Date: 2013-09-06
Type of information: Licensing agreement
Compound: Facilitating Cell Therapy (FCRx) platform
Company: Novartis (Switzerland) Regenerex (USA)
Therapeutic area: Transplantation
Type agreement: R&D
licensing
Action mechanism: FCRx is a novel allogeneic hematopoietic stem cell based therapy platform that also contains facilitating cells derived from a donor. The platform supports the development of tolerance, or \"bone marrow chimerism,\" in transplant recipients, providing a better side effect profile than current human hematopoietic stem cell transplantation protocols. Chimerism may eventually render the recipient tolerant to cell, tissue or organ transplants from the same donor, thereby enabling transplant patients to discontinue immunosuppressive medications after building stable immunological tolerance. Results from a Phase II study in 15 kidney transplant recipients are extremely encouraging with six patients fully withdrawn from immunosuppression without loss of engraftment, and a further two with planned full withdrawal at 1 year. This approach may also allow for treatment of inherited metabolic diseases like metachromatic leukodystrophy or sickle cell disease.
Disease:
Details: * On September 6, 2013, Novartis has announced that it has entered into an exclusive global licensing and research collaboration agreement with Regenerex LLC, a biopharmaceutical company based in Louisville, Kentucky, for use of the company\'s novel Facilitating Cell Therapy (FCRx) platform. The hematopoietic stem cell-based FCRx platform has been investigated in kidney transplantation to induce stable immunological tolerance, resulting in graft survival without the need for lifelong immunosuppression. This collaboration reaffirms the Novartis commitment to transplant. Beyond transplant, Regenerex\'s novel platform potentially has curative potential for multiple underserved diseases and will be investigated in the rescue of serious genetic deficiencies such as inherited metabolic storage disorders and hemoglobinopathies. FCRx will broaden the current Novartis cell therapy portfolio, which includes two novel cell therapy platforms initially being investigated in hematological malignancies. HSC835 is a novel cell therapy approach that enables an expanded single umbilical cord blood derived hematopoietic stem cell transplant in patients with limited treatment options. HSC835 is currently in a Phase II trial in patients with high-risk hematological malignancies. A second cell therapy product, CTL019 is a chimeric antigen receptor T cell therapy currently in Phase II development in acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia (CLL).
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