Provention Bio Announces Breakthrough Therapy Designation for Teplizumab

Provention Bio announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to teplizumab (PRV-031) for the prevention or delay of clinical type 1 diabetes (T1D) in individuals at-risk of developing the disease.

BTD is an FDA program designed to expedite the development and review of therapeutic candidates intended to treat serious or life-threatening diseases. To qualify for this designation, preliminary clinical evidence has to indicate that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint. The benefits to Provention of this BTD include more intensive and interactive guidance from FDA on an efficient drug development program, access to a scientific liaison to help expedite review time, and eligibility for Priority Review if relevant criteria are met.

The FDA decision on BTD was based on clinical data from the "At-Risk" Study conducted by TrialNet, which showed that a single 14-day course of PRV-031 (teplizumab) significantly delayed the onset and diagnosis of clinical T1D, as compared to placebo, by a median of at least 2 years in children and adults considered to be at high risk of developing clinical T1D.

"We are pleased that the FDA recognizes the transformative potential of PRV-031 in T1D, and we are committed to working with the Agency to bring this candidate to patients as quickly as possible," said Ashleigh Palmer, CEO of Provention Bio. "The groundbreaking data from the 'At Risk' trial showed, for the first time, the potential to delay the onset of T1D in individuals who are almost certain to develop clinical disease. With this designation in place, we plan to leverage that landmark data, as well as the robust safety database from prior teplizumab studies, to support a registration filing. We look forward to discussing next steps with the FDA and providing an update on the process in the coming months."

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. 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.

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