EU Market Welcomes Zemcelpro, a Pioneering Blood Cord Expansion Therapy

In a significant development for the field of transplant medicine, ExCellThera, a leading biotech company, has announced plans to expand the Phase-3 trial for its product Zemcelpro in additional indications within the United States. This announcement was made by David Millette, the CEO of both ExCellThera and Cordex Biologics, in a press release on August 27, 2023.

Zemcelpro is a groundbreaking therapy that aims to increase transplant access in cases where no matched donor is available. It achieves this by making smaller cord blood units viable for use in adults. The therapy combines cord blood-derived stem cells expanded ex vivo using UM171, a small molecule that promotes stem cell self-renewal, with unmodified cells from the same unit.

UM171, a valuable tool across diverse cell and gene therapies, not only drives stem cell expansion but also enhances the metabolic fitness of blood stem cells. It reduces oxidative stress and preserves mitochondrial function, contributing to improved cellular resilience that is crucial for transplant outcomes. This resilience contributes to durable engraftment, lowers the risk of graft failure, and may reduce complications associated with delayed immune recovery.

The European Commission has already granted conditional marketing authorization for Zemcelpro therapy for adult patients with blood cancers who lack a suitable donor. This makes Zemcelpro the first treatment of its kind in the European Union, targeting the challenge of donor availability in transplant medicine. It is also the second product of its kind to reach the market, and the first in Europe.

ExCellThera is open for collaborations with anyone interested in using UM171. The company's Enhance program leverages UM171 to improve gene-modified stem cells and CAR-T cell products. The company is also exploring pediatric and non-malignant blood disorder indications for Zemcelpro.

As ExCellThera prepares filings in key jurisdictions, including the U.S., the successful powering of multiple downstream therapies could position the company as a partner for wider cell and gene therapy development. The timing of Zemcelpro availability in individual countries will depend on the completion of national reimbursement procedures.

In conclusion, the advancement of Zemcelpro in the U.S. and EU marks a significant step forward in addressing the challenge of donor availability in transplant medicine. The potential of UM171 to power multiple downstream therapies could pave the way for improved outcomes and expanded access to life-saving transplants.