Diamyd Medical to seek expedited approval for Type 1 Diabetes precision therapy

STOCKHOLM, Sept. 9, 2024 -- Diamyd Medical is set to pursue a fast-tracked approval route in the U.S. for its antigen-specific immunotherapy, Diamyd® (rhGAD65/alum). This therapeutic aims to preserve the body's own insulin production in patients diagnosed with Stage 3 Type 1 Diabetes. An interim analysis from an ongoing Phase 3 trial, DIAGNODE-3, will serve as the basis for a Biologics License Application (BLA) under this accelerated pathway. The results from this interim analysis are anticipated around March 2026.

Ulf Hannelius, CEO of Diamyd Medical, stated, "We are determined to work intensively with our partners and maintain close communication with the FDA to capitalize on this rare opportunity to make Diamyd® available to patients as swiftly as possible." Hannelius emphasized the significance of the earlier readout from the DIAGNODE-3 trial and expressed confidence in Diamyd®'s potential benefits, bolstered by a recently announced positive futility analysis. He highlighted the importance of collaborating with the FDA to meet all regulatory requirements.

Diamyd Medical recently received Fast Track designation in the U.S. for Diamyd®, with the FDA acknowledging the potential for accelerated approval, contingent on demonstrating significant treatment-related benefits on C-peptide levels, a measure of endogenous insulin production, in response to Diamyd® therapy. The FDA's Accelerated Approval program permits drugs for serious conditions that address an unmet medical need to be approved based on a surrogate endpoint. This allows for faster approval of these drugs.

To prepare the BLA under this program, Diamyd Medical plans an interim study readout that will include efficacy data from approximately 170 participants who have completed their 15-month assessment, along with safety data from more participants in the DIAGNODE-3 trial. The primary endpoint for this analysis will be C-peptide, which the FDA now recognizes as likely to predict clinical benefit. Following this interim readout, the DIAGNODE-3 trial will continue to monitor all enrolled patients until the 24-month end-of-study assessment. As of now, 148 patients have been randomized in the DIAGNODE-3 trial. The results of the interim study readout are expected around March 2026, followed by the submission of a BLA under the accelerated approval pathway.

The DIAGNODE-3 trial is being conducted in eight European countries and the United States and aims to fully recruit approximately 330 patients by the end of 2025. All patients are expected to be fully treated by the time the earlier readout is performed. The trial targets recently diagnosed patients with Stage 3 Type 1 Diabetes carrying the HLA DR3-DQ2 genotype, a subgroup that represents about 40% of Stage 3 Type 1 Diabetes patients and has shown a response to Diamyd® in previous trials.

About Diamyd Medical:
Diamyd Medical focuses on developing precision medicine therapies for preventing and treating Type 1 Diabetes and LADA (Latent Autoimmune Diabetes in Adults). Diamyd® is an antigen-specific immunomodulatory therapy designed to preserve endogenous insulin production and 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. DIAGNODE-3, a confirmatory Phase III trial, is actively recruiting patients with recent-onset (Stage 3) Type 1 Diabetes in eight European countries and in the U.S. Significant results have been demonstrated in a large genetically predefined patient group through a large-scale meta-analysis and the Company’s Phase IIb trial in Europe, where Diamyd® was administered directly into a superficial lymph node in recently diagnosed patients. The Company is also developing a biomanufacturing facility in Umeå, Sweden, to produce the recombinant GAD65 protein, the active ingredient in Diamyd®. Additionally, Diamyd Medical is working on GABA-based investigational drug Remygen® for metabolic disease treatments and holds significant shares in the stem cell company NextCell Pharma AB and the AI company MainlyAI AB.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!