6 month follow-up data expected in H1 2014
Amsterdam, The Netherlands, November 18, 2013; Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing treatments for blood cancers, today announces the successful Phase II enrollment of approximately ten patients of whom the majority have already been transplanted and received ATIR. Kiadis Pharma’s lead program, ATIR, is a cell-based product designed to enable stem cell transplantations from partially mismatched (haploidentical) family donors for blood cancer patients who do not have a standard of care stem cell donor available. Topline data from the trial (CR-AIR-007) is expected in the first half of 2014.
The CR-AIR-007 Phase II clinical study is an international multi-center study undertaken to corroborate and extend the safety and efficacy results from the previous successful Phase I/II dose-escalation study with ATIR. A total of 23 leukemia patients, including patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and myelodysplastic syndrome (MDS) will be enrolled and treated with the optimal dose identified in the Phase I/II study. The primary endpoint of the trial is the rate of transplant related mortality six months post a haploidentical stem cell transplant. Secondary endpoints include overall survival (OS) at one year after transplant as well as relapse rated mortality, non-relapse related mortality, incidence of acute and chronic Graft versus Host Disease (GvHD) and rate of severe infections. Enrollment of patients commenced in Canada and Belgium in April and August 2013, respectively, with the Maisonneuve-Rosemont Hospital in Montreal and Hamilton Health Sciences participating in Canada and the University Hospitals Leuven, the Institute Jules Bordet in Brussels and the Sint-Jan Academic Hospital in Bruges participating in Belgium.
Manfred Ruediger, PhD, Chief Executive Officer of Kiadis Pharma, commented: “We are delighted that the enrollment into CR-AIR-007 is progressing according to plan. This demonstrates the demand for ATIR and its significant potential as a lifesaving blood cancer treatment. All patients currently enrolled in the study are alive and doing well and none of them have yet to experience any acute GvHD or serious infections. The majority of patients have been on the study for between two to seven months. We look forward to reporting the topline Phase II data in the first half of 2014.” He continues: “Having successfully transferred our manufacturing process for ATIR to our European CMO in Frankfurt am Main, Germany, we now have two manufacturing facilities using our robust process, one for our European sites and one in Montreal for our Canadian sites.”
ATIR is a 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 a 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 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 confirm and extend the data from the Phase I/II study, is now ongoing with topline results of the first phase expected in H1 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. ATIR is currently being studied in an international Phase II study. 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.