The completed animal study is based on a cancer model in which human cells from an aggressive form of chronic lymphocytic leukemia were introduced to immune-deficient mice. This animal model is considered by leading scientists to be of clinical relevance and therefore suitable for evaluation of new drugs for the treatment of chronic lymphocytic leukemia, despite the inherent limitations and variations seen in the model*.
After 14 days of treatment with KAN0439834 the number of leukemia cells was reduced by an average of 50% in the treated animals as compared to the control group that did not receive treatment. This is a statistically significant effect. A newly developed crystalline formulation of KAN0439834 was orally administered to the animals once daily resulting in a blood drug concentration that was sufficient for a significant reduction in number of leukemia cells. A toxicological evaluation of the new formulation was conducted by histopathology on 10 organs. The results from this evaluation indicated a possible mild side effect in the kidney but no tissue damage in the examined organs. The analyses of the liver histology and blood markers of liver function show that the indications of effects on the liver that were observed in November 2014 when using a different formulation, are not present with the new crystalline formulation of KAN0439834.
Protein analyses showed that the amount of activated ROR1 was significantly reduced, suggesting that also in this study, treatment with KAN0439834 has had the desired effect on leukemia cells with ROR1 as target.
On November 3, 2014, Kancera reported that a seven-day treatment with KAN0439834 significantly reduced leukemia cells in the lymphatic system in an animal model of a progressive phase of chronic lymphocytic leukemia. The study described in the current press release has showed that KAN0439834 is also effective in a more aggressive phase of chronic lymphocytic leukemia characterized by a genetic change (17p deletion), which makes the disease more difficult to treat. Additionally, the current study showed that a single dose per day of a crystalline formulation of KAN0439834 yielded a significant effect against leukemia and that the used dose schedule (60-80 mg/kg per day) was well tolerated.
The recently submitted national patent application (EP15153394.0), which includes newly invented small molecule ROR inhibitors, will be converted to an international patent application after 12 months. In parallel with the submission of this new patent application, Kancera has managed to postpone the publication of the company's first patent application EP13180941.0. The purpose of this is to extend the time when it is possible to broaden the scope of the patent applications and thus increase their commercial value.
* Reference: Bertilaccio et al.: Xenograft models of chronic lymphocytic leukemia: problems, pitfalls and future directions. Leukemia 27:534-540.2013
About the ROR project
ROR is a family of receptors, ROR1 and ROR2. The ROR receptors mediate signals for growth and survival. Originally ROR was linked to fetal development, but it is now known that they also contribute to cancer cell development and proliferation. Professor Håkan Mellstedt, Kancera´s co-founder and professor at the Karolinska Institute, and his colleagues have shown that Kancera´s ROR inhibitors have the ability to kill cells from tumors in pancreas, and leukemia cells. Professor Mellstedt and his colleagues as well as independent researchers have shown that ROR is also active as a target in prostate, breast, skin and lung cancer.
Because ROR primarily generates a survival and growth signal to tumor cells but is inactive in healthy cells in adults, there are good prospects that a drug directed against ROR hit the tumor much harder than the surrounding healthy cells. Kancera and Professor Mellstedt have shown that inhibition of ROR leads to that cancer cells eliminate themselves by cellular suicide. Against this background, there are reasons to anticipate that a ROR-targeted drug is both safer and more effective than several chemotherapies currently used to treat cancer.
About Kancera AB (publ)
Kancera develops the basis for new therapeutics, starting with new treatment concepts and ending with the sale of a drug candidate to international pharmaceutical companies. Kancera is currently developing drugs for the treatment of leukemia and solid tumors, based on blocking survival signals in the cancer cell and on addressing cancer metabolism. Kancera’s operations are based in the Karolinska Institutet Science Park in Stockholm and the company employs around 10 people. Kancera shares are traded on NASDAQ OMX First North and are held by around 6700 shareholders as of December 30, 2014. Remium Nordic AB is Kancera’s Certified Adviser. Professor Carl-Henrik Heldin and Professor Håkan Mellstedt are board members and Kancera´s scientific advisers.
For additional information, please contact:
Thomas Olin, CEO: Tel +46 735 20 40 01
Karolinska Institutet Science Park
SE 171 48 Solna,Sweden
Please visit the company’s web www.kancera.com