Diamyd Medical AB Class B · OMXSTO:DMYD_B
Diamyd Medical AB Class B — Investor Presentation (February 2021)
37 slides from Diamyd Medical AB Class B's investor presentation, February 2021
Slide 1: Leading Developer of Disease Modifying Therapies for Autoimmune Diabetes
Leading Developer of Disease Modifying Therapies for Autoimmune Diabetes 1 1
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® • Upcoming pivotal program with a precision medicine approach • Addressable market encompassing up to 25% of all diabetes patients De-risked developme
Slide 4: Clinical stage disease modifying therapies
Clinical stage disease modifying therapies Inducing immunological tolerance to preserve endogenous insulin production. Stimulating regeneration of insulin producing beta cells and alleviating beta cell stress. The opportunity for Primary indication regeneration and market Approx. 25% of all > 1.5 mi
Slide 5: Prioritizing unmet medical need
Prioritizing unmet medical need 25% of the patients, autoimmune and severely insulin deficient diabetes, lack treatment options that address their underlying disease pathology Prioritized subgroups Autoimmune diabetes** Approximately 10% of all diabetes patients have severe autoimmune diabetes (SAID
Slide 6: Priority #1 - Autoimmune diabetes
Priority # 1 - Autoimmune diabetes Type 1 Diabetes LADA ~ 300,000 new cases every year* >1.2 million new cases every year** 132,000 children and adolescents (0-20 years of It is estimated that up to 10% of all type 2 age) and equally many adults are diagnosed with diabetes patients have autoimmune d
Slide 7: Annual economic burden of Type 1 Diabetes (T1D)
Annual economic burden of Type 1 Diabetes (T1D) 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. 7
Slide 8: Leading pipeline in autoimmune diabetes
Leading pipeline in autoimmune diabetes Clinical Trials Product Indication Trial Participants Sponsor Status Preclinical I IIa IIb III Diamyd Diamyd® T1D, intervention DIAGNODE-2 109 Medical Results available Linköping Diamyd® T1D, intervention DIAGNODE-1 12 Results available University NTNU, Diamyd
Slide 9: Diamyd
Recombinant GAD65 Formulated in Alum (rhGAD65/alum) Diamyd® Primary Indication New-onset type 1 Diabetes with HLA type DR3-DQ2 Label expansion Type 1 Diabetes prevention, LADA Mechanism of Action Induce immunological tolerance against GAD65 Clinical Effect and Benefit Preserve the endogenous insulin
Slide 10: Acknowledged Precision Medicine approach
Acknowledged Precision Medicine approach Highlights • New medical consensus regarding genetically defined groups of T1D • Strong case for the emerging precision medicine – in line with Diamyd Medical’s approach Battaglia et al, Introducing the endotype concept to address the challenge of disease het
Slide 11: Responders to Diamyd treatment identified
Responders to Diamyd® treatment identified Effect on preserving endogenous insulin production in genetically predefined group of T1D Hannelius et al, Efficacy of GAD-alum immunotherapy associated with HLA-DR3-DQ2 in recently diagnosed type 1 diabetes, Diabetologia 2020. 11
Slide 12: Phase IIb topline results demonstrate significant treatment effect of Diamyd
Phase IIb topline results demonstrate significant treatment effect of Diamyd® in genetically predefined group of individuals recently diagnosed with T1D
Slide 13: Topline results – significant treatment effect of Diamyd in genetically predefined group
Topline results – significant treatment effect of Diamyd® in genetically predefined group • Phase IIb verified meta-analysis – effect of Diamyd® was seen in HLA DR3-DQ2 positive individuals. No observed effect in individuals negative for HLA DR3-DQ2 • More than 50% greater preservation (p < 0.01) of
Slide 14: DIAGNODE-2 results support large-scale meta-analysis
DIAGNODE-2 results support large-scale meta-analysis Diamyd® Placebo Effect estimate (95% CI) HLA DR3-DQ2 absent Low dose (sc) 96 89 High dose (sc) 70 89 DIAGNODE-2 (il) 26 30 HLA DR3-DQ2 present Low dose (sc) 90 97 High dose (sc) 79 97 DIAGNODE-2 (il) 29 17 sc = 20 μg subcutaneous* il = 4 μg intral
Slide 15: Combined analysis including DIAGNODE-2 further supports precision medicine approach
Combined analysis including DIAGNODE-2 further supports precision medicine approach Meta-analysis based on more than 600 individuals from four placebo-controlled randomized intervention trials (Phase III Europe, Phase II Sweden, Phase II US, Phase IIb Europe) Placebo better Diamyd better Low dose =
Slide 16: Secondary endpoints in meta-analysis support effect on primary endpoint
Secondary endpoints in meta-analysis support effect on primary endpoint in DR3-DQ2 individuals HbA1c (mmol/mol) Insulin dose (IU/kg/24h) IDAAC1 Note: Preliminary unpublished results 16 Diamyd better Placebo better
Slide 17: 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 18: Intralymphatic administration route enhances effect of Diamyd
Intralymphatic administration route enhances effect of antigen-specific immunotherapy Diamyd® in type 1 diabetes 18
Slide 19: More efficient uptake in and drainage to lymph nodes following intralymphatic administration
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 20: Simple outpatient procedure enhances value proposition for Diamyd
Simple 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 radiologis
Slide 21: Dose-response relationship
Dose-response relationship Intralymphatic injections are superior to subcutaneous injections Comparison of three SC injections vs three IL (Bayesian analysis) → 99% probability that IL is superior to SC regarding C-peptide retention → 98% probability that IL is superior to SC regarding reduction of
Slide 22: Clinical and immunological results support mechanistic rationale for antigen-specific reprogramming
Clinical and immunological results from Diamyd® trials support the mechanistic rational for antigen-specific reprogramming of the immune response 1. GAD65 is taken up, processed and presented by 3. T-cell deactivation APCs to T-cells by MHC Class II protein complexes. Immunological results from clin
Slide 23: Superior safety profile
Superior safety profile Patient drop-out rate in clinical trials Total patient exposure 14% Diamyd Placebo 12% 12% 10% 376 844 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 24: Vaccine Manufacturing – Control and Predictability
Vaccine Manufacturing – Control and Predictability • 10,000 square feet site comprising clean rooms, laboratory facilities and office space • Transferring manufacturing of recombinant GAD65 (active pharmaceutical ingredient in the diabetes vaccine Diamyd®) to own facility • Making Diamyd Medical ind
Slide 25: Diamyd market exclusivity and manufacturing
Diamyd® market exclusivity and manufacturing Core Intellectual Property • Substance of matter in the US until 2032 • Intralymphatic administration of Diamyd® in Europe, Japan, Australia and Russia, additional countries pending, expiry 2035. HLA subgroups and biomarkers in national phase with expiry
Slide 26: Comprehensive knowledge ahead of Phase III
Comprehensive knowledge ahead of Phase III 1 2 3 4 Presence of HLA Additional Superior safety Intralymphatic DR3-DQ2 increases subcutaneous profile and injections of GAD- effect of GAD-alum injections further convenience alum superior to increases effect of subcutaneous GAD-alum injections Diamyd® h
Slide 27: 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 28: Clinical results with attractive path to market for Remygen
Clinical results with attractive path to market for Remygen® ▪ Ongoing clinical Phase IIa 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)
Slide 29: 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 Growth Hormone (µg/L) Normoglycemia Normoglycemia 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 30: Positive allosteric modulators enhance GABA's beta cell regenerative effects
Positive allosteric Effect of PAMs on INS-1 cell proliferation. INS-1 cells were cultured with the indicated PAM at a dose range of 10 −9 to 10−6 M and assessed for their proliferation. Data shown are the average rate of proliferation relative to that of cultures with media alone (designated as 1).
Slide 31: 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 32: Board, Management and Scientific Advisors
Board, Management and Scientific Advisors 32
Slide 33: Board of Directors
Board of Directors Erik Nerpin Anders Essen-Möller Maria-Teresa Essen-Möller Torbjörn Bäckström Mark A. Atkinson Independent of the Company Founder of and CEO during CEO of Health Solutions AB. CEO of Umecrine AB. Professor of Diabetes Research, and its principal owners. 1996-2007 of Diamyd Medical
Slide 34: Management
Management Ulf Hannelius Martina Widman Anna Styrud Anton Lindqvist Maja Johansson Eva Karlström Chief Executive Officer Director Clinical Development Chief Financial Officer Chief Scientific Officer Facility Manager Regulatory Manager PhD in Molecular Biology from M.Sc. in Mechanical Engineering fr
Slide 35: 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 36: Diamyd Medical
DIAMYD MEDICAL • Swedish clinical phase pharmaceutical company, founded 1994 • NASDAQ First North Growth Market, ticker DMYD B FINANCES • Market Cap Feb 8, 2021 ~ MSEK 1 800 • Cash Nov 30, 2020: MSEK 173 INDICATIONS • Diabetes • Autoimmunity PRODUCT CANDIDATES • Diamyd® (Phase IIb) • Remygen® (Phase
Slide 37: Diamyd Medical
Diamyd Medical www.diamyd.com
Diamyd Medical — Investor Presentation (February 2021) on 6ix