Equillium’s phase 3 graft-versus-host disease (GVHD) trial has missed its primary endpoint and two key secondary objectives. But the biotech brushed off the setback, highlighting other secondary endpoints and post hoc analyses to make the case it could file for FDA approval next year.
The study randomized 158 adults and adolescents with acute GVHD to receive the anti-CD6 antibody itolizumab or placebo. Equillium advanced itolizumab into the late-phase trial in the belief the antibody can inhibit T effector cells while preserving T regulatory cells. Ono Pharmaceutical dropped out of an alliance last year, leading cash-strapped Equillium to pause enrollment and bring forward the readout.
Thursday, Equillium reported the complete response (CR) rate at Day 29 was 43% on itolizumab and 48.1% on placebo, causing the trial to miss its primary endpoint. Two key secondary endpoints looked at overall response rate at Day 29 and durable CR at Day 99. Neither result was statistically significant.
The biotech reported significant improvements on the secondary endpoints median duration of CR—336 days for itolizumab versus 72 days for placebo—and failure-free survival. Post hoc analyses—durable CR evaluating Day 29 complete responders, and CR at Day 99 after removing three patients who did not receive any study treatment—also came out positive.
Itolizumab failed to improve overall survival. However, with the death rate favoring itolizumab, 24.4% playing off against 32.5%, Equillium added a “positive trend” in the data to its list of encouraging areas.
Based on the data, Equillium has filed for breakthrough therapy designation and has secured a meeting with the FDA to discuss the potential for accelerated approval. The biotech expects to receive feedback from the agency in May. If the FDA offers encouragement, Equillium plans to file for approval in the first half of next year.
The biotech needs both the FDA and people with money to buy into its plan. Equillium ended last year with $22.6 million, a sum it expects to keep the lights on into the third quarter. Investors are skeptical, wiping 13% off the biotech’s stock in premarket trading to send the share price down to $0.66.
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