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AUTM Panel: Platform Techs Best Bet for Deals Between Biopharma, Academia

SAN DIEGO – Biopharmaceutical companies and venture capitalists have become more likely in recent years to seek out and license drug “platform” technologies such as RNAi from universities, non-profit research institutes, and smaller biotechnology companies, a panel of tech-transfer experts said at a conference held here this week.
During an industry trends workshop at the Association of University Technology Managers meeting, experts said that the developing trend is a marked change over past tech-transfer deals, which tended to revolve around one or a few promising small-molecule compounds.
Increasingly, such small-molecule discoveries are too early stage and high-risk for large and even medium-sized biopharma companies to license, the panelists said. However, small molecules become much more attractive if they are linked to a clearly defined and patent-protected method or pathway by which the molecules work or can be discovered.
“Early-stage alliances are a shrinking percentage of all deals in the past five years,” said Mark Edwards, managing director of San Francisco Bay Area-based biotech business consulting firm Recombinant Capital. “We’ve seen an increase in mid-stage deals, where a compound is approaching clinical trials, but that has been at the expense of early-stage deals.”
However, Edwards added, one of the biggest upticks has been in the area of drug platform technologies. “Big technology platform deals are being struck on both a ‘standalone’ and ‘anchor compound’ basis,” he said, meaning that companies are interested in licensing both discovery platforms by themselves or in association with one or a few compounds that are based on the platform.
Panelist Lita Nelsen, director of the technology licensing office at the Massachusetts Institute of Technology, said that companies are more frequently approaching MIT to see “what technologies it has sitting around,” and asking to see platform-based technologies instead of individual early-stage small molecules or even more advanced drug leads.
“When I started hearing this, I almost fell over,” Nelsen said. “It’s a complete 180 from four or so years ago.”
Nelsen also said that there has been a noticeable increase in the number of VCs seeking to fund early-stage university spinouts based on drug platforms at MIT.
In addition, the panelists said that many larger pharmaceutical companies – which have traditionally eschewed licensing directly from academic entities – have become more interested in forging broad-based discovery partnerships with universities and non-profits to exploit platform technologies that can identify promising drug leads.
Merck has been one of the busier companies in this respect, having forged multi-million dollar drug-discovery partnerships in recent years with San Francisco’s Gladstone Institute to develop drugs directed to the ApoE pathway in cells; with Harvard to identify inhibitors of the visual cycle for the treatment of age-related macular degeneration; with Dana-Farber Cancer Institute to tackle cancer genomics; and with the H. Lee Moffitt Cancer Center in the area of personalized medicine.
Panelist Greg Wiederrecht, vice president and head of external scientific affairs, worldwide licensing, and external research for Merck, said that in general, some of the aspects Merck looks for in platform technologies include novel, robust methods to identify and validate targets; formulation and delivery technologies; improved manufacturing methods for chemicals or biologics; and new therapeutic modalities.
Wiederrecht also mentioned “automation at all levels, and data management, interrogation, and sharing” as examples of choice platform technologies.
RNAi is a recent example of a platform technology that Merck and other pharmas are more interested in partnering with academic entities and early-stage biotech companies to develop, Wiederrecht said, adding that it is a technology that “has the potential to change the game.”
Platform technologies besides RNAi of high interest to Merck include imaging technologies, such as those used in PET, SPECT, CT, and MRI, as well as optical reporters of any kind; biologics, such as platforms for identifying and generating monoclonal antibodies and aptamers; delivery systems, especially for biologics like RNAi and viral vectors; and oral, IV, and inhalation drug-delivery systems.

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