U3 Pharma AG
German start-up U3 Pharma AG is hoping that its focus on, and understanding of, full biological systems rather than simply on chemistry will give it a chance to win the race to find new cancer drugs.
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The $752 million worth of new funds raised by leading European biotechnology VCs over the past three months suggests that Europe's private biotech market is picking up. But these funds won't simply land in companies' laps. Europe's biotech managers, many of whom have raised funds only during boom times, will have to satisfy increasingly stringent criteria to secure their slice of the pie. Yet although late-stage products and critical mass still top some investors' wish lists, selected platform and early-stage groups capable of rapidly advancing a broad range of compounds into the clinic have also found funding.
It has always been popular to start companies around families and subfamilies of targets, banking on the similarity among the receptors to speed drug discovery. But the idea largely hasn't panned out, in part because of the new target risk--sometimes they're not pharmaceutically relevant and sometimes they resist the available chemistries. That's why G-protein coupled receptors (GPCRs) have been so important: they're clearly relevant (many, perhaps most, blockbusters come from this class of receptor) and their unique structure makes them both relatively easy to hit with ligands and likely to do something when hit. But the same structural advantages turn into scientific disadvantages for researchers: they resist screening and other techniques of modern drug discovery. We explore some of the newest approaches to mining this rich vein of opportunity.
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.