Objective

M4K Pharma, a for-profit drug company that is owned entirely by a charitable trust, is inventing open-access “first in class” therapeutics to enable experimental medicine studies and drug discovery, with a commitment to affordability. Its first drug discovery project is structure-enabled and targets the ALK2 kinase, which has an activating mutation in a rare brain cancer called DIPG. The chemistry plans and progress are open for all to see (www.m4kpharma.com). The project involves a large number of collaborators, including the Ontario Institute for Cancer Research (Canada), Wellcome (UK), Charles River (UK), Reaction Biology (U.S.), Brain Tumour Charity (UK) and several universities (Toronto, Oxford, McGill etc.). M4K Pharma’s IP and business model is predicated on the fact that the various forms of data and market exclusivity protections granted by regulators to newly approved drug products have significant, and under-appreciated, value in protecting drug marketing rights - and thus have significant commercial value. Importantly, unlike business models based on patents, these sui generis forms of intellectual property protection are compatible with real-time sharing of the science, as they are not invalidated by prior disclosures. Thus, M4K Pharma provides a unique vehicle to develop tools and affordable new medicines with a strong IP market position, and also provides a mechanism to share the science for collective scientific, social and economic benefits.