AMSTERDAM, December 9, 2014 /PRNewswire/ --
~ Data confirms the potential of ATIR™ in partially matched hematopoietic stem cell transplants ~
Kiadis Pharma B.V. ("Kiadis Pharma"), a clinical stage biopharmaceutical company developing T-cell immunotherapy treatments for blood cancers, today announces positive interim data from the ongoing Phase II clinical study with its lead product ATIR™. The data were presented by Dr. Denis-Claude Roy, Professor of Medicine at the University of Montreal and Chair of the study, at the American Society of Hematology (ASH) Annual Meeting in San Francisco (Abstract 314).
ATIR™ is a cell-based product designed to enable stem cell transplantations from partially matched (haploidentical) family donors for blood cancer patients who do not have a standard of care stem cell donor available. The Phase II study is an international multi-center study in which 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 ATIR™ as part of the transplant regimen.
The primary endpoint of the study is the rate of Transplant Related Mortality (TRM) at 6 months after hematopoietic stem cell transplantation (HSCT). Data from the pre-specified interim analysis of the first 10 patients showed only 2 cases of TRM at 6 months post-HSCT and both TRM cases resulted from a viral infection. Overall, 6 out of the 10 patients were completely free from severe infections (grade 3-5) after ATIR™ administration and up to 6 months after the transplant.
These data confirm the safety and efficacy of ATIR™ administration with no grade III-IV (life-threatening) acute Graft versus Host Disease (GvHD) occurring, despite the fact that no prophylactic immune suppressants were used. Only a limited number of grade I-II GvHD events were reported, all of which were treated successfully. Furthermore, no relapses were reported in any of the first 10 patients, with 4 out of the 10 patients in follow-up for more than one year at the time of the interim analysis.
These data are in line with the data previously reported from Kiadis Pharma's Phase I/II study with 19 patients. The current study is progressing well with further enrollment of patients in Canada (Hôpital Maisonneuve-Rosemont (Montreal), Juravinski Hospital and Cancer Centre (Hamilton) and Princess Margaret Cancer Centre (Toronto)), Belgium (University Hospitals Leuven, AZ Sint Jan (Bruges) and Institut Jules Bordet (Brussels)) and Germany (University Hospital of Würzburg).
Dr. Denis-Claude Roy, Professor of Medicine at the University of Montreal and Chair of the study, said: "ATIR™ has demonstrated its potential to provide potent immune protection against lethal infectious complications and residual tumor cells without causing clinically critical GvHD as an add-on for blood cancer patients who have received a stem cell transplantation from an only partially matched family member. This offers hope to patients who currently cannot be treated with a transplantation because of the limited availability of suitable matched donors."
Manfred Ruediger, PhD, Chief Executive Officer of Kiadis Pharma, commented: "The results from this interim analysis are very encouraging and show the significant potential of ATIR™ as a treatment option to enable stem cell transplantations from partially matched family donors for blood cancer patients who do not have a standard of care stem cell donor available. We also believe ATIR™ has potential as a curative treatment for blood cancer patients who have relapsed already once or more times on other treatments and who would currently not receive a stem cell transplantation because of limited donor availability. These data, presented at ASH by Dr. Denis-Claude Roy, were very well received and we look forward to providing further updates on the ongoing study in due course."
ATIR™ is a T-cell immunotherapy based medicinal product enabling stem cell transplantations using partially matched (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™ thus has the potential to address this unmet medical 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™. ATIR™ is administered as an adjunctive treatment after a T-cell depleted haploidentical HSCT, facilitating early immune reconstitution without causing life-threatening (acute) GvHD.
ATIR™ dovetails on 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™.
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 (TRM) and high overall survival. Positive follow-up results from this study demonstrate 78% survival after one year, 67% survival after five years and no TRM in the nine patients who received a higher dose of ATIR™.
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.
ATIR™ has been granted Orphan Drug Designations 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 T-cell immunotherapy based medicinal product designed to enable stem cell transplantations from partially matched (haploidentical) family donors. Kiadis Pharma is collaborating with internationally renowned clinical centers in Europe and North America for the successful development of ATIR™. Kiadis Pharma is working under a GMP manufacturing license and GMP certificate for its Quality Control laboratory. The German Red Cross Blood Donor Service in Frankfurt manufactures ATIR™ for the European sites whereas ATIR™ is manufactured at the Hôpital Maisonneuve-Rosemont in Montreal for the Canadian sites participating in the ongoing Phase II study.
Kiadis Pharma is supported by a strong group of leading international investors including LSP, DFJ Esprit, Alta Partners, Quest for Growth, NOM and MedSciences Capital. Kiadis Pharma is based in Amsterdam, The Netherlands. Further information can be found at: http://www.kiadis.com
Manfred Ruediger, CEO
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SOURCE Kiadis Pharma