Phase 2 Trial of Bertilimumab in Patients with Bullous Pemphigoid Yields Positive Results

This morning, May 15, 2018, Immune Pharmaceuticals Inc. released updated, positive results from its phase 2 study of bertilimumab in patients with moderate-to-extensive bullous pemphigoid (BP). The results are being presented at the 2018 Pre-IID Pemphigus and Pemphigoid Symposium in Orlando, Florida, today.

Bertilimumab is a first-in-class human monoclonal antibody engineered to deflect the protein eotaxin-1, which is responsible for inflammation in a significant number of diseases.

In the study, overall, participants experienced positive outcomes. At day 84 of the study, subjects experienced a decline in the Bullous Pemphigoid Disease Area Index (BPDAI) Activity Score of 81% (p=0.015) from a mean baseline score of 67. Eighty-six percent of subjects also displayed a minimum of a 50% improvement in the BPDAI Activity Score, and 57% displayed a minimum improvement of 90%.

Throughout the study, participants also experienced improvements in pruritus and quality of life. With a mean reduction in BPDAI Activity Score of 70% by day 42, these results were seen quickly. Additionally, in a subgroup of 6 patients in which lesion healing was evaluated, all patients displayed signs of healing of prior lesions by day 28.

Although subjects only received 3 doses of bertilimumab (on days 0, 14, and 28) and modest doses of prednisone that were aggressively tapered, improvements were observed. The mean starting dose of prednisone was 28 mg (0.33 mg/kg); however, it was reduced to 17 mg (0.19 mg/kg) by day 42 (p=0.022) and to 12 mg (0.15 mg/kg) by day 84 (p=0.005). Forty percent of subjects also had a prednisone dose of 10 mg/day or less by day 42, and 58% had achieved 10 mg/day or less by day 84.

Approximately, participants received 2,900 mg less prednisone than called for by the regimen of Joly et al and 1,700 mg less prednisone than called for by British treatment guidelines. Currently, BP patients treated with systemic steroids’ standard of care is a starting dose of 0.5 to 1.0 mg/kg that is diminished slowly over the course of 6-12 months.

“I am gratified to have brought this important study to a close,” closed Tony Fiorino, MD, PhD, Immune’s Chief Medical and Operating Officer in a recent statement. “The subjects in this study showed rapid improvement despite low doses of prednisone, which strongly suggests bertilimumab is active in bullous pemphigoid. Our next study will include a much longer treatment period, which could provide further benefits.”

Dr Fiorino added that Immune intends to discuss with the FDA plans for a randomized, controlled phase 2/3 study of bertilimumab in bullous pemphigoid with the FDA later this year. “We believe this next trial could serve as a registrational study in this orphan disease with no FDA-approved therapies,” he said. “We expect to launch this pivotal study in 2019, using bertilimumab produced by our new manufacturing process.”

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