STOCKHOLM, Jan. 10, 2025 /PRNewswire/ -- Diamyd Medical provides an update following the receipt of final meeting minutes from a positive Type C meeting with the U.S. Food and Drug Administration (FDA) held in December 2024. The meeting focused on refining study protocol and analysis strategies for Diamyd® (rhGAD65/alum) in preparation for Accelerated Approval of the antigen-specific immunotherapy targeting Stage 3 Type 1 Diabetes. The final minutes from the Type C meeting confirm alignment on critical development milestones and the statistical plan, setting the stage for an expedited review pathway.
As previously announced on December 13, Diamyd Medical had held a positive in-person meeting with the FDA. Following the meeting, Diamyd Medical has now received the final minutes. The FDA provided affirmative feedback on several pivotal aspects of Diamyd Medical's development program, including:
Accelerated Approval Pathway: Confirmation that the ongoing DIAGNODE-3 Phase 3 trial aligns with requirements for an Accelerated Approval, leveraging interim efficacy data based on stimulated C-peptide as the primary endpoint from approximately 170 evaluable participants who will complete their 15-month assessments by early 2026.
Safety Dataset and Confirmatory Evidence: Concurrence on the suitability of both the safety dataset and the confirmatory evidence for a potential accelerated approval. The safety dataset includes data from DIAGNODE-3 at filing, along with data from other intralymphatic trials with Diamyd® and the three placebo-controlled trials evaluating subcutaneous injections of Diamyd® from the original meta-analysis published by Hannelius et al. in Diabetologia (2020). Furthermore, concurrence on that DIAGNODE-3 serves as the pivotal trial to demonstrate substantial evidence of effectiveness, supported by DIAGNODE-2 and the aforementioned placebo-controlled trials as confirmatory evidence.
Co-Primary Endpoints: Agreement on simultaneous evaluation of stimulated C-peptide levels and HbA1c as co-primary endpoints at the 24-month final analysis.
Statistical Analysis Plan: Acceptance of the testing strategy for endpoint evaluation, ensuring robust data integrity for both accelerated and full approvals.
The FDA emphasized that the analysis presented in the Trial Outcome Markers Initiative (TOMI) meta-analysis, published by Taylor et al. in Lancet (2023), provides critical insights into the association between C-peptide preservation and clinical outcomes in type 1 diabetes. The FDA agreed that the justification for the required level of C-peptide preservation to support an accelerated approval, including its relationship to a clinically meaningful reduction in HbA1c, could be further discussed at a pre-BLA meeting and included in the Biologics License Application (BLA).
Additionally, the FDA reaffirmed the Orphan Drug Designation for Diamyd® for treating a subset of Type 1 Diabetes patients with residual beta cell function. This designation highlights the therapy's potential to address significant unmet needs in this well-defined patient population.
About Diamyd Medical
Diamyd Medical develops precision medicine therapies to prevent and treat Type 1 Diabetes and LADA (Latent Autoimmune Diabetes in Adults). Diamyd® is an antigen-specific immunomodulatory therapeutic for the preservation of endogenous insulin production. Diamyd® has been granted Orphan Drug Designation in the U.S. as well as Breakthrough Designation and Fast Track Designation by the U.S. FDA for the treatment of Stage 3 Type 1 Diabetes. Diamyd® has also been granted Fast Track Designation for the treatment of Stage 1 and 2 Type 1 Diabetes. DIAGNODE-3, a confirmatory Phase III trial is actively recruiting patients with recent-onset (Stage 3) Type 1 Diabetes at 60 clinics in eight European countries and in the US. Significant results have previously been shown in a large genetically predefined patient group - in a large-scale meta-analysis as well as in the Company's prospective European Phase IIb trial, where Diamyd® was administered directly into a superficial lymph node in children and young adults with recently diagnosed Type 1 Diabetes. Injections into a superficial lymph node can be performed in minutes and are intended to optimize the treatment response. A biomanufacturing facility is under development in Umeå, Sweden, for the manufacture of recombinant GAD65 protein, the active ingredient in the antigen-specific immunotherapy Diamyd®. Diamyd Medical is a major shareholder in the stem cell company NextCell Pharma AB and in the artificial intelligence company MainlyAI AB.
Diamyd Medical's B share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company's Certified Adviser.
For further information, please contact:
Ulf Hannelius, President and CEO
Phone: +46 736 35 42 41
E-mail: ulf.hannelius@diamyd.com
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