EMA Accepts ExCellThera's Accelerated MAA for UM171 Cell Therapy for Hematological Malignancy Patients Without Suitable Donors

ExCellThera Inc. (ExCellThera), a pioneering company in the field of blood stem cell expansion and therapies, has announced a significant development concerning its UM171 Cell Therapy (INN-dorocubicel). The European Medicines Agency (EMA) has accepted the Market Authorisation Application (MAA) for this therapy, under an accelerated assessment procedure. This specific treatment targets adult patients with hematological malignancies who require a stem cell transplant but do not have a readily available suitable donor.

The acceptance of UM171 Cell Therapy for accelerated assessment by the EMA’s Committee for Medicinal Products for Human Use (CHMP) underscores its importance from a public health perspective. This accelerated process is designed to shorten the timeframe for the CHMP to review a MAA, as compared to the usual procedure. This follows the therapy's earlier recognition, when it was granted Orphan Medicinal Product designation and access to the Priority Medicines scheme (PRIME) by the EMA back in 2020.

David Millette, CEO of ExCellThera, expressed enthusiasm over this regulatory milestone, highlighting the therapy's potential to meet the significant unmet needs of patients requiring stem cell transplants without suitable donors. He emphasized the company's eagerness to collaborate with the EMA to expedite the availability of this therapy to the patients in need.

Developed by Cordex Biologics, a subsidiary of ExCellThera, UM171 Cell Therapy has undergone evaluation in clinical trials involving 120 patients across the United States, Europe, and Canada. The therapy has received multiple designations, including orphan drug designation and regenerative medicine advanced therapy (RMAT) from the FDA, and orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification, and PRIME designation from the EMA.

The Phase 2 trials of UM171 Cell Therapy have shown promising results in patients with high and very high-risk acute leukemias and myelodysplasias. These patients typically have limited treatment options, low survival rates, and high relapse rates under the current standard of care. Notably, the trials included patients with refractory or active diseases, those requiring a second transplant, and those with TP53 mutations or other genetic abnormalities. Looking ahead, a Phase 3 trial is slated to begin in the latter half of 2024 for this patient cohort.

Moreover, the potential application of UM171 Cell Therapy extends beyond its current scope. Research is ongoing to explore its effectiveness in other patient groups, such as those with multiple myeloma, pediatric patients, and individuals with non-malignant hematological diseases.

It is important to note that UM171 Cell Therapy remains an investigational treatment, and its safety and efficacy have not yet been established by health authorities, including the FDA, EMA, and Health Canada.

ExCellThera continues to be at the forefront of blood stem cell expansion technology. Their EnhanceTM platform aims to provide higher doses of functional therapeutic stem cells by expanding hematopoietic stem cells (HSCs) from various sources while counteracting the negative effects of culture or gene editing-induced stress. This platform includes the proprietary molecule UM171, which is recognized for its unique mechanism in ex vivo expansion and enhancing the metabolic fitness of HSCs. Through collaborations with biopharmaceutical companies, ExCellThera strives to develop leading cell and gene therapies utilizing their cutting-edge technologies.