=BL-7010&rank=1).
Press release March 10, 2014