Corporate Presentation
March 2024
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 harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, any statements relating to our growth strategy, products and product development programs , including the timing of and our ability to make regulatory filings such as INDs and other applications and to obtain regulatory approvals for our product candidates, statements concerning the potential of therapies and product candidates, statements regarding the potential addressable market of our therapies and any other statements that are not descriptions of fact. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include , among other things, risks related to whether the Company's third-party manufacturer is able to successfully perform its obligation to produce the Company's products under the manufacturing services agreement on a timely basis and to acceptable standards, disruption from the sale of the Company's manufacturing facility making it more difficult to maintain business and operational relationships, negative effects of the announcement of the consummation of the sale of the Company's manufacturing facility on the market price of the Company's common stock, significant transaction costs, the development stage of the Company's primary product candidates, our ability to obtain, perform under, and maintain financing and strategic agreements and relationships, risks relating to the results of research and development activities, risks relating to the timing of starting and completing clinical trials, uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers, our ability to attract, integrate and retain key personnel, the early stage of products under development, our need for substantial additional funds, government regulation, patent and intellectual property matters, competition, as well as other risks described in Part I, Item 1A, "Risk Factors," in our Annual Report on Form 10-K filed on March 30, 2023, subsequent Reports on Form 10-Q, and our other filings we make with the SEC. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this presentation should be read as applying mutatis mutandis to every other instance of such information appearing herein, as required by law.
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Certain information contained in this presentation relates to or is based on studies, publications, surveys and other data obtained by third-party sources and the Company's own internal estimates and research. While the Company believes these third-party sources to be reliable as of the date of this presentation, it has not independently verified and makes not representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third- party sources.
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Mustang Bio is Pioneering Innovative CAR-T Therapies for Cancer and Gene Therapies for Primary Immunodeficiencies
Milestones
over next 12
months
- Expect additional disclosure of data from MB-106 indolent lymphoma Arm of Mustang-sponsored trial, with start of first pivotal trial in Waldenstrom macroglobulinemia mid-2024
- Expect to treat 1st pt on groundbreaking combination Ph 1 trial of CAR-T + oncolytic virus in malignant brain tumors
- Expect to publish proof-of-concept data in a murine cancer model for in vivo CAR-T program
Key Programs
▪ First-in-classCD20-targetedCAR-T for non-Hodgkin lymphoma & chronic lymphocytic leukemia | |
MB-106 | - First multicenter Mustang data presented at ASH in December 2023 provide early confirmation of Phase 1 Fred |
Hutch trial across multiple institutions & multiple indolent histologies | |
- Expect to treat 1st pt in pivotal Ph 2 trial for Waldenstrom macroglobulinemia mid-2024;top-line data mid-2026 | |
MB-101 + | ▪ First-in-class combination of CAR-T therapy + an oncolytic virus for malignant brain tumors |
MB-108 | ▪ FDA granted safe-to-proceed for phase 1 trial October 2023; expect to treat first patient in 2024 |
In vivo | ▪ Novel platform to make CAR-Ts in the patient's own body - i.e., not in a manufacturing facility |
CAR-T | ▪ Expect to publish first proof-of-concept data in 2024 |
MB-117 / | ▪ Transformational gene therapies for XSCID* (rare genetic disease) |
MB-217 | ▪ Phase 1 investigator IND trials with modified lentiviral vector expected to start in 2024 |
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* XSCID = X-linked severe combined immunodeficiency
Robust Pipeline of Therapies Addressing Highly Challenging Diseases
Therapeutic | Product (Target) | Pre-IND | Phase 1 | Registrational | |
Modality | Phase 2 | ||||
Hematologic CAR-T | MB-106 for NHL (including WM) & CLL (CD20) | Expect to start pivotal Waldenstrom | |||
macroglobulinemia (WM) trial in 2024 | |||||
Combination CAR-T | MB-101CAR-T for GBM | COH first-in-human (FIH) MB-101 Phase 1 complete | |||
(IL13Rα2) | Combination | Expect to treat first | |||
+ | |||||
= MB-109 | patient in 2024 | ||||
oncolytic virus (OV) | |||||
MB-108 OV for GBM | |||||
UAB FIH MB-108 Phase 1 ongoing* | |||||
In vivo CAR-Ts | TBD | Publication expected | |||
2024 | |||||
Ex-vivoGene | MB-117 (modified vector) for newborn XSCID | ||||
(IL2RG) | Investigator IND trials | ||||
Therapy | |||||
MB-217 (modified vector) for previously | expected to start 2024 | ||||
transplanted XSCID (IL2RG) | |||||
CLL = Chronic lymphocytic leukemia | OV = Oncolytic virus | |
COH = City of Hope National Medical Center | UAB = University of Alabama at Birmingham | |
FIH = First-in-human | WM = Waldenstrom macroglobulinemia | |
GBM = Glioblastoma | XSCID = X-linked severe combined immunodeficiency | |
NIH = National Institutes of Health | * Partially or totally supported by grants` | |
4 | NHL = Non-Hodgkin lymphoma |
Leadership Team with Extensive Cell, Gene & Rare Disease Therapy Experience
Lynn E. Bayless, MS
VP & Head,
Regulatory Affairs
Greg Furrow, MS,
FRQA
Chief Quality Officer
Kerry Biron, BBA
VP & Head,
Clinical Operations
Manuel Litchman, MD
President & Chief Executive Officer
Richard Bodmer, MS | Bruce Dezube, MD |
Head of CMC | Chief Medical Officer |
Development |
Debra Manning, SPHR | James Murphy, BA |
Senior VP & Head, | Ad interim Chief |
Human Resources | Financial Officer |
James Edinger, PhD
Chief Scientific
Officer
Robert Sexton, MS,
MBA
VP & Head, Program &
Alliance Leadership
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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 Brown | Dr. Brian Till | Dr. Kevin Cassady | Dr. Brian Sorrentino | Dr. Larry Pease |
City of Hope | City of Hope | FHCC | Nationwide | St. Jude (1958-2018) | Mayo Clinic |
MB-101 | MB-106 | MB-108 | MB-117,MB-217 | In vivo CAR-Ts |
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We Engineer Patients' Cells with Goal of Long-Term Benefit
Editing T cells harnesses the immune system for precise targeting of the patient's tumor
Editing blood stem cells restores the patient's immune system crippled by genetic defect
- Infuse cells back into patient
5. Cyclophosphamide & fludarabine
- Cryopreservation
CAR-T therapy
(MB-101,MB-106)
1. Leukapheresis & T cell enrichment
2. Engineer T cells to express CAR
1. Bone marrow harvest or leukapheresis
Gene therapy
(MB-117,MB-217,MB-110*)
Stem | Stem | ||
Stem | cell Stem | cell | |
cell | |||
cell | |||
Stem | |||
Stem | cell | ||
cell | |||
2. Engineer stem cells to | |||
express normal gene |
3. Expand the modified T cells
3. Cryopreservation
5. Infuse cells back into patient
7 Source: Duan Q, et al. Trends in Cancer. 2020;6:605-618
4. Low-dose busulfan
*MB-110 = RAG1-SCID gene therapy in-licensed from Leiden Univ. Medical Centre
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 clinical trials | |
▪ | Comprehensive cell processing capabilities, including | |
sterility testing of final product |
Office | Labs | ▪ Lentiviral vectors to carry genetic payload are | |
(Quality Control, Process Devt, | produced at academic partners & CDMOs | ||
Analytical Devt & Research) | ▪ | Successful manufacturing of MB-106 product for all | |
pts enrolled to date on Mustang-sponsored trial |
Mustang has developed a universal platform process for autologous CAR-Ts
Day -1 | Day 0 Day 2 Day 3 | Day 7 | Day 15+ Day 19+ |
8-day sterility |
Leukapheresis
Enrichment | Transduction | Expansion | Fill | Shipment |
Infusion
CoA
Shipment | Activation | Wash | Harvest | Cryopreservation | Thawing |
8 | * Details of this Agreement may be found in our SEC filings |
MB-106 Overview
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MB-106:CAR-T for Non-Hodgkin Lymphoma (NHL) & Chronic Lymphocytic Leukemia (CLL)
MB-106
MB-106
First-in-classCD20-targetedCAR-T cell therapy for treatment of relapsed/refractory NHL & CLL
- Competitive safety & efficacy profile vis-à-vis approved & investigational cell therapies & bispecific antibodies
- Clear regulatory pathway for autologous CAR-Ts based on FDA approval to date of 6 autologous CAR-Ts
- Currently enrolling multicenter Phase 1 Mustang- sponsored trial
- BLA strategy is to pursue indications where there are no approved CAR-Ts:
- Waldenstrom macroglobulinemia (WM)
- DLBCL relapsed from CD19 CAR-Ts
- CLL
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Clinical development: 2 trials in progress
- Ongoing Fred Hutch IND study: Plan to continue enrollment; expect publication of follicular lymphoma data in 2024
- Mustang IND 6-center Phase 1/2 trial: Currently enrolling*
- 3-armstudy targeting relapsed/refractory disease
- Aggressive NHL
- Indolent NHL
- CLL
- Relapses from CD19 CAR-Ts eligible in all arms
- Indolent NHL arm includes Waldenstrom (fast-to-marketstrategy)
- Since Mustang IND trial is using same lentiviral vector as Fred Hutch trial, FDA has allowed dose escalation to start higher than initial FHCC dose
- Additional data disclosures from indolent NHL arm expected in 2024; expect to start pivotal WM Ph 2 trial mid-2024
- https://clinicaltrials.gov/ct2/show/NCT05360238
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Mustang Bio Inc. published this content on 01 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 01 March 2024 22:33:00 UTC.