Dianthus Therapeutics, Inc. · NASDAQ:DNTH
Dianthus Therapeutics — Magenta Therapeutics — Investor Presentation (January 2020)
43 slides from Dianthus Therapeutics's investor presentation, Jan 13, 2020
Slide 1: Magenta Therapeutics
Exhibit 99.2 ($) magenta THERAPEUTICS January 2020 (NASDAQ:MGTA)
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “an
Slide 3: Multiple Sclerosis The Patient Journey
Multiple Sclerosis The Patient Journey
Slide 4: The Promise of a New Immune System: Jennie's Transplant Journey
The Promise of a New Immune System: Jennie’s Transplant Journey
Slide 5: Approved One-Time Curative Treatment for MS Does Not Exist Today
Approved One-Time Curative Treatment for MS Does Not Exist Today 2.3M Affected worldwide, or 1 in 700 adults One of most prevalent autoimmune diseases Leading cause of non-traumatic neurological disability in young adults Life-long, chronic therapies create high burden. 75% Unable to work within 5 y
Slide 6: Magenta will Revolutionize Immune Reset for All Patients
Magenta will Revolutionize Immune Reset for All Patients CONDITIONING Antibody Drug Conjugates MOBILIZATION Biologic Combo Drugs NEW CLASS OF MEDICINES
Slide 7: Immune Reset through Stem Cell Transplant: Currently Reserved for Limited Number of Patients Only
Immune Reset through Stem Cell Transplant: Currently Reserved for Limited Number of Patients Only 65,000 patients receive a transplant worldwide* *Includes Autoimmune Diseases, Blood Cancers, Genetic Diseases; not to scale
Slide 8: Immune Reset through Stem Cell Transplant: Currently Reserved for Limited Number of Patients Only
Immune Reset through Stem Cell Transplant: Currently Reserved for Limited Number of Patients Only 65,000 patients receive a transplant worldwide* 150,000 patients eligible today* *Includes Autoimmune Diseases, Blood Cancers, Genetic Diseases; not to scale
Slide 9: Immune Reset through Stem Cell Transplant: Currently Reserved for Limited Number of Patients Only
Immune Reset through Stem Cell Transplant: Currently Reserved for Limited Number of Patients Only 65,000 patients receive a transplant worldwide* 150,000 patients eligible today* Annual patients eligible for a transplant worldwide* *Includes Autoimmune Diseases, Blood Cancers, Genetic Diseases; not
Slide 10: From Platform to Medicines and One-time Cures
Platform Pipeline Vision From Platform to Medicines and One-time Cures Discovery Engine Differentiated first-in-class medicines Total patient care in outpatient setting
Slide 11: We Are Magenta
($) magenta THERAPEUTICS We Are Magenta | 6 Magenta (NASDAQ:MGTA)
Slide 12: Delivering Immune System Reset to Make Cures Possible for More Patients
Delivering Immune System Reset to Make Cures Possible for More Patients Only company comprehensively transforming transplant across autoimmune diseases, genetic diseases and blood cancers People, platforms and product engine to change practice of transplant medicine Portfolio of innovative, first-in
Slide 13: 2019 Accomplishments Fuel Momentum Ahead
2019 Accomplishments Fuel Momentum Ahead Preclinical IND-Enabling Phase I Phase II CD45 MGTA-145* Genetic Diseases Preclinical IND-Enabling Phase I Phase II CD117 MGTA-145* MGTA-456 Blood Cancers Preclinical IND-Enabling Phase I Phase II CD117 MGTA-145* MGTA-456 REMOVE REBUILD REMOVE REBUILD REMOVE
Slide 14: 2019 Accomplishments Fuel Momentum Ahead
2019 Accomplishments Fuel Momentum Ahead Preclinical IND-Enabling Phase I Phase II CD45 MGTA-145* Genetic Diseases Preclinical IND-Enabling Phase I Phase II CD117 MGTA-145* MGTA-456 Blood Cancers Preclinical IND-Enabling Phase I Phase II CD117 MGTA-145* MGTA-456 REMOVE REBUILD REMOVE REBUILD REMOVE
Slide 15: 2019 Accomplishments Fuel Momentum Ahead
2019 Accomplishments Fuel Momentum Ahead Preclinical IND-Enabling Phase I Phase II CD45 MGTA-145* Genetic Diseases Preclinical IND-Enabling Phase I Phase II CD117 MGTA-145* MGTA-456 Blood Cancers Preclinical IND-Enabling Phase I Phase II CD117 MGTA-145* MGTA-456 REMOVE REBUILD REMOVE REBUILD REMOVE
Slide 16: 2019 Accomplishments Fuel Momentum Ahead
2019 Accomplishments Fuel Momentum Ahead Preclinical IND-Enabling Phase I Phase II CD45 MGTA-145* Genetic Diseases Preclinical IND-Enabling Phase I Phase II CD117 MGTA-145* MGTA-456 Blood Cancers Preclinical IND-Enabling Phase I Phase II CD117 MGTA-145* MGTA-456 REMOVE REBUILD REMOVE REBUILD REMOVE
Slide 17: Delivering Immune Reset to Patients
Delivering Immune Reset to Patients
Slide 18: Platform Screening of Agents and ADC Payloads for Human Stem Cell Potency
Platform Screening of Agents and ADC Payloads for Human Stem Cell Potency Discovery Biology Biotherapeutics Discovery Stem Cell Biology Payload Class (Potency) RNA Polymerase Inhibitor (40pM) Microtubule Inhibitor (17nM) Ribosomal Inhibitor (-) Naked (-) Discovery Engine Delivers Candidates
Slide 19: Platform Screening Magenta ADCs in Primates
Platform Screening Magenta ADCs in Primates DRUG DOSE Therapeutic Index (TI) TI = <1 THERAPEUTIC INDEX = MTD/MED MTD = Maximum Tolerated Dose MED = Minimum Effective Dose Therapeutic Index (TI) TI = 6* MGTA-117 Amanitin ADC Therapeutic Index (TI) TI = 30**
Slide 20: Magenta will Revolutionize Immune Reset for All Patients
MOBILIZATION Biologic Combo Drugs Magenta will Revolutionize Immune Reset for All Patients CONDITIONING Antibody Drug Conjugates NEW CLASS OF MEDICINES
Slide 21: A Single Dose of CD45-ADC Enables Immune Reset to Treat Autoimmune Disease
Highly active on disease-causing T cells from patients Single-dose of CD45-ADC resets autoimmunity in all three disease models: Multiple Sclerosis Systemic Sclerosis Inflammatory Arthritis MS Patient T Cells MS Disease Model A Single Dose of CD45-ADC Enables Immune Reset to Treat Autoimmune Disease
Slide 22: Next steps for CD45-ADC
Potential opportunity Autoimmune Disease Future Next steps for CD45-ADC GMP manufacturing started Advance IND-enabling studies Initiate Phase I/II clinical trials in patients Transplants today * Not to scale
Slide 23: CD117-ADC is a Medicine for Blood Cancers and Genetic Diseases
CD117-ADC is a Medicine for Blood Cancers and Genetic Diseases (with Gene Therapy) A single dose of CD117-ADC: robustly depleted stem cells in vivo spared the immune system was well-tolerated was cleared rapidly to allow for optimal timing Transplant and gene marking was successful Results validate
Slide 24: Single Dose of CD117-ADC Selectively Depletes Stem Cells in Primates and is Rapidly Cleared
Single Dose of CD117-ADC Selectively Depletes Stem Cells in Primates and is Rapidly Cleared CD117-ADC Depletes Functional HSC CFU (x103) / mL of Bone Marrow Assess HSC depletion in bone marrow Primates CD117-ADC (Single dose, i.v.) Day 6 Day 0 Plasma ADC (ng/ml) Days Engineered CD117-ADC Lower limit
Slide 25: First Successful Autologous Transplant and Gene Therapy in Primates Conditioned without Chemotherapy
First Successful Autologous Transplant and Gene Therapy in Primates Conditioned without Chemotherapy Single Dose CD117-ADC Cohort Multi-Dose Busulfan Cohort (Clinical Regimen) Uchida et al. Mol Ther 2019 Primate Primate
Slide 26: Single Dose of CD117-ADC is Sufficient to Enable Gene Therapy and Transplant in Primates
Single Dose of CD117-ADC is Sufficient to Enable Gene Therapy and Transplant in Primates Days Post-Transplant Neutrophils (103/ul) NHP #1 NHP #2 Fast Neutrophil Recovery ANC 500 Vector Copy Number Days Post-Transplant NHP #1 NHP #2 Gene therapy range with busulfan conditioning Stable Gene Marking
Slide 27: CD117-ADC is Well-Tolerated in Primates
Busulfan Side Effects Outcomes With CD117-ADC Emesis Not observed Diarrhea Not observed Mucositis Not observed Wasting Syndrome Not observed Seizures Not observed Veno-occlusive Disease Not observed Pulmonary Fibrosis Not observed CD117-ADC is Well-Tolerated in Primates
Slide 28: CD117-ADC Future: Next steps for MGTA-117
CD117-ADC Future Next steps for MGTA-117 Complete GMP manufacturing Complete GLP tox study Begin clinical trial in patients Blood Cancers (allogeneic) Genetic Diseases/ Gene Therapy
Slide 29: Magenta will Revolutionize Immune Reset for All Patients
Magenta will Revolutionize Immune Reset for All Patients CONDITIONING Antibody Drug Conjugates NEW CLASS OF MEDICINES MOBILIZATION Biologic Combo Drugs
Slide 30: Rebuild: Current State of Stem Cell Mobilization: Inefficient, Multi-Day Process
Rebuild: Current State of Stem Cell Mobilization: Inefficient, Multi-Day Process
Slide 31: MGTA-145: A Novel Medicine
Faster mobilization Safer for donor Higher quality stem cells MGTA-145: A Novel Medicine
Slide 32: MGTA-145 Demonstrates Single Agent Activity and Robust Mobilization of CD34+ Cells
MGTA-145 Demonstrates Single Agent Activity and Leads to Robust Mobilization of CD34+ Cells in Healthy Subjects in Combination with Plerixafor Mobilization Regimen MGTA-145 dose (mg/kg) Timing Peak CD34+ (#/µL) Median (range) % ≥ 20/µL MGTA-145 + plerixafor 0.03 Staggered (2hr) 40 (18-63) 83% (5/6)
Slide 33: MGTA-145 + Plerixafor Enabled Single-Day Dosing, Mobilization and Collection
MGTA-145 + Plerixafor Enabled Single-Day Dosing, Mobilization and Collection of Robust Numbers of CD34+ Cells Subject Total CD34+ Yield (x106 cells) Body weight (kg) CD34+/kg (x106 cells) CD90+ (%)a 801 319 78.3 4.1 39% 807 322 72.6 4.4 41% 817 500 94.2 5.3 26% 821b 239 89.6 2.7 19% Median 321 - 4.3
Slide 34: Mobilized Human CD34+ Cells Rapidly Engraft in Humanized Mouse Model
Human CD34+ Cells from Healthy Subjects Mobilized with MGTA-145 + Plerixafor Rapidly Engraft in Humanized Mouse Model Radiation Transplant into NSG mice Collect mobilized CD34+ cells Low Mid High Superior Engraftment compared to G-CSF mobilized CD34+ cells
Slide 35: MGTA-145: Single-Day Dosing and Collection of High-Quality Stem Cells
has the potential to scale immune reset via transplant for autoimmune diseases, genetic diseases and blood cancers. MGTA-145: Single-Day Dosing and Collection of High-Quality Stem Cells Faster mobilization Safer for donor Higher quality stem cells
Slide 36: MGTA-145 Clinical Development Path
MGTA-145 Clinical Development Path Healthy Volunteers PHASE I TODAY PHASE II 2020 PHASE III Patients in different disease settings Engraftment
Slide 37: Stem Cell Mobilization Future and Next steps for MGTA-145
Transplants in the future with better conditioning 150,000 Transplants eligible today Stem Cell Mobilization Future ~$2B Total Addressable Market Next steps for MGTA-145 Complete Phase I trial Initiate Phase II studies in patients Demonstrate engraftment of CD34+ cells in patients Dialogue with agen
Slide 38: Looking Ahead
Looking Ahead (NASDAQ:MGTA) 'e) magenta THERAPEUTICS
Slide 39: Key Goals and Milestones Ahead in 2020
Key Goals and Milestones Ahead in 2020 (NASDAQ: MGTA) CD45-ADC Advance GMP manufacturing MGTA-117 Complete manufacturing and prepare to enter clinical trials MGTA-145 Launch Phase II studies to show engraftment in patients MGTA-456 Complete Phase II trial and define registration path in US and Europ
Slide 40: Large, Growing Market Opportunity and Commercial Ecosystem
Large, Growing Market Opportunity and Commercial Ecosystem US & EU Transplant Centers Are Concentrated; 20% of Centers Perform 50% of Transplants ~170 Total NMDP* Accredited US Centers ~205 Total JACIE** Accredited Centers Source: Huron Consulting Research, 2017 * National Marrow Donor Program ** Jo
Slide 41: Long-Term Vision for Total Patient Care and Cures
1st Horizon 2nd Horizon 3rd Horizon Long-Term Vision for Total Patient Care and Cures First global commercial launch of medicine Multiple first-in-class therapies across programs Total patient care with outpatient setting for all transplants
Slide 42: The Promise of a One-Time Curative Therapy
The Promise of a One-Time Curative Therapy
Slide 43: Magenta Therapeutics
(NASDAQ:MGTA) (e magenta THERAPEUTICS,
Magenta Therapeutics — Investor Presentation (January 2020) on 6ix