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Major licensing deal for novel blood disorder inhibitors

The Australian Cooperative Research Centre for Cancer Therapeutics (CTx) with support from the Wellcome Trust and Cancer Research Technology (CRT, UK), have developed a series of novel compounds that inhibit protein arginine methyl transferase 5 (PRMT5) which have potential application as therapeutics for cancer and non-cancer blood disorders.

CRT, on behalf of the CTx, has licensed the rights to develop inhibitors of PRMT5 to MSD (known as Merck in the US and Canada) in a major license agreement. Under the terms of the licence, MSD will be responsible for research and development, including clinical development, with CRT receiving an upfront payment of US$15 million and potential payments of up to US$0.5 billion for the achievement of development, regulatory and commercialisation milestones. All payments will be shared between CRT, CTx and the Wellcome Trust with the majority being returned to CTx and its Australian research partners.

PRMT5 is a protein with a significant role in regulating other genes, including the tumor suppressor protein p53 which helps protect cells against cancer-causing mutations but is found to be faulty in nine out of ten cancers. High levels of PRMT5 are found in many cancers including mantle cell lymphoma, chronic lymphocytic leukaemia, melanoma, lung and breast cancers, and are linked to poor survival. In addition, PRMT5 has a role in regulating genes involved in the development of blood, and blocking PRMT5 has potential therapeutic applications in non-cancer blood disorders such as sickle cell disease and beta thalassemia.

Designing potent inhibitors of PRMT5 with selectivity and acceptable drug-like properties is key in the discovery of potential therapeutics targeting this protein.

The CTx brought together a multi-institutional collaborative project team, including medicinal chemists, biologists and pharmaceutical scientists, to design new molecules with the right balance of biological activity, selectivity and drug-like properties. The CDCO was an integral part of the project team bringing expertise in assessing the physicochemical characteristics, absorption, metabolism, distribution, excretion (ADME) and pharmacokinetic properties of potential drug compounds.

Dr Warwick Tong, CTx Chief Executive, says: "This is a great result for Australian science and the CRC Programme as a whole and further demonstrates what can be achieved when science and commercialisation capabilities unite."