Following a previously reported interim analysis and subsequent evaluation of the interim results, including independent external statistical validation, the company has concluded that the interim results meet the pre-specified futility criteria and do not support continuation.

The results showed no clinically meaningful effect on C-peptide

The interim analysis results, first announced on Friday March 27, were based on 174 evaluable participants followed from baseline to month 15. The results showed no clinically meaningful effect on C-peptide, a marker for endogenous insulin secretion, in the overall population or in pre-specified subgroups. In addition to this, the pre-specified criteria required to support continuation of the trial were not met.

In an evaluation that included review of the data derivation, patient randomization, and statistical assumptions used for the interim analysis, no factors were identified that meaningfully affected the interpretation of the results. No new safety concerns have been identified. Available glycemic data, specifically HbA1c and Time in Range, are consistent with the C-peptide results and indicate a well-controlled patient population with no observable differences between the active and placebo arm at month 15.

An orderly wind-down of the trial

Diamyd Medical will initiate an orderly wind-down of the trial that prioritizes patient safety and meets all ethical and regulatory requirements, it states. The company will proceed with unblinding of the dataset and conduct a comprehensive analysis to better understand the outcome, it states further.

The company will assess the future potential and direction of its GAD-based immunotherapy program based on insights from this analysis.

Strategic review and operational focus

In parallel, the Board of Directors has initiated a formal review of the organization and strategic alternatives aimed at maximizing shareholder value. This includes evaluation of external assets, partnerships, and potential corporate transactions. The company will also adjust its staffing and other costs to address the situation that has arisen.

The company will explore opportunities for the Umeå facility as a stand-alone biomanufacturing company.

As part of this review, Diamyd Medical will continue to maintain and evaluate its manufacturing capabilities at its fully owned biomanufacturing facility in Umeå, Sweden, including the ongoing GMP certification process. The company will explore opportunities for the facility as a stand-alone biomanufacturing company.