CB-103

Project "CB-103" data sheet

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 Partnership

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 Project objective

T-cell Acute Lymphoblastic Leukemia (T-ALL) is an aggressive NOTCH-dependent cancer that affects the blood and bone marrow especially in children aged 0-14 years, and currently has no effective cure. T-ALL accounts for about 25% of the total cases of Lymphoblastic Leukemia (ALL); additionally, an estimated 250,000 people are afflicted by NOTCH-dependent cancers every year. Chemotherapy, the standard-of-care treatment for T-ALL, can reduce tumor growth, but it is ineffective at killing the cancer stem cells that drive tumor progression. Moreover, it can lead to adverse toxicological effects as a result of off-target toxicity, as the drug cannot discriminate between healthy and cancerous cells. Small molecule drugs help to solve this issue thanks to their high efficiency in targeting tumor cells. Cellestia may have found the answer. Its novel approach targets a tumor development pathway, the NOTCH, which play crucial oncogenic roles in tumorigenesis, tumor expansion and survival. By inhibiting the NOTCH signaling in its most downstream part, Cellestia’s small molecule compound, CB-103, blocks the problem at the roots (i.e. specifically at the level of the NOTCH transcription complex) stopping cancer cells growth and survival. Pre-clinical results show CB-103 to have a high therapeutic index relative to conventional cancer drugs. Most importantly, it significantly prolongs survival rates (median improvement of 75%) with no adverse side effects or dose-limiting toxicity (DLT). Results indicate it may have benefit for other cancers too – any in which NOTCH pathway is highly activated, including forms of leukemia and other cancers such as lung, breast and colorectal. The Phase 1 project will be focused on establishing a complete and scalable supply chain, verifying the business model and commercialization strategy, planning new collaborations and partnerships, including the ones to implement the next clinical trials stage.