A classification for innovative new treatments
Amsterdam, The Netherlands, December 14, 2007 – Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing treatments for blood cancers, today announces that its lead product ATIR has received approval for regulatory classification as a “cell based” medicinal product by the Innovation Task Force (ITF), a division of the European Medicines Agency (EMEA). Based upon this regulatory classification ATIR is eligible for EMEA procedures. As a next step Kiadis Pharma will file for orphan drug designation with the EMEA to obtain additional product protection upon marketing approval. In November this year ATIR has been granted orphan drug designation by the US Food and Drug Administration (FDA).
ATIR is under development to prevent acute Graft versus Host Disease (GvHD) allowing the use of a mismatched donor for bone marrow transplantations. It is a personalized cell-based treatment prepared according to a proprietary protocol using both a novel small molecule substance and a proprietary medical device. With the official classification of ATIR as a cell based medicinal product by the EMEA, it is now categorized as an Advanced Therapy, and considered to be a highly innovative treatment. The benefits are a centralized marketing authorization procedure, which harmonizes and facilitates access to the European market. In addition, it provides access to an expert Committee for Advanced Therapies within the EMEA, to address scientific, legal and regulatory issues during product development.
“We are very pleased having received a cell based medicinal product classification for ATIR. The regulatory guidelines that will rule ATIR are therefore clear and we have subsequently started with the application of the orphan drug designation with the EMEA which will be filed before the end of this year”, said Manja Bouman, CEO Kiadis Pharma.
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.