West Coast Editor

For the second time in about a week, the feds tapped a biotech firm for research into hemorrhagic fever virus, including Ebola, and Alnylam Pharmaceuticals Inc. stands to gain $23 million over the next four years from the anti-terror contract.

Cambridge, Mass.-based Alnylam is putting its expertise in small interfering RNAs to work on the project, and establishing Alnylam Biodefense, through which the firm plans to build a platform for RNAi drugs against potential bioterrorism.

"[Defense experts] do believe Ebola is a potentially weaponizable virus," said Zachary Zimmerman, director of external alliances for Alnylam, who was instrumental in gaining the grant. "You can imagine, if it's able to be aerosolized and spread, it could be devastating."

Some reports suggest an aerosol already has been made, and the government lists Ebola as a "Category A" potential threat, one of the worst. Funders in Washington are particularly interested in platform technologies such as RNAi that can be used not only for defense, but also for public health threats such as pandemic influenza (against which Alnylam also has a drug candidate), Zimmerman said.

Once Alnylam started with the flu effort, "it was very clear to us that the major customer, if not the sole customer, would be the federal government," he said, and Alnylam started investigating other grant possibilities. The latest money comes from the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health.

The Alnylam Biodefense initiative "is offering us near-term product opportunities through government stockpiling contracts," Zimmerman said. "And what we can learn from this [Ebola] program, we'll be able to transfer to other programs."

Earlier this month, Protiva Biotherapeutics Inc., of Vancouver, British Columbia, got $1.4 million of a $3.6 million grant from the U.S. Defense Threat Reduction Agency, and will use the company's Stable Nucleic Acid Lipid Particle technology to develop a therapy the likes of Ebola and Marburg viruses.

Ebola is particularly fearsome in bioterror because of its quick onset and horrific symptoms, such as bleeding from orifices and destruction of internal tissue. The virus, named after a river in Africa where it was first found, kills most of its victims. Outbreaks are believed to start by contact with infected animals.

Others investigating Ebola drugs include AVI BioPharma Inc., of Portland, Ore., which is preparing for a Phase I trial with its Neugene antisense product, AVI-6002, and San Diego-based Vical Inc., recently reporting positive Phase I data with a vaccine administered using Vical's DNA delivery technology.

Alnylam also made news this month by way of a potential $56 million deal with Biogen Idec Inc., also of Cambridge, to find RNAi-based drugs for progressive multifocal leukoencephalopathy, the typically fatal brain infection linked in rare cases to the use of Biogen's multiple sclerosis drug Tysabri (natalizumab). Last year, Biogen and partner Dublin, Ireland-based Elan Corp. plc voluntarily withdrew Tysabri after two patients died from PML, but limited quantities of the drug have since been allowed back on the market. (See BioWorld Today, June 6, 2006, and Sept. 22, 2006.)

The RNAi approach in biodefense also has the advantage of speed, from lead candidate to drug development, Zimmerman said.

"Small molecules typically take between a year and two years to get to a lead compound," he said, whereas Alnylam's drugs can go from project initiation into humans in 15 to 18 months.

"We know pandemic flu is on the horizon," Zimmerman said. ALN-FLU01 for flu is partnered with Basel, Switzerland-based Novartis AG. The companies hope to file an investigational new drug application for ALN-FLU01 by the end of this year. Alnylam's ALN-RSV01 for respiratory syncytial virus is undergoing Phase I study. (See BioWorld Today, Feb. 22, 2006.)

Alnylam's stock (NASDAQ:ALNY) closed Thursday at $14.48, down 2 cents.

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