Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing T-cell immunotherapy treatments for blood cancers, today announces that its lead product ATIR(TM) has been granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for the treatment of acute myeloid leukemia (AML). Kiadis Pharma has previously been granted an ODD for ATIR(TM) by both the EMA and the FDA for the prevention of acute Graft versus Host Disease (GvHD) following an allogeneic bone marrow transplantation.
The EMA's ODD is reserved for new therapies being developed to treat life-threatening or chronically debilitating diseases or conditions that are relatively rare in the European Union and for which no satisfactory therapy is available. The ODD designation provides for incentives to support development, including fee reductions and a ten-year period of market exclusivity in the European Union after product approval.
Manfred Ruediger, PhD, Chief Executive Officer of Kiadis Pharma, commented:"This second Orphan Drug Designation for our lead product ATIR(TM) in the European Union represents another very important milestone in the development of our product. This, coupled with the previously granted ODDS for ATIR(TM) by the FDA and EMA, further highlights our product's importance as a novel approach which may help provide a potentially life-saving transplant from a family member to those patients who otherwise do not find a matched donor in time. In many cases, this will be the only remaining option for these patients."
"This news comes directly after having successfully closed an equity financing round with our existing investors which emphasizes the belief our investors have in both the Company and ATIR(TM)."
About ATIR(TM)
ATIR(TM) is a T-cell immunotherapy based medicinal product enabling stem cell transplantations using partially mismatched (haploidentical) family members as donors for patients suffering from blood cancer who do not have a standard of care matching stem cell donor available. A hematopoietic stem cell transplantation (HSCT) is the only potentially curative option for many patients but a matching donor is available for only half of the patients in need. ATIR(TM) thus has the potential to address this unmet need and to make a HSCT available for virtually all patients worldwide.
Those T-cells in a haploidentical donor graft which would cause Graft-versus-Host-Disease (GvHD) are selectively eliminated using proprietary technology to produce ATIR(TM). ATIR(TM) is administered as an adjunctive treatment after a T-cell depleted haploidentical HSCT, facilitating early immune reconstitution without causing life-threatening (acute) GvHD.
ATIR(TM) dovetails current approaches that render a patient eligible for a stem cell transplantation. Usually, only patients in remission are considered eligible for such a transplantation and so approaches that put refractory patients into remission, such as Chimeric Antigen Receptor (CAR) T-cell therapies, will increase the need for donors and potentially, therefore, the need for ATIR(TM).
In a Phase I/II study in which high-risk leukemia patients with very poor prognosis were treated with escalating doses of ATIR(TM) after a haploidentical HSCT, long-term safety, efficacy and proof of concept were confirmed in terms of absence of Grade III/IV (life-threatening) acute GvHD, reduced rates of infection, reduced Transplant Related Mortality and high Overall Survival. Positive follow-up results from this study demonstrate 78% survival after one year, 67% survival after five years and no Transplant Related Mortality in the nine patients who received a higher dose of ATIR(TM).
An international multi-center Phase II study including patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and myelodysplastic syndrome (MDS), to corroborate and extend the safety and efficacy results from the Phase I/II study, is now ongoing with interim data expected in the second half of 2014.