Onward Therapeutics Announces IMPD Approval for Its Subsidiary Emercell to Start Clinical Investigation of Allogeneic NK Cell Therapy OT-C001 in Combination with Rituximab

EPALINGES, Switzerland and PARIS, April 22, 2025 /PRNewswire/ -- Onward Therapeutics SA, a biotechnology company dedicated to advancing innovative cancer immunotherapies, announced that its subsidiary, Emercell SAS, has received Investigational Medicinal Product Dossier (IMPD) approval from the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of OT-C001, an allogeneic natural killer (NK) cell therapy, in combination with an anti-CD20 monoclonal antibody, Rituximab.

The upcoming phase 1 study (C00101 study) will enroll patients with diffuse large B-cell lymphoma (DLBCL), who have relapsed after, or are refractory to CAR-T cell therapy, or ineligible for CAR-T cell infusion. OT-C001 will be evaluated with Rituximab across various dose levels. The trial will also examine cellular kinetics and biomarkers to characterize the specific mechanism associated with therapeutic responses.

"We are delighted to have reached an important milestone of entering clinical development of OT-C001 following our strategic investments in Emercell since 2021," said Dr. C. Grace Yeh, Chairman and CEO of Onward Therapeutics,"This Phase 1 trial may validate Emercell's patented platform technology for meaningful clinical outcomes. Along with the continued optimization of industrial manufacturing, these efforts will strengthen our ability to form strategic partnerships and advance this promising therapy."

"OT-C001 sets itself apart by utilizing a pool of multiple umbilical cord blood (UCB) samples, offering both diversity in the product and consistency across GMP batches," said Dr. Patrick Henno, Founder and ex-President of Emercell, "unlike the complex manufacturing processes of many engineered cell therapy products, OT-C001 provides a safe and scalable solution to unmet medical needs in targeted indications."

"We are excited to initiate this multicenter phase 1 trial in one of the most common and aggressive forms of non-Hodgkin lymphoma," said Dr. Alain Herrera, President of Emercell and CMO of Onward Therapeutics, "despite challenges in the cell therapy market, this achievement advances our mission to provide novel treatments for DLBCL patients in need of new therapeutic options. OT-C001 has the versatility to act as a universal platform through combinations with various monoclonal antibodies or targeted therapies; it may benefit patients across multiple indications beyond DLBCL."

About OT-C001

OT-C001 is an allogeneic cell therapy of highly activated and expanded NK cells from UCB, manufactured using a patented expansion and activation process. It targets tumor cells through direct and indirect cytotoxicity, when combined with a monoclonal antibody targeting tumor specific antigens. OT-C001 is being evaluated as a potential new treatment for advanced hematologic malignancies with monoclonal antibodies. In non-clinical in vitro, in vivo models, and toxicology studies, it was well tolerated and displayed promising anti-tumor activities.

About Onward Therapeutics

Onward Therapeutics (www.onward-therapeutics.com) is a clinical-stage biotechnology company focused on developing innovative cancer therapies. Led by a seasoned team 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, alongside an equity investment; and an allogeneic NK cell therapy (OT-C001) developed via its majority-owned subsidiary, Emercell. Additional programs in immunotherapy, targeted therapy, and onco-metabolism are in early development. Headquartered in Switzerland, Onward Therapeutics also operates in Paris and Taipei.

About Emercell

Emercell (www.emercell.com) develops off-the-shelf allogeneic NK cell therapies. Based at the IRMB at Saint-Éloi University Hospital Center in Montpellier, France, its lead candidate OT-C001 targets lymphoma patients resistant to standard treatments. The patented platform pre-activates and expands NK cells. In 2023, Emercell received support from Bpifrance under the France 2030 "Biotherapies and bioproduction of innovative therapies" acceleration strategy and the Occitanie Region.

SOURCE Onward Therapeutics