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Alnylam Sets Goals for 2010 as It Adds Huntington’s to Pipeline

Alnylam Pharmaceuticals this week provided details on a long-term plan that includes advancing at least four RNAi therapeutic programs into clinical trials, as well as forming four or more new corporate partnerships, by the end of 2010.
Also this week, Alnylam provided new details about its drug-development plans and financial goals for 2008, including the expansion of its pipeline to include a Huntington’s disease drug candidate termed ALN-HTT — a move that further positions the company as a competitor to one-time partner Merck, which is developing its own siRNA-based Huntington’s disease drug through its Sirna Therapeutics subsidiary.
“What we accomplished in 2007 and where we are … in our level of confidence in this technology has now allowed us to look forward in a more distant manner … [to] where we think [our RNAi] technology will go,” Alnylam CEO John Maraganore said during a presentation this week at the JPMorgan Healthcare conference in San Francisco, which was webcast.
Under its so-called RNAi 2010 initiative, Alnylam will focus on “specific and key goals across science, [the] clinic, and business,” he added. These goals include having at least four drug candidates in the clinic, including directly and systemically delivered siRNA, as well as microRNA-based therapeutics.
Currently, Alnylam has one drug in clinical trials: the inhaled respiratory syncytial virus drug ALN-RSV01. During his JPMorgan presentation, Maraganore noted that the company expects to see top-line results from a phase II trial of ALN-RSV01 early in the first quarter, giving Alnylam “the first opportunity to demonstrate human proof of concept for an RNAi therapeutic.”
Assuming the data are positive, Alnylam expects to begin another phase II trial of the drug in adult patients this year.
The company also anticipates filing an investigational new drug application this year for either its liver cancer drug candidate, ALN-VSP01, or its hypercholesterolemia therapy, ALN-PCS (see RNAi News, 11/8/2007).


On the business side of the RNAi 2010 plan, Alnylam expects to “form four new major partnerships,” Maraganore said, noting that such deals could be structured like the company’s deal with Roche, which included non-exclusive access to the RNAi shop’s intellectual property (see RNAi News, 7/12/2007); or its recently renewed alliance with Medtronic, which focuses on developing drug/delivery device combinations for neurodegenerative diseases (see RNAi News, 8/2/2007).


Maraganore said that other possible partnership structures include the out-licensing of overseas rights to its various drug candidates, IP licenses under Alnylam’s InterfeRx program (see RNAi News, 12/19/2003), and deals for miRNA-targeting drugs being developed by Alnylam/Isis Pharmaceuticals joint venture Regulus Therapeutics (see RNAi News, 9/13/2007).


At least two of the four planned alliances are expected to be forged some time this year, Maraganore noted.
As for other goals in 2008, Alnylam said it will publish 10 or more scientific papers related to in vivo efficacy and systemic delivery of RNAi and miRNA drugs.

“What we accomplished in 2007 and where we are … in our level of confidence in this technology has now allowed us to look forward in a more distant manner … [to] where we think [our RNAi] technology will go.”

In addition, Alnylam said that it expects to end the year with more than $390 million in cash and raised its 2007 cash guidance to more than $450 million from previous estimates of more than $435 million.
Hunting Huntington’s
When Alnylam and Medtronic renewed their 50/50 alliance in July, they agreed to focus their initial efforts on Huntington’s disease. In the six months since, the companies have apparently made enough headway for Alnylam to add the effort to its formal drug pipeline.
According to Maraganore, Alnylam has “generated some exciting preclinical data for this program … which demonstrates the type of work we’re doing with Medtronic to understand the distribution of our drug into the brain of larger organisms, therefore modeling what our drug/device combination will look like when used in a human setting.”
Among these data is a demonstration by Alnylam researchers and collaborators at Massachusetts General Hospital that cholesterol-conjugated siRNA, when delivered into the brain of a Huntington’s disease mouse model, could silence the mutant form of the gene responsible for the condition, reduce the neural pathology, and delay the onset of abnormal behavior.
With these data, “we’ve shown not only the ability to silence the huntingtin gene, but also the ability of achieving significant benefits,” Maraganore added.
He did not provide a timeline for when ALN-HTT might reach clinical trials.
With the addition of Huntington’s disease to its pipeline, Alnylam has also put itself in direct competition with Merck, which officially went from close collaborator to rival last year when the companies terminated an alliance (see RNAi News, 9/20/2007) after Merck acquired Sirna (see RNAi News, 11/2/2006).


Sirna began developing a Huntington’s disease treatment in 2005 through a partnership with Targeted Genetics (see RNAi News, 1/4/2005).


Although Merck has made no mention of any of Sirna’s RNAi drug programs since the acquisition closed about a year ago, in May TGen’s President and CEO H. Stewart Parker told investors that she anticipated a clinical trial of Sirna’s Huntington’s disease therapy to begin sometime in 2008 (see RNAi News, 5/24/2007).

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