Wugen announced Wednesday that it secured $115 million in equity financing to advance its pivotal clinical trial of WU-CART-007, an off-the-shelf CAR-T therapy that could become the first approved allogeneic treatment for T-cell malignancies. The financing was led by Fidelity Management & Research Company, with participation from RiverVest Venture Partners, Lightchain Capital, Abingworth, and other leading life sciences investors.
The St. Louis-based biotechnology company plans to use the proceeds to fund its ongoing pivotal T-RRex trial evaluating WU-CART-007 in patients with relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL). Wugen aims to file a Biologics License Application (BLA) with the FDA in 2027.
Breakthrough Efficacy in Heavily Pretreated Patients
WU-CART-007, also known as soficabtagene geleucel, demonstrated remarkable clinical activity in a completed global Phase 1/2 study. The therapy achieved an overall response rate of 91% and a composite complete remission rate of 73% at the recommended Phase 2 dose in patients who had already tried multiple other therapies. The median duration of response exceeded six months with manageable safety profiles.
"WU-CART-007's robust response in a heavily pretreated patient population—coupled with manageable safety and scalable manufacturing—positions it as a potential first-in-class therapy," said Cherry Thomas, M.D., chief medical officer of Wugen. These results, presented at the 2024 American Society of Hematology Annual Meeting, substantially surpass outcomes achieved with current standard-of-care therapies.
Revolutionary Off-the-Shelf Approach
Unlike conventional CAR-T treatments that require extraction and manipulation of a patient's own cells in a lengthy laboratory process, WU-CART-007 uses donor-derived cells that can be administered to any qualified patient. This off-the-shelf approach addresses key limitations of first-generation cell therapies by providing immediate availability and scalable manufacturing.
The therapy employs CRISPR/Cas9 gene editing technology to delete the CD7 and T cell receptor alpha constant (TRAC) genes, preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host disease. By using healthy donor-derived T cells, WU-CART-007 eliminates the risk of malignant cell contamination historically observed in autologous CAR-T settings.
Addressing Critical Unmet Medical Need
"Relapsed and refractory T-ALL/T-LBL are aggressive malignancies resistant to current treatment options," said Kumar Srinivasan, Ph.D., MBA, president and chief executive officer of Wugen. "We are committed to delivering an accessible, off-the-shelf therapy that can significantly improve the trajectory of patients' care."
The pivotal T-RRex trial is designed to evaluate WU-CART-007 in a single study for both pediatric and adult patients, with the goal of offering a potentially curative option where current salvage therapies fail.
Regulatory Recognition and Market Validation
WU-CART-007 has received multiple regulatory designations recognizing its therapeutic potential, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, as well as Priority Medicines (PRIME) Scheme designation in the European Union. These designations provide increased agency support to expedite development and review.
The significant investor interest reflects growing confidence in the off-the-shelf CAR-T approach. Last year, Roche agreed to pay approximately $1 billion to acquire San Diego-based Poseida Therapeutics, which also specializes in allogeneic cell therapy, demonstrating major pharmaceutical companies' commitment to this technology platform.
"RiverVest has been impressed by the Wugen team's efforts advancing WU-CART-007 into this pivotal study, and we are pleased that several of the world's leading cancer centers are participating," said Niall O'Donnell, Ph.D., Managing Director at RiverVest. "We are optimistic about Wugen's potential to transform care for patients who currently face poor outcomes and limited treatment options."
