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Alnylam Provides Clinical, Financial Goals for 2010, Reiterates Previous Guidance

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Alnylam Pharmaceuticals this week released an update for its key financial and clinical goals for 2010. However, the guidance was primarily a confirmation of previous estimates.

"We are … excited about the next 12 to 18 months as we … fulfill the RNAi 2010 goals that we described two years ago," Alnylam President and COO Barry Greene said in a statement. "Over this period, we expect to have four RNAi therapeutic programs in clinical development … providing us with the opportunity to demonstrate human proof of concept for multiple programs."

Alnylam noted that the clinical programs could include both in-house drug-development efforts, as well as ones that are part of 50/50 alliances or handled by its microRNA therapeutics joint venture Regulus Therapeutics.

Alnylam said that it continues to expect it will advance its siRNA-based respiratory syncytial virus therapy, ALN-RSV01, into a new phase IIb trial during the first quarter of this year in adult lung transplant patients infected with the virus.

In July, Alnylam released data from a phase IIa study of the drug, also in RSV-infected adult lung transplant patients, showing that ALN-RSV01 was safe and well tolerated (see RNAi News, 7/23/2009). At the time, the company also said that the data indicated a certain level of efficacy, but this demonstration was less robust than many industry watchers had expected.

Tellingly, Cubist Pharmaceuticals, which is Alnylam's partner on the RSV program worldwide except for Asia, later decided to hand back ALN-RSV01 to Alnylam and instead focus on a next-generation molecule, ALN-RSV02 (see RNAi News, 11/12/2009).

But Alnylam has remained committed to ALN-RSV01, and this week the company said that the planned phase IIb study would be conducted to "repeat and extend the clinical results observed in a recently completed Phase IIa study."

Meanwhile, Alnylam said that enrollment in a phase I study of its liver cancer treatment ALN-VSP, which began early last year (see RNAi News, 4/9/2009), continues apace. The trial is expected to enroll an estimated 55 patients with advanced liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement, who are refractory to the standard of care. It will evaluate the systemic drug's safety, tolerability, pharmacokinetics, and pharmacodynamics.

Alnylam said that preliminary data from the study is likely to be released in mid-2010.

In terms of future programs, Alnylam said that it remains on track to move its third proprietary program, an siRNA-based treatment for the hereditary disease TTR amyloidosis, into phase I testing in the first half of this year.

In August, Alnylam announced that it had formally added the program to its pipeline. The move was widely expected after Alnylam Vice President of Drug Discovery Muthiah Manoharan told RNAi News in June that the company deemed the delivery hurdles facing the TTR program to be lower than those facing its other lead preclinical program in hypercholesterolemia (see RNAi News, 6/11/2009).

This week, the company noted that it has already filed the necessarily regulatory applications for the drug, dubbed ALN-TTR.

As for its hypercholesterolemia drug, ALN-PCS, Alnylam said that it expects to move the compound into human trials sometime in 2011. At the same time, Alnylam's program in Huntington's disease, being conducted with long-time partner Medtronic, continues to make its way toward the clinic, the company said. However, Alnylam did not provide any guidance on when that might occur.

On the business side, Alnylam said that it continues to expect that it will forge at least four new alliances before the end of this year, in line with the guidance it provided in 2008. These could take the form of platform deals, product partnerships, or collaborations with Regulus or Alnylam's biologics manufacturing initiative, Alnylam Biotherapeutics (see RNAi News, 11/19/2009).

Financially, Alnylam reiterated its expectation that it will post a cash position of greater than $430 million for 2009, and said it end 2010 with more than $325 million in cash, excluding a potential payment from partner Novartis related to the companies' ongoing alliance, which was just renewed for a fifth year (see RNAi News, 7/23/2009).