A bold new business model for drug discovery

Published: March 16th 2021
Dr. Aled Edwards

In a world of skyrocketing drug costs, a revolutionary new business model is being trialed in Canada that promises to spur the pace of drug development and price the results within reach.

The concept will form the basis of Dr. Aled Edwards’ virtual presentation, Open Drug Discovery – A Business Model that can Discover New Medicines and Price Them Affordably, 12 p.m. April 7. The talk is part of the Centre for Genomics Enhanced Medicine (CGEM) Seminar Series, of which Genome Atlantic is a sponsor.

Dr. Edwards, a protein biochemist, based at the University of Toronto, is a leading thinker in the field of chemical biology and drug discovery. He is best known as the founding and current CEO of the Structural Genomics Consortium, or SGC, a charitable open science research organization with university-based sites in Canada, the United Kingdom, Brazil, Sweden, United States and Germany.

Essentially, SGC is a public-private partnership intent on using open science, sharing data and materials fast and publicly, unencumbered by the peer review publishing process, to speed up discoveries of new therapeutic drugs. SGC’s pre-competitive research in structural biology and medicinal chemistry is funded by a range of large pharmaceutical companies, governments, charities, and Genome Canada.  

In addition, Dr. Edwards is a director of Toronto-based M4K Pharma, which also uses open science principles to partner with organizations that contribute their scientific expertise and research services.

In this case, the focus is on developing a cure for DIPG–diffuse intrinsic pontine glioma–an aggressive form of pediatric brain cancer for which there is no effective therapy for the small population of children affected by the disease. 

What makes M4K Pharma so radically different is it combines open science with reliance on regulatory exclusivity as its primary intellectual property and commercial asset instead of patents.

This new business model is attracting interest from government, foundations and individual funders that have different expectations compared with biotech, pharmaceutical companies or venture capitalists, when it comes to return on investment. Because of this difference, Dr. Edwards is convinced that therapeutics commercialized on the basis of open science and regulatory exclusivity mean discoveries can be speeded up, and the resulting drugs can be affordably priced.

Regulatory exclusivity refers to incentives awarded under many governments’ drug approval regimes. These inducements aim to stimulate the discovery of new drugs by shielding the developers from competition.

On the list is data protection for drugs with new active ingredients. This form of regulatory exclusivity effectively blocks competition from generic drug manufacturers. Some governments give regulatory exclusivity to orphan drugs developed for rare diseases, whether or not they contain new active ingredients and there are other forms of regulatory exclusivity as well.

The argument is that these forms of market protection compare quite well with the average length of patent protection, which, unlike regulatory exclusivity measures, can be costly for companies to obtain, defend and extend.

Dr. Edwards and others associated with M4K Pharma expect its success will reinforce their conclusions about the benefits of open science and regulatory exclusivity for quicker development of new affordable drugs.

M4K Pharma has been making headway dealing with DIPG, now the leading cause of death among pediatric patients with brain tumours. The company stepped in to try and come up with a drug therapy because the market for those who need it is too small and unprofitable for conventional pharmaceutical companies to direct research to the problem.

Using open science, M4K Pharma’s collaborators have already identified the ALK2 kinase receptor as a key pathway along with a potential therapeutic approach to exploit it with precision medicine.

Toronto’s Krembil Foundation has already given the given the open science – no patents approach its seal of approval with a $1.5 million donation to set up Medicines 4 Neurodegenerative Diseases, known as M4ND Pharma. The new company’s brief highlights their plan to pursue new genetic drug targets for Parkinson’s disease and Amyotrophic Lateral Sclerosis or ALS modelled on the M4K concept.

The number of attendees at Dr. Edwards’ April 7 virtual presentation is limited. Register here.

[https://www.eventbrite.com/e/virtual-cgem-seminar-series-april-7th-dr-aled-edwards-tickets-142934229127.] Dr. Edwards is a professor at the University of Toronto, and Adjunct Professor at the University of Oxford and McGill University.