Dianthus Therapeutics, Inc. · NASDAQ:DNTH
Dianthus Therapeutics — Magenta Therapeutics — Investor Presentation (January 2019)
40 slides from Dianthus Therapeutics's investor presentation, Jan 7, 2019
Slide 1: Magenta Therapeutics January 2019
Magenta Therapeutics January 2019 Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements and information. The use of words such as "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar
Slide 3: The Promise of a One-Time Curative Therapy
The Promise of a One-Time Curative Therapy
Slide 4: Bone Marrow Transplant: The Patient Journey to a Cure
Bone Marrow Transplant: The Patient Journey to a Cure CONDITIONING Antibody Drug Conjugates EXPANSION Small Molecule Ex Vivo Expansion MOBILIZATION Biologic Combo Drugs Post-Transplant Complications Novel Therapeutics EXPANSION Small Molecule Ex Vivo Expansion CAR T Therapy
Slide 5: The Rising Impact of Transplant-Based Therapies: Autoimmune Disease
The Rising Impact of Transplant-Based Therapies: Autoimmune Disease Sullivan et al, New England Journal Medicine 2019 Multiple Sclerosis >2000 pts transplanted Crohn’s Disease Type I Diabetes Systemic Lupus Erythematosus Atkins et al, Lancet 2016 TRANSPLANT Systemic Sclerosis >1000 pts transplanted
Slide 6: The Rising Impact of Transplant-Based Cell and Gene Therapies
The Rising Impact of Transplant-Based Cell and Gene Therapies Gene Therapy 1 approved medicine Clinical trials for more than 20 diseases 7 programs for SCD Barriers: conditioning, mobilization, cell dose CAR T 2 approved medicines >240 ongoing clinical trials Barriers: conditioning
Slide 7: The Rising Use of Transplant-Based Therapies for Blood Cancers
The Rising Use of Transplant-Based Therapies for Blood Cancers Annual Number of Transplants by Type (US) Increased number of transplants, curative benefit Barriers: Conditioning, finding matched donor/cell dose (>50% patients do not get a matched donor)
Slide 8: Barriers to Transplant Limit Eligible Patients Who Could Benefit Today
Barriers to Transplant Limit Eligible Patients Who Could Benefit Today Only ~40% of Eligible Patients Are Transplanted Today* *Based on treatment protocols in eight diseases that comprise ~70% of total transplants (NHL, MM, AML, MDS, IMDs, SCD, MS, and SSc) Malignant Non-malignant 65,000 Transplants
Slide 9: Magenta's Portfolio Could Allow Every Eligible Patient to Benefit
Magenta’s Portfolio Could Allow Every Eligible Patient to Benefit Potential for All Eligible Patients to Be Transplanted Magenta Portfolio Addresses Transplant Barriers Targeted conditioning Higher cell doses Better-matched cells Prevent GVHD Increased system capacity 65,000 Transplants Malignant No
Slide 10: Magenta's Portfolio Could Allow More Patient Cures, Earlier in Disease Course
Magenta’s Portfolio Could Allow More Patient Cures, Earlier in Disease Course With Increasing Experience with Transplant and Magenta Medicines Potential to move transplant earlier in treatment paradigm Increase patient eligibility Grow transplant-addressable population Today’s >150,000 Eligible Pati
Slide 11: Magenta Engine Drives Innovative Programs
Magenta Engine Drives Innovative Programs Discovery Biology * To be developed in partnership for E478-expanded gene therapies ** Investigator-initiated study Biotherapeutics Discovery Stem Cell Biology Targeted Conditioning Mobilization Expansion Post- Transplant Complications Program C100 C200 C300
Slide 12: 2018: A Transformative Year for Magenta
2018: A Transformative Year for Magenta Built unique platform for targeted conditioning Selected first development candidate for targeted conditioning (C200) Completed IND-enabling studies for first-line mobilization product candidate, MGTA-145 Initiated Phase 2 study of cell therapy MGTA-456 in inh
Slide 13: Key Takeaways Today
Key Takeaways Today Potential patient impact Next steps for each program Near-term milestones and long-term path
Slide 14: Targeted Conditioning: Transforming Patient Preparation for Transplant, Gene Therapy and CAR T
Targeted Conditioning: Transforming Patient Preparation for Transplant, Gene Therapy and CAR T
Slide 15: An Overdue Revolution in Patient Preparation
An Overdue Revolution in Patient Preparation Deplete stem cells Make space for incoming donor stem cells Prevent rejection by host immune cells Prevent relapse in blood cancers Remove pathogenic immune cells in autoimmune disease Current Agents Old Not selective Toxic Severe Side Effects Organ toxic
Slide 16: Innovative Platform Designed for Targeted Patient Conditioning
Innovative Platform Designed for Targeted Patient Conditioning Payload Potent: Depletes target cells Selective: Binds only to target cells Engineered: Fast-acting and safe
Slide 17: New Targeted Agents for Conditioning – Two Profiles
New Targeted Agents for Conditioning – Two Profiles C200 Lead target CD117 Cells removed Stem cells Disease-causing cells Diseases Blood cancers Genetic diseases (gene therapy) C100 Lead target CD45 Cells removed Stem and immune cells Disease-causing cells Diseases Autoimmune diseases Blood cancers
Slide 18: A Single Dose of C200 or C100 Selectively Removes Target Cells in Non-human Primates
A single dose of fast-half life anti-CD117–Amanitin shows greater than 95% depletion of HSC and CFU in the bone marrow of NHPs 7 days after treatment Well tolerated in non-human primates A Single Dose of C200 or C100 Selectively Removes Target Cells in Non-human Primates 18 Swap out for Brad’s graph
Slide 19: C200 Development Candidate is Well Tolerated in Non-Human Primates
C200 Development Candidate is Well Tolerated in Non-Human Primates C200 Control Liver (10x) Heart (10x) Stain: Hematoxylin &Eosin H: Hemorrhage H H All animals healthy All tissues normal Effective dose of C200 or Busulfan C200 Busulfan No animals survived Multiple organs damaged Control Busulfan
Slide 20: Clinical Development Paths for C200 and C100
Clinical Development Paths for C200 and C100 C100: Acute Leukemias (CD45+) C100: Autoimmune Disease Safety and tolerability Level of target cell depletion Safety and tolerability Engraftment C200: Stem Cell Gene Therapy C200: Acute Leukemias (CD117+) C100: CAR T Phase II Efficacy C200 Phase 1: Acute
Slide 21: Targeted Conditioning Programs Will Have Significant Patient Impact
Targeted Conditioning Programs Will Have Significant Patient Impact 65,000 Transplants ~60% Additionally Eligible 100% Transplanted (>150,000) Future Eligible Patients Today’s >150,000 Eligible Patients Future Eligible Patients Today’s >150,000 Eligible Patients 65,000 Transplants ~60% Additionally
Slide 22: Magenta Will Optimize Stem Cell Collection
Magenta Will Optimize Stem Cell Collection
Slide 23: MGTA-145 Mobilization Addresses Challenges for Donors and Patients
MGTA-145 Mobilization Addresses Challenges for Donors and Patients Plus Increases Transplant Center Capacity Limitations to current Standard of Care Requires 4-6 days Unpredictable yields Side effects Not used in some diseases Benefits of novel mobilization regimen Mobilize higher doses of high-qual
Slide 24: A Single Injection of MGTA-145 + Plerixafor Rapidly Mobilizes Large Numbers of Stem Cells
A Single Injection of MGTA-145 + Plerixafor Rapidly Mobilizes Large Numbers of Stem Cells in Nonhuman Primates Stem Cells G-CSF G-CSF
Slide 25: A Single Injection of MGTA-145 + Plerixafor Rapidly Mobilizes Sufficient Cells for Transplant
A Single Injection of MGTA-145 + Plerixafor Rapidly Mobilizes Sufficient Cells for Transplant in Four Hours EXPERIMENTAL DESIGN MOBILIZATION & COLLECTION (4 hours) AUTOLOGOUS TRANSPLANTATION CD34+ SELECTION MGTA-145 + plerixafor In collaboration with Dr. Hans-Peter Kiem; Fred Hutch Inst. MOBILIZATIO
Slide 26: MGTA-145: Ideal Mobilization Agent to Scale Transplant
MGTA-145: Ideal Mobilization Agent to Scale Transplant BENEFITS OF MGTA-145 + PLERIXAFOR Minutes Stem Cells Magenta Mobilization MGTA-145 + plerixafor Single injection of MGTA-145 + plerixafor Rapid and robust mobilization of stem cells Mobilized cells block graft vs host disease Rapid engraftment M
Slide 27: Next Steps for MGTA-145
Next Steps for MGTA-145 2020 2019 Allogeneic Donors Safety/tolerability Dose selection Number of stem cells mobilized Safety/tolerability Number of stem cells mobilized Engraftment Phase 3: MM and NHL First clinical data expected ASH 2019 First MM/NHL data expected ASH 2020 Phase 2: MM and NHL Autol
Slide 28: MGTA-145 Will Have Significant Impact in Transplant and Gene Therapy
MGTA-145 Will Have Significant Impact in both Allogeneic and Autologous Transplant and Gene Therapy 65,000 Transplants Malignant Non-malignant 100% Transplanted (>150,000) Future Eligible Patients Today’s >150,000 Eligible Patients Future Eligible Patients Today’s >150,000 Eligible Patients Not to s
Slide 29: Magenta Expansion Will Enable More Patients to be Transplanted
Magenta Expansion Will Enable More Patients to be Transplanted
Slide 30: MGTA-456 Enables Better Matched Transplants, Larger Cell Doses
MGTA-456 Enables Better Matched Transplants, Larger Cell Doses
Slide 31: MGTA-456 Enables Better Matched Transplants, Larger Cell Doses
MGTA-456 Enables Better Matched Transplants, Larger Cell Doses 36 Patients Treated in Phase I/II Hem/Onc Study 5 Patients Treated in Phase II IMD Study 100% of patients engrafted Current practice Expanded cords: MGTA-456 Percent of patients with better matched units Source: CIBMTR report on inventor
Slide 32: Rapid Resolution of Brain Inflammation in Patients with cALD
Rapid Resolution of Brain Inflammation in Patients with cALD Contrast enhancement in brain MRI images from cALD patients at screening and at day +28 post-transplant showing resolution. Red arrows indicate areas of inflammation on screening and resolution of contrast-enhancement by day +28. cALD Pati
Slide 33: Next Steps for MGTA-456
Next Steps for MGTA-456 Continue enrolling patients with leukodystrophies in Phase 2 study 2020 2019 IMDs SCD Initiate Phase 2 study (1H19) Acute Leukemia and MDS Phase 2 ongoing 36 patients treated to date Endpoints: Engraftment Time to immune recovery Days in hospital Biomarkers Disease-specific e
Slide 34: Concluding Remarks
(S) magenta THERAPEUTICS Concluding Remarks © magenta
Slide 35: Key Takeaways Today
Key Takeaways Today Potential patient impact Next steps for each program Near-term milestones and long-term path
Slide 36: 2019-2020: Focused Execution on Value-Driving Programs
2019-2020: Focused Execution on Value-Driving Programs C200 IND-enabling data C200 gene therapy preclinical data C100 development candidate C100 autoimmune preclinical data MGTA-145 Phase 1 data MGTA-456 Phase 2 data C200 IND filed C100 IND-enabling data MGTA-145 Phase 2 data in MM and NHL Additiona
Slide 37: Bone Marrow Transplant: The Patient Journey to a Cure
Bone Marrow Transplant: The Patient Journey to a Cure CONDITIONING Antibody Drug Conjugates EXPANSION Small Molecule Ex Vivo Expansion MOBILIZATION Biologic Combo Drugs Post-Transplant Complications Novel Therapeutics EXPANSION Small Molecule Ex Vivo Expansion CAR T Therapy
Slide 38: Magenta Therapeutics: Transforming the Field of One-Time Curative Therapies
Magenta Therapeutics: Transforming the Field of One-Time Curative Therapies 65,000 Transplants Malignant Non-malignant 100% Transplanted (>150,000) Future Eligible Patients Today’s >150,000 Eligible Patients Future Eligible Patients Today’s >150,000 Eligible Patients Not to scale Today’s >150,000 El
Slide 39: Magenta Vision: Integrated Company for Total Patient Care and Cures
Magenta Vision: Integrated Company for Total Patient Care and Cures Third Horizon Total patient care Outpatient setting for all transplants Second Horizon Multiple first-in-class products across programs First Horizon First global product launched
Slide 40: The Promise of a One-Time Curative Therapy
The Promise of a One-Time Curative Therapy
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