Kiadis Pharma proceeds with pivotal trial ATIR™
Amsterdam, The Netherlands, December 4, 2008 – Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing treatments for blood cancers, today announces that it has successfully completed treatment of the last patient on its ATIR™ phase I/II clinical trial. ATIR™, a donor lymphocyte cell based product, is under development to prevent acute Graft versus Host Disease (GvHD) and allow early immune reconstitution following a mismatched bone marrow transplantation. Nineteen end stage blood cancer patients were treated with no cases of grade III/IV acute GvHD occurring within the first 100 days after ATIR™ infusion, showing the feasibility and tolerability of the addition of ATIR™ in combination with a mismatched transplant procedure.
Following scientific advice from the EMEA Kiadis Pharma is preparing to initiate a multi center multinational pivotal study starting recruitment early 2009. In this study the efficacy of ATIR™ will be investigated in patients diagnosed with AML, ALL or MDS eligible for an allogeneic bone marrow transplantation but for whom no matching donor is available.
“Bone marrow transplantations are often the only treatment option left for end-stage blood cancer patients. But too many patients simply do not find a suitable matched donor in time” says Dr. Denis-Claude Roy of the Maisonneuve-Rosemont Hospital in Montreal, the principal investigator on the study. “Our study shows a rapid immune reconstitution in transplanted patients provided with ATIR™ treated immune cells from mismatched donors. Without ATIR™ treatment this would not be possible because of the high risk of acute Graft versus Host Disease with mismatched donor immune cells. This is obviously a very hopeful development for a large patient group.”
Dr. Manja Bouman, CEO of Kiadis Pharma, says “The results of this clinical trial are very encouraging and it is another important milestone in the development of ATIR™ as a novel approach which may enable a safe and potentially life-saving mismatched bone marrow transplantation as a treatment option for end-stage blood cancer patients.”
ATIR™ is a T‑cell 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 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™ thus has the potential to address this unmet need and to make an HSCT available for all patients worldwide.
Those T‑cells in a haploidentical graft which would cause Graft-versus-Host-Disease (GvHD) are selectively eliminated using proprietary technology to produce ATIR™. ATIR™ is administered as an adjunctive treatment after a T‑cell depleted haploidentical HSCT facilitating early immune reconstitution without causing life‑threatening (acute) GvHD.
In a Phase I/II study in which high‑risk leukemia patients with very poor prognosis were treated with escalating doses of ATIR™ 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 demonstrating 67% survival after five years and no Transplant Related Mortality in the nine patients who received an efficacious dose of ATIR™ were recently reported.
An international multi‑center Phase II study including patients with acute myeloid leukemia, acute lymphoblastic leukemia and myelodysplastic syndrome, to corroborate and extend the safety and efficacy results from the Phase I/II study, is now ongoing with topline data expected in the first half of 2014.
ATIR™ has been granted Orphan Drug Designation both in the EU and the USA. Together, both regions represent a combined primary market potential of more than EUR 1 billion per year.
About Kiadis Pharma
Kiadis Pharma B.V. is a private, clinical stage biopharmaceutical company focused on the development of innovative and potentially life‑saving therapies for patients with late stage blood cancers and related disorders, an area of significant unmet medical need.
Kiadis Pharma’s lead product is ATIR™, a cell based product designed to enable stem cell transplantations from partially mismatched (haploidentical) family donors. Kiadis Pharma is collaborating with internationally renowned centers in Europe and North America for the successful development and manufacturing of ATIR™. Kiadis Pharma recently obtained a GMP manufacturing license and GMP certificate for its Quality Control laboratory.
Kiadis Pharma is supported by a strong group of leading international investors including LSP, Alta Partners, DFJ Esprit, Quest for Growth, MedSciences Capital and NOM. Kiadis Pharma is based in Amsterdam, The Netherlands.