BioLineRx has announced positive Phase IIa results for BL-7040, an orally available drug for treating inflammatory bowel disease (IBD). The study showed that BL-7040 is safe and effective in treating ulcerative colitis, a form of IBD.
The Phase IIa trial was an open-label, proof-of-concept study to evaluate the efficacy, safety and tolerability of BL-7040 in patients with moderately active ulcerative colitis.
Patients were treated for up to five weeks with BL-7040: 12 mg/day for up to three weeks, followed by 40 mg/day for two additional weeks. The clinical trial was carried out at five leading medical centers in Israel.
Sixteen of the 22 patients who were enrolled in the clinical trial completed the full five-week course of treatment and two-week follow-up.
The primary clinical endpoint in the study - a reduction in the Mayo score between baseline and completion of treatment - was achieved.
Fifty percent of patients (8 patients) met the primary endpoint, while the remaining 8 patients demonstrated a stable clinical condition or minor improvement.
Fifty-six percent of patients (9 patients) demonstrated decreases of at least 1 point in the rectal-bleeding sub-score and 69% (11 patients) had rectal-bleeding sub-scores of ≤1 (in 6 of the 11 patients, no rectal bleeding was seen at all).
Fifty percent of the patients completing study treatment also met certain secondary endpoints, such as a partial Mayo score reduction and mucosal healing evaluated by endoscopy sub-score measurements.
Additional secondary endpoints in the study were the IBD Quality-of-Life Questionnaire, and the serum CRP and fecal calprotectin measurements.
The results of these additional secondary endpoints were not conclusive, although certain positive trends were noted.
BL-7040 was highly safe and well tolerated by the study participants, with a very low incidence of drug related, mild-to-moderate adverse events (AEs), as well as one serious adverse event (SAE) not related to the treatment.
Both patients and investigators were very satisfied with the safety and tolerability profile of the treatment and, in particular, emphasized the ease of oral administration.
"Ulcerative colitis and Crohn's disease are prevalent conditions that affect the quality of life of millions across the globe. It is estimated that as many as 1.4 million individuals in the U.S. suffer from these diseases. In addition to discomfort, which can be quite extreme, IBD can cause significant complications, including anemia, intestinal abscesses, intestinal perforation and more. Current treatments are far from satisfactory, and many people stand to benefit from a new and effective treatment," said Dr. Kinneret Savitsky, Chief Executive Officer of BioLineRx.
“Based on historical data, current steroidal or biological treatment regimens have yielded clinical response rates of between 30% and 70% in studies with significantly longer treatment periods; thus, the results of our five-week, proof-of-concept study suggest positive efficacy for BL-7040. A number of experts in the IBD field who have reviewed the results of our study all agree that these are very encouraging and positive results, and that the reduction in rectal bleeding is particularly impressive. Our immediate next steps include evaluating the most advantageous ways to progress with this therapeutic candidate from a clinical and business perspective, including examining potential additional indications. In parallel, we also plan to accelerate discussions with potential co-development and licensing partners for this asset," concluded Dr. Savitsky.