The clinical development plan for BC-819 consists of two clinical studies that together constitute a straightforward pathway to potential approval.
The two trials will support independent but related indications.
The proposed 204 study is a single arm trial for registration. It will enroll approximately 140 patients with NMIBC who have been treated with two courses of BCG, who experienced subsequent tumor recurrence; this population is considered to have BCG-unresponsive disease. After resection of tumor, patients will be treated with BC-819 administered intravesically for two years or until disease recurrence. The primary endpoint of the study is the one-year tumor recurrence rate.
Our second registrational trial, the proposed 301 study, is a randomized clinical trial. Study 301 has been granted a Special Protocol Assessment (SPA) by the FDA, a regulatory endorsement that the study design will support approval if it meets its endpoints.
The patient population of the 301 trial is at an earlier stage in their treatment course than in the 204 study. Patients eligible for 301 will have NMIBC and have been treated with a single course of BCG and suffered recurrence. The study treatment will comprise BC-819 and BCG administered together. After resection of recurrent tumor, subjects will be randomized to the combination or to a standard second course of BCG alone. The primary endpoint of this randomized trial is disease free survival.
At present, we plan to initiate both the studies in 2017, and begin patient enrollment in the first half of 2018.