Provention Bio announced a temporary pause in the randomization of patients with newly diagnosed type 1 diabetes (T1D) into its global Phase 3 PROTECT study of PRV-031 (teplizumab). This pause is being taken out of an abundance of caution to protect patients, caregivers, clinical site staff, company employees and contractors at this critical juncture in the collective global efforts to combat the COVID-19 pandemic. Patients currently undergoing study therapy will be allowed to complete their course, as recommended by the PROTECT study's Data Safety Monitoring Board, which was recently expanded to include infectious diseases expertise. The Company remains on track to complete its rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for teplizumab for the prevention or delay of T1D in at-risk individuals in the fourth quarter of 2020.
"Our decision to pause randomization of new patients into the PROTECT study follows COVID-19-related State of Emergency declarations in many countries where our study is taking place, including the United States," said Ashleigh Palmer, CEO of Provention Bio. "Our decision was not based on any study-related COVID-19 infections or other safety events, but rather a preponderance of caution relating to the ongoing pandemic, and our concern for the well-being of recently diagnosed T1D pediatric patients and their caregivers. The demands on medical institutions and their clinicians during this unprecedented global crisis were also a main consideration in this decision. For avoidance of any doubt, we believe teplizumab will prove to be a well-tolerated therapy with a favorable safety profile as evidenced by our database of more than 800 patients. We will continue to monitor the situation closely and will provide an updated timeline for the PROTECT study at a future date."
Provention remains on track to complete its rolling BLA submission for teplizumab in T1D patients at-risk of developing clinical stage disease in the fourth quarter of 2020. As Provention has stated previously, the critical path to completion of the rolling BLA submission will be the filing of the chemistry, manufacturing and controls (CMC) module. The Company continues to expect to proceed with three commercial scale process performance qualification (PPQ) batches this summer, supporting the CMC module and enabling the completion of the rolling BLA submission as planned. The Company has not experienced any interruption in its supply chain or manufacturing efforts at this time.
As of this date, none of the Company's other clinical and pre-clinical operations have been significantly impacted by the COVID-19 Public Health Crisis.
The global PROTECT (PROvention T1D trial Evaluating C-peptide with Teplizumab) Phase 3 clinical trial is a randomized, double‑blind, placebo-controlled, multicenter trial that is expected to enroll approximately 300 patients ages 8-17 with recent onset T1D at approximately 90 centers worldwide. The patients will be randomized 2:1 to either two 12-day cycles, six months apart, of intravenous PRV-031 (teplizumab) or placebo. The primary efficacy endpoint is C-peptide change. Secondary endpoints include insulin use, HbA1c, hypoglycemic episodes, and safety.
PRV-031, also known as teplizumab, is an anti-CD3 monoclonal antibody (mAb), which is being developed for the interception and prevention of type 1 diabetes (T1D). The candidate has been the subject of multiple clinical studies involving more than 1,000 subjects with more than 800 patients receiving PRV-031 in those studies. Teplizumab has been shown to delay the onset of end-stage T1D in at-risk individuals with pre-symptomatic disease. In previous studies of newly diagnosed patients, PRV-031 has consistently demonstrated the capability of preserving beta cell function and reducing the need for exogenous insulin usage. Teplizumab has been granted Breakthrough Therapy designation from the U.S. Food and Drug Administration. Provention is currently evaluating PRV-031 in patients newly diagnosed with clinical T1D (the Phase 3 PROTECT Study).
