Diamyd Medical AB Class B · OMXSTO:DMYD_B
Diamyd Medical AB Class B — Investor Presentation (July 2023)
47 slides from Diamyd Medical AB Class B's investor presentation, July 2023
Slide 1: Transformational Precision Medicine for Autoimmune Diabetes
Transformational Precision Medicine for Autoimmune Diabetes Stockholm NASDAQ First North Growth Market – DMYD B
Slide 2: Disclaimer
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 e
Slide 3: Targeting Autoimmune and Insulin Deficient Diabetes
TARGETING AUTOIMMUNE AND INSULIN DEFICIENT DIABETES Leading clinical stage pipeline • First-in-class disease modifying therapies Diamyd® and Remygen® • R&D partnership with JDRF De-risked development program • Responder patients identified for Diamyd®, significantly increasing likelihood for success
Slide 4: Autoimmune Diabetes
AUTOIMMUNE DIABETES Significant unmet medical need and health economic burden Type 1 Diabetes ~ 500,000 new cases every year* 184,100 children and adolescents (0-19 years of age) and 329,000 adults are diagnosed with type 1 diabetes every year. It is more prevalent in western countries with the high
Slide 5: Significant Annual Economic Burden of Type 1 Diabetes
SIGNIFICANT ANNUAL ECONOMIC BURDEN OF TYPE 1 DIABETES Disease modifying therapies for T1D are predicted to have a multibillion-dollar economic impact in the US alone 1) Modelling the total economic value of novel T1D therapeutic concepts, January 2020, Health Advances. 5
Slide 6: Recent deals emphasize the value of novel innovative therapies for T1D
Recent deals emphasize the value of novel innovative therapies for T1D $2.9 billion acquisition of ProventionBio by Sanofi. FDA-approved immunotherapy “TZIELD” to delay onset of T1D. Vertex Pharmaceuticals acquisition of ViaCyte ($320M) and Semma Therapeutics ($950M) as well as $100M upfront licensi
Slide 7: Leading pipeline targeting autoimmune diabetes
Leading pipeline targeting autoimmune diabetes PROGRAMME DEVELOPMENT STATUS Study / Indication Preclinical Phase 1 Phase 2 Phase 3 Global Rights Milestones DIAGNODE-3 Ongoing in Europe, US start Q3 Diamyd®, Recent-onset Stage 3 2023, interim analysis Q4 Type 1 Diabetes (Treatment) 2024/Q1 2025, topl
Slide 8: Diamyd®
Diamyd® Recombinant GAD65 Formulated in Alum (rhGAD65/alum) Primary Indication Diamyd® New-onset (stage 3) Type 1 Diabetes with HLA type DR3-DQ2 Label Expansion Type 1 Diabetes prevention (stage 1 & 2), LADA Mechanism of Action Induce immunological tolerance against GAD65 Clinical Effect and Benefit
Slide 9: Multibillion total addressable market for Diamyd®
Multibillion total addressable market for Diamyd® >500 000 new cases of T1D globally every year ~ 130 000 in the US and • Estimated pricing in US in line with Europe existing drugs on the market (65k – 100k+) ~ 50 000 carry • Market for Stage 3 T1D with HLA DR3- HLA DR3-DQ2 DQ2 in US and Europe > $2
Slide 10: Focus on preemptive medicine
Focus on preemptive medicine Diamyd® is designed to prevent diabetes complications and improve glucose control by stopping the autoimmune destruction of beta cells Reduced risk of1 ● Severe hypoglycemia Illustrative decline of functional beta ● Life-threatening ketoacidosis cell mass in autoimmune d
Slide 11: Significant label expansion opportunities for Diamyd®
Significant label expansion opportunities for Diamyd® Stage 3 Stage 1&2 LADA Boosters Combinations T1D T1D
Slide 12: Position Diamyd® to maximize efficacy, safety, convenience
POSITION DIAMYD® TO MAXIMIZE EFFICACY, SAFETY, CONVENIENCE Disease modifying therapies Added value compared to standard of care Immunosuppressive Antigen-specific treatments Cell therapies technologies Effect, Benefit Antigen-specific immunotherapy with Diamyd® Artificial targets the body’s immune s
Slide 13: Diamyd® in Pivotal Phase 3 Program aligned with FDA and EMA
Diamyd® (rhGAD65/alum) In Pivotal Phase 3 Program aligned with FDA and EMA ● Strong safety profile – evaluated in almost 1,000 persons aged 4-70 years ● Compelling efficacy for preserving insulin producing capacity and improving glucose control based on data from >600 patients ● Simple and short tre
Slide 14: New-onset (Stage 3) Type 1 Diabetes with HLA type DR3-DQ2
New-onset (Stage 3) Type 1 Diabetes with HLA type DR3-DQ2 14
Slide 15: Responders to Diamyd® treatment
RESPONDERS TO DIAMYD® TREATMENT Genetic variants influence T1D is a collection GAD-specific immunotherapy is influenced by the same genetic the appearance of of distinct variants that influence autoimmunity. Individuals with GAD autoimmunity endotypes autoimmunity respond best to GAD immunotherapy S
Slide 16: Crucial research advances in precision medicine for type 1 diabetes
CRUCIAL RESEARCH ADVANCES IN PRECISION MEDICINE FOR TYPE 1 DIABETES Antigen- Antigen- reactive presenting cell T cell Significant treatment effects on: 1. Preservation of endogenous insulin production HLA is central to response 2. Improved HbA1c GAD65 3. Less glycemic variability 4. More time spent
Slide 17: HLA influences effect of Diamyd®
HLA INFLUENCES EFFECT OF DIAMYD® Significant and dose-dependent treatment effect of Diamyd® (GAD-alum) in HLA DR3-DQ2 positive individuals on preservation of own insulin production and HbA1c Meta-analysis with >600 recent-onset T1D patients 4 RCTs (Phase III EUR, Phase II SWE, Phase II US, Phase IIb
Slide 18: Correlated treatment effects on C-peptide and HbA1c
CORRELATED TREATMENT EFFECTS (CHANGE FROM BASELINE TO MONTH 15 VERSUS PLACEBO) ON C-PEPTIDE AND HBA1C 3 or 4 doses of Diamyd® vs Placebo Without HLA DR3-DQ2 With HLA DR3-DQ2 Sensitivity analyses, including adjustment for insulin dose, confirm robust effect Size of circle is corresponding to the rela
Slide 19: The first ever precision medicine Phase III trial in Type 1 Diabetes
The first ever precision medicine Phase III trial in Type 1 Diabetes • Diamyd® in individuals recently diagnosed with type 1 diabetes and positive for the HLA DR3-DQ2 haplotype
Slide 20: DIAGNODE-3 Pivotal Precision Medicine Phase 3 trial
DIAGNODE-3 Pivotal Precision Medicine Phase 3 trial Ongoing at just over 50 clinical sites in Europe Germany Czech Rep Estonia Spain Hungary Netherlands Sweden Poland Approved by FDA to start in US, ca. 10 US sites planned, starting summer of 2023 Joint press release, 4 April 2023 www.diagnode-3.com
Slide 21: Results support design of pivotal, global Phase III trial DIAGNODE-3
RESULTS SUPPORT DESIGN OF PIVOTAL, GLOBAL PHASE III TRIAL DIAGNODE-3 • Responder population HLA DR3-DQ2 (40-50%) with GADA, 12-28 yr, <6m T1D DR3-DQ2-pos/ • Intralympatic injections (superior to subcutaneous injections) DR4-DQ8-neg • 3 monthly injections (superior to 2 injections) DR3-DQ2-pos H2 𝛂2
Slide 22: Latent Autoimmune Diabetes in Adults (LADA)
Latent Autoimmune Diabetes in Adults (LADA)* *Also called Slowly progressing Autoimmune Diabetes (SAID) or Slowly progressing insulin- dependent diabetes mellitus (SPIDDM) 22
Slide 23: Background
Background In highly autoimmune LADA individuals: treatment that directly targets autoimmunity is needed *p< 0.007 Hals IK, Fiskvik Fleiner H, Reimers N, Astor MC, Filipsson K, Ma Z, Grill V, Björklund A. Investigating optimal β-cell-preserving treatment in latent autoimmune diabetes in adults: Resu
Slide 24: Levels vs. Baseline (%)
Glucagon-stimulated C-peptide levels unchanged at 12 months vs Baseline (0 months) in the HLA-DR3DQ2 positive subgroup Phase 2 trial with Diamyd in up to 70 year-old LADA patients Glucagon-stimulated C-peptide * * 100 DR3DQ2 negative (n=7) Levels vs. Baseline (%) DR3DQ2 positive (n=7) 95 90 85 80 0
Slide 25: Conclusions
Conclusions • Treatment with intralymphatic GAD is well tolerated in LADA individuals – no safety concerns • GAD-induced immune responses appear compatible with those in studies with type 1 diabetes • Results on C-peptide suggest an HLA-dependent beneficial effect akin to type 1 diabetes Also see •
Slide 26: Type 1 Diabetes prevention (Stage 1 & 2)
Type 1 Diabetes prevention (Stage 1 & 2) 26
Slide 27: Diamyd Medical coordinates the ASSET milieu
DIAMYD MEDICAL COORDINATES THE ASSET MILIEU A T1D Forum to drive precision medicine, prevention and screening Contact with T1D research community Aim for a European-level Partnerships in contact network developing AI algorithms Discuss best practices for Integration of data from screening programs d
Slide 28: About ASSET
ABOUT ASSET The innovation milieu ASSET (AI for Sustainable Prevention of Autoimmunity in the Society – www.asset.healthcare) will develop and evaluate new algorithms based on AI to be able to assess the individual risk of developing Type 1 Diabetes (T1D), and the likelihood of responding to differe
Slide 29: Safety and administration of Diamyd®
Safety and administration of Diamyd® 29
Slide 30: Intralymphatic administration route enhances effect of Diamyd®
Intralymphatic administration route enhances effect of Diamyd® antigen- specific immunotherapy in Type 1 Diabetes 30 30
Slide 31: More efficient uptake in and drainage to lymph nodes
MORE EFFICIENT UPTAKE IN AND DRAINAGE TO LYMPH NODES FOLLOWING INTRALYMPHATIC COMPARED TO SUBCUTANEOUS ADMINISTRATION Lessons Learned from Allergy Immunotherapy Trials Subcutaneously (S.C.) injected large molecules including proteins do not effectively spread to the draining lymphnodes. Intranodal (
Slide 32: Convenient outpatient procedure enhances value proposition for Diamyd®
CONVENIENT OUTPATIENT PROCEDURE ENHANCES VALUE PROPOSITION FOR DIAMYD® Potential to reach patients outside specialized clinics and avoiding costs related to hospitalization Intralymphatic Appointment Ultrasound Treated patient administration (minutes) (seconds) The procedure is performed by a radiol
Slide 33: Superior safety profile
SUPERIOR SAFETY PROFILE Patient drop-out rate in clinical trials Total patient exposure 14% Diamyd Placebo 12% 12% 10% 990 513 8% 7% 6% 4% Most commonly reported adverse events: • tenderness, injection site edema, injection site pain 2% and injection site reaction. 0.8% • no difference in the rate o
Slide 34: Manufacturing and Market Exclusivity of Diamyd®
Manufacturing and Market Exclusivity of Diamyd® 34
Slide 35: Full Control and Predictability of the Manufacturing Process
Full Control and Predictability of the Manufacturing Process A nascent biomanufacturing plant in Umeå - Northern Sweden’s cultural capital • 20,000 square feet facility comprising clean rooms, laboratory facilities and office space • Manufacturing facility property fully acquired in 2022. Goal to be
Slide 36: Diamyd® (rhGAD65/alum) manufacturing
DIAMYD® (rhGAD65/ALUM) MANUFACTURING Baculovirus expression system Upstream & process: Insect cells Clarification DP formulation Downstream Capture process: Polish Nanofiltration 36
Slide 37: Diamyd® IP & market exclusivity
DIAMYD® IP & MARKET EXCLUSIVITY Core Intellectual Property • Substance of matter in the US until 2032 • Intralymphatic administration of Diamyd® in Europe, Japan, China, Australia and Russia, additional countries pending, expiry 2035. • Intralymphatic administration of additional betacell antigens (
Slide 38: Remygen®
Modified Release GABA Primary Indication Type 1 diabetes Remygen® Label expansion LADA, Insulin-deficient type 2 diabetes Mechanism of Action Activate GABA-receptors in the pancreas Clinical Effect • Regenerate endogenous insulin production, reduce short- and long-term complications • Prevention of
Slide 39: Clinical results with attractive path to market for Remygen®
CLINICAL RESULTS WITH ATTRACTIVE PATH TO MARKET FOR REMYGEN® ▪ Phase Ib/IIa first in man trial ▪ ReGenerate-1 at the University of Uppsala where Remygen® (proprietary formulation of GABA) alone and in combination with low-dose alprazolam (GABA receptor modulator to enhance effect, see next slide) ev
Slide 40: GABA publications
VA biomedicines (ia) Article GABA and Combined GABA with GAD65-Alum Treatment Alters Th1 Cytokine Responses of PBMCs from Children with Recent-Onset Type 1 Diabetes Katie E. Heath "*, Joseph M. Feduska »*0, Jared P. Taylor #®, Julie A. Houp 2, Davide Botta *®, Frances E. Lund *©, Gail J. Mick *, Ger
Slide 41: GABA treatment improves the hormonal response to hypoglycemia
GABA TREATMENT IMPROVES THE HORMONAL RESPONSE TO HYPOGLYCEMIA GH response to hypoglycemia Glucagon response to hypoglycemia 10 8 Normoglycemia Normoglycemia Growth Hormone (µg/L) Glucagon (pmol/L) 8 Hypoglycemia Hypoglycemia 6 * 6 * 4 4 2 2 0 0 e e e e e se se se lin os os os in do do do e d d d el
Slide 42: Remygen® market exclusivity and manufacturing
REMYGEN® MARKET EXCLUSIVITY AND MANUFACTURING Core Intellectual Property • Exclusive license from UCLA on treating diabetes and other inflammatory diseases with GABA • Formulation patent application (Remygen®). Application in national phase. • Exclusive license from UCLA on GABA in combination with
Slide 43: Organization, Board, Management and Scientific Advisors
Organization, Board, Management and Scientific Advisors 43
Slide 44: Management, Board of Directors, Scientific Advisory Board
Management Board of Directors Scientific Advisory Board Dr. Ulf Hannelius, PhD, MBA Erik Nerpin, LL.M. Professor Dr. Mark Atkinson, PhD SWE President & Chief Executive Officer Chairman of the Board (Chair) University of Florida Martina Widman, MSc Anders Essen-Möller, MSc SWE Professor Dr. David Les
Slide 45: Top worldwide experts
TOP WORLDWIDE EXPERTS Covering the areas of clinical practice and scientific excellence in Type 1 Diabetes and LADA Prof. Johnny Ludvigsson Prof. David Leslie Prof. Åke Lernmark Prof. Daniel Kaufman Prof. Mark A. Atkinson Professor of Pediatrics. First Professor of Diabetes and Professor in Experime
Slide 46: Diamyd Medical
DIAMYD MEDICAL • Swedish clinical phase pharmaceutical company, founded 1994 • NASDAQ First North Growth Market, ticker DMYD B FINANCES • Market Cap Jul 17, 2023 ~ MSEK 843 • Cash May 31, 2023: MSEK 77 (+ MSEK ~70 from share issue July 2023) INDICATIONS • Diabetes • Autoimmunity PRODUCT CANDIDATES •
Slide 47: Diamyd Medical
Diamyd Medical www.diamyd.com