お知らせ • Jun 12
Medexus Pharmaceuticals Secures Exclusive Canadian Rights to Commercialize Um171 Cell Therapy as Zemcelpro
Medexus Pharmaceuticals had secured exclusive Canadian rights to commercialize UM171 Cell Therapy, a proprietary advanced clinical stage investigational drug product that recently received conditional marketing authorization in Europe from the European Commission as Zemcelpro (dorocubicel), in a license and supply deal with ExCellThera and Cordex Biologics, its wholly owned subsidiary. Zemcelpro is a novel personalized cryopreserved hematopoietic 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. The UM171 molecule and technology behind Zemcelpro was discovered and developed in Canada by scientists at the Universite de Montreal. The product is used to treat hematological malignancies (blood cancers), such as leukemias and myelodysplasias. Given its current stage of development in Canada, Medexus does not expect to begin commercializing the product before calendar year 2028 or, depending on available regulatory pathways, possibly calendar year 2031. There were more than 843 allogeneic hematopoietic stem cell transplantation (allo-HSCT) procedures in Canada during calendar year 2024. Depending on the future product label approved by Health Canada, and considering the specialized nature and expected contribution of the treatment to the allo-HSCT field, Zemcelpro (if approved by Health Canada) is expected to be an appropriate option for a significant portion of Canadian patients requiring these allo-HSCT procedures annually. Zemcelpro recently received conditional marketing authorization in Europe from the EC for the treatment of adults with hematological malignancies requiring an allo-HSCT procedure following myeloablative conditioning for whom no other type of suitable donor cells is available. In addition to this first conditionally approved indication, Cordex expects to initiate, as soon as possible, an international multi-center phase 3 clinical trial in patients with high- and very high-risk acute leukemias and myelodysplasias, which is a post-authorization measure for the product's conditional marketing authorization in Europe. The phase 3 clinical trial for this second indication is expected to be completed within four years. Subject to an assessment of available regulatory pathways in Canada, Medexus intends to submit UM171 Cell Therapy for Health Canada approval as soon as possible, and no later than the successful completion of the phase 3 clinical trial by Cordex. As part of the regulatory process for UM171 Cell Therapy, Medexus intends to seek Health Canada approval of the brand name Zemcelpro, which has not yet been reviewed or accepted by Health Canada nor approved for use in Canada. Zemcelpro is a registered trademark of Cordex or its related companies in Canada. Subject to regulatory approval and subsequent engagement with relevant Canadian payer bodies, Medexus expects that the net price of Zemcelpro in Canada will be competitively positioned relative to other cell and gene therapy products available in Canada. Medexus's pricing analysis in advance of any commercial launch in Canada will be informed by, and subject to, the expected continued evolution of the allo-HSCT field in Canada and globally in the coming years, among other factors. Medexus will pay to ExCellThera quarterly royalty payments on net sales of the product and various one-time milestone payments that share the potential rewards from this opportunity and align the parties' interests around long-term net revenue performance. Cordex will manage the phase 3 clinical trial and will be responsible for the manufacturing and supply of the product to Medexus on a cost-plus basis. Medexus made an initial USD 2 million payment to Cordex and has agreed to sponsor the new drug submission seeking Health Canada approval of UM171 Cell Therapy in Canada as Zemcelpro (dorocubicel). The initial term of the license and supply agreements will extend through to the 10-year anniversary of Health Canada approval (if any) with successive two-year extension terms thereafter. Zemcelpro (dorocubicel), also known as UM171 Cell Therapy, is a novel personalized cryopreserved hematopoietic 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 over 120 patients with hematologic malignancies in clinical trials in the United States, Europe, and Canada. The product has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designations from the US Food and Drug Administration and orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification, and priority medicines (PRIME) designations from the European Medicines Agency. Zemcelpro has been tested in phase 2 clinical trials in 60 adult patients with high- and very high-risk acute leukemias (AL) and myelodysplastic syndromes (MDS) who have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care, including patients with TP53 mutations or other genetic abnormalities, patients requiring a second transplant, and patients with refractory or active disease. Positive topline results from the phase 2 clinical trials were recently announced. Outcomes were assessed in 60 adults with high- or very high-risk acute leukemias (AL) and myelodysplastic syndromes (MDS) who received Zemcelpro in two prospective phase 2 trials conducted in the United States, Canada, and Europe. High-risk AL/MDS was defined as disease with an expected 2-year progression-free survival (PFS) <40% after conventional allogeneic hematopoietic stem cell transplantation (allo-HSCT). Very high-risk disease was defined as a second allo-HSCT, AL with active disease, acute myeloid leukemia (AML) with TP53 mutation and complex karyotype, AML with EVI1 mutation, or MDS with multi-hit TP53 mutation. The primary endpoints were safety and NRM; secondary endpoints included PFS, overall survival (OS), relapse incidence, engraftment, and graft-versus-host disease (GVHD). Sixty (60) patients (median age 43 years; range 19-66) were transplanted; 87% had AL (63% myeloid, 37% lymphoid) and 13% had MDS. Thirty percent (30%) had failed a prior allogeneic transplant and 57% met very high-risk criteria.