BioLineRx Ltd. has announced unblinded results from its Phase 1/2 study for BL-7010, a novel polymer for the treatment of celiac disease.
The Phase 1/2 study, taking place at Tampere Hospital in Finland, a world-leading site for celiac disease research, is a two-part (single and repeated administration), double-blind, placebo-controlled, dose escalation study of BL-7010 in up to 32 well-controlled celiac patients. The primary objective of the study is to assess the safety of single and repeated ascending doses of BL-7010. Secondary objectives include an assessment of the systemic exposure, if any, of BL-7010.
During the single-administration part of the study, six dose levels of BL-7010 were evaluated compared to placebo in a 6+2 standard design, with six patients on BL-7010 and two patients on placebo. This escalation stage reached the highest planned dose with no serious or dose-limiting adverse events.
All planned doses were safe and well-tolerated with all patients completing this part of the study. During the second, repeated-administration part of the study, each patient received either 3 grams of BL-7010 or placebo for 14 days, three times per day, in the same 6+2 standard design.
BL-7010 was well-tolerated over 14 days of treatment, with only one patient not completing the 14-day treatment period. Gastrointestinal-related adverse events (primarily diarrhea) were reported in six out of eight patients, though none were considered serious or dose-limiting and were also observed in one of the two patients on placebo. In light of these findings, and based on pre-clinical studies where the efficacious dose is predicted to be lower than the dose tested in the repeated administration stage of the study, the Company intends to further investigate lower repeated doses of BL-7010 in order to select the optimal dose for the upcoming efficacy study, which is expected to commence in early 2015.
Importantly, pharmacokinetic analyses revealed no systemic exposure of BL-7010 in plasma and urine samples from all patients at all doses and time points tested, both in the single- and repeated-dose regimens. Based on previous communications with a Notified Body in the European Union, the lack of systemic exposure will likely support a medical-device classification in Europe for BL-7010.
Dr. Kinneret Savitsky, CEO of BioLineRx, said, “We are pleased with these unblinded results. In particular, we are very encouraged that the results support previous pre-clinical results showing that BL-7010 is excreted in the feces and is not absorbed systemically. This will most likely support a medical device classification for this promising product in Europe, which can significantly accelerate and reduce the cost of development. Though not serious or dose-limiting, our decision to expand the dosing range in order to potentially mitigate the gastrointestinal events observed in the study is based on preclinical evidence suggesting efficacy at lower repeat doses. In the next few months we will concentrate on selecting the optimal dose of BL-7010, and, following this selection, we plan to commence in Europe a randomized, placebo-controlled efficacy study in celiac patients early next year.”