Onward Therapeutics' Emercell Gains Approval to Begin Clinical Trial of OT-C001 with Rituximab

EPALINGES, Switzerland and PARIS, April 22, 2025 — Onward Therapeutics SA, a biotechnology company focused on pioneering cancer immunotherapies, has announced a significant development in its subsidiary, Emercell SAS. The company has received approval from the European Medicines Agency (EMA) for the Investigational Medicinal Product Dossier (IMPD) to commence a Phase 1 clinical trial of OT-C001. This therapy is an allogeneic natural killer (NK) cell treatment, intended to be used in conjunction with Rituximab, an anti-CD20 monoclonal antibody.

The forthcoming Phase 1 trial, referred to as the C00101 study, is set to enroll patients suffering from diffuse large B-cell lymphoma (DLBCL). These patients have either relapsed after receiving CAR-T cell therapy, are unresponsive to it, or are not suitable candidates for CAR-T cell infusion. The study aims to assess the effectiveness of OT-C001 when combined with Rituximab across different dosage levels. Furthermore, it will investigate cellular kinetics and biomarkers to better comprehend the mechanisms that contribute to the therapeutic responses observed.

Dr. C. Grace Yeh, Chairman and CEO of Onward Therapeutics, expressed her excitement about the progress, noting the milestone of moving OT-C001 into clinical development following strategic investments in Emercell since 2021. The Phase 1 trial has the potential to validate Emercell's patented platform technology, which could lead to significant clinical outcomes. This move, along with the ongoing optimization of industrial manufacturing processes, is expected to bolster Onward Therapeutics’ ability to establish strategic partnerships and advance this promising therapy.

Dr. Patrick Henno, Founder and former President of Emercell, highlighted the uniqueness of OT-C001. The therapy is distinguished by its use of multiple umbilical cord blood (UCB) samples, providing product diversity and consistency in GMP batches. Unlike the complex manufacturing processes of many engineered cell therapy products, OT-C001 offers a safe and scalable solution to unmet medical needs in targeted indications.

Dr. Alain Herrera, President of Emercell and Chief Medical Officer of Onward Therapeutics, shared his enthusiasm about the launch of this multicenter Phase 1 trial for one of the most prevalent and aggressive forms of non-Hodgkin lymphoma. Despite the challenges in the cell therapy market, this progress aligns with Onward Therapeutics’ mission to deliver novel treatments for DLBCL patients who require new therapeutic options. OT-C001 is versatile enough to serve as a universal platform, potentially benefiting patients across various indications beyond DLBCL when used in combination with different monoclonal antibodies or targeted therapies.

OT-C001 is an allogeneic cell therapy that utilizes highly activated and expanded NK cells derived from UCB. It is manufactured through a patented process of expansion and activation and targets tumor cells through both direct and indirect cytotoxicity when paired with a monoclonal antibody targeting tumor-specific antigens. The therapy is under evaluation as a potential new treatment for advanced hematologic malignancies alongside monoclonal antibodies. In non-clinical in vitro, in vivo models, and toxicology studies, OT-C001 has shown to be well-tolerated and has demonstrated promising anti-tumor activities.

Onward Therapeutics, a clinical-stage biotechnology company, is dedicated to developing innovative cancer therapies. With a team experienced in translational science and drug development, the company advances promising candidates through a fast-track development model. Its portfolio includes a bispecific antibody (OT-A201) licensed from Biomunex and an allogeneic NK cell therapy (OT-C001) developed through its majority-owned subsidiary, Emercell. Additional programs in immunotherapy, targeted therapy, and onco-metabolism are in early development. The company is headquartered in Switzerland with operations in Paris and Taipei.

Emercell specializes in developing off-the-shelf allogeneic NK cell therapies. Based at the IRMB at Saint-Éloi University Hospital Center in Montpellier, France, its leading candidate, OT-C001, targets lymphoma patients resistant to standard treatments. The company's patented platform pre-activates and expands NK cells. In 2023, Emercell received support from Bpifrance as part of the France 2030 "Biotherapies and bioproduction of innovative therapies" acceleration strategy and the Occitanie Region.