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Opko Invests in New Technologies, Raising Questions About the Company's Focus on RNAi

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By Doug Macron

Although it bills itself primarily as an ophthalmic drug and instrumentation company, Opko Health has long been focused on the development of siRNA-based therapeutics, including the wet age-related macular degeneration treatment bevasiranib.

But with the failure of that drug in a phase III trial, Opko has lately been making investments in other technologies in earnest, raising questions about how much longer RNAi will remain as the centerpiece of the company's drug-development portfolio.

Opko was created in early 2007 when Acuity Pharmaceuticals, which originally developed bevasiranib, was merged with privately held ocular disease firm Froptix and publicly traded shell company Exegenics (see RNAi News, 3/29/2007).

At the time, bevasiranib was already in late phase II development and poised to become the first siRNA drug to enter phase III testing. As such, much of Opko's activities were centered around advancing the drug, as well as continuing work on other RNAi programs inherited from Acuity.

In fact, about six months after the merger, Sam Reich, an Acuity co-founder who became Opko's executive vice president of ophthalmics, told the investment community at the UBS 2007 Global Life Sciences conference in New York that the company was "proud and excited about its siRNA portfolio," which was going to remain a "high priority" (see RNAi News, 9/27/2007).

But bevasiranib remained the only siRNA drug in Opko's official pipeline until earlier this year when Opko announced that it had shut down a phase III study of the drug after preliminary data indicated that it was "unlikely" to meet its primary endpoint (see RNAi News, 3/12/2009). Reich has since left Opko.

At the time, Opko Chairman and CEO Phillip Frost said in a statement that although the early data from the trial were "disappointing … the indications of [the drug's] activity are encouraging, and we look forward to fully analyzing the data in the coming weeks." Since then, the company has made no statements regarding bevasiranib and all mention of the drug has disappeared from Opko's corporate website.

An overview of the RNAi mechanism remains on the site, and Opko announced in February the presentation of early-stage data on next-generation siRNAs (see RNAi News, 2/12/2009), but it has been the company's interest in other technologies that has moved to the forefront. This month alone, Opko announced that it had either invested in or acquired technologies related to several non-RNAi drugs.

Specifically, Opko said that it had invested $2.3 million in Sorrento Therapeutics, a privately held firm with a "proprietary platform technology for the generation of fully human monoclonal antibodies." In exchange, Opko acquired roughly one-third of Sorrento's outstanding common shares and took an exclusive license to its antibody library for "the discovery and development of therapeutic antibodies in the field of ophthalmology," according to the company.

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Days later, Opko reported that it had agreed to make an undisclosed investment in Cocrystal Discovery, a private developer of antiviral drugs that was founded last year with $10 million from an investor group led by Opko's Frost.

And that same week, Opko said that it had acquired the worldwide, exclusive rights to "a new platform technology for the rapid identification of molecules that can be useful as vaccines and new drugs, and to create new diagnostic tests."

In a statement, Frost noted that the initial focus for the technology will be on "the development of … quantitative blood tests for several diseases for which none are presently available and for which early diagnosis is difficult." These include Alzheimer's disease, multiple sclerosis, Parkinson's disease, and lung cancer, he noted.

Opko also stated in that announcement that while its key interest is in ophthalmics, it is "developing new technologies for products of use in other areas of medicine, as well."

Amid the new investments and expanding interests, Opko has also hired former Locus Pharmaceuticals CEO Jamie Freedman as its new executive vice president of business development and research and development, possibly signaling a change in direction for the company.

Multiple requests to Opko for comment were not returned.

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