Zemcelpro® (UM171 Cell Therapy) receives EC authorization as the first and only cell therapy for blood cancer patients without access to suitable donor cells

MONTREAL, Quebec, Canada — Aug. 27, 2025

• Zemcelpro® is conditionally authorized by the European Commission for adults with haematological malignancies who require an allogeneic haematopoietic stem cell transplantation after myeloablative conditioning and for whom no other suitable donor cells are available.
• Zemcelpro® will increase access to allogeneic stem cell transplantation for life‑threatening haematologic malignancies, including leukemias and myelodysplastic syndromes.
• Availability in each country will depend on factors such as completion of national reimbursement procedures; Cordex Biologics is seeking partnerships to accelerate commercialization.

ExCellThera Inc. (ExCellThera) and its wholly owned subsidiary Cordex Biologics announced today that the European Commission (EC) has granted a conditional marketing authorization for Zemcelpro® (UM171 Cell Therapy). The authorization covers adults with haematological malignancies who require an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning and for whom no other type of suitable donor cells is available. The EC decision authorizes marketing across the European Union and in Iceland, Norway and Liechtenstein.

Zemcelpro®, also called UM171 Cell Therapy, is a personalized, cryopreserved haematopoietic stem cell transplantation product consisting of two components derived from the same cord blood unit: UM171‑expanded CD34+ cells (dorocubicel) and unexpanded CD34‑ cells.

In Europe, more than 10,000 patients each year are diagnosed with haematological malignancies that may require stem cell transplant. Despite improvements in access, many patients still cannot obtain suitable donor cells due to lack of a compatible donor or inability to secure donor cells within the narrow therapeutic window in urgent cases. Patients who miss the opportunity for potentially curative transplant often include a disproportionately high number of individuals from ethnic minority backgrounds.

“The curative potential of allogeneic stem cell transplantation is still limited by access to a suitable donor. Access to stem cell donation according to the stringent disease course is one of the major limiting factors for success,” said Dr. Fabio Ciceri, Professor of Hematology, Vita‑Salute San Raffaele University, Milan, Italy. “UM171 Cell Therapy offers an option timely available for patients in need.”

“Each year, thousands of people across Europe are diagnosed with blood cancers that require potentially life‑saving allogeneic stem cell transplantation. Yet some are left without access to suitable donor‑derived blood stem cells, facing a critical and unmet medical need,” said Dr. J. (Jurjen) Versluis, Internist‑Hematologist and Principal Investigator, Erasmus MC, Rotterdam, The Netherlands.

Zemcelpro® is a one‑time cell therapy developed with curative intent to enable transplants for patients who cannot access suitable donor cells. By expanding access to transplant, Zemcelpro® has the potential to save lives and reduce the healthcare and societal burdens associated with these conditions.

The timing of Zemcelpro® availability in specific countries will depend on national reimbursement and other local requirements. Cordex Biologics is working with national health authorities to enable early access for eligible patients ahead of reimbursement decisions and is engaging leading transplantation centers to establish a network of treatment sites.

“This authorization marks a pivotal milestone in delivering on the therapeutic promise of Zemcelpro® for adults battling life‑threatening haematological malignancies. As the first and only cell therapy approved for patients without access to suitable donor cells, Zemcelpro® offers a vital new stem cell transplant option — and renewed hope — for those who have been left without one,” said David Millette, CEO of ExCellThera and Cordex Biologics.

Additional regulatory filings are planned with other health authorities, including in the United States, Canada, the UK and Switzerland. Cordex Biologics is actively seeking strategic partnerships to support and accelerate commercialization in Europe and other international markets.

The safety profile of Zemcelpro® is consistent with the well‑characterized safety profile of conventional allogeneic blood stem cell transplantation after myeloablative conditioning. Full details on warnings, precautions and adverse reactions are described in the EU Summary of Product Characteristics (SmPC) for Zemcelpro®.

About Conditional Marketing Authorizations (CMAs)

CMAs are used for medicines that address a significant unmet medical need (for serious or life‑threatening diseases where no satisfactory treatments exist or where the medicine offers a major therapeutic advantage). A CMA may be granted when comprehensive clinical data are not yet complete but the benefit of immediate availability outweighs the need for further data. CMAs are valid for one year and renewable annually while additional data are collected and reviewed.

About Zemcelpro®

Zemcelpro® (UM171 Cell Therapy) is a personalized haematopoietic stem cell transplantation product containing UM171‑expanded CD34+ cells (dorocubicel) and unexpanded CD34‑ cells from the same cord blood unit. Developed by Cordex Biologics (a wholly owned subsidiary of ExCellThera), Zemcelpro® has been evaluated in clinical trials involving approximately 120 patients in the United States, Europe and Canada. The product has received orphan drug and RMAT designations from the FDA, and orphan medicinal product, ATMP classification and PRIME designation from the EMA.

Zemcelpro® has been tested in Phase 2 studies in high and very high‑risk acute leukemias and myelodysplasias, including patients with TP53 mutations, those requiring a second transplant, and patients with refractory or active disease. A pivotal Phase 3 trial in this population will be initiated as soon as feasible. Use in other populations, including pediatric and non‑malignant haematological diseases, is under investigation.

About ExCellThera and UM171 Technology

ExCellThera is a leader in enhanced blood stem cell therapies. Its proprietary Enhance™ platform aims to expand functional therapeutic haematopoietic stem cells (HSCs) from any source and improve metabolic fitness. The platform centers on the molecule UM171, which has a first‑in‑class mechanism for ex vivo expansion and metabolic fitness of HSCs. ExCellThera collaborates with biopharma partners to develop cell and gene therapies leveraging the Enhance™ platform. For more information, visit excellthera.com.

Zemcelpro® is a registered trademark of Cordex Biologics.