FDA Positive on Diamyd Medical's Accelerated Approval for Type 1 Diabetes Drug

STOCKHOLM, July 23, 2024 -- Diamyd Medical has received positive feedback from the U.S. Food and Drug Administration (FDA) regarding an Accelerated Approval pathway for its immunotherapy, Diamyd®, aimed at treating patients with Stage 3 Type 1 Diabetes with the genotype HLA DR3-DQ2.

In a recent face-to-face Type C Meeting, the FDA acknowledged that Diamyd Medical could use C-peptide as a surrogate endpoint to predict the clinical benefit, which involves preserving the endogenous insulin production. This approval pathway could be pursued if significant treatment-related benefits on C-peptide levels are demonstrated through the administration of Diamyd®. Diamyd Medical will continue discussions with the FDA to establish the requirements for a Biologics License Application (BLA) via the Accelerated Approval pathway, including the possibility of an earlier readout of stimulated C-peptide from the ongoing Phase 3 trial, DIAGNODE-3.

Ulf Hannelius, the CEO of Diamyd Medical, stated that aligning with the FDA on the Accelerated Approval pathway for Diamyd is a crucial step toward making this treatment available for Type 1 diabetes patients. The use of C-peptide as a surrogate endpoint for accelerated approval is an important milestone. The company is committed to working closely with the FDA to expedite regulatory approval for this promising therapy.

Professor Mark Atkinson, a Diamyd Medical Board Member and a veteran investigator in Type 1 diabetes research, expressed significant optimism after the FDA meeting, noting that a feasible approval pathway for Diamyd appears achievable. He emphasized the urgent need for such a therapy, highlighting that the biggest beneficiaries of this potential approval would be individuals living with Type 1 diabetes, including those not yet diagnosed.

About Accelerated Approval and Surrogate Endpoints:
The FDA's Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions and address an unmet medical need, based on a surrogate endpoint. A surrogate endpoint is a marker anticipated to predict clinical benefit but is not a direct measure of clinical benefit. Using a surrogate endpoint can significantly reduce the time required to gain FDA approval.

About Diamyd Medical:
Diamyd Medical specializes in developing precision medicine therapies for the prevention and treatment of Type 1 Diabetes and LADA (Latent Autoimmune Diabetes in Adults). Diamyd® is an antigen-specific immunomodulatory therapeutic designed to preserve endogenous insulin production. It has been granted Orphan Drug Designation and Fast Track Designation by the U.S. FDA for treating Stage 1, 2, and 3 Type 1 Diabetes. The DIAGNODE-3 Phase III trial is recruiting patients with recent-onset (Stage 3) Type 1 Diabetes across eight European countries and the U.S. Previous studies have shown significant results in a large genetically predefined patient group, including a large-scale meta-analysis and a European Phase IIb trial. In these studies, Diamyd® was administered directly into a superficial lymph node in youth with newly diagnosed Type 1 Diabetes, optimizing treatment response. The company is also developing a biomanufacturing facility in Umeå, Sweden, for producing recombinant GAD65 protein, the active ingredient in Diamyd®. Moreover, Diamyd Medical is developing the GABA-based investigational drug Remygen® for treating metabolic diseases. The company holds significant shares in the stem cell company NextCell Pharma AB and the artificial intelligence company MainlyAI AB.

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