Amsterdam, The Netherlands, September 22, 2008 – Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing treatments for blood cancers, today announces that its lead product ATIR™ has been granted orphan drug designation (ODD) by the European Medicines Agency (EMEA) for the prevention of acute Graft versus Host Disease (GvHD) following an allogeneic bone marrow transplantation.
“Following the orphan drug designation granted by the FDA for our lead product ATIR™, this 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” says Dr. Manja Bouman, Chief Executive Officer of Kiadis Pharma.
The EMEA’s orphan drug designation 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 orphan drug designation provides for incentives to support research and development, exemption from user fees and a ten-year period of market exclusivity in the European Union after product approval.
ATIR™ is under development to allow early immune reconstitution while preventing life-threatening GvHD, a major complication of allogeneic bone marrow transplantations. GvHD is a condition caused by the donor immune cells attacking the patient’s tissues and organs. Allogeneic bone marrow transplantations today require that the patient and donor immune systems are highly similar in order to reduce the risk of GvHD and therefore these transplantations rely on matching donors. By eliminating those immune cells which could attack the patient’s body and thus preventing the occurrence of GvHD, ATIR™ enables the use of a mismatched donor. This means ATIR™ will address a significant limitation in bone marrow transplantations, which is the timely availability of a donor.
Every year more than 70,000 patients die of blood cancers in the European Union alone. For these end-stage and high-risk blood cancer patients, a bone marrow transplantation might be the only treatment option which can lead to prolonged survival or cure. Annually about 9,000 patients in the European Union receive a bone marrow transplant from a matched donor. However, this is only 1/3 of the patients in the European Union who are in need of such a transplant, leaving the majority of patients without the option of a potential curative therapy.
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.