Benzinga | Jul 12, 2021 04:01PM EDT

Equillium Announces Plans to Initiate Phase 3 Pivotal Study of Itolizumab in First-line Treatment of Acute Graft-Versus-Host Disease Following End-of-Phase 1 Meeting with the FDA

Equillium, Inc. (NASDAQ:EQ), a clinical-stage biotechnology company developing itolizumab to treat severe autoimmune and inflammatory disorders, today announced the completion of an End-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) for itolizumab in first-line treatment of patients with acute graft-versus-host disease (aGVHD). The meeting confirmed a path to advance itolizumab into a single Phase 3 pivotal study to support the Biologics License Application (BLA) filing for itolizumab in first-line treatment of aGVHD patients. The company plans to initiate the Phase 3 study in Q4 2021.

"Following the positive outcome of our meeting with the FDA, we will immediately advance to a pivotal clinical study, moving one step closer to developing the first approved therapy to treat aGVHD patients in the first-line setting," said Dolca Thomas, executive vice president of research and development and chief medical officer of Equillium. "This extremely ill patient population is significantly underserved by today's standard of care -- high-dose corticosteroids. Our EQUATE Phase 1b study demonstrated rapid and durable complete clinical responses and a swift reduction in systemic corticosteroid use that are critical for positive longer-term patient outcomes. Itolizumab has received FDA fast track and orphan drug designations for the treatment of aGVHD, and with this feedback from the FDA we are immediately transitioning to late-stage development and look forward to collecting the data needed to support a BLA filing."

The meeting provided guidance from the FDA on the pivotal study design, as well as advice on chemistry, manufacturing and controls (CMC), nonclinical and regulatory-related topics to support Equillium's proposed single pivotal clinical study and BLA submission of itolizumab for the first-line treatment of aGVHD in combination with corticosteroids. The randomized double-blinded pivotal study will evaluate one dosing regimen of itolizumab versus standard of care (high-dose corticosteroids) and will include complete response at Day 29 as the primary endpoint, with an interim evaluation for futility and efficacy at 50% patient enrollment. Equillium is finalizing details of the Phase 3 protocol based on feedback and guidance from the FDA.