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In 2005, RNAi Science Advances and Big Pharma Takes Notice; As Usual, More Work Left to Be Done


In the nascent RNAi therapeutics field, getting recognition from well-established industry players has proven to be perhaps the most important piece of the overall business puzzle. Aside from the upfront revenue a big pharma/biotech alliance can bring, the investment community often sees a nod from a large company as one of the best indications of a firm's health since a business partner is privy to scientific and corporate information not available to the public.

Although almost all RNAi drug companies say that they are in negotiations with potential partners, over the past year it has been Alnylam Pharmaceuticals and Sirna Therapeutics that have actually cemented deals with big names in the biopharmaceutical arena — and this follow through has helped maintain the two firms' dominance of the field, both in terms of investor confidence and clinical development progress.


Holding on as king of the RNAi hill is Alnylam, which became the highest profile RNAi drugs developer after being founded in 2002 by some of the biggest names in RNAi research and launching an initial public offering — the industry's first — that netted $26 million for the then-pre-clinical company last year (see RNAi News, 6/4/2004).

"The investment community often sees a nod from a large company as one of the best indications of a firm's health since a business partner is privy to scientific and corporate information not available to the public."

Although Alnylam was not the first to get an RNAi drug into the clinic (see RNAi News, 11/12/2004), this year it persuaded Medtronic that it was the go-to company in field, inking a deal that calls for the companies to work together on developing RNAi/medical device-based treatments for neurodegenerative disorders such as Huntington's disease, Alzheimer's disease, and Parkinson's disease (see RNAi News, 2/11/2005).

More significantly, Alnylam also cinched a broad drug discovery and development deal with Novartis (see RNAi News, 9/9/2005), which agreed to buy a 19.9-percent stake in the RNAi company, worth $58.5 million, in connection with the alliance. Around the same time, Novartis also dropped its opposition to an Alnylam-controlled patent in Europe, leaving only five of the original eight opposing parties still trying to have the patent pulled (see RNAi News, 1/16/2004) and 1/14/2005).

Alnylam also recently said that it has received a $240,000 grant from the US Department of Defense's Defense Advanced Research Projects Agency to fund its early development of an RNAi-based treatment for pandemic influenza. While the US government doesn't qualify as a big pharma, its coffers can be a significant source of revenue. Furthermore, Alnylam's receipt of the DARPA funding marks a small but definite step forward in its effort to edge start-up Galenea out of the flu market. As reported by RNAi News, Galenea, which is working on its own RNAi-based flu drug, had been in discussions to collaborate with Alnylam, but Alnylam ultimately chose to work on its own (see RNAi News, 6/3/2005 , and story, this issue, for further details on Galenea's flu program.)

Subsequently, Alnylam has not only seen its stock climb over 70 percent since the beginning of the year, but the company name has found its way onto the lips of a growing number of equity analysts and into the articles of key financial publications — an important driver of financial performance for a public company.

Additionally, Alnylam has initiated US and European phase I trials of an RNAi therapy for respiratory syncytial virus, and said it plans to file an investigational new drug application for a flu drug in the second half of 2006 — a timeline that, if met, will keep the company well-positioned amongst its peers.


Meanwhile, Sirna, which became an RNAi drugs company in 2002 after reorganizing itself from its former incarnation Ribozyme Pharmaceuticals, has been holding on to the number-two position in part because of its ability in wooing bigger partners — though not with the success Alnylam has had.

So far this year, Sirna has announced only one major collaboration — but it was a significant one. In October, the company reported that it had licensed its phase I age-related macular degeneration treatment to Allergan. Under the deal, the companies will also collaborate on new RNA-based drugs for other ophthalmic diseases, with Sirna providing optimized siRNA compounds against Allergan targets.

Although the upfront cash Sirna received through the agreement, $5 million, pales in comparison with the cash Alnylam secured through its Novartis deal, Sirna stands to receive up to $245 million in clinical milestones should its drug successfully clear all regulator hurdles, as well as royalties, research funding, and manufacturing revenues.

Since signing its deal with Allergan, Sirna has said it expects to move its next clinical candidate, a treatment for hepatitis C, into phase I testing in the second half of 2006 and to sign another collaboration by May.


Though not a drug company, Fisher Scientific subsidiary Dharmacon this year expanded the role of the traditional RNAi research products provider through an alliance with Alcon (see RNAi News, 10/21/2005).

Under this arrangement, the companies will jointly identify ocular disease-related gene targets for potential siRNA drugs. Dharmacon will come up with siRNA drug candidates, and Alcon will handle development and commercialization efforts.

Thus far, no other RNAi oligo and tool company has followed suit, but as the market for such research products becomes increasingly saturated with competition, it seems likely that ones will as they search for new avenues of opportunity.

— Doug Macron ([email protected])

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