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Alnylam Plans to File Influenza IND in Second Half of 06; US Government Will Fund Program

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Alnylam Pharmaceuticals this week updated its general timeline for several RNAi-based drug candidates it is developing in-house and with collaborators, and disclosed that it will advance its "pandemic" flu candidate with the help of US government funding.

In a three-hour R&D presentation held in New York this week, Alnylam said it expects to submit an investigational new drug application for an RNAi-based for a pandemic flu candidate "as early as the end of 2006." The company said the drug would be designed to treat and prevent illness brought on by a wide variety of influenza viruses, including bird flu.

In addition, Alnylam said that Dowpharma, Dow Chemical's contract manufacturing services business, "intends" to work with Alnylam to manufacture an RNAi therapeutic for influenza, which would "create the opportunity for domestic production."

The disclosure was part of a broad update Alnylam provided for its ongoing drug-discovery and -development efforts, including RNAi-based candidates for respiratory syncytial virus, influenza, cystic fibrosis, neuropathic pain, spinal cord injuries, Parkinson's disease, and at least three undisclosed alliances with three big pharmas [see accompanying table].


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But industry insiders will likely hone in on Alnylam's RSV and flu updates, which are the farthest along in the developmental cycle than any other of the company's candidate compounds.

'Pandemic' Influenza

In an information packet that was part of its R&D show, Alnylam said it expects to submit an investigational new drug application for its influenza candidate in the second half of next year. The company said that the "final selection of [a] clinical candidate is in progress," and that it expects to begin GMP manufacturing in the first half of 2006.

"We believe that RNAi therapeutics can play an important role in effective preparedness for a global influenza pandemic, providing a new option for the treatment and prevention of highly virulent strains of flu," Alnylam President and CEO John Maraganore said in a statement coinciding with the R&D presentation.

"With initial federal funding, the support of Dowpharma, and our relationships with world-leading academic centers, we are building a strong collaborative network in both the private and public sectors to address a potentially major global health issue," Maraganore added.

Alnylam said the influenza program, which will be developed both internally and with Dowpharma, will look at drugs that could be inhaled or delivered intranasally.

The company said RNAi technology could improve the treatment and prevention of influenza because "optimized RNAi [that] targets highly conserved regions within mRNA [is] essential for viral replication."

Alnylam said its flu drug will be designed to target sequences that are highly conserved across all flu genomes, including those of avian origin such as the H5N1 strain that has grabbed headlines in recent months. The company expects this candidate to have antiviral activity against "any newly emerging strain of influenza that could cause human disease," Alnylam said in the statement.

The company said it believes that such an RNAi drug could be stockpiled in advance of a flu pandemic as a complement to vaccines and other antivirals.


In a three-hour R&D presentation held in New York this week, Alnylam said it expects to submit an investigational new drug application for an RNAi therapeutic for "pandemic" flu "as early as the end of 2006."

Alylam will conduct its influenza research with "initial" funding from the US Department of Defense's Defense Advanced Research Projects Agency. In the R&D presentation, Barry Greene, Alnylam's COO, said the DARPA grant was for $240,000. Additionally, the company said that RNAi therapeutics were "specifically recognized" in President Bush's strategy for combating influenza, which includes a $7.1-billion request for "emergency funding" from Congress [see related article].

RSV

According to Maraganore, Alnylam's plan to develop an influenza product can be traced to its ability begin clinical studies for its RSV candidate, ALN-RSV01. These studies, he said in the statement, "[laid] the groundwork for rapidly developing a therapeutic for flu."

At its R&D show, Alnylam reiterated that it has begun its European phase I trial of ALN-RSV01, and that a US phase I study of the drug is set to begin before the end of the year [see RNAi News, 12/9/2005].

Updating this timeline, the company said it expects to have preliminary phase I data in the first half of 2006, and begin a phase I inhalation study in the second half of 2006.

Alnylam also said it is considering an "experimental infection" human study for 2006, and plans to initiate a phase II trial in naturally infected patients in the first half of 2007.

In September, Alnylam started its ALN-RSV01 program after halting development of its most advanced RNAi therapeutic candidate to date, the age-related macular degeneration treatment ALN-VEG01 [see RNAi News, 9/23/05].

— Kirell Lakhman ([email protected])

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