Novartis, Regenerex in Cell Therapy Deal
Novartis announces an exclusive global licensing and research collaboration with Regenerex, leveraging a novel cell platform to broaden presence in the cell therapy space
Research collaboration advances company goal to deliver an innovative portfolio of novel cell therapy therapeutics for conditions with a high unmet need
Strategic research collaboration enlarges scope of the Novartis novel cell therapy portfolio with the potential to build expertise and synergies across the programs
For kidney transplantation this has the potential to induce immunological tolerance, resulting in long-term graft survival without the need for lifelong immunosuppression
Basel, September 6, 2013 - Novartis today 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.
"As the field of biomedicine sits on the cusp of a new transformation, we are excited to announce this agreement which supports the Novartis leadership position in cell therapy," said Dr. Timothy Wright, Global Head Development, Novartis Pharmaceuticals. "Thirty years ago, Novartis developed ciclosporin, which changed transplantation treatment paradigms and enabled countless lives to be saved. Now, this collaboration, along with our internal cell therapy assets, has the potential to transform medicine once again through innovation."
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).
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. Currently, solid organ transplant recipients must take immunosuppressive drugs for life to prevent rejection. This approach may also allow for treatment of inherited metabolic diseases like metachromatic leukodystrophy or sickle cell disease.
The foregoing release contains forward-looking statements that can be identified by terminology such as "goal," "potential," "commitment," "potentially," "will," "on the cusp," "being investigated," "may," "eventually," "encouraging," "planned," or similar expressions, or by express or implied discussions regarding potential approvals for the FCRx platform, HSC835 or CTL019, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that the FCRx platform, HSC835 or CTL019 will be approved for use or sale in any market, or at any particular time. Nor can there be any guarantee that such products will achieve any particular levels of revenue in the future. In particular, management's expectations regarding such products could be affected by, among other things, unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; unexpected manufacturing issues; the impact that the foregoing factors could have on the values attributed to the Novartis Group's assets and liabilities as recorded in the Group's consolidated balance sheet, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines and diagnostic tools, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas. In 2012, the Group achieved net sales of USD 56.7 billion, while R&D throughout the Group amounted to approximately USD 9.3 billion (USD 9.1 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 131,000 full-time-equivalent associates and operate in more than 140 countries around the world. For more information, please visit http://www.novartis.com.
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 Leventhal J, Abecassis M, Miller J. Gallon L, Ravindra K, Tollerud DJ, King B, Herzig G, Herzig R, Ildstad ST. Chimerism and tolerance without GVHD or engraftment syndrome in HLA-mismatched combined kidney and hematopoietic stem cell transplantation. Science Translational Medicine 2012 Mar 7; 4(124): 124ra28.
Posted: September 2013