Diamyd Medical have announced the completion of a pre-specified interim efficacy analysis on C-peptide in the ongoing Phase 3 DIAGNODE-3 trial evaluating retogatein (rhGAD65) in individuals with recent-onset Stage 3 type 1 diabetes carrying the HLA DR3-DQ2 haplotype.

The interim analysis that included 174 out of 321 study participants did not demonstrate statistical significance on the primary endpoint at this timepoint. In addition to this, the pre-specified criteria required to support continuation of the trial were not met. No safety concerns were identified during the interim review.

“This outcome is highly unexpected and not consistent with the totality of prior data,” said Ulf Hannelius, CEO of Diamyd Medical. “The absence of any observable effect on C-peptide, including in pre-specified subgroups, makes it critical to conduct a thorough evaluation. Our priority is to understand these surprising findings that we received late on Friday and determine the most appropriate path forward.”

The company will conduct a comprehensive assessment of the analysis results that were received late on Friday March 27. Its core operations, including manufacturing activities, will continue as planned during this period, they state.

To ensure patient safety and full compliance with ethical and regulatory standards Diamyd Medical will engage with regulatory authorities to determine appropriate next steps.

“The interim outcome is disappointing and not aligned with earlier results,” says Dr. Joshua Vieth, Senior Director of Research at Breakthrough T1D. “A thorough evaluation of the interim results will be essential to understand these findings. We support Diamyd Medical in this work that will inform next steps in the development of retogatein.”