real target
TRANSCRIPT
Kholodenko Roman, phD, IBCh RAS.
[email protected] ; +7 903 221 02 89
RealTarget: New approach for immunotherapy of GD2-positive
tumors
ProblemAccording to the WHO about 500 thousand new cancer patients were diagnosed in Russia in 2012. In Russia five-year survival rate of cancer patients does not reach 43%. (USA – 62%).
Most of them are solid tumors that are difficult to treat with standard methods.
Мen:
Statistics of occurrence of cancer in Russia (GD2-
positive):
Deaths from cancer in 2012:
1
GD2-positive tumors are about 10% of all cancers, including neuroblastoma, small cell lung cancer, melanoma, glioma, number of sarcomas, and retinoblastoma.
Mortality 10 000 p **:
Market analysis
2
IMS Health: the global market of anticancer drugs ~ $ 66 billion in 2013,
CAGR about 12-15%The Russian market is $ 0.55
billion (retail market) + $ 0.74 billion (DLO).
CAGR 10%-16%.
In Russia we focus on the segment of the market about
$ 60 million.
Our segment is therapy of particularly dangerous tumors including neuroblastoma in children, small cell lung cancer and melanoma in adults. In Russia immunotherapeutic approaches for these cancer are absent. Modern foreign immunotherapies based on full length antibodies have serious limitations.
SolutionOur project aims to create an effective drug for the GD2-positive tumor treatment, which action is based on the functional properties of ganglioside GD2, which serves as receptor
of tumor cell death.* Our approach is to use modified fragments of GD2-specific antibodies devoid of whole immunoglobulin molecules drawbacks. Effective anti-tumor effect will be achieved through induction of cell death upon binding of antibody fragments with ganglioside GD2 without activating CDC and ADCC. The patented drug (PCT) will have lack of side effects associated with hyperactivation of the immune system due to the absence of Fc-fragment. High penetration of our drug will be developed due to the smaller size compared with whole antibodies.Modified fragments of GD2-specific antibodies will be highly effective in
targeted elimination of GD2-positive tumor cells without affecting the healthy cells.*Doronin II, Vishnyakova PA, Kholodenko IV, Ponomarev ED, Ryazantsev DY, Molotkovskaya IM, Kholodenko RV. // BMC Cancer.
2014;14(1):295
GD2-specific fragments of monoclonal antibodies
+
3
GD2-positive tumor cell
Core team
4 IBCh RAS
Cell biologist, is an expert in the field of immunology with 15 years experience. Senior Researcher of laboratory of lipid modulators of the immune system.
Expert in the field of biomedical cell technologies, an immunologist. Familiar with the various stages of drug development from early studies to starting clinical trials.
Assistant Professor, head of laboratory of The Chinese University of Hong Kong, Faculty of Medicine, School of Biomedical Sciences CUHK.
PhD Eugene D.
PonomarevCSO
PhD Roman V.
KholodenkoCEO
PhD Irina V.
Kholodenko
CFO
Specialist in immunology and immunotherapy of oncology. Familiar with the various stages of drug development from early research to the start of clinical trials.
Igor I. Doronin
CTO
Publications in:Nature Medicine, BMC (Cancer, Cell Biol., Biotechnol.), Journal of Immunology, Journal of Immunology Methods, PLOS ONE, Neurochemical Research, Cell Proliferation etc.
What do we need
5
Choosing the most
effective ligand:2016 г.
Preclinical trials: 2017-
2018 г.
Phase I, Phase II (A and B) of
clinical trials: 2018-2022 г.
M&A / license sale.
Based on our results, evaluation of published data and descriptions of
used/developed modified antibody-based drugs, we identified the main characteristics
of the prototype drug effective in the treatment of GD2 positive tumors:
• recombinant modified protein;• ligand of ganglioside GD2 directly induces cell death of tumor cells;• the molecular weight of 45-60 kDa.
We need investments and partners.
