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Founded with the Mission to Cure Type 1 Diabetes April 2025
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DISCLAIMER This Presentation (”Presentation”) has been prepared by Diamyd Medical AB (“Diamyd” or “the Company”). By accepting this Presentation, the recipient acknowledges and agree to the following: The information contained herein have been prepared to assist interested parties making their own evaluations of the Company and does not purport to be all-inclusive, or to contain all the information that the prospective investor may desire. In all cases the interested parties should conduct their own investigations and analyses of the Company, and the data set forth in this Presentation. Diamyd does not make any representation or warranty as to the accuracy or completeness of the information contained in this Presentation. Diamyd expressly disclaim any and all liability based on, or relating to, any representations or warranties contained in, or errors or omissions from, this Presentation or any other written or oral communications transmitted to the recipient, or any of its affiliates, or representatives, in the course of its investment evaluation of the Company. 2
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Precision medicine to prevent and cure Type 1 Diabetes Clinically validated and de-risked immunology platform • Antigen-specific immunotherapy targeting genetic subgroups • Durable effect and favorable safety profile based on 16 trials and more than 1,000 treated patients • Potential to extend health and life span by lowering risk for cardiovascular disease and other long-term complications Precision medicine pipeline spanning prevention and intervention • Clinical development program, Diamyd® (targeting 40 % of Type 1 Diabetes) • Phase 3 program: Stage 3 Type 1 Diabetes • Phase 2 program: Stage 1 & 2 Type 1 Diabetes • Discovery program (targeting 50 % of Type 1 Diabetes) • Precision medicine ecosystem: AI driven risk prediction, disease screening, and in-house biologics manufacturing Significant commercial potential, strong regulatory alignment, near-term milestones • >$2 billion sales potential in the US for Diamyd® launch indication • Significant upsides: RoW, adult- onset Type 1 Diabetes, Stage 1 and 2 Type 1 Diabetes • FDA Fast Trackand Orphan designation, alignment for an accelerated approval pathway • Phase 3 readout March 2026 to support potential accelerated BLA 30+ years of Scientific and Clinical development
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>500 000 new cases of Type 1 Diabetes annually >$90 USD billion economic burden Life long dependence on insulin treatment and blood glucose measurements High risk for serious complications incl. cardiovascular disease 35 years shorter health span 15 years shorter life span Addressing a significant unmet medical need and economic burden T1Dindex.org; Healthadvances 2020; IDF Atlas 2022; Rafshani et al 2019, Lancet
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Stage 4 Long-term treatment Complications Autoimmunity Type 1 Diabetes – Progressive and irreversible autoimmune destruction of insulin-producing cells Genetic risk Environment Stage 0 Insulin production Stage 2 No symptoms Abnormal blood glucose Autoimmunity Stage 3 Symptoms Elevated blood glucose Autoimmunity Type 1 Diabetes Insulin dependent Stage 1 No symptoms Normal blood glucose Autoimmunity Diamyd Medical addresses the full spectrum of Type 1 Diabetes
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Program Discovery Preclinical Phase 1 Phase 2 Phase 3 Status Diamyd® Stage 3 Type 1 Diabetes (HLA DR3-DQ2 positive) Orphan designation; Fast Track designation Ongoing in EU & US, early readout March 2026 Stage 1&2 Type 1 Diabetes (HLA DR3-DQ2 positive) Fast Track designation Started Q4, 2023 Adult-onset Type 1 Diabetes / LADA (HLA DR3-DQ2 positive) Completed, published in 2023 Evaluation of booster doses Completed, published in 2024 Insulin antigen Stage 1,2,3 Type 1 Diabetes (HLA DR4-DQ8 positive) DIAGNODE-3 DIAPRECISE GADinLADA Pipeline Overview Targeting full spectrum of autoimmune diabetes through HLA-Specific antigen therapies Global rights available DIAGNODE-B
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Diamyd® Recombinant GAD65 Formulated in Alum (rhGAD65/alum) Primary Indication (Fast Track and Orphan designation) Type 1 Diabetes (Stage 3) with residual beta cell function and HLA type DR3-DQ2 Label Expansion Type 1 Diabetes prevention (Stage 1 & 2), Fast Track designation Adult-onset Type 1 Diabetes / LADA Mechanism of Action Induce immunological tolerance against GAD65 Clinical Effect and Benefit Preserve endogenous insulin production, delay or prevent isease progression, reduce or prevent short- and long-term complications Mode of Administration Three targeted intranodal injections one month apart, outpatient treatment Development Status Phase 3 – Stage 3 Type 1 Diabetes Phase 2 – Stage 1&2 Type 1 Diabetes Phase 2 - Adult-onset Type 1 Diabetes / LADA Licensing Status Global rights available Diamyd®
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GAD HLA presents GAD T cell receptor Antigen-presenting cell Auto-reactive T cell HLA is central to autoimmunity against GAD Response Selected for Phase 3 trial No Response HLA DR3-DQ2 present Individuals with Type 1 Diabetes Diamyd® HLA DR3-DQ2 absent Diamyd® targets the GADA-first Type 1 Diabetes endotype with HLA DR3- DQ2 positivity IAA-first disease • HLA DR4-DQ8 (60%) • Enterovirus B • INS , PTPN22, UBASH3A • Likely responders to an insulin-based antigen- specific therapy GADA-first disease • HLA DR3-DQ2 (40%) • Adenovirus F • BACH2 • Likely responders to Diamyd® Courtesy of Prof. Åke Lernmark. Graphs based on data from the TEDDY study. Age of auto-antibody occurrence Diamyd® responders
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Regulatory and Commercial strategy
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Pathway to market for Diamyd® • Aligned with both FDA and EMA Single pivotal Phase 3 trial (DIAGNODE-3) • For the treatment of Stage 1, 2 & 3 Type 1 Diabetes with HLA DR3-DQ2 Fast Track designation • For the treatment of Type 1 Diabetes with residual beta cell function Orphan designation • Alignment with the FDA Interim readout to support accelerated BLA planned for March 2026 Accelerated approval potential
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Estimated > $2 Billion Peak Sales in the US Alone Note: Base case assumptions informed by US payer and HCP Research Nov-24 • 60k+ patients (Stage 3 Type 1 Diabetes with residual beta cell function, HLA DR3-DQ2 positive, Age >= 12) Diamyd® Launch indication • WAC (gross) price $157K/course, grown at 2% p.a. • Limited Gross-to-Net discounts, max 20%. • > 80% access (high Type 1 Diabetes insurance coverage and expected high prior authorization) • At least 30% market penetration US Pricing, formulary status & market share • Ex-US sales (40% of global sales based on Type 2 Diabetes analogs) • Life Cycle Management – Stage 1,2 Type 1 Diabetes, Adult-onset T1D / LADA, booster courses • Adult-onset T1D / LADA base case US peak sales estimated at > $2 billion Significant Upsides
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Strong IP position and regulatory exclusivity • Substance of matter in the US until 2032 • Intralymphatic administration of Diamyd® in Europe, Japan, China, Hong Kong, Australia, South Africa, Eurasia and Canada, additional countries pending, expiry 2035. • Intralymphatic administration of additional betacell antigens (proinsulin, preproinsulin etc) approved in Australia, Israel, Russia, additional countries pending. • Treatment/prevention of HLA DR3-DQ2 subgroup with Diamyd® approved in Europe, Eurasia, Israel, Hong Kong, South Africa, Japan, South Korea, expiry 2038, additional countries pending. • Treatment/prevention of HLA DR4-DQ8 with insulin as an antigen approved in South Korea, expiry 2038, and pending in several territories. Core Intellectual Property • US BLA approval provides 12 years exclusivity • US orphan designation provides 7 years exclusivity • European approval provides 10 years of exclusivity Regulatory exclusivity
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Significant momentum paves way for potential Accelerated Approval March 2022 Phase 3 starts DIAGNODE-3 is initiated in Europe and expands to the US in September 2023 Present Focus on expedited approval Potential for accelerated approval. Interim readout, aligned with the FDA, planned for March 2026. February – June 2024 Fast Track Stage 1,2 & 3 T1D FDA grants Fast Track designation for Diamyd® across all stages of Type 1 Diabetes July 2024 Positive non-futility analysis DSMB recommendation to continue the trial unmodified July 2024 Accelerated pathway FDA acknowledges in Type C meeting C-peptide as surrogate endpoint for accelerated approval for Diamyd® BLA Potential for Priority and Rolling review Blockbuster potential for launch indication
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Clinical Data supporting launch indication for Diamyd®
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Favorable safety and tolerability profile No major safety signals in >1,000 patients exposed to Diamyd®. Drop-out rate <1% in trials with targeted administration (intralymphatic, IL) of Diamyd®. 4% <1% 12% 7% 0% 5% 10% 15% Patient drop-out rate in clinical trials Diamyd® - total Diamyd® - IL Vaccines Metabolic/CVD 1096601 Total patient exposure in 16 trials Diamyd® Placebo Summary of clinical safety data • Most common adverse events: transient tenderness at injection site, injection site edema, mild injection site pain and injection site reaction (< 7 days) • No major safety signals • No drug-related SAEs in intralymphatic (IL) program (1 in total, LADA population) • <1% subject drop-out rate in IL program • Safety assessed in persons aged 4 – 70 years, with Stage 1 to Stage 3 Type 1 Diabetes or Adult- onset Type 1 Diabetes / LADA
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Meta-analysis of 3 pre-2014 trials identified responder patients Meta-analysis of 3 randomized controlled clinical trials with subcutaneous Diamyd® conducted before 2014 with >500 individuals identified patients carrying HLA DR3-DQ2 gene as responders 44% reduction in C-peptide decline from Baseline to Month 15 compared to placebo in patients carrying the HLA DR3-DQ2 gene who received 3 or 4 injections of Diamyd® Hannelius et al. Diabetologia 2020 Significant treatment effect in subgroup of patients positive for HLA DR3-DQ2 gene (responder patients) 1 1 2 High dose = 3 or 4 injections; Low dose = 2 injections; Combined = 2, 3 or 4 injections Mixed meal tolerance test (MMTT) stimulated C-peptide Even larger treatment effect in ca. 50% of responder patients with HLA DR3- DQ2 who lack the HLA DR4-DQ8 gene (super responder patients) 2
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DIAGNODE-2 Phase 2b Trial Confirmed Responder Patients European, multinational, randomized, placebo-controlled, 2-arm trial assessing 3 targeted injections of Diamyd® given on top of standard of care Primary Endpoint • Change from Baseline to Month 15 in Mixed Meal Tolerance Test (MMTT) stimulated C-peptide Area under the Curve Key Secondary Endpoint • Change in Hemoglobin A1c (HbA1c) between baseline and Month 15 • Change in insulin-dose-adjusted HbA1c (IDAA1c) between Baseline and Month 15 • Change in daily exogenous insulin consumption between Baseline and Month 15 Population • Persons diagnosed with Type 1 Diabetes less than 6 months ago aged 12-24 years and positive for GAD antibodies • Residual beta cell function: fasting C-peptide ≥ 0.12 nmol/L • Pre-specified subgroup added to topline readout before database lock: responder patients with HLA DR3-DQ2 genotype Diamyd®*Screen Run-in Placebo 3 monthly intralymphatic injections *4 µg / inj, combined with oral Vitamin D 0 2 6 15 24 months Follow-up Open-label extension** **Subgroup of patients (50 out of 109)
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DIAGNODE-2 Phase 2b trial confirmed responder patients Diamyd® achieved statistically significant preservation of C-peptide secretion, numerical improvement in HbA1c compared to placebo at Month 15 in patients with HLA DR3-DQ2 Mixed meal tolerance test (MMTT) stimulated C-peptide Glycated haemoglobin (HbA1c) Pre-specified subgroup of patients positive for HLA DR3-DQ2 gene 56% reduction in C-peptide decline from Baseline to Month 15 compared to placebo treatment in patients carrying the HLA DR3-DQ2 gene Ludvigsson et al. Diabetes Care 2021
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DIAGNODE-2 Phase 2b Trial confirmed responder patients In exploratory analyses, Diamyd® achieved statistically significant benefit on Continuous Glucose Monitoring (CGM) outcomes in patients carrying the HLA DR3-DQ2 responder gene • Better Time in Range • Less time in severe hyperglycaemia • Less glycaemic variability Nowak et al. JCEM 2022 Independent Commentary by Lunati & Fiorina, JCEM 2022 Time in Range Glycaemic variability Time in severe hyperglycaemia % change from Baseline to Month 15 >250 mg/dL (>13.9 mmol/L) % change from Baseline to Month 15 Change in standard deviation from Baseline to Month 15
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0 3 6 9 12 15 0 500 1000 0 2 4 6 8 20000 40000 60000 Months GADA (mean change from baseline, IU/ml) Proliferation SI (mean change from baseline) GADA GAD-alum DR3-DQ2 (n=29) GADA GAD-alum Not DR3-DQ2 (n=27) GADA Placebo (n=52) Pr SI Placebo (n=52) Pr SI GAD-alum Not DR3-DQ2 (n=27) Pr SI GAD-alum DR3-DQ2 (n=29) # * * * * * * * * * * * * p<0.001 for difference to Placebo # p=0.0210 for difference between DR3-DQ2 and Not DR3-DQ2 groups 0 3 6 9 12 15 0 25 0 10 20 30 200 400 600 Time (months) GAD-stimulated IL-13 (change from baseline, pg/mL) GAD-stimulated IL-10 (change from baseline, pg/mL) IL10 GAD-alum DR3-DQ2 (n=29) IL10 GAD-alum Not DR3-DQ2 (n=27) IL10 Placebo (n=52) IL13 GAD-alum DR3-DQ2 (n=29) IL13 GAD-alum Not DR3-DQ2 (n=27) IL13 Placebo (n=52) # ## * * * * * * * * * * p<0.0001 for difference to Placebo # p=0.0095 for difference between DR3-DQ2 and Not DR3-DQ2 groups ## p=0.0080 for difference between DR3-DQ2 and Not DR3-DQ2 groups Median change from baseline of anti-GAD65 antibodies (GADA) and Proliferation of PMBC (Stimulation Index, SI) (A), and GAD-stimulated secretion by PBMC of IL-10 and IL-13 levels (B) for GAD-alum treated subjects with and without the DR3-DQ2 haplotype Placebo treatment subjects. P values, Wilcoxon test, are indicated. DIAGNODE-2 Phase 2b trial biomarker data support HLA-specific response GAD-specific immune response differentiates responders from non-responders
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Correlated Diamyd® treatment effects on C-peptide and HbA1c Updated meta-analysis including the Phase 2b trial shows correlated treatment effects on C-peptide and HbA1c – the two co-primary endpoints of the Phase 3 trial Nowak et al. Diabetes Obesity and Metabolism 2022 48% reduction in C- peptide decline, 4.8 mmol/mol (0.5% DCCT units) lower HbA1c from Baseline to Month 15 compared to placebo in patients carrying the HLA DR3-DQ2 gene who received 3 or 4 injections of Diamyd® Without HLA DR3-DQ2 3 or 4 doses of Diamyd® vs placebo With HLA DR3-DQ2 Larger C-peptide effect Better HbA1c
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• Procedure performed by a radiologist or endocrinologist with ultrasound training • Pain level equal to taking a blood sample • No pre-medication (only local anesthetic) 22 Ultrasound (minutes) Intralymphatic administration (seconds) Appointment Treated patient Intralymphatic (IL)-injection with needle placed in plan with the ultrasound probe Monitoring of IL injection using ultrasound Flory, S. et al(2024).Allergy, 79(8), 2222–2234 Ultrasound-guided targeted injection Quick, low-key outpatient procedure with discomfort comparable to venepuncture. Targets superficial lymphnode to enhance immunological response.
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The first ever precision medicine Phase 3 trial in Type 1 Diabetes Diamyd® in individuals recently diagnosed with Stage 3 type 1-diabetes and positive for the HLA DR3-DQ2 haplotype Partner since 2023
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DIAGNODE-3 Single Pivotal Precision Medicine Phase 3 trial Aligned with the FDA and EMA. 60 clinics in the United States and Europe. Randomized, placebo-controlled, 2-arm trial to confirm the effect and safety of 3 targeted injections of Diamyd® given on top of standard of care. Diamyd®Screen Run-in 2:1 rand. Placebo 3 monthly injections Combined with oral Vitamin D in both arms 0 2 6 15 Follow-up Interim analysis in March 2026 based on ~170 patients and 15-month follow-up to support a potential Accelerated BLA in the US 24 Co-Primary Endpoints • Stimulated C-peptide area under the curve, change from Baseline to Month 24 in Mixed Meal Tolerance Test (MMTT) • HbA1c, change from Baseline to Month 24 Key Secondary Endpoint • Time in glycemic target range 3.9-10 mmol/L (70-180 mg/dL) assessed by CGM, change from Baseline to Month 24 • Proportion of patients with insulin dose-adjusted HbA1c (IDAA1c) ≤9 (partial remission) at Month 24 • Number of episodes per patient of severe hypoglycemia between Baseline and Month 24 • Number of episodes per patient of diabetic ketoacidosis (DKA) between Baseline and Month 24 Population • Persons diagnosed with T1D less than 6 months ago aged 12-29 years who are positive for GAD antibodies and positive for HLA DR3-DQ2 • Residual beta cell function: fasting C-peptide ≥ 0.12 nmol/L
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Supportive clinical data in Stage 1 & 2 Type 1 Diabetes & Adult-onset Type 1 Diabetes (LADA)
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Phase 2 trial with Diamyd in up to 70 year-old LADA patients 1-year pilot study of targeted injections of Diamyd® in individuals with adult-onset Type 1 Diabetes (latent autoimmune diabetes in adults (LADA)) . No safety concerns. Glucagon-stimulated C-peptide *p< 0.03 for median 13.3% reduction at 12 months vs. Baseline (0 months) in the DR3DQ2 negative subgroup (n=7). *p< 0.04 for difference between HLA subgroups in change at 12 months vs. Baseline (0 months). Hals et al., Diabetes, Obes Metab. 2023 Unchanged glucagon- stimulated C-peptide levels at 12 months vs Baseline (0 months) in the HLA DR3-DQ2 positive subgroup. 0 5 12 0 5 12 80 85 90 95 100 DR3DQ2 positive (n=7) DR3DQ2 negative (n=7) Months Levels vs. Baseline (%) * *
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Analysis shows that 2 subcutaneous injections of Diamyd® may delay Type 1 Diabetes onset by nearly 7 years in children with the HLA DR3-DQ2 genotype – reinforcing its preventive potential and precision medicine approach. KM plot of time to Type 1 Diabetes in HLA DR3-DQ2 (Diamyd ® n=12, Placebo n=15). The arrow highlights the difference in median time to stage 3 Type 1 Diabetes. Performed in 2024 based on data from the Swedish National Diabetes Registry combined with phone interviews. The study was performed by Prof. Helena Elding Larsson, Lund University. Poster presented at ISPAD 2024, unpublished DiAPREV-IT: 2 subcutaneous injections of Diamyd® in 50 children positive for two or more islet autoantibodies. Clinical Data in Stage 1/ Stage 2 Type 1 Diabetes Long-Term follow-up of DiAPREV-IT shows that two subcutaneous injections of Diamyd® may delay Type 1 Diabetes onset by nearly 7 years
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DIAMYD MEDICAL COORDINATES THE ASSET MILIEU A Type 1 Diabetes Forum to drive precision medicine, prevention and screening Contact with Type 1 Diabetes research community Partnerships in developing AI algorithms Integration of data from different cohort studies Aim for a European-level contact network Discuss best practices for screening programs www.asset.healthcare
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Manufacturing of Diamyd® Wholly-owned biomanufacturing plant
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Diamyd Medical has established a biomanufacturing plant for GMP commercial scale production of recombinant GAD65 Commercial-scale production of rhGAD65 planned to be ready for BLA/MAA and market entry • 24,000 square feet facility in Umeå, Northern Sweden, comprising clean rooms, laboratory facilities and office space • Manufacturing facility property fully acquired in 2021 • Full control over the manufacturing of recombinant GAD65 • Independence from CDMOs, third parties • In control of costs and resource allocation • Potential beyond GAD manufacturing
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Full Control and Predictability of the Manufacturing Process Diamyd Medical’s Umeå facility uses the Baculovirus Expression Vector System (BEVS) in the complex manufacturing process of recombinant human GAD65 protein Baculovirus expression system & insect cells Upstream process Clarification Capture Polish Nanofiltration Downstream process Drug Product formulation
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DIAMYD MEDICAL • Swedish clinical phase pharmaceutical company, founded in 1994 • NASDAQ First North Growth Market, ticker DMYD B FINANCES • Market Cap April 15, 2025 ~ MSEK 810 • Cash Apr 14, 2025: MSEK 93.0 • Preferential rights issue, MSEK 208, incl warrants TO5 exercisable in April 2026. Subscription period April 15-29, 2025.
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Board of Directors SWE Anders Essen-Möller, MSc Chairman, Founder SWE Erik Nerpin, LL.M. Vice Chairman SWE Maria-Teresa Essen-Möller, MSc SWE Dr. Torbjörn Bäckström, MD, PhD Dr. Mark Atkinson, PhD Dr. Karin Hehenberger, MD, PhD Dr. Karin Rosén, MD, PhD Scientific Advisory Board Professor Dr. Mark Atkinson, PhD (Chair) University of Florida Professor Dr. David Leslie, MB BS, MRCS, MD, FRCP , FAoP University of London Professor Dr. Åke Lernmark, MD, PhD Lund University Associate Professor, Dr. Emily Sims, MD Indiana University School of Medicine Dr. Alice Long, PhD Benaroya Research Institute, Seattle Management Dr. Ulf Hannelius, PhD, MBA President & Chief Executive Officer Martina Widman, MSc Chief Operating Officer Anna Styrud, BSc Chief Financial Officer Anton Lindqvist, MSc Chief Scientific Officer Dr. Maja Johansson, PhD Chief Operating Officer – Manufacturing Site Team with extensive experience from biotech and pharma including Horizon Pharma, GSK, Genentech, Johnsson & Johnsson, Sanofi
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Diamyd Medical www.diamyd.com Ulf Hannelius, President & CEO ulf.hannelius@diamyd.com