Diamyd Medical AB: Diamyd Medical updates on the registrational Phase 3 trial; upcoming interim analysis and enrollment

Interim Analysis outcome scheduled for beginning of August
The Interim Analysis will be conducted in July by ICON plc, the appointed global CRO for the Phase 3 trial, and will be assessed by an independent Data Safety and Monitoring Board (DSMB). The DSMB is currently scheduled to convene on July 30[th] to evaluate the Interim Analysis outcome. The DSMB has approximately a week to inform Diamyd Medical of the conclusion of their meeting.

As previously announced, an Interim Analysis of the ongoing precision medicine Phase 3 trial DIAGNODE-3 in Type 1 Diabetes will assess 6-month data from approximately 70-80 patients. The trial specifically enrolls patients that carry the genetic HLA DR3-DQ2 haplotype and the interim analysis evaluates the likelihood of the trial to achieve one of the primary endpoints, endogenous insulin production measured as stimulated C-peptide. The analysis will guide decisions on the continuation of the trial and is designed to maintain the registrational integrity of the trial. Specifically, the analysis will inform whether the trial is still likely to reach its endpoint regarding C-peptide while the exact efficacy observed at the interim readout is blinded.

Activities to accelerate patient enrollment
At the date of this press release, May 16, 2024, 109 patients have been enrolled and 27 patients are in active screening phase. A total of 335 patients have so far been screened in the trial and no patients have discontinued the trial. As expected, approximately 50 % of all the screened individuals carry the genetic HLA DR3-DQ2 haplotype that has been associated with a positive response to Diamyd[®] treatment. Also as expected, up to 40 % of screened patients fulfill all inclusion criteria for the trial. Current enrollment projections indicate that the trial will be fully enrolled during 2025. Activities are ongoing to accelerate recruitment, both in the US and Europe.

Patient enrollment in Europe has steadily increased and screening efforts in the US are improving. In total 55 clinics are currently open for screening patients in the trial and an additional 6 clinics are preparing to start screening.

Diamyd Medical's commitment to reach underserved patient communities
Activities to reach underserved patient communities in the US are ongoing. Recent research in the US has highlighted significant inequities among diabetes patients across racial/ethnic groups or insurance types, which is reflected in worse glycemic control and lower use of novel diabetes technologies. Also, it is a well known fact that people from racial and ethnic minorities and other diverse groups are underrepresented in clinical research. The efforts to inform clinical sites serving patients from underrepresented groups underscores Diamyd Medical's commitment to a robust and diverse participant base to ensure comprehensive assessment of Diamyd[®].

Discussions emphasizing C-peptide as a standalone endpoint
As previously announced, discussions are ongoing regarding the potential for accelerated approval of Diamyd[®] based on C-peptide as a key endpoint. This could entail a smaller number of patients completing the ongoing Phase-3 trial, and an earlier read-out. Specifically for C-peptide as a standalone endpoint, about 150 patients are needed to achieve at least 90% statistical power in DIAGNODE-3. Diamyd Medical will announce if and when any progress towards making Diamyd[®] available to patients earlier than currently expected is made.

The importance of C-peptide as a reasonably likely surrogate endpoint for Type 1 Diabetes amongst others, is increasingly being discussed by the regulatory and other authorities in the field. These discussions are based on the correlation between preserved C-peptide levels and improved glycemic control exemplified by a recent meta-analysis in The Lancet Diabetes & Endocrinology (Taylor et al 2023 (https://www.thelancet.com/journals/landia/article/PIIS2213-8587(23)00267-X/fulltext)) where data from about 2,700 Type 1 Diabetes patients across 21 trials were analyzed. Similar to earlier findings with Diamyd[®] (Nowak et al 2022 (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9540800/)), the analysis confirms the importance of preserved C-peptide. Specifically the study linked preserved C-peptide to better HbA1c levels, with notable improvements seen as soon as 6 months post-baseline.

About DIAGNODE-3
The confirmatory Phase III trial DIAGNODE-3 (www.diagnode-3.com), evaluating the safety and efficacy of the antigen-specific immunotherapy Diamyd[®] in individuals diagnosed with Type 1 Diabetes is ongoing in the United States and in eight European countries: Sweden, Spain, the Czech Republic, the Netherlands, Germany, Poland, Hungary and Estonia.

DIAGNODE-3 will enroll up to 330 individuals aged 12 to 29 years, recently diagnosed (within 6 months) with Type 1 Diabetes, who carry the HLA DR3-DQ2 haplotype, a certain genetic risk factor for Type 1 Diabetes. A further stratification for HLA haplotypes is included in order to evaluate the potential super responder group of individuals who are positive for HLA DR3-DQ2 and negative for HLA DR4-DQ8. HLA testing is well established and widely available.

This patient population is based on clinical efficacy and safety results from the Phase IIa and Phase IIb trials DIAGNODE-1 and DIAGNODE-2, as well as on the large-scale meta-analysis encompassing data from more than 600 individuals from previous Phase II and Phase III trials using Diamyd[®]. The trial design provides a high probability to reach its co-primary endpoints of preservation of endogenous insulin producing capacity measured as stimulated C-peptide and improved blood glucose control as determined by HbA1c.

DIAGNODE-3 is supported in part by funding from JDRF, the leading global type 1 diabetes research and advocacy organization.

About Diamyd Medical
Diamyd Medical develops 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 for the preservation of endogenous insulin production that has been granted Orphan Drug Designation in the U.S. as well as Fast Track Designation (February2024) by the U.S. FDA. DIAGNODE-3, a confirmatory Phase III trial is actively recruiting patients with recent-onset Type 1 Diabetes 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 lymphnode 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 also develops the GABA-based investigational drug Remygen[®] as a component in the treatments of metabolic diseases. Diamyd Medical is a major shareholder in the stem cell company NextCell Pharma AB as well as 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.