Netherlands-based Kiadis Pharma has secured regulatory approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and Ethics Committees of the Royal Manchester Children’s Hospital and the Birmingham Children’s Hospital to initiate a Phase I/II clinical trial of ATIR201 to treat thalassemia.

Based on the company’s Theralux platform, ATIR201 has been developed to provide allodepleted T-cell immunotherapeutics (ATIR), which supports the patient’s newly transplanted immune system before it is fully functional.

ATIR201 is a cellular product for infusion consisting of donor lymphocytes derived from a healthy, haploidentical stem-cell donor and created for each individual patient.

The Phase I/II clinical trial will test the safety and feasibility of ATIR201 in treating ten pediatric and adult patients suffering from beta-thalassemia major.

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The patients will be administered with ATIR201 as an adjunctive immuno-therapeutic coupled with hematopoietic stem-cell transplantation (HSCT).

The therapy is expected to provide functional, mature immune cells to protect against infections eliminating the risk of severe graft-versus-host-disease (GVHD).

“We believe ATIR201 has the potential to make curative HSCT a viable option to many more patients suffering from inborn disorders of the blood like thalassemia.”

Kiadis Pharma CEO Manfred Rüdiger said: “It is well established that a stem-cell transplant, once engrafted, can be functional for life and so the aim of our approach is to not just ameliorate symptoms or reduce the need for transfusions, but to provide patients with an enduring, lifelong cure.

“Our approach could provide an alternative to various gene-therapy-based strategies, which, for example, may suffer from gene silencing or suboptimal expression levels over time.

“We believe ATIR201 has the potential to make curative HSCT a viable option to many more patients suffering from inborn disorders of the blood like thalassemia.”

The trial’s first safety and efficacy result is expected to be released in the second half of next year.

Image: Blood sample of person with delta beta thalassemia. Photo: courtesy of Prof Osaro Erhabor.