Diamyd Medical (DMYD) Lilla Lördag summary

Mission and disease overview

• Focused on curing type 1 diabetes, a chronic autoimmune disease with significant health and lifespan impacts.

• Type 1 diabetes often misdiagnosed as type 2 in adults, leading to underestimation of prevalence.

• Standard care cannot fully replicate natural insulin production, resulting in high complication risks.

• Early diagnosis, especially in young girls, leads to dramatically reduced lifespan due to complications.

• Preserving pancreatic beta cell function is central to improving glycemic control and reducing complications.

Precision medicine approach and research findings

• Employs a precision medicine strategy targeting a genetic subgroup defined by HLA genetics.

• Therapy aims to reprogram the immune system using a recombinant protein, acting as a reverse vaccine.

• Previous phase III trial failed to show efficacy in the general population; current trial focuses on HLA-selected patients.

• HLA type is critical for therapeutic response, explaining previous subgroup effects by gender and geography.

• Post hoc analyses revealed significant effects in males and non-Nordic populations, later attributed to HLA distribution.

Clinical development and regulatory pathway

• Ongoing phase III trial is the first precision medicine study in type 1 diabetes globally.

• Interim readout expected at the end of March, with full results by the end of next year’s second quarter.

• Single registrational study accepted by FDA and EMA due to extensive prior clinical data.

• Fast Track and Orphan Drug Designations in the U.S. provide regulatory and reimbursement advantages.

• Accelerated approval possible if interim results show statistical significance on C-peptide, a marker for insulin production.