Avanzanite Licenses European Rights to Blood Cancer Cell Therapy Zemcelpro

Rare Daily Staff

Avanzanite Bioscience has secured exclusive rights to commercialize the stem cell therapy Zemcelpro across 32 European countries under an agreement with Cordex Biologics, an ExCellThera company, expanding the rare disease drugmaker’s business into cell therapy.

The agreement covers the European Economic Area, the United Kingdom and Switzerland, as well as potential future indications. Financial terms were not disclosed. The partnership allows ExCellThera to focus on scientific and clinical development while drawing on Avanzanite’s European commercial and market-access capabilities. The companies did not discolose financial details.

Zemcelpro received conditional European Union marketing authorization in August 2025 for adults with blood cancers who need a donor stem cell transplant and have no other suitable donor cells available. Its approved use follows myeloablative conditioning, an intensive treatment given before transplantation to destroy bone marrow cells and prepare patients to receive donor cells.

Zemcelpro is made for each patient from a single donated umbilical cord blood unit. It contains two components: dorocubicel, a population of blood-forming stem cells expanded using ExCellThera’s UM171 technology, and a second population of cells that is not expanded.

After infusion, the blood-forming stem cells travel to the bone marrow, where they multiply and develop into blood and immune cells. The unexpanded component consists primarily of T cells, which help rebuild the immune system and can contribute to an immune response against leukemia after transplantation.

The therapy provides an additional transplant option for patients who lack other suitable donor cells. The companies estimate that about 15,000 adults across the licensed territories need a donor stem cell transplant each year for blood cancers and other diseases.

Zemcelpro has orphan drug designation and received development support through the European Medicines Agency’s PRIME program for promising medicines addressing unmet medical needs.

Avanzanite will take over the existing EU marketing authorization following a regulatory transfer and plans to seek approval in the United Kingdom and Switzerland. Montreal-based ExCellThera will retain responsibility for manufacturing and supply and continue to lead global development.

Avanzanite will also contribute to the cost of a planned phase 3 trial comparing Zemcelpro with transplantation using cells from half-matched donors or mismatched unrelated donors. The study will enroll adults with high- and very high-risk acute leukemias and myelodysplastic syndromes, a group of bone marrow cancers.

The trial is intended to provide confirmatory evidence of safety and efficacy and could support broader use of the therapy. The announcement did not specify the size of Avanzanite’s financial contribution or when the study would begin.

“Our commitment also extends to supporting the funding of the planned phase 3 trial to gather further evidence and explore the therapy’s potential for broader use,” said Adam Plich, co-founder and CEO of Avanzanite.

Photo: Adam Plich, co-founder and CEO of Avanzanite