Umoja Biopharma Announces Activation of First ENLIGHTen Phase 1 Trial Site in Study of UB-TT170, a TumorTag that Targets Folate Receptors to Mark Tumors for Clearance by CAR T Cells

The Phase 1 clinical trial will evaluate UB-TT170 in osteosarcoma and follows the recent approval of Umoja’s first IND application by the FDA

SEATTLE, May 3, 2022 – 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 today the Seattle Children’s activation of the Phase 1 ENLIGHTen clinical trial in patients with osteosarcoma to assess the safety and tolerability of autologous “universal” CAR T cells when administered with UB-TT170, the Company’s proprietary small molecule fluorescein tag. This follows the recent clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for UB-TT170. The trial is being conducted in partnership with Seattle Children’s Therapeutics, a venture at Seattle Children’s, bringing cutting edge, curative technologies and therapies to defeat pediatric cancer and other diseases that impact children.

“We are excited to receive IND clearance of our first TumorTag product candidate UB-TT170,” said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja Biopharma. “The ability to target solid tumors with CAR T technology remains a large barrier for patients with cancer. Our TumorTag technology is intended to overcome treatment limitations due to antigen heterogeneity of tumors and the restrictive solid tumor microenvironment with the goal to provide better treatment outcomes for patients. This marks a great start for Umoja’s three integrated core programs, the TumorTag, VivoVec, and RACR/TagCAR platforms, to meaningfully advance the cancer immunotherapy field.”

The ENLIGHTen trial (NCT05312411) is an open-label Phase 1 feasibility and safety study of fluorescein-specific CAR T cells in combination with folate-fluorescein (UB-TT170) for patients with refractory/recurrent osteogenic sarcoma. All patients in the trial will be administered autologous CAR T cells engineered to express an anti-fluorescein chimeric antigen receptor that were developed by Seattle Children’s Therapeutics and will be manufactured in their Cure Factory Good Manufacturing Practices facility. Following that, each patient will receive escalating doses of Umoja’s UB-TT170 TumorTag molecules, followed by fixed dosing. The primary endpoint is safety (adverse events tracking) with secondary endpoints focused on the ability to manufacture anti-fluorescein CAR T cells. The trial will only be open at Seattle Children’s and the age range for enrollment is people aged 15-30 years old.

Umoja’s unique TumorTag technology consists of a bispecific small molecule, which can be either antigen-specific or -independent, that selectively binds to tumor cells or immunosuppressive tumor stromal cells and labels their cell surface with fluorescein. Upon labelling, tumor and local stromal cells become marked for recognition and destruction by CAR T cells engineered to bind to fluorescein. The UB-TT170 TumorTag targets folate receptors (FR) with an initial indication in osteosarcoma (OS) where up to 80% of OS tumor samples show FR expression1. The prognosis for patients with advanced metastatic OS is poor with a 5-year survival rate of 27%2.

“Seattle Children’s is dedicated to pursuing the advancement of next-generation therapies,” said Dr. Julie Park, a pediatric oncologist at Seattle Children’s who heads clinical and translational research at Seattle Children’s Therapeutics. “The launch of the ENLIGHTen trial underscores our commitment to innovation in the fight against pediatric cancer.”

1 Yang R, et al. The folate receptor alpha is frequently overexpressed in osteosarcoma samples and plays a role in the uptake of the physiologic substrate 5-methyltetrahydrofolate. Clin Cancer Res. 2007 May 1;13(9):2557-67. doi: 10.1158/1078-0432.CCR-06-1343. PMID: 17473184.

2 Bone Cancer (Sarcoma of Bone): Statistics. https://www.cancer.net/cancer-types/bone-cancer-sarcoma-bone/statistics (accessed March 24, 2022).

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. 

About Seattle Children’s
Seattle Children’s mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Children’s Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research and raise funds to create better futures for patients. Ranked as one of the top children’s hospitals in the country by U.S. News & World Report, Seattle Children’s serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho – the largest region of any children’s hospital in the country. As one of the nation’s top five pediatric research centers, Seattle Children’s Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Children’s Foundation works with the Seattle Children’s Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research. For more information about Seattle Children’s, visit seattlechildrens.org.

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. 

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LifeSci Communications
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