Mustang Bio, Inc. · NASDAQ:MBIO
Mustang Bio — Investor Presentation (February 2024)
47 slides from Mustang Bio's investor presentation, February 2024
Slide 1: Corporate Presentation
Corporate Presentation February 2024
Slide 2: Cautionary Statement
Cautionary Statement This presentation contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. For such forward-looking statements, we claim the protection of the safe harbo
Slide 3: Mustang Bio is Pioneering Innovative CAR-T Therapies for Cancer and Gene Therapies
Mustang Bio is Pioneering Innovative CAR-T Therapies for Cancer and Gene Therapies for Primary Immunodeficiencies ▪ Expect additional disclosure of data from MB-106 indolent lymphoma Arm of Mustang-sponsored trial, with start of Milestones first pivotal trial in Waldenstrom macroglobulinemia mid-202
Slide 4: Robust Pipeline of Therapies Addressing Highly Challenging Diseases
Robust Pipeline of Therapies Addressing Highly Challenging Diseases Therapeutic Registrational Product (Target) Pre-IND Phase 1 Modality Phase 2 Expect to start pivotal Waldenstrom Hematologic CAR-T MB-106 for NHL (including WM) & CLL (CD20) macroglobulinemia (WM) trial in 2024 MB-101 CAR-T for GBM
Slide 5: Leadership Team with Extensive Cell, Gene & Rare Disease Therapy Experience
Leadership Team with Extensive Cell, Gene & Rare Disease Therapy Experience Lynn E. Bayless, MS Kerry Biron, BBA Richard Bodmer, MS Bruce Dezube, MD James Edinger, PhD VP & Head, VP & Head, Head of CMC Chief Medical Officer Senior VP & Head, Regulatory Affairs Clinical Operations Development Preclin
Slide 6: R&D Collaborators: World Class Team of Scientific Experts
R&D Collaborators: World Class Team of Scientific Experts ▪ Technology licensed from City of Hope (COH), Fred Hutch Cancer Center (FHCC), Nationwide Children’s Hospital, St. Jude Children’s Research Hospital, & Mayo Clinic ▪ Research based on pioneering work by: Dr. Stephen Forman Dr. Christine Brow
Slide 7: We Engineer Patients' Cells with Goal of Long-Term Benefit
We Engineer Patients’ Cells with Goal of Long-Term Benefit Editing T cells harnesses the immune system Editing blood stem cells restores the patient’s for precise targeting of the patient’s tumor immune system crippled by genetic defect CAR-T therapy Gene therapy (MB-101, MB-106) (MB-117, MB-217, MB
Slide 8: Asset Purchase Agreement Closed July 28, 2023, with uBriGene
Asset Purchase Agreement Closed July 28, 2023, with uBriGene – US Division of Highly Experienced Chinese CDMO*; Cell Processing Has Continued Seamlessly ▪ 27,000 square foot potential commercial launch site currently owned by Mustang – will retain Mustang Cleanroom space personnel & support our clin
Slide 9: MB-106 Overview
MB-106 Overview 9
Slide 10: MB-106: CAR-T for Non-Hodgkin Lymphoma (NHL) & Chronic Lymphocytic Leukemia (CLL)
MB-106: CAR-T for Non-Hodgkin Lymphoma (NHL) MB-106 & Chronic Lymphocytic Leukemia (CLL) Clinical development: 2 trials in progress MB-106 1. Ongoing Fred Hutch IND study: Plan to First-in-class CD20-targeted CAR-T cell therapy continue enrollment; expect publication for treatment of relapsed/refrac
Slide 11: MB-106: Potent Cell Killing; Limited Cell Therapy Competition for Target
MB-106: Potent Cell Killing; Limited Cell Therapy Competition for Target ▪ While approved CAR-Ts for NHL target CD19, CD20 has been validated as a clinically relevant target by decades of commercial experience with CD20 antibodies1 CD20-targeting CAR molecule on surface of Binding of CAR to CD20 cau
Slide 12: MB-106: Dose Escalation Complete in Fred Hutch Trial
MB-106: Dose Escalation Complete in Fred Hutch Trial 6-Center Mustang-sponsored Phase 1 trial also now enrolling all 3 arms ▪ Single institution phase 1/2 study at Fred Hutch Cancer Center (NCT03277729)* ▪ Relapsed/refractory CD20+ B-cell NHL & CLL ▪ Prior to infusion of MB-106, all patients receive
Slide 13: MB-106: Follicular Lymphoma Updated at ICML June 2023 Patient Characteristics
MB-106: Follicular Lymphoma (FL) Updated at ICML* June 2023 Patient Characteristics N = 20 Age, median (range) 63 (44-81) > 65, n(%) 9 (45%) > 80, n(%) 3 (15%) Female sex, n(%) 11 (55%) Stage at initial diagnosis ▪ Median time between leukapheresis and lymphodepletion was 15 days 1-2 2 (11%) (range:
Slide 14: MB-106: FL Efficacy Updated at ICML June 15, 2023
MB-106: FL Efficacy Updated at ICML June 15, 2023 Best Responses Best responses by PET scan N=20 Best Dose Dose Dose Dose Dose Response by All dose PR: 3 Histology level 0 level 1 level 2 level 3 level 4 Lugano PET levels (n=1) (n=3) (n=5) (n=8) (n=3) (15%) criteria † 1 x 105 3.3 x 105 1 x 106 3.3 x
Slide 15: MB-106: FL Durability June 15, 2023 (10 CRs > 1 year)
MB-106: FL Durability June 15, 2023 (10 CRs > 1 year) CR PR Pseudo PD PD Death Prior CD19 Ongoing CAR-T Response (10 CRs ≥ 1 yr) 15 Presented by Mazyar Shadman, MD (FHCC), at the 17th International Conference Time (months) on Malignant Lymphoma (https://www.icml.ch/icml/home.html)
Slide 16: MB-106: FL Safety Data Updated June 15, 2023
MB-106: FL Safety Data Updated June 15, 2023 Cytokine release syndrome (CRS) & Immune effector cell-associated neurotoxicity syndrome (ICANS) G1 G2 G3 G4 All grades CRS* 5 (25%) 1 (5%) 0 0 6 (30%) ICANS* 0 0 0 0 0 ▪ No dexamethasone prophylaxis ▪ Median time to CRS, post CAR-T day (range): 7 (1-8) ▪
Slide 17: MB-106: FL Safety: Other Events (first 28 days)
MB-106: FL Safety Data Updated June 15, 2023 Safety: Other Events (first 28 days) N=20 Grade 3 Grade 4 Grade 3-4 Leukopenia 7 (35%) 9 (45%) 16 (80%) Lymphopenia 4 (20%) 15 (75%) 19 (95%) Neutropenia 4 (20%) 15 (75%) 19 (95%) Thrombocytopenia 1 (5%) 2 (10%) 3 (15%) Anemia 4 (20%) 1 (5%) 5 (25%) Febri
Slide 18: MB-106: FL CAR-T Cell Expansion & Persistence
MB-106: FL CAR-T Cell Expansion & Persistence Expansion by flow cytometry Absolute CAR+ cells by dose level 1000 10000 DL0 CD3+CD19+ cells/l CD3+CD19+ cells/l 100 1000 DL1 100 10 DL2 10 1 DL3 1 DL4 0.1 0.1 0.01 0.01 0.001 0.001 0 10 20 30 0 10 20 30 Days post CAR-T infusion Days post CAR-T infusio
Slide 19: MB-106: Waldenstrom Data Updated at EHA June 9, 2023
MB-106: Waldenstrom Data Updated at EHA June 9, 2023 Patient characteristics (N = 6) Age, median (range) 69 (51-79) Female sex, n (%) 2 (33%) Prior lines of therapy, median (range) 7.5 (2-12) Prior Bruton tyrosine kinase inhibitor 6 (100%) Best response by IWWM-7* CR 2 (33%) Response rate: VGPR 1 (1
Slide 20: MB-106: Durable Responses (10 CRs ≥ 1 year) Across All Histologies
MB-106: Durable Responses (10 CRs ≥ 1 year) Across All Histologies at IWWM-11*, October 2022 (no CRS or ICANS ≥ grade 3) Investigator-Assessed ResponseA CR PR PD Undetectable minimal residual disease to 10-4 sensitivity in (uMRD4) in blood and marrow B uMRD4 in Blood B Prior CD19 CART A Evaluated ac
Slide 21: MB-106 Priorities: Indications Where CAR-Ts Not Yet Approved
MB-106 Priorities: Indications Where CAR-Ts Not Yet Approved Phase 1 End of Ph 1 Phase 2 • 1,400 new US cases per year 2-3 dose levels per arm FDA mtgs Registrational Priority #1: • Orphan Drug Designation granted Waldenstrom (WM) • Expect Fast Track filing Arm 2 Arm 2E • Topline data expected 1H26
Slide 22: MB-106: Mustang Trial Enrolling in All 3 Arms at 6 Centers
MB-106: Mustang Trial Enrolling in All 3 Arms at 6 Centers* Expect 6 centers by year end Study Progress and Milestones Active sites Fred Hutch, Mass General, UC Irvine, Cleveland Clinic, Univ. of Rochester, Memorial Sloan Kettering Dose escalation Safety Review Committee unanimously approved dose es
Slide 23: Arm 2: Baseline Patient Characteristics
Arm 2: Baseline Patient Characteristics 23 Source: Presentation by M. Shadman at ASH 2023: www.mustangbio.com/wp-content/uploads/2023/12/MB-106_ASH-Poster_Final_2023-12-08.pdf
Slide 24: Arm 2 / Indolent Lymphoma: Compelling Responses Seen in Heavily Pre-Treated Population
Arm 2 / Indolent Lymphoma: Compelling Responses Seen in Heavily Pre-Treated Population at DL1 (3.3 x 106 cells/kg) Prior Lines of Site Age/Gender Indication Infusion Status CRS/ICANS Therapy Hairy Cell Ongoing SD at Month 6 with Grade 1 CRS Fred Hutch 79/Male 5 November 2022 Leukemia transfusion ind
Slide 25: Arm 2 – Efficacy [Combined Results for DL1 and DL2]
Arm 2 – Efficacy [Combined Results for DL1 (N=4) and DL2 (N=5)] ▪ A 9th patient (heavily transfusion- Best Responses to Date‡ FL, N=5 WM, N=3 dependent hairy cell leukemia – variant) treated at DL1 achieved transfusion Overall response rate (ORR)† 100% (5/5) 3 (100%) independence & maintained stable
Slide 26: Arm 2 – Safety [Combined Results for DL1 and DL2]
Arm 2 – Safety [Combined Results for DL1 (N=4) and DL2 (N=5)] Grade ≥3 Adverse Events (First 28 Days), CRS & ICANS Regardless of Causality Grade 1 Grade 2 Grade 3 Grade 4 Grade 3 Grade 4 CRS 5 (56%) 0 0 0 Neutrophil count decreased 1 5 ICANS 0 0 0 0 Febrile Neutropenia 0 0 • Numbers in tables are in
Slide 27: All NHL Patients Who Relapsed from Prior CD19 CAR-T Achieved CR with MB-106
All NHL Patients from Both Trials Who Relapsed from Prior CD19 CAR-T Therapy Achieved CR with MB-106 Prior Lines of Indication Age/Gender Dose of MB-106 Status Therapy Diffuse Large B-Cell 1×107 cells/kg 63/Male Unknown Ongoing CR at Month 20+ Lymphoma (FHCC Trial) Mantle Cell 1×106 cells/kg 59/Male
Slide 28: Summary: Multicenter Arm 2 Data Provide Early Confirmation
Summary: Multicenter Arm 2 Data Provide Early Confirmation of Single-Center Indolent Lymphoma Patients ▪ Compelling efficacy in 2 lead subsets of indolent lymphoma: FL & WM – Notable for the FL patients are CRs despite poor prognostic indicators – VGPR** in WM provides additional strong support for
Slide 29: MB-106 Initial Addressable Populations, With Opportunity To Expand
MB-106 Initial Addressable Populations, With Opportunity To Expand To Earlier Lines Of Therapy MB-106 U.S. addressable markets1, 2 Initial Addressable Patients Expanded Addressable Patients Indication (line of therapy)3 (line of therapy)4 ~1,200 (CD19 CAR-T Relapsed 3L+) DLBCL 7,000-8,000 (2L+) no a
Slide 30: Autologous CAR-Ts Experiencing Accelerating Revenue Growth
Autologous CAR-Ts Experiencing Accelerating Revenue Growth1 Reflects increasing acceptance among prescribers, patients & payors 1. The revenue growth depicted below represents aggregate worldwide sales of the drugs identified for the periods indicated. All of such drugs approved to treat B-cell mali
Slide 31: Yescarta Global Sales
Yescarta® Global Sales1 Source: Evaluate Pharma and Gilead 1. Yescarta is a CD-19 directed CAR-T cell therapy for the treatment of (i) adult patients with LBCL that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy, (ii) adult patients
Slide 32: MB-109 Overview
MB-109 Overview (combination of MB-101 + MB-108) 32
Slide 33: MB-101: IL13Rα2 CAR-T for Glioblastoma (GBM)
MB-101: IL13Rα2 CAR-T for Glioblastoma (GBM) MB-101 + MB-108 • More than 13,000 new patients diagnosed in the US annually; 7% 5-year survival • IL13R⍺2 is an attractive target: Overexpressed in >75% of GBM, not on normal brain cells Few CAR-T programs competing for target1 • However, GBM is a notori
Slide 34: MB-108: HSV-1 Oncolytic Virus Turns Cold Glioblastoma Tumors Hot
MB-108: HSV-1 Oncolytic Virus Turns Cold Glioblastoma Tumors Hot ▪ MB-108 was in-licensed from Nationwide Children’s Hospital in 20191 Phase 1 investigator-sponsored trial underway at the University of Alabama at Birmingham2 ▪ Single patient data were disclosed at the 2022 meeting of the American As
Slide 35: If We Could Turn Cold GBM Tumors Hot, Would They Respond Better?
If We Could Turn Cold GBM Tumors Hot, Would They Respond Better? Injecting an oncolytic virus followed by a CAR-T may be best strategy to test hypothesis 4. Inject MB-101 CAR-T cells into 1. Inject MB-108 & around “hot” tumor leading oncolytic virus to complete tumor killing into tumor mass MB-101 C
Slide 36: MB-101: 3 Approaches at COH to Enhancing Efficacy
MB-101: 3 Approaches at COH to Enhancing Efficacy MB-101 IL13Rα2-Directed CAR-T for GBM Monotherapy Patient Selection Strategy Combination Strategy Leptomeningeal disease (CAR-T alone) Combine with Oncolytic Virus (OV) Combine with Checkpoint Inhibitors • Opportunity for activation of CAR-T cells •
Slide 37: In vivo CAR-T Overview
37 In vivo CAR-T Overview se AS MUSTANGBIO|
Slide 38: Overview of in vivo CAR-T Platform Technology
In vivo Overview of in vivo CAR-T Platform Technology CAR-T Clinical development overview In vivo CAR-T • In vivo proof-of-concept established in mouse model of cancer via 2- step process (unpublished) Novel platform to make CAR-Ts in the patient’s own body to treat hematologic • Following further p
Slide 39: XSCID Program Overview
XSCID Program Overview ▪ Modified lentiviral vector will replace vector used to date in MB-107 (newborn XSCID) & MB-207 (previously transplanted XSCID) Phase 1 clinical trials ▪ Nomenclature for new products with modified vector will be MB-117 & MB-217 39
Slide 40: X-Linked Severe Combined Immunodeficiency (XSCID): Rare Genetic Disease
MB-107 / X-Linked Severe Combined Immunodeficiency (XSCID): MB-207 Rare Genetic Disease Due to Mutations in the IL2RG Gene ▪ IL2RG codes for a protein (c) that is critical for development of normal immune cells (T cells, B cells, NK cells) ▪ Early diagnosis & treatment possible in areas with newbor
Slide 41: XSCID & RAG1-SCID Represent a Substantial Addressable Population
XSCID & RAG1-SCID Represent a Substantial Addressable Population With High Unmet Need MB-117 / MB-217 / MB-110 (RAG1-SCID)† U.S. addressable markets* Indication Initial Addressable Patients Expanded Addressable Patients XSCID 20-25 (annual newborn incident patients) ~400^ (pool of previously transpl
Slide 42: MB-107: 100% Response Rate with Excellent Safety Profile in 23 Newborn Patients
MB-107: 100% Response Rate with Excellent Safety Profile MB-107 in 23 Newborn Patients Treated on St. Jude-Sponsored Trial* ▪ Multicenter trial: St. Jude, Seattle Children’s, UCSF Benioff Children’s; median follow-up of 2.6 years ▪ First successful use of targeted low-exposure busulfan + a lentivira
Slide 43: MB-207: Results in Previously Transplanted Patients on NIH-Sponsored Trial
MB-207: Results in Previously Transplanted Patients on NIH- MB-207 Sponsored Trial1 are Consistent with Newborn Data2,3 • Promising safety & efficacy profile, but time to recovery from chronic viral gastrointestinal (GI) infections was slow • To address this challenge, transduction enhancers (TE), i
Slide 44: XSCID Program: Now Pivoting to Modified Lentiviral Vector
XSCID Program: Now Pivoting to Modified Lentiviral Vector ▪ Based on review of the data from the 2 investigator-sponsored clinical trials, the respective investigators have elected to pause enrollment to these trials – The NIH has posted on ClinicalTrials.gov that “Clones representing 10% or more of
Slide 45: Anticipate initiating first pivotal MB-106 trial in WM in mid-2024
Anticipate initiating first pivotal MB-106 trial in WM in mid-2024 ▪ MB-106 (CD20 CAR-T): Continue to enroll patients in Mustang-sponsored multicenter trial − Await enrollment of 6th & final patient to Arm 2 (indolent lymphoma), dose level 2, in 1Q 2024 − Expect End-of-Phase 1 meeting for Arm 2 in 1
Slide 46: Annex – Background and Limitations Regarding Estimated Market Potential for MB-106
Annex – Background and Limitations Regarding Our Estimated Market Potential for MB-106 Background Cancer therapies are sometimes characterized as “first line,” “second line” or “third line” or later therapies, and the FDA often approves new therapies initially only for last line use. When blood canc
Slide 47: Corporate Presentation
Corporate Presentation February 2024