Avanzanite Announces Exclusive Licence Agreement to Commercialise Zemcelpro® in Europe

Zemcelpro is authorised in the European Union for eligible adults with blood cancers who need an allogeneic stem cell transplant and have no other suitable donor cells available. Avanzanite secures exclusive commercialisation rights to Zemcelpro across 32 countries covering the European Economic Area, UK and Switzerland including potential indication expansion. Avanzanite will become the EU marketing authorisation holder following regulatory transfer and support a planned Phase 3 trial. Zemcelpro becomes Avanzanite’s fifth rare disease launch and first advanced therapy medicinal product (ATMP).

Avanzanite Bioscience B.V. (“Avanzanite” or “Company”) a European pharmaceutical company focused on rare diseases, today announced an exclusive licence agreement with Montreal-based Cordex Biologics, an ExCellThera company (“ExCellThera”), a leader in enhanced blood stem cell therapies, to commercialise Zemcelpro^®. Zemcelpro received conditional marketing authorisation from the European Commission on 25 August 2025 for the treatment of adult patients with haematological malignancies requiring an allogeneic haematopoietic stem cell transplant (allo-HSCT) following myeloablative conditioning for whom no other type of suitable donor cells is available.

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Adam Plich, Co-founder and CEO, Avanzanite Bioscience

Under the agreement, Avanzanite will lead commercialisation across 32 countries including the European Economic Area, UK and Switzerland. Avanzanite will become the holder of the existing EU marketing authorisation for Zemcelpro and plans to submit marketing authorisation applications in the UK and Switzerland. ExCellThera will retain responsibility for manufacturing and supply and will continue to lead global development across potential indication expansion in specific patients.

Furthermore, Avanzanite will contribute to the cost of a planned Phase 3 trial evaluating Zemcelpro against haploidentical or mismatched unrelated donor transplantation in adult patients with high-and very high-risk acute leukaemias and myelodysplastic syndromes (MDS). The trial is intended to provide confirmatory evidence of efficacy and safety and may support a potential indication expansion.

“This partnership reflects why Avanzanite was founded: to unlock the full potential of innovative medicines for patients with rare diseases in Europe,” said Adam Plich, Co-founder and CEO, Avanzanite Bioscience. “We believe that while discovering breakthrough medicines requires one kind of genius, ensuring those medicines reach patients who can benefit from them requires another. This partnership unites these strengths in a shared sense of purpose.” Plich continues: “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.”

Zemcelpro has orphan drug designation and was part of the European Medicines Agency’s PRIority MEdicines (PRIME) scheme during development. This scheme provides early and enhanced scientific and regulatory support for promising medicines addressing unmet medical needs.

“Our team and collaborators have worked relentlessly for many years to advance Zemcelpro from scientific discovery towards patient care,” said David Millette, CEO of ExCellThera. “Avanzanite brings a pan-European footprint with deep expertise in rare disease launches and market access. We believe they are the ideal partner to help turn the promise of this science into impact for patients in need while allowing us to continue focusing on scientific and clinical development.”

“For patients with blood cancers who require a donor stem cell transplant, finding suitable donor cells is a vital step in the treatment pathway,” said Dr Jurjen Versluis, Internist-Haematologist, Erasmus MC Cancer Institute, Rotterdam, the Netherlands. “When no other suitable donor cells are available, additional transplant options are important. Erasmus MC contributed to the clinical trials evaluating Zemcelpro, and we are pleased to have played a role in its clinical development.”

The agreement advances Avanzanite’s ambition to become a European rare disease champion. With this fifth rare disease launch in four years, Zemcelpro extends the company’s haematology portfolio into stem cell therapy and haemato-oncology, alongside its ophthalmology and nephrology franchises. Founded in 2022, Avanzanite now has more than 110 employees across Europe and serves more than 400 patients.

About Zemcelpro^®

Zemcelpro is an allogeneic haematopoietic stem cell therapy authorised as a medicinal product in the European Union for use in eligible adults requiring allogeneic haematopoietic stem cell transplantation (allo-HSCT). It is made specifically for each patient from a single umbilical cord blood unit and contains two components: expanded CD34+ haematopoietic stem cells using proprietary UM171 technology (dorocubicel) and unexpanded CD34–cells. Following infusion, the CD34+ stem cells travel to the bone marrow where they divide and mature into specialised blood and immune cells. The unexpanded CD34–cells, consisting primarily of CD3+ T cells, play a complementary role by supporting immune reconstitution and providing graft-versus-leukaemia effects post-transplantation.

About Allogeneic Stem Cell Transplantation

Each year around 15,000 adults in the licensed territories require allogeneic haematopoietic stem cell transplantation (HSCT) as a potentially curative treatment for blood cancers such as acute myeloid leukaemia (AML), acute lymphoblastic leukaemia (ALL), myelodysplastic syndromes (MDS), lymphoma and other diseases. However, one third of these patients urgently need a transplant but have no matched related donor. Most receive transplants from haploidentical or mismatched unrelated donors, which have an increased risk of serious complications, including graft-versus-host disease (GVHD) and infections, contributing to non-relapse mortality. Some are unable to receive a transplant at all and face the risk of relapse with little prospect of survival.

About Avanzanite Bioscience

Avanzanite is a next-generation pharmaceutical company purpose-built to unlock the full potential of rare disease medicines across Europe. Founded in 2022 and headquartered in Amsterdam, the Netherlands, Avanzanite was created around a belief that scientific innovation creates patient impact only when medicines reach those who can benefit from them. Through tailored partnerships, biopharma innovators entrust Avanzanite with the European future of their medicines. Avanzanite combines pan-European expertise, bold commercial and access strategy, together with high-performance execution, to transform Europe’s complexity into opportunity, ensuring no patient is left behind and creating lasting long-term value for partners.

For more information, visit www.avanzanite.com and follow us on LinkedIn.

About ExCellThera

ExCellThera is a world leader in enhanced blood stem cell therapies. Its technology platform is designed to expand HSCs from any source while counteracting the cellular stress associated with cell culture or gene editing.

For additional information, visit www.excellthera.com and follow on LinkedIn.

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