Kindred Spirits in Small Molecule Drug Discovery
This article was originally published in Start Up
Kinetix Pharmaceuticals hoped that a focus on a particular class of targets that shares structural similarities--the protein kinases--would yield small molecules for a variety of therapeutic areas. But in order to realize synergies between multiple research projects in protein kinases, the company couldn't partner the projects out to numerous pharmaceutical partners, each requiring exclusivity and confidentiality. After technological rival Vertex signed a multi-million dollar protein kinase development partnership with Novartis, Kinetix began to rethink its original business model. A $170 million acquisition offer by Amgen Inc. made strategic sense for the start-up.
You may also be interested in...
Thanks to productivity, lower-risk follow-on drugs and access to a Big Pharma balance sheet, Theravance is trying to demonstrate the possibility of getting paid for doing what biotech does best: research.
The low-risk model--don't develop drugs, develop technology and sell it to the drug industry--is on rapid rewind. The latest casualty: Aurora Biosciences, which has given up its lower-risk model of selling technology to throw its lot in with high-risk, high-value Vertex Pharmaceuticals. The moral of this story seems to be that it's better to find a seat at the table in a game with big stakes than simply get paid for dealing the cards.
Pharmaceutical companies face two giant risks; compound risk and target risk. So, despite an abundance of so-called validated targets emerging from genomics efforts, and novel high throughput tools for compound synthesis and screening that yield plenty of hits against each target, at the end of the day, pharmaceutical company productivity, as measured by approved drugs, remains low. Recently, several former big-pharma executives have founded private companies that hope to speed up the time it takes to come up with optimized small molecule leads. Each has staked out a particular niche where it thinks it can do better than big pharma at coming up with clinic-ready compounds. Kinetix Pharmaceuticals and Triad Pharmaceuticals hope to leverage their knowledge of particular gene families to come up with optimized leads; Enanta Pharmaceuticals hopes to morph peptides into drugs with a combinatorial approach to binding pharmacophores, and Sunesis hopes to tackle some of the targets that prove intractable for others, or in which only large molecules have been able to intervene. The new small molecule companies share a vision of drug discovery that is based on targets, rather than diseases.