AMSTERDAM, The Netherlands I December 14, 2016 I Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, today announces 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, according to schedule.

Thalassemia is an inherited blood disorder which results in abnormal hemoglobin formation, leading to improper oxygen transport and destruction of red blood cells. In this new trial the safety and feasibility of using ATIR201™ in pediatric and adult patients suffering from beta-thalassemia major, the most severe form of the disease, will be studied.

There is currently no approved curative medicine for beta-thalassemia major. 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.”

About Kiadis Pharma

Kiadis Pharma is focused on cell-based immunotherapy products for the treatment of blood cancers and inherited blood disorders. The Company’s products have the potential to address the risks and limitations connected with allogeneic hematopoietic stem cell transplantation (HSCT), namely Graft-versus-Host-Disease (GVHD), cancer relapse, opportunistic infections and limited matched donor availability. The Company believes that HSCT could become a first-choice treatment for blood cancers, inherited blood disorders and possibly autoimmune diseases and solid organ transplantations.

On December 5, 2016 at the Annual Meeting of the American Society of Hematology (ASH), the Company reported positive Phase II results with its lead product ATIR101™ in patients with blood cancer. The data showed that ATIR101™ significantly reduced Transplant Related Mortality, significantly improved Overall Survival and did not elicit grade III-IV GVHD in any patient. ATIR101™ has been granted Orphan Drug Designations both in the US and Europe. The Company’s second product candidate, ATIR201™, addresses inherited blood disorders with an initial focus on thalassemia, a disease which results in destruction of red blood cells in patients.

Kiadis Pharma, based in Amsterdam, The Netherlands, was granted an Advanced Therapy Medicinal Product (ATMP) certificate for manufacturing quality and non-clinical data by the European Medicines Agency (EMA). The Company’s shares are listed on Euronext Amsterdam and Euronext Brussels. For more information visit

SOURCE: Kiadis