Autoimmune diabetes vaccine (Diamyd)

STOCKHOLM, Jan. 13, 2025 /PRNewswire/ -- Diamyd Medical has released an updated analysis of the commercial potential for its lead product candidate, the precision immunotherapy Diamyd® (rhGAD65/alum) in the U.S. market. The addressable patient population for its launch indication is estimated at over 60,000 individual patients, with annual peak sales projections well exceeding block buster potential*. Diamyd® has Orphan Drug Designation status in the U.S. for its launch indication, and follow-on indications represent a market potential that is expected to equal or even exceed its primary indication. *Defined as annual sales of at least $1 billion. Addressable market at launch The addressable market for Diamyd® at launch consists of two distinct populations, both comprising patients aged 12 years and older. Newly diagnosed Stage 3 Type 1 Diabetes patients In 2024, approximately 47,000 patients in the U.S. are expected to be newly diagnosed with Stage 3 T1D. Of these, an estimated 19,000 individuals (40 %) are HLA DR3-DQ2 positive and eligible for Diamyd® treatment. Recently diagnosed Type 1 Diabetes patients with retained beta cell function An additional opportunity exists among recently diagnosed Type 1 Diabetes patients who retain beta cell function for up to two years following diagnosis. This population is estimated at 62,000 individuals in 2024, of whom approximately 25,000 are HLA positive. While this group is expected to shrink as Diamyd® is adopted in newly diagnosed patients, it will remain a significant contributor to the addressable market. These combined populations will annually total over 60,000 patients in the U.S. the coming years when accounting for incidence and population growth. Market assumptions Pricing: Diamyd®'s base case gross price is projected at $157,000 per course, supported by market research with payers and providers. Limited standard Gross-to-Net discounts, which account for rebates, discounts, and other payer incentives, are expected. The final gross price will be dependent on the Phase 3 data, with potential for a gross price up to $200,000 per course. Price increases of 2% per year are expected. Access and coverage: Diamyd® is expected to achieve over 80 % market access. This is based on broad insurance coverage of Type 1 Diabetes and expected high prior authorization approvals, reflecting its strong clinical differentiation as a disease-modifying therapy and its status having an FDA granted orphan drug designation. Market penetration: Qualitative market research supports an estimate of at least 30 % of Type 1 Diabetes patients being a reasonable penetration assumption for base case projections. Clear growth drivers and upsides Latent Autoimmune Diabetes in Adults (LADA): LADA, a condition affecting approximately 7 % of the 1.2 million adults diagnosed annually with Type 2 Diabetes in the U.S., represents an addressable market that is equal or larger than the Stage 3 Type 1 Diabetes market. Earlier Type 1 Diabetes Stages: Expanding Diamyd®'s indication to include Stage 1 and Stage 2 Type 1 Diabetes patients offers opportunities for early intervention. The size of the addressable market is heavily dependent on the availability and coverage of screening programs. Booster courses: Booster treatments for patients previously treated with Diamyd® present an additional market segment. Ex-U.S. market: Global opportunities could account for an additional 40 % of Diamyd®'s total sales potential, based on analogs from the Type 2 Diabetes market. The market research underscores the importance of enhancing Health Care Practitioner (HCP) education and engagement, as many physicians lack familiarity with the genetic and clinical profiling necessary to identify eligible patients, such as HLA testing and C-peptide measurements. Given that Diamyd® could be the first or among the first therapies approved to preserve insulin-producing capacity in Type 1 Diabetes, measured by C-peptide, it is essential to educate HCPs, patient advocacy groups, and payers about the critical value of maintaining endogenous insulin production and its long-term benefits for patient outcomes. The market research was conducted in collaboration with the Kondrad Consulting Group, LLC, Sarasota, FL USA, . 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: [email protected] This information was brought to you by Cision . The following files are available for download: SOURCE Diamyd Medical AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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: [email protected] This information was brought to you by Cision . The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED