Washington Editor

Alnylam Pharmaceuticals Inc. and Novartis AG are taking a tag-team approach to pandemic influenza.

The early stage biotech company and the large pharmaceutical firm formed a new collaboration to develop RNAi therapeutics in this space, which continues to garner attention these days. Among latest developments, the H5N1 bird flu has moved into Africa and Western Europe.

RNAi represents an opportunity for a whole new class of drugs "to treat diseases in a different way," explained John Maraganore, Alnylam’s president and CEO. Relative to combating various flu strains in particular, including H5N1 and its potential variants, he spoke to BioWorld Today about the importance of "harnessing the RNAi pathway" to destroy viral RNA.

"New approaches are urgently needed," he said of developing antiviral products for pandemic flu, in addition to vaccines. An RNAi therapeutic could stop the virus by incapacitating several genes, and the partners believe such drugs could be adapted to new strains as they surface.

Alnylam’s deal with Novartis is in addition to their previous agreement from six months ago, a broadly focused RNAi agreement that could be worth more than $700 million, and taps into Alnylam’s existing pandemic flu program that was divulged just months ago. Those efforts already have earned U.S. government money for the Cambridge, Mass.-based company, and Maraganore said the new commercial agreement "brings substantial support" in the form of "significant funding."

Alnylam in December received an initial government grant for the pandemic flu program from the Department of Defense’s Defense Advanced Research Projects Agency (DARPA) to focus on developing RNAi therapeutic targeting sequences both specific for particular strains and conserved across all flu strains, including those of avian origin. The seedling grant, worth $250,000, potentially represents the first of further government funding for the program, as Maraganore said Alnylam has "active requests" for additional public support.

The RNAi therapeutic is being developed to have antiviral activity against any newly emerging strain of influenza capable of causing human disease and leading to a pandemic, including any H5N1 variant.

In this latest collaboration, Alnylam and Novartis will advance RNAi therapeutics for pandemic flu to initial clinical testing and, if successful, regulatory approval. Maraganore said Alnylam, which is in the process of selecting a lead candidate, expects to be in a position to file an investigational new drug application with the FDA by the end of this year.

The partners are working in concert toward that goal, while Novartis, of Basel, Switzerland, is taking the program’s lead outside the U.S.

Specific financial terms were not disclosed, although Maraganore noted that Alnylam stands to earn "a significant share" of profits. The economic part of the deal, he said, was structured to reflect the nature of the product’s likely markets - government stockpiles. The company made no changes to its financial guidance as a result of the agreement, however.

Rodent studies are ongoing, and Maraganore expressed confidence in identifying a candidate in the planned time frame. After all, Alnylam’s lead program for respiratory syncytial virus (RSV) infection already has an antiviral product, ALN-RSV01, in Phase I. It took 15 months after beginning that program to get into human studies, and that success, he said, "gives us confidence" that Alnylam can create a pandemic flu antiviral, too.

Already, there is a contract manufacturing arrangement in principle for a pandemic flu RNAi therapeutic with Dowpharma, a unit of The Dow Chemical Co. in Midland, Mich. Alnylam’s program also has academic support from researchers at the University of Georgia in Athens, and St. Jude Children’s Research Hospital in Memphis, Tenn.

Alnylam’s relationship with Novartis goes back to last year, when the companies unveiled an agreement in which Novartis bought almost one-fifth of Alnylam. That equity purchase was part of $56.8 million worth of initial payments to Alnylam, giving Novartis a nonexclusive option to integrate Alnylam’s RNAi platform into its internal efforts. (See BioWorld Today, Sept. 8, 2005.)

On Tuesday, Alnylam’s shares (NASDAQ:ALNY) gained $1.42, to close at $15.46.

No Comments