Diamyd Medical (DMYD) Status Update summary

Disease overview and unmet need

• Type 1 diabetes affects around 9 million people globally, with 300,000-500,000 new diagnoses each year.

• The disease is caused by an autoimmune attack on insulin-producing beta cells, leading to insulin deficiency and serious complications.

• HLA genotyping helps identify individuals at higher risk due to genetic predisposition.

Advances in disease understanding and measurement

• C-peptide is a key marker for measuring endogenous insulin production and is central to clinical trial endpoints.

• Preserving C-peptide is crucial for maintaining metabolic control and reducing complications.

• Precision medicine is guiding the development of targeted therapies based on genetic and immunological profiles.

Diamyd Medical’s positioning and clinical development

• Diamyd Medical is conducting the first precision medicine-based phase III trial in Type 1 diabetes, targeting patients identified by HLA genetics.

• Their GAD65-based therapy is the only antigen-specific immunotherapeutic in phase III, aiming to reprogram the immune system without broad immunosuppression.

• The phase III trial includes patients aged 12-28, with potential to expand to younger children in future studies.