September 2023 – August 2024, Diamyd Medical AB (publ), Fiscal year 2023/2024
Diamyd® vials Precision Medicine for Autoimmune Diabetes Aiming for Accelerated Market Approval Diamyd Medical’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B. Further information is available on https://www.diamyd.com September 1, 2023 – August 31, 2024 Net result: MSEK -151.8 (-116.1), fourth quarter: MSEK -49.7 (-37.5). The higher costs compared to previous year is mainly due to that all 60 clinics in Europe and U.S. in the Phase III trial DIAGNODE-3 are activated and recruiting patients. Result per share: SEK -1.6 (-1.5), fourth quarter: SEK -0.5 (-0.5) Cash flow from operating activities: MSEK -129.2 (-111.0), fourth quarter: MSEK -29.2 (-34.5) Cash and short-term investments at August 31, 2024: MSEK 132.4 (127.5). After the period a milestone amount from Breakthrough T1D and an additional amount to a previous sale of shares in Companion Medical to Medtronic were received, corresponding to a total amount of MSEK 17.5. In October 2024, MSEK 48 were received through redemption of warrants. Significant events during the fourth quarter, June 1, 2024 – August 31, 2024 Positive feedback from FDA on potential Accelerated Approval for Diamyd® in Type 1 Diabetes Diamyd Medical received second U.S. FDA Fast Track designation for Diamyd® - for the prevention of Type 1 Diabetes Other events during the fourth quarter Diamyd Medical announced positive interim analysis results for the Phase 3 Trial DIAGNODE-3 Other events after the fourth quarter Diamyd Medial raised approximately SEK 48 million through redemption of warrants Diamyd Medial announced the intention to pursue accelerated approval pathway for Diamyd® Comments by CEO Ulf Hannelius Dear Shareholders, The year has been characterized by a significant breakthrough in our discussions with the U.S. FDA. A major highlight of the year was our decisive step towards an accelerated approval pathway in the United States for Diamyd®. This strategy was bolstered by the positive outcome of our recent FDA discussions, where the Agency acknowledged that C-peptide, a marker for endogenous insulin production, could serve as a surrogate endpoint reasonably likely to predict clinical benefit. This recognition is critical as it allows us to pursue an earlier interim readout from the ongoing DIAGNODE-3 Phase 3 trial, expected around March 2026??, to support a potential accelerated Biologics Licensing Application (BLA) in the U.S., the world's by far most dominant pharmaceutical market. Another key regulatory milestone this year was securing Fast Track designation from the FDA for the treatment of individuals with Stage 3 Type 1 Diabetes, as well as for the potential use of Diamyd® in delaying the onset of clinically diagnosed insulin requiring Type 1 Diabetes in pediatric patients with Stage 1 or Stage 2 Type 1 Diabetes. These designations underline the critical need for new treatment options across the entire spectrum of autoimmune diabetes and provide an opportunity to work closely with the FDA to expedite Diamyd®’s development?. Moreover, the positive outcome of the futility analysis in July of the DIAGNODE-3 trial marked a pivotal milestone. The independent Data Safety Monitoring Board's recommendation to continue the trial as planned reaffirmed our confidence in the potential of Diamyd® to address the unmet medical need in Type 1 Diabetes. The six-month data from 74 patients indicated that the trial is on track to meet its objectives, particularly regarding the preservation of endogenous insulin production?. In preparation for the BLA and the potential commercialization of Diamyd®, our biologics manufacturing facility in Umeå plays a crucial role. The facility is currently engaged in producing technical rhGAD65 batches (recombinant human GAD65 protein, the active ingredient in Diamyd®), as well as preparing and establishing procedures to ensure future compliance with Good Manufacturing Practice (GMP). Continued activities at the facility aim to demonstrate that we can reliably and reproducibly manufacture rhGAD65 at the quality and scale required to meet regulatory demands and future market demand for Diamyd®. This work is being carried out in parallel with manufacturing activities for the final drug formulation, as well as clinical operations, in order to optimize timelines with the available resources ahead of a possible BLA submission. We have also strengthened the Umeå team on the business development side to assess future opportunities for external manufacturing. Moreover, the recent warrant exercise this September strengthened our cash position, raising approximately SEK 48 million before issue costs. Additionally, we received this month a milestone payment from our existing collaboration with Breakthrough T1D (formerly JDRF), along with additional proceeds from the previous sale of Companion Medical shares, totaling SEK 17.5 million. This further supports our clinical and manufacturing operations. Looking ahead, the planned earlier readout from the DIAGNODE-3 trial in March 2026, represents a critical step in our journey to making Diamyd® available to patients as swiftly as possible. We remain fully committed to advancing this promising therapy through ongoing dialogue and collaboration with the FDA. With all 60 clinical sites activated in Europe and the U.S., and with 161 patients recruited to date, we are well on our way to achieve important recruitment milestones that are aligned with the planned earlier readout. In summary. Our progress this year has been nothing short of remarkable. DIAGNODE-3 stands as the only ongoing Phase 3 trial worldwide for a disease-modifying therapy in Type 1 Diabetes, placing Diamyd Medical at the absolute forefront of innovative treatments. Stockholm, October 9, 2024 Ulf Hannelius, President and CEO Significant events during the fourth quarter June 1, 2024 – August 31, 2024 Positive feedback from FDA on potential Accelerated Approval for Diamyd® in Type 1 Diabetes Diamyd Medical received encouraging feedback from the U.S. Food and Drug Administration (FDA) regarding an Accelerated Approval pathway for its immunotherapy, Diamyd® ( rhGAD65/alum), for treating patients with Stage 3 Type 1 Diabetes carrying the genotype HLA DR3-DQ2. Diamyd Medical received second U.S. FDA Fast Track designation for Diamyd® - for the prevention of Type 1 Diabetes The FDA granted Fast Track designation for Diamyd® to treat Type 1 Diabetes in pediatric patients with Stage 1 or Stage 2 Type 1 Diabetes carrying the genotype HLA DR3-DQ2. Earlier this year, Diamyd® received Fast Track designation for the treatment of individuals with Stage 3 Type 1 Diabetes carrying the HLA DR3-DQ2 genotype. The FDA grants Fast Track designation to facilitate the development and expedite the review of medicines to treat serious conditions and fill an unmet medical need. Fast Track designation is intended to bring promising medicines to patients sooner. Other events during the fourth quarter June 1, 2024 – August 31, 2024 Positive Interim Analysis Results for Phase 3 Trial DIAGNODE-3 Diamyd Medical announced a successful interim analysis (non-futility test) for its ongoing precision medicine Phase 3 DIAGNODE-3 trial. The interim analysis, reviewed by an independent Data Safety Monitoring Board (DSMB), resulted in a favorable recommendation to continue the trial without any modifications. Other events after the fourth quarter Diamyd Medical announced the final outcome of the exercise of series TO 3 warrants Diamyd Medical issued a total of 9,165,682 series TO 3 warrants in 2023 as part of a rights issue of units. During the exercise period, which ran from September 2, 2024, to September 30, 2024, a total of 4,365,200 shares were subscribed for by exercise of warrants TO 3, corresponding to an exercise rate of approximately 95 percent. The Company will thereby receive issue proceeds of approximately SEK 48 million before deduction of related issue costs. Diamyd Medical to pursue accelerated approval pathway for Type 1 Diabetes precision medicine Diamyd Medical announced that the Company will pursue an accelerated approval pathway in the U.S. for its antigen-specific immunotherapy, Diamyd®, aimed at preserving endogenous insulin production in patients with Stage 3 Type 1 Diabetes. An earlier interim study readout from the ongoing Phase 3 trial, DIAGNODE-3, is planned to serve as the basis for a Biologics License Application (BLA) under the accelerated approval pathway. Results from the interim readout are expected around March 2026. Drugs in clinical development Diamyd® and Remygen® are drugs in clinical development that focus on the underlying disease mechanisms of diabetes; the dysfunction and loss of insulin-producing beta cells in the pancreas. Diamyd® is an antigen-specific immunomodulating precision medicine therapy for the treatment and prevention of autoimmune diabetes (type 1 diabetes and LADA, Latent Autoimmune Diabetes in Adults). Clinical data indicate the potential of Diamyd® to halt or stop the autoimmune destruction of insulin-producing beta cells in individuals that carry the HLA DR3-DQ2 haplotype. The effect is achieved by antigen-specific reprogramming of immune cells by administration of low doses of Diamyd® in superficial lymph nodes. By maintaining the endogenous insulin production, Diamyd® has the potential to make a significant difference in the daily life of patients as well as significantly reduce the complications of type 1 diabetes. Topline results from the Phase IIb trial DIAGNODE-2 demonstrated a significant treatment effect of Diamyd® in the predefined genetic patient group. A confirming Phase III trial, DIAGNODE-3, is on-going. Remygen® is an oral GABA-based regenerative and immunomodulatory drug candidate for the treatment of autoimmune- and type 2 diabetes. The safety of Remygen® has been demonstrated in a Phase 1/2 clinical trial with Remygen® in patients who have had type 1 diabetes for several years. In addition to safety, the study also collected data on restoring or stimulating the body's insulin production and preventing hypoglycaemia. Clinical trials Type 1 Diabetes is a devastating disease which requires daily treatment with insulin to sustain life. The importance of finding a drug that improves the prospects for patients with diabetes is of utmost importance. The effect of intralymphatic administration of Diamyd®, an antigen-specific precision medicine immunotherapy aimed at stopping the immune system's attack on insulin-producing beta cells in autoimmune diabetes, is evaluated in the Phase III trial DIAGNODE-3 and in the Phase II trial DiaPrecise. Ongoing clinical trials Trials with Diamyd® in lymph nodes DIAGNODE-3 – DIAMYD® IN LYMPH NODES WITH ORAL SUPPLEMENTATION OF VITAMIN D The placebo-controlled Phase III trial DIAGNODE-3 will include approximately 330 individuals aged 12 to 29 who have been recently diagnosed with Type 1 Diabetes and who carry the genetically defined haplotype HLA DR3-DQ2. The trial is currently ongoing at approximtely 60 clinics in eight European countries and the United States, where about 40% of all individuals with Type 1 Diabetes are estimated to carry the current haplotype. After an initial month in which all trial participants receive vitamin D, the individuals will be randomized 2:1, i.e. two out of three trial participants will receive three intralymphatic injections of Diamyd® and one in three will receive the corresponding placebo at one-month intervals, with one primary reading 24 months after trial start. The design provides, based on efficacy data from previous studies on the HLA-restricted patient population, a high probability of reaching the primary endpoints; preservation of stimulated C-peptide and lower HbA1c. The Coordinating Investigator for the trial is Professor Johnny Ludvigsson at Linköping University. The Sponsor of the trial is Diamyd Medical. DiaPrecise – DIAMYD® IN LYMPH NODES DiaPrecise is an open-label clinical trial where Diamyd® (GAD-alum) is given directly into a lymph node in 10 to 16 children aged 8 to 18 years who are at high risk of being diagnosed with clinical Type 1 Diabetes (so called Stage 1 or Stage 2 Type 1 Diabetes), and who also carry the genetically defined haplotype HLA DR3-DQ2, associated with clinical response to Diamyd®. The aim of the trial is to evaluate the safety and feasibility of two or three intralymphatic injections with Diamyd® as well as the effect on the immune system and clinical parameters including endogenous insulin production and blood glucose control. The Principal Investigator of DiaPrecise is Dr. Markus Lundgren, Researcher at the Department of Clinical Sciences at Lund University and consultant pediatrician at Kristianstad hospital, Sweden. Sponsor of the trial is Diamyd Medical. About Diamyd MedicalDiamyd 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 by the U.S. FDA for the treatment of 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 at 60 sites 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 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 more information, please contact: Ulf Hannelius, President and CEO, phone: +46 736 35 42 41 Diamyd Medical AB (publ), Box 7349, SE-103 90 Stockholm, Sweden Phone: +46 8 661 00 26 E-mail: info@diamyd.com Reg. no: 556242-3797
The information was submitted for publication, through the agency of the contact person set out above, at 08.15 CET on October 9, 2024.
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