Stem cell therapeutics (ExCellThera)

Cordex Biologics' stem cell treatment uses unique technology to multiply the number of cells that can be used from a donor's umbilical cord blood unit. A new treatment option for certain patients with blood cancers has passed muster with the European Commission (EC).Zemcelpro, made by ExCellThera subsidiary Cordex Biologics, is a one-time cell therapy meant to offer a curative treatment for those with hematological malignancies (also known as blood cancers) who don’t have access to necessary donor cells.After winning an endorsement from the Europe’s Committee for Medicinal Products for Human Use (CHMP) in June, the EC has now followed suit with a nod authorizing the therapy to treat adults with blood cancers who require an allogeneic hematopoietic stem cell transplantation following myeloablative conditioning (chemotherapy or radiotherapy) and for whom no other type of suitable donor cells are available.“This authorization marks a pivotal milestone in delivering on the therapeutic promise of Zemcelpro for adults battling life-threatening haematological malignancies,” ExCellThera and Cordex’s CEO David Millette said in an Aug. 27 press release. “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.”Zemcelpro is a unique personalized treatment made up of stem cells derived from a donor’s umbilical cord blood, some of which have been grown and multiplied. Under this approach, Zemcelpro renders the stem cells from a small blood cord unit more effective, according to a June European Medicines Agency release on the CHMP decision. In clinical studies, 21 of the 25 patients that ExCellThera evaluated in a pooled analysis of two single-arm, open-label studies achieved neutrophil engraftment, the term for when donor stem cells successfully produce the white blood cells neutrophils, within a median time of 20 days. The European approval is conditional, meaning the company will have to provide more comprehensive clinical data in the future.A phase 3 study will be initiated “as soon as possible,” according to ExCellThera. Zemcelpro’s potential use in other patient populations, such as pediatric patients and those with non-malignant hematological diseases, is also being investigated.  With the nod, Zemcelpro offers a first-of-its-kind option to many of the thousands of patients who are diagnosed each year in Europe with blood cancers—such as leukemias and myelodysplastic syndromes—as well as the only potential curative option for some blood cancers. Many patients face barriers that prevent them from accessing suitable donor cells, such as a lack of compatible donors or trouble securing cells within the often-short window required for transplant. No official statistics exist to track the number of patients who miss out on potentially curative transplants, but the group includes a disproportionately high number of people from ethnic minority backgrounds, ExCellThera pointed out in its release.Patients who do not receive needed stem cell transplants face disease progression or complications and may need hospitalization and supportive care. Zemcelpro, for its part, "has the potential not only to save lives but also to reduce the healthcare and societal burden associated with these devastating conditions,” the company noted. The drug’s launch hinges on several factors, including wrapping up national reimbursement procedures. Meanwhile, ExCellThera’s Cordex is currently working “closely” with authorities in individual countries to enable early access for eligible patients as the reimbursement talks progress.The company is also in discussions with leading stem cell transplant centers to set up a network of treatment centers for the future administration of Zemcelpro.Beyond Europe, additional regulatory filings are planned for the U.S., Canada, the U.K. and Switzerland, according to the company. Cordex is “actively seeking” strategic partnerships to ramp up the commercialization efforts in Europe and other markets, its parent company said.

- Zemcelpro® is conditionally authorized by the European Commission for adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available - Zemcelpro® will increase access to allogeneic stem cell transplantation to treat life-threatening haematologic malignancies, including leukemias and myelodysplastic syndromes - Timing of Zemcelpro® availability in individual countries will depend on several factors, including the completion of national reimbursement procedures - Cordex Biologics, an ExCellThera company, is actively seeking strategic partnerships to accelerate the commercialization of Zemcelpro® in Europe and other markets MONTREAL, August 27, 2025 /PRNewswire/ -- ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, and its wholly owned subsidiary Cordex Biologics, announced today the conditional marketing authorization of Zemcelpro® by the European Commission (EC) for the treatment of adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. This EC decision authorizes the marketing of Zemcelpro® in all European Union member states, as well as Iceland, Norway and Liechtenstein. Continue Reading ExCellThera logo Zemcelpro®, also known as UM171 Cell Therapy, is a novel personalized cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit. In Europe, over 10,000 patients each year are diagnosed with haematological malignancies—including leukemias and myelodysplastic syndromes—that require stem cell transplant. While access to transplant has improved in recent years, a number of patients still cannot access suitable donor cells. Barriers include the absence or unavailability of a compatible donor or the inability to secure donor cells within the therapeutic window for transplant, which can be especially short in urgent cases. Unlike documented transplant procedures which are well-tracked through national registries, no official statistics exist for patients who miss the opportunity for a potentially curative transplant—a group that includes a disproportionately high number of individuals from ethnic minority backgrounds. "The curative potential of allogeneic stem cell transplantation is still limited by the access to a suitable donor. The 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, Millan, Italy. "UM171 Cell Therapy offers an option timely available for patients in need." Patients with blood cancers who do not receive the stem cell transplant they require represent a significant burden to healthcare systems, driven by ongoing medical needs, disease progression, complications, hospitalizations and supportive/palliative care. Beyond this, the societal impact is profound, with premature deaths leading to lost productivity and considerable emotional and economic strain on families and communities. "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 an innovative, one-time cell therapy with curative intent, developed to give blood cancer patients without access to suitable donor cells the transplant they urgently need. By enabling more patients to receive a life-saving transplant, Zemcelpro® has the potential not only to save lives but also to reduce the healthcare and societal burden associated with these devastating conditions. The timing of Zemcelpro® availability in individual countries will depend on several factors, including the completion of national reimbursement procedures. In the meantime, Cordex Biologics is working closely with national health authorities to enable early access for eligible patients ahead of the reimbursement process, and is engaging with leading stem cell transplantation centers to establish a network of treatment centers for the future administration of Zemcelpro®. "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 for Zemcelpro® with other health authorities, including in the US, Canada, the UK, and Switzerland. Cordex Biologics is also actively seeking strategic partnerships to support and accelerate the commercialization of Zemcelpro in Europe and other international markets. The safety of Zemcelpro® is consistent with the well-characterized safety profile of conventional allogeneic blood stem cell transplantation for haematological malignancies following myeloablative conditioning. For full details on the Warnings and Precautions for Use and Adverse Reactions (including appropriate management), please refer to the EU Summary of Product Characteristics (SmPC) of Zemcelpro®. About Conditional Marketing Authorizations (CMAs) CMAs are for medicines that fulfil a significant unmet medical need such as being for serious and life-threatening diseases, where no satisfactory treatment methods are available or where the medicine offers a major therapeutic advantage. A CMA is granted where comprehensive clinical data is not yet complete, but the benefit of the medicine to address a significant unmet need outweighs the need for data that will become available in the future. CMAs are valid for one year and renewable annually with ongoing regulatory review of data. About Zemcelpro® Zemcelpro®, also known as UM171 Cell Therapy, is a novel personalized cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit. Zemcelpro®, developed by Cordex Biologics, a wholly owned subsidiary of ExCellThera, has been evaluated in 120 patients with haematologic malignancies in clinical trials in the United States, Europe and Canada. Zemcelpro® has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designations from the FDA as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the EMA. Zemcelpro® has been tested in Phase 2 trials in patients with high and very high-risk acute leukemias and myelodysplasias who have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care, including patients with patients with TP53 mutations or other genetic abnormalities, patients requiring a second transplant, and patients with refractory or active disease. A pivotal Phase 3 trial in this patient population will be initiated as soon as possible. The use of Zemcelpro® in other patient populations, including pediatric patients and patients with non-malignant haematological diseases, is also being investigated. Zemcelpro® is a registered trademark of Cordex Biologics. The product safety and efficacy have not yet been established by other regulatory agencies, such as the FDA, the MHRA and Health Canada. About ExCellThera and UM171 Technology ExCellThera is a world leader in enhanced blood stem cell therapies. ExCellThera's proprietary EnhanceTM platform for cell expansion and metabolic fitness is designed to deliver a greater dose of functional therapeutic stem cells by expanding haematopoietic stem cells (HSCs) from any source and counteracting the effects of culture or gene editing induced stress. ExCellThera partners with biopharmas to help them develop best-in-class cell and gene therapies by leveraging the technologies that form the EnhanceTM platform, including the proprietary molecule UM171 which has a first-in-class mechanism of action for ex vivo expansion and metabolic fitness of HSCs. For additional information, visit excellthera.com, and follow us on LinkedIn. 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