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Alnylam Unveils New Vaccine Initiative, Signs GSK as First Partner

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By Doug Macron

Alnylam Pharmaceuticals this week unveiled an initiative to apply its RNAi technology to the production of vaccines and announced that GlaxoSmithKline will be its first partner for the platform.

The RNAi drug developer, which disclosed its third-quarter financial results this week, also provided updates on some of its pipeline programs and reported a widened net loss during the three-month period amid lower revenues.

The new vaccine effort, dubbed VaxiRNA, focuses on silencing genes related to the growth of viruses in vaccine-manufacturing systems such as cell cultures and chicken eggs, Alnylam CEO John Maraganore said during the firm's earnings conference call this week.

“We purposely kept this effort stealth over the past couple of years to advance our data and our IP estate,” he said. “Our goal in this effort is to increase the production of virus particles that are used in the manufacture of vaccine products.

VaxiRNA stems from work conducted by Alnylam Biotherapeutics, a business unit launched in late 2009 to leverage the company's RNAi expertise to “improve the quantity and quality of biologics-manufacturing processes using mammalian cell culture,” Alnylam said at the time (GSN 11/19/2009). Generally, the biotherapeutics effort focuses on Chinese hamster ovary cells, a workhorse cell line used in the biologics industry.

“In the case of vaccine production, we're targeting genes in either cell-culture systems or even in the egg, which is another manufacturing vessel used for … certain vaccines … [to silence] key inhibitory pathways involved in controlling virus replication,” Maraganore noted during the call. “By targeting those types of genes — and we have a proprietary set that we've identified in our work — we're able to augment virus production.”

A “very significant objective” for vaccine producers, he said, is the ability to increase the productivity of cells so that the “viral titer that is generated and ultimately used to purify the vaccine components is as robust as possible.”

As an example of when certain vaccines are in short supply because the “viral titers that are provided to the manufacturers may not grow well in cell-culture systems,” Maraganore pointed to influenza vaccine production — an area of focus for GlaxoSmithKline.

“This approach could significantly change that whole landscape of growing viruses,” he said, adding that the opportunities for VaxiRNA are especially compelling because of the “relatively short development-cycle time for vaccines.

“That lends itself very nicely to potential opportunities" for this technology to be more rapidly commercialized, Maraganore said.

While Alnylam did not provide specific data related to the VaxiRNA platform, it has managed to secure interest from GlaxoSmithKline, which has more than 30 vaccines on the market.

Under that deal, which initially focuses on flu vaccine production in cell-culture systems, the British drug maker will provide undisclosed funding and milestone payments to Alnylam for access to its RNAi technology.

If the technology is successfully used in the manufacture of a commercial product, Alnylam will receive certain payments on unit product sales, the company said.

Alnylam has also granted GlaxoSmithKline options to use the VaxiRNA approach for two additional, unnamed vaccine products.

During the conference call, Alnylam officials also updated the company's progress with its RNAi drug programs.

The first candidate from the company's transthyretin-mediated amyloidosis program, called ALN-TTR01, is currently in a phase I study, data from which remains on track for presentation later this month at the International Symposium on Familial Amyloidotic Polyneuropathy, CMO Akshay Vaishnaw said.

Meanwhile, an investigational new drug application for a second-generation version of the drug, which uses a proprietary lipid nanoparticle delivery system, is expected to be filed by the end of this year.

Phase I human proof-of-concept data on the company's hypercholesterolemia drug ALN-PCS will be released sometime this quarter, and data from a phase IIb trial of the company's respiratory syncytical virus therapy ALN-RSV01, which is being tested in adult lung transplant patients, is slated for presentation in 2012.

Alnylam also expects to file INDs for its refractory anemia treatment ALN-HPN and its Huntington's disease agent ALN-HTT next year, with an IND for its earlier-stage hemophilia drug ALN-APC coming sometime in 2013.

Vaishnaw added that the company remains on track to announce another new pipeline program during the fourth quarter.

Q3 Financials

For the three-month period ended Sept. 30, Alnylam's net loss rose to $13.2 million, or $0.31 per share, from a year-ago loss of $9.6 million, or $0.23 a share.

Revenues fell to $20.8 million from $27.7 million the year before, while research and development spending shrank to $24.3 million from $27.5 million.

General and administrative costs edged up slightly to $9 million from $8.9 million year over year, and Alnylam recorded an equity loss of $500,000 during the quarter related to its microRNA drugs joint venture Regulus Therapeutics.

At the end of the third quarter, Alnylam had $286.2 million in cash, cash equivalents, and marketable securities. It said it expects to have more than $250 million in the bank at the end of 2011.


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