Diamyd Medical (DMYD) Study result summary

Interim phase III trial results

• Interim analysis of the phase III DIAGNODE-3 trial for retogatein in type 1 diabetes showed futility, with no statistically significant effect observed on C-peptide in the full population or any pre-specified subgroup, including potential super responders.

• The results are in stark contrast to previous trials and analyses, which had shown clear trends or significant effects in the targeted genetic subgroup.

• 174 patients with the right genetic profile were followed for 15 months, and the data suggest a very low likelihood of a positive outcome at full readout in 2027.

• No safety concerns have been identified; the investigational therapy remains considered safe.

• The trial continues as planned until further analysis clarifies the findings, with patient safety and regulatory compliance as top priorities.

Investigation and next steps

• The immediate focus is on verifying the integrity of the data and analysis, with a systematic root cause investigation and comprehensive assessment underway.

• If errors in data handling or analysis are found and corrected, the trial may continue; otherwise, discontinuation and unblinding will be considered.

• An external scientific advisory board, the CRO, and regulatory authorities are involved in reviewing all aspects, including production and regional differences.

• Transparency and timely communication are emphasized, with live conference calls and updates planned as analyses progress.

• The company is considering alternative development pathways, including preventive approaches in earlier disease stages and rare neurological disorders, but will await further analysis before making decisions.

Financial position and shareholder communication

• The current cash position is approximately $40 million, with a recent $25 million equity investment completed and no conditions attached.

• The cash runway is estimated at 18-24 months, but this may change depending on the outcome of the ongoing analysis and next steps.

• Management acknowledges the significant share price drop and is committed to regaining shareholder trust through diligent analysis and strategic planning.

• The board and management emphasize their personal commitment to the type 1 diabetes community and the importance of continuing efforts in precision medicine.

• The broader field of disease-modifying therapies for type 1 diabetes is seen as resilient, with ongoing need and opportunity for innovation despite this setback.