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Releasing 2006 Guidance, Alnylam Stays On Track as CytRx Continues to Move from RNAi


Alnylam Pharmaceuticals released this week details about its product-development and business goals for 2006, announcing, among other things, that it will add an additional RNAi-based drug to its formal pipeline and release non-human primate data from its systemic RNAi program.

Also this week, early RNAi mover CytRx released guidance for the year, showing that it continues to distance itself from the gene-silencing technology as a therapeutic modality by focusing on its newer small-molecule drug and DNA vaccine programs.


Currently, Alnylam has two programs in its development pipeline: its RSV program, which is examining an intranasal siRNA drug in phase I trials in both the US and Europe; and its pandemic influenza program, which is expected to yield an investigational new drug application before the end of 2006.

In the discovery-phase of development are programs for cystic fibrosis and neuropathic pain, as well as spinal cord injury in collaboration with Merck, and Parkinson's disease and Huntington's disease with Medtronic.

The company said this week that it "intends to announce an additional development candidate in the second half of 2006," but did not specify whether this new candidate would fall under its program developing directly administered RNAi drugs or its systemic RNAi program.

The company said this week that it "intends to announce an additional development candidate in the second half of 2006" to join RSV and flu, but did not specify whether this new candidate would fall under its program developing directly administered RNAi drugs or its systemic RNAi program.

Alnylam did note that it intends to "continue its efforts to develop systemic RNAi therapeutics and expects to present systemic non-human primate data in the first half of 2006," either at a conference or in a peer-reviewed publication.

Alnylam reiterated its plan to file an IND on a pandemic flu therapy "as early as the end of 2006," and noted that it expects to obtain additional funding for this program during the first half of the year. In December, the company reported that it had secured a $240,000 grant from the US Department of Defense's Defense Advanced Research Projects Agency for its flu program (see RNAi News, 12/16/2005). It was not clear whether the additional funding would come from a public or private source.

The company also reiterated that it expects to launch a phase I trial of an inhalable version of its RSV treatment in the second half of 2006. It added that it will "evaluate the possibility of conducting an experimental infection clinical study in the second half of 2006," which would "enable initiation of a phase II trial in naturally infected RSV patients in the first half of 2007."

From a business standpoint, Alnylam said it anticipates receiving $15 million in alliance-based funding during 2006, and that it expects to grant five new licenses to its intellectual property during the year, bringing to 22 the total number of such deals the company has inked since 2003.

Alnylam noted that it expects to finish 2006 with more than $50 million in cash.

An Alnylam spokeswoman told RNAi News that company officials were not available for comment.


Although it was one of the first companies to embrace the use of RNAi as a potential therapeutic, CytRx's interest in the field has seemingly waned lately as it focuses on its other drug programs.

In line with this trend, the company reported this week that it expects to begin enrolling patients in a phase IIa study of the small molecule arimoclomol for amyotrophic lateral sclerosis in the first quarter of this year, with a phase IIb study beginning in the third quarter. The company also said it expects results from a phase I trial of its DNA and protein vaccine for HIV in the second quarter of 2006.

Absent from its 2006 guidance was any specific mention of its RNAi drug programs, despite the company's announcement in September that it had identified an siRNA drug candidate for type II diabetes and obesity (see RNAi News, 9/9/2005).

In its guidance, the company did note that it "hopes to move a lead candidate from its RNAi therapeutics drug development program ... through the development phase toward an IND submission," but provided no timeline for such an effort.

In November, CytRx President and CEO Steven Kriegsman told RNAi News on the sidelines of the Rodman & Renshaw Techvest 7th Annual Healthcare Conference in New York that the company was considering spinning out a separate, publicly traded, pure-play RNAi drugs firm in order to focus on its nearer-term small-molecule and DNA vaccine work (see RNAi News, 11/11/2005).

Officials from CytRx were not available for comment as of press time.

- Doug Macron ([email protected])

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