Based on the FDA’s response, XTL plans to file its IND, and in the coming quarters initiate a global clinical trial for hCDR1 in the treatment of systemic lupus erythematosus (SLE) in the U.S., Europe and Israel.
The FDA provided encouraging guidance on several key aspects of XTL’s proposed clinical trial including: the primary efficacy endpoint to be based on the BILAG index, a measure of lupus disease activity which was the secondary efficacy endpoint in a prior Phase 2 study of hCDR1; the appropriate patient population; and total number of patients required to prove safety for a new drug application (NDA) for marketing approval. The FDA recommended that the trial be a Phase 2 study.
The FDA has also provided additional guidance on other aspects of the trial design, which XTL intends to review with its Clinical Advisory Board as it finalizes the study protocol including doses and study duration.
XTL CEO Josh Levine said: "We are very pleased with the FDA’s written response to our pre-IND filing meeting package and see it as a vote of confidence in our ability to bring a much needed treatment to people living with lupus.
"This study has an increased likelihood to succeed, in our view, because the FDA’s guidance encourages the study to be substantially similar to the prior Phase 2 trial which demonstrated efficacy in the 0.5 mg dose using the BILAG index, the secondary endpoint of that trial.
"We believe the FDA’s guidance validates the value and relevance of the safety and efficacy data from the prior Phase 2 trial including data on the patient population and statistically significant effect of a 0.5mg dose of hCDR1 on the BILAG endpoint. Using a primary efficacy endpoint based on the BILAG index, we are hopeful that our upcoming study will produce robust data towards supporting a future NDA filing."
hCDR1 is a novel compound with a unique mechanism of action and with clinical data on over 400 patients in 3 clinical studies. The drug has a favorable safety profile, is well tolerated by patients and has demonstrated efficacy in at least one and possibly more clinically meaningful endpoints.