Kezar’s Autoimmune Drug Hit With Second FDA Clinical Hold in as Many Months

 

Following patient deaths in a lupus trial that led to the termination of that program, Kezar’s autoimmune candidate zetomipzomib faces a partial clinical hold barring four trial participants from continuing treatment in the open-label portion of the trial, though the trial itself will continue as planned.

Kezar Life Sciences can’t catch a break this fall with its autoimmune candidate zetomipzomib. Just over a month after the FDA issued a clinical hold on zetomipzomib in lupus, the agency has placed a partial hold on a mid-stage trial of the drug in autoimmune hepatitis, Kezar said Tuesday in its Q3 business update.

Last month, Kezar made the decision to discontinue the lupus program after four patient deaths occurred during the Phase IIb PALIZADE trial. Now, a partial clinical hold will block the remaining four patients in the double-blind treatment period (DBTP) of the Phase IIa PORTOLA trial in autoimmune hepatitis from continuing treatment with zetomipzomib by moving on to the open-label extension portion of the trial. The FDA is allowing enrolled participants to complete the 24-week DBTP and for those currently enrolled in the open-label extension to continue treatment.

“This additional precaution was added to ensure any potential placebo patients still in the DBTP not be initiated on zetomipzomib, despite the IDMC recommending the PORTOLA trial proceed as planned and no reported adverse events of interest or deaths as seen in the PALIZADE trial,” William Blair analysts wrote in an investor note Wednesday. No Grade 4 or 5 serious adverse events have been observed in PORTOLA, according to Kezar.

Kezar plans to report topline data from the PORTOLA trial in the first half of 2025. The William Blair analysts emphasized the importance of this readout. “Following discontinuation of the PALIZADE study during the quarter, there is clearly significant dependence on positive results from the PORTOLA study in the first half of 2025 to spur a reversal in share price,” they wrote.

Kezar’s shares have been on a rollercoaster ride this year, falling from a high of $10.50 per share in February to a low of $5.20 per share in September. The company was trading at $7.28 per share pre-market Wednesday.

Kezar CEO Chris Kirk said in October that the decision to discontinue the lupus program was difficult as the company had seen a favorable safety profile and clinical activity in an earlier trial called MISSION. “However, a focused development effort in AIH [autoimmune hepatitis] extends our cash runway and provides flexibility as we work to bring zetomipzomib forward as a treatment for patients living with this life-threatening disease,” Kirk said in a statement at the time.

Kezar had $148 million in cash, cash equivalents and marketable securities as of September 30.

https://www.biospace.com/fda/kezars-autoimmune-drug-hit-with-second-fda-clinical-hold-in-as-many-months