Umoja Biopharma Presents New Preclinical Data at ASGCT 2022 Annual Meeting on its VivoVec Platform Describing Preclinical Safety Data for UB-VV100 Product Candidate, VivoVec Engineering for Improved in vivo CAR T cell Generation and Development of a Scalable Manufacturing Platform

UB-VV100 preclinical safety data demonstrates overall low risk for off target transduction and has a favorable safety and biodistribution profile

Next generation VivoVec surface engineering promotes “immune synapse”-formation for improved in vivo CAR T cell generation

Manufacturing process development demonstrates steps for scalable manufacturing of cGMP-grade lentiviral vector drug product

SEATTLE, May 19, 2022 (GLOBE NEWSWIRE) — Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo for patients with solid and hematologic malignancies, announced data from three presentations at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting on its scalable, off-the-shelf in vivo lentiviral vector-based platform, termed VivoVec, that has the potential to achieve efficient T cell transduction upon direct administration to patients.

“At this year’s ASGCT conference we’re excited to share for the very first time preclinical data showing our efforts to engineer lentiviral surface particles to structurally mimic the immune synapse. The immune synapse is the space formed between antigen presenting cells and T cells during T cell activation and we show that optimization of this synapse can enhance the efficiency of our in vivo CAR T cell platform, VivoVec, to create therapeutic T cells,” said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja. “Our research team is also sharing updates on improvements made to our proprietary manufacturing process for VivoVec particles that supports scalable cGMP production for clinical trials. Altogether these studies continue to provide additional evidence that our multi-component in vivo CAR T cell therapeutics platform, which also includes universal TumorTag small molecules and the RACR/CAR for natural expansion and proliferation of therapeutic CAR T cells, has great potential to overcome critical obstacles facing the cell therapy industry.”

Umoja’s one oral and two poster presentations demonstrate progress in UB-VV100 IND-enabling studies, manufacturing platform development, and next generation VivoVec surface engineering. Preclinical data provides early evidence of the anti-tumor activity and preliminary preclinical safety of UB-VV100, Umoja’s preclinical candidate for the treatment of B cell malignancies. Preclinical safety studies completed to-date demonstrate the overall low risk for off target transduction after in vivo administration of UB-VV100 in two animal models, including a model of intranodal delivery. Additional development of next generation VivoVec particle surface engineering with costimulatory ligands to promote T cell binding, activation, and transduction by replicating immune synapse formation will enable efficient in vivo CAR T cell generation and subsequent anti-tumor immune activity. Umoja further describes a scalable, suspension cell culture-based manufacturing process capable of producing cGMP-grade lentiviral vector product for in vivo CAR T cell therapy. 

Presentation highlights:

Abstract #: 1242
Title: Preclinical Activity and Safety of UB-VV100, A Novel Lentiviral Vector Product Designed for Selective and Effective In Vivo Engineering of Therapeutic Anti-CD19 CAR T Cells for B cell Malignancies
Presenter: Alissa Brandes, Ph.D., Principal Scientist, Umoja Biopharma
Key Highlights:

• Presentation describes the preclinical activity and safety of UB-VV100, a VivoVec particle containing a payload encoding an anti-CD19 4-1BBz CAR and a novel synthetic receptor, rapamycin-activated cytokine receptor (RACR).
  • Administration of UB-VV100 in a humanized mouse model of B cell malignancies results in the activation and transduction of T cells resulting in CAR expression, RACR enhanced CAR T cell expansion, and anti-tumor activity
  • UB-VV100 preliminary toxicology studies demonstrate a favorable safety and biodistribution profile in two preclinical animal models: intranodal administration to canines and systemic administration to humanized mice
  • Intranodal administration in canines, the proposed clinical route of administration, resulted in transduction that was largely restricted to the injected lymph nodes and no quantifiable transduction of non-immune tissues

Abstract #: 879
Title: A Lentiviral-Based In Vivo CAR T Cell Generation Platform with Viral Particle Surface Engineering Incorporating Anti-CD3 Single Chain Variable Fragment and T Cell Costimulatory Molecules
Presenter: Christopher Nicolai, Ph.D., Senior Scientist, Umoja Biopharma
Key Highlights:

• Investigation of multiple novel surface particle engineering approaches for VivoVec lentiviral vectors to replicate immune synapse formation, resulting in enhanced particle-T cell binding, T cell activation, transduction, and transduced T cell quality.
  • VivoVec particles pseudotyped with the Cocal fusion glycoprotein and engineered to express T cell costimulatory ligands exhibited enhanced activation and transduction efficiency
  • CAR T cells generated with next generation VivoVec particles demonstrate enhanced anti-tumor activity and persistence in preclinical models of hematologic malignancy

Abstract #: 1166
Title: Development of a Scalable, Suspension Cell Culture-Based Manufacturing Process for VivoVec, a Lentiviral Vector Platform for In Vivo CAR-T Cell Generation
Presenter: Jeff Plomer, Ph.D., Senior Director Process Development, Umoja Biopharma
Key Highlights:

• Poster describes the development of a scalable, suspension cell culture-based manufacturing process capable of producing lentiviral vector product with quality characteristics suitable for direct injection
  • The VivoVec platform is built on 3^rd generation lentiviral vector gene delivery technology
  • Establishes a Quality Target Product Profile (QTPP) providing lentiviral vector product design that forms the basis for development
  • Utilizes well established biopharmaceutical manufacturing methods to control lentiviral vector production and reduce process-related impurities
  • Results will be used to identify critical process parameters and establish the operating ranges for the clinical and commercial manufacturing control strategy

Presentations can be accessed from the ASGCT website at https://annualmeeting.asgct.org/.

About Umoja Biopharma
Umoja Biopharma, Inc., is developing treatments for solid tumors and hematologic cancers that reprogram the patient’s immune system in vivo. Our technology platforms are designed to work synergistically as part of a therapeutic regimen that can be delivered to any patient, with any tumor, at any time. Based on pioneering work performed at Seattle Children’s Research Institute and Purdue University, Umoja’s approach is powered by novel cellular immunotherapy technologies including the VivoVec in vivo delivery platform, the RACR/CAR in vivo cell expansion/control platform, and the TumorTag targeting platform. Umoja believes its approach can broaden access to advanced immunotherapies to give more patients the hope of lasting remission. To learn more, visit http://www.umoja-biopharma.com.

Cautionary Note Regarding Forward-Looking Statements 
This press release contains forward-looking statements about Umoja Biopharma, Inc. (the “Company,” “we,” “us,” or “our”). The Company has based these forward-looking statements largely on its current expectations, estimates, forecasts and projections about future events and financial trends that it believes may affect its financial condition, results of operations, business strategy and financial needs. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. These statements are subject to risks and uncertainties that could cause the actual results to vary materially, including, among others, the risks inherent in drug development such as those associated with the initiation, cost, timing, progress and results of the Company’s current and future research and development programs, preclinical and clinical trials, as well as the economic, market and social disruptions due to the ongoing COVID-19 public health crisis. Except as required by law, the Company undertakes no obligation to update publicly any forward-looking statements for any reason. 

Media Contact:
Darren Opland, Ph.D.
LifeSci Communications
darren@lifescicomms.com