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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 first pivotal trial in Waldenstrom macroglobulinemia in second half of 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-class CD20-targeted CAR-T for non-Hodgkin lymphoma & chronic lymphocytic leukemia - 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 2H 2024; top-line data 2H 2026

  • First-in-class combination of CAR-T therapy + an oncolytic virus for malignant brain tumors

  • FDA granted safe-to-proceed for phase 1 trial October 2023; expect to treat first patient in 2024

  • Novel platform to make CAR-Ts in the patient's own body - i.e., not in a manufacturing facility

  • Expect to publish first proof-of-concept data in 2024

  • Transformational gene therapies for XSCID* (rare genetic disease)

  • Phase 1 investigator IND trials with modified lentiviral vector expected to start in 2024

* XSCID = X-linked severe combined immunodeficiency

Robust Pipeline of Therapies Addressing Highly Challenging Diseases

Therapeutic

Modality

Product (Target)

Pre-IND

Phase 1

Registrational

Phase 2

Hematologic CAR-T

MB-106 for NHL (including WM) & CLL (CD20)

Expect to start pivo macroglobulinemia

tal Waldenstrom (WM) trial in 2024

Combination CAR-T + oncolytic virus (OV)

MB-101 CAR-T for GBM

(IL13Rα2)

Combination

COH first-in-human (FIH) MB-

Expect to treat first

101 Phase 1 complete

= MB-109

MB-108 OV for GBM

patient in 2024

UAB FIH MB-108 Phase 1 ong

oing*

In vivo CAR-Ts

TBD

Publication expected 2024

Ex-vivo Gene

Therapy

MB-117 (modified vector) for newborn XSCID

(IL2RG)

Investigator IND trials

MB-217 (modified vector) for previously transplanted XSCID (IL2RG)

expected to start 2024

CLL = Chronic lymphocytic leukemia

COH = City of Hope National Medical Center FIH = First-in-human

GBM = Glioblastoma

NIH = National Institutes of Health NHL = Non-Hodgkin lymphomaOV = Oncolytic virus

UAB = University of Alabama at Birmingham WM = Waldenstrom macroglobulinemia

XSCID = X-linked severe combined immunodeficiency

* Partially or totally supported by grants`

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

City of Hope

Dr. Christine Brown

City of Hope

Dr. Brian Till

FHCC

Dr. Kevin Cassady

Nationwide

Dr. Brian Sorrentino St. Jude (1958-2018)

Dr. Larry Pease

Mayo Clinic

MB-106

MB-108

MB-117, MB-217

In vivo CAR-Ts

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

Gene therapy

(MB-117, MB-217, MB-110*)

1. Bone marrow harvest or leukapheresis

5. Infuse cells back into patient

Stem cellStem cell

Stem Stem cell cell

Stem cell

4. Low-dose busulfan

Stem cell

2. Engineer stem cells to express normal gene

3. Cryopreservation

*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 sitecurrently owned by Mustang - will retain Mustang personnel & support our clinical trials

  • Comprehensive cell processing capabilities, including sterility testing of final product

Lentiviral vectors to carry genetic payload are produced at academic partners & CDMOs

  • 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+

8-day sterility

Day 19+

MB-106 Overview

MB-106: CAR-T for Non-Hodgkin Lymphoma (NHL) & Chronic Lymphocytic Leukemia (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:

    • (1) Waldenstrom macroglobulinemia (WM)

    • (2) DLBCL relapsed from CD19 CAR-Ts

    • (3) CLL

10

Clinical development: 2 trials in progress

1.

Ongoing Fred Hutch IND study: Plan to

continue enrollment; expect publication of follicular lymphoma data in 2024

2. Mustang IND 6-center Phase 1/2 trial: Currently enrolling*

  • 3-arm study 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-market strategy)

  • 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 in the second half of 2024

*https://clinicaltrials.gov/ct2/show/NCT05360238

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Mustang Bio Inc. published this content on 01 April 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 01 April 2024 13:26:07 UTC.