Commercializing first-in-class dctpase inhibitors for treatment of hematological cancers
In our ERC grant GEnetic NEtworks as a tool for anti-CAncer Drug Development we used siRNA screening and genetic
networks to identify dCTPase being involved in DNA repair and replication. Further studies revealed that dCTPase plays a
role in the degradation of nucleoside analogues used in cancer treatment. We then used an RNAi approach to validate
dCTPase as a target to improve nucleoside analogue therapy. The dCTPase protein was purified and we set up an
enzymatic high-throughput assay to screen >65,000 compounds, which generated hits that inhibit dCTPase
(IC50~1-10μM). Using in house medicinal chemistry we developed TH1217, a low nM potent and selective dCTPase
inhibitor with favourable pharmacokinetic properties. TH1217 synergistically induces apoptosis and cell death in
combination with cytidine analogue treatment in cancer cells in vitro and in vivo, but shows no increased toxicity in nontransformed
dividing cells. Here, we want to explore the commercial potential of the dCTPase inhibitors identified in the
ERC grant.
In this programme, we will, with the company Oxcia AB, establish the viability of the business programme using technical
analysis, develop a business strategy and direction, specifically secure IP, perform market analysis, develop a business
plan, manage preclinical development and prepare for clinical trials in collaboration with clinicians and regulatory bodies,
IMPD application to Medical Products Agency and identify and discuss with potential commercialization partners and
funding agencies to support cost of clinical trials. We have a non-profit foundation that owns our IP rights in an effort to
secure long term support for translational science aimed at bringing new therapies to patients. In our planned business
model, we start a new company that holds an exclusive license to the IP from the foundation which is used to develop the
overall business programme and attract investments.