Aventis/Regeneron: Validation by Association
In the past, drug companies paid big fees up front only for drugs that had demonstrated clear clinical validation. But in paying $80 million cash up front for Regeneron's VEGF Trap cancer candidate--the highest up-front fee ever for a Phase I--Aventis is relying on data generated not by the compound in question, but by another product targeting the same receptor.
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The fundamental business model for platform technology companies hasn't changed: Build the platform, and apply the resulting discovery capability to internal product development while leveraging its value to raise cash or barter for resources. But investors today prefer companies that can articulate a short route to proof-of-concept data,. And unlike 6-10 years ago, more start-ups are now being formed around clinically savvy drug development teams that are better able to select alternative paths to near-term opportunities--be it in-licensing or even M&A--that allows companies greater flexibility.
Despite the success of Gleevec, developing drugs that inhibit complex signaling pathways, while simultaneously trying to understand the biology around a drug's target, remains a challenge. Without biomarkers to help establish dosing and identify likely responding patients, clinical development of targeted cancer drugs will remain challenging. AstraZeneca's recent experience with Iressa bears this out. It's likely that single markers will not be sufficient to stratify patients by their tumor types; rather, patterns of gene and protein expression will be required. The technology to take these measurements is making its way from academia to industry, but the process is slow, and needs encouragement and better coordination between academia, regulators, and industry. Meanwhile, clinical trials themselves remain the best target validation tools. When all is said and done, efficacy is the best biomarker.
After more than a decade of anticipation, pharmas have now clearly recognized the value of antibodies as therapeutics. And biotechs can offer discovery tools and development expertise.