Kiadis Pharma N.V., a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders will start Phase I/II clinical trial with its product ATIR201 for thalassemia, according to schedule.
Kiadis Pharma said in its press release that it has obtained regulatory approval from the national authority in the United Kingdom (the MHRA, the Medicines and Healthcare products Regulatory Agency), as well as approval from the Ethics Committees of the Royal Manchester Children’s Hospital and the Birmingham Children’s Hospital to start a Phase I/II clinical trial with its product ATIR201 for thalassemia.
The company said that in this new trial it will study the safety and feasibility of using ATIR201 in pediatric and adult patients suffering from beta-thalassemia major, the most severe form of the disease.
There is currently no approved curative medicine for beta-thalassemia major, the company noted and further said that the current standard of care treatment is purely symptomatic, requiring lifelong blood transfusions and iron chelators, with drug support to improve hematopoiesis. Replacing the diseased system and restoring the proper production of hemoglobin through an allogeneic hematopoietic stem cell transplantation (HSCT) from a healthy half-matched family donor could provide a cure for this disease. After an HSCT treatment, however, it usually takes the patient several months to recover to near-normal blood cell levels and immune cell functions, during which time the patient is vulnerable to infections caused by bacteria, viruses and fungi.
The addition of ATIR201, administered as an adjunctive immuno-therapeutic on top of an HSCT, is intended to provide the patient with functional, mature immune cells that can fight infections while not eliciting severe Graft-versus-Host-Disease (GVHD), thereby bridging the time until the immune system has fully re-grown from stem cells in the transplanted graft.
The new trial will commence in the United Kingdom and the Company plans to expand the trial into Germany pending regulatory approval. A total of up to ten beta-thalassemia major patients will be enrolled. Kiadis Pharma expects first safety and efficacy results to become available in the second half of 2017.
Manfred Rüdiger, PhD, Chief Executive Officer of Kiadis Pharma, commented: “We are excited to initiate this new Phase I/II study with ATIR201 for thalassemia patients on track as planned. 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, life-long 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.”