Alnylam Pharmaceuticals Inc. and Isis Pharmaceuticals Inc. are pooling their expertise in microRNA (miRNA) to launch a joint venture, Regulus Therapeutics LLC.
"This is certainly in our view a clear case where one plus one equals five," said John Maraganore, president and CEO of Alnylam, in a reference during a conference call to the value created by combining the resources of the two companies.
Cambridge, Mass.-based Alnylam and Carlsbad, Calif.-based Isis will both exclusively license their miRNA intellectual property to Regulus, including the Tuschl III patent. Isis also will provide a license to several hundred patents covering oligonucleotides so that Regulus can develop second-generation antisense compounds to antagonize its miRNA targets. Alnylam will invest $10 million to level the playing field, and the two companies will share equally in future funding.
Unlike other types of RNA, miRNAs do not encode proteins. Instead, the 500 or so miRNAs identified to date are believed to control the expression of up to one-third of all human genes. The mutation or inappropriate expression of miRNA has been linked to cancer, metabolic disease, viral infections, diabetes, cardiovascular disease and inflammation. In vivo studies have indicated that antagonism of mutated miRNA targets may correct an entire disease pathway rather than just a single disease target.
Regulus will begin its miRNA efforts by targeting miR-122 for the treatment of hepatitis C virus. Maraganore said the program could advance into the clinic "over the next couple of years." Beyond that, the company is exploring 60 miRNAs as potential targets in cancer, viral diseases, metabolic disorders and inflammatory diseases.
As programs advance into the clinic, Regulus may develop them internally or seek pharmaceutical partners. Any programs Regulus elects not to pursue or partner will be available to Alnylam and Isis under pre-negotiated terms.
Named for the star in the heart of the Leo constellation, Regulus will be located with Isis in Carlsbad. The company expects to employ approximately 20 researchers within the next two years, while Isis and Alnylam will provide support for administrative functions. Yet the joint venture will be independent, with its own management team, board of directors and scientific advisory board.
The scientific advisory board will be chaired by David Baltimore, a Noble Prize winner and professor of biology at California Institute of Technology. Additional members will include David Bartel of MIT, Scott Hammond of the University of North Carolina School of Medicine, Markus Stoffel of the Swiss Federal Institute of Technology, Thomas Tuschl of Rockefeller University and Phillip Zamore of the University of Massachusetts Medical School.
Baltimore also will serve on the board of directors, along with Maraganore; Barry Greene, chief operating officer of Alnylam; Stanley Crooke, chairman and CEO of Isis; and Lynne Parshall, executive vice president, chief financial officer and secretary of Isis. Regulus' management team has yet to be disclosed.
Alnylam and Isis have plenty of experience working together. The two companies formed a collaboration in 2004 to combine their knowledge in the field of RNAi. (See BioWorld Today, March 15, 1004.)
Jefferies & Co. Inc. analyst Salveen Kochnover called the new joint venture a "novel approach with mid- to long-term promise," but news of the deal didn't stimulate much of a reaction on Wall Street. Shares of Alnylam (NASDAQ:ALNY) rose $1.12 to close at $26.97 on Friday, while shares of Isis (NASDAQ:ISIS) closed at $12.74, unchanged.