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Alnylam Licenses RSV Drug Rights to Cubist, Maintains Cash Guidance and Eye on Alliances


Alnylam Pharmaceuticals last week announced that it has licensed the commercialization rights to its phase II respiratory syncytial virus drug ALN-RSV01 for all markets outside of Asia to Cubist Pharmaceuticals.

At the same time, the companies — which both have roots extending to two other Bay State biotechs, Millennium Pharmaceuticals and Biogen Idec — will collaborate on follow-on RNAi-based RSV drugs currently under preclinical development at Alnylam.

Separately this week, Alnylam also said that it remains on track to meet its previously stated goal of ending 2008 with more than $500 million in cash and expects to sign at least two new industry partnerships during 2009.

According to a Cubist official, partnering with Alnylam gives the company a new technological angle from which to approach the RSV field, but is not signal that it plans to jump into the RNAi drugs space.

"Cubist made this deal because of our specific interest in the need for RSV treatment," Eileen McIntyre, senior director of corporate communications for Cubist, told RNAi News in an e-mail this week.

As a company focused on developing anti-infectives for acute care settings, Cubist is "interested in building our pipeline in a way that leverages our development and commercialization expertise," she wrote. "The unmet need/potential commercial opportunity [of RSV], plus our high scientific interest in the [ALN-RSV01] program, make this a good fit for us."

That said, the company has "no plans today … to get into RNAi outside of this," McIntyre added.

To Alnylam, the deal with Cubist represents "additional critical mass to the advancement of ALN-RSV01 and/or second-generation RSV-specific RNAi therapeutics … which we aim to advance through ongoing preclinical studies toward potential clinical studies as early as 2010," Alnylam CEO John Maraganore said in a statement. "The partnership also provides Alnylam with yet additional financial flexibility to invest beyond RSV."

Under the terms of Alnylam's latest deal, it will receive $20 million upfront from Cubist for the commercialization rights to ALN-RSV01 worldwide except for Asia, where the drug has already been licensed to Japanese drugmaker Kyowa Hakko Kogyo (see RNAi News, 6/19/2008).

In North America, the deal is structured as a 50/50 joint-development and -profit share arrangement. In other markets outside of Asia, Cubist will handle the drug's development in exchange for milestones of up to $82.5 million and double-digit royalties to Alnylam.

Upon the achievement of undisclosed development milestones, Alnylam has the option to convert the North American co-development and profit-share portion of the deal to a royalty-bearing license with development and sales milestones.

ALN-RSV01 is currently in a phase II trial in adult lung transplant patients naturally infected with RSV (see RNAi News, 4/10/2008). In November, Alnylam said that it would push back the start of a planned phase II study of the drug in a pediatric population — the drug's primary market — until the completion of the lung transplant trial (see RNAi News, 11/21/2008).

A Shared History

In partnering on ALN-RSV01 with Cubist, Alnylam's management is continuing a long-standing practice of maintaining close ties with those in the industry they have worked with before — in this case, with familiar faces from Millennium Pharmaceuticals and Biogen, which later became Biogen Idec.

From the start, Alnylam was created around a core team from those two companies. Before joining Alnylam in 2001, Maraganore was Millennium's senior vice president of strategic product development, while Alnylam President and COO Barry Greene joined the RNAi shop a year later after serving as Millennium's general manager of oncology.

Later on, Alnylam added a number of staffers who had previously worked for Biogen Idec including Antonin de Fougerolles, Alnylam's vice president of research for immunology, metabolic, and viral diseases; David Konys, Alnylam's vice president of manufacturing; Victor Kotelianski, senior vice president at Alnylam; Stuart Pollard, Alnylam's vice president of scientific and business strategy; Dinah Sah, Alnylam's vice president of research for central nervous system diseases and oncology; and Akshay Vaishnaw, Alnylam's senior vice president of clinical research. Additionally, Alnylam co-founder Phillip Sharp was a co-founder of Biogen.

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And those associations have been apparent in several of the deals Alnylam has struck. In May, Alnylam announced that it had signed a deal giving Japan's Takeda Pharmaceutical non-exclusive access to its RNAi intellectual property and technology in exchange for up to $1 billion in upfront and technology-transfer payments, milestones, and royalties (see RNAi News, 5/29/2008). That same month, Takeda closed its acquisition of Millennium.

And in late 2006, Alnylam said it would begin working with Biogen Idec to develop RNAi treatments for progressive multifocal leukoencephalopathy, an opportunistic viral infection of the brain that is a complication of treatment with Biogen Idec's multiple sclerosis drug Tysabri (see RNAi News, 9/21/2006).

Now, Alnylam has inked its deal with Cubist, a company with a senior management team largely comprising Millennium and Biogen Idec alums including President and CEO Michael Bonney, COO Robert Perez, General Counsel Tamara Joseph, and Vice President of Non-Clinical Development Frederick Oleson, who all previously worked from Biogen Idec. Cubist's CSO Steven Gilman and Lindon Fellows, senior vice president of technical operations, both previously worked for Millennium.

According to Cubist's McIntyre, "trust is key to any deal getting done, so the fact that some folks know each other never hurts." Still, the RSV program and RNAi technology were the primary drivers of the deal with Alnylam, she stressed.

"We are excited to have formed this new alliance with Cubist — a like-minded organization that shares our commitment to bringing a novel RNAi therapeutic to patients infected with RSV," Maragenore noted in his statement. "We have tremendous respect for the Cubist team."

Officials from Alnylam were not available for additional comment.

Looking Ahead

This week, Alnylam said that it continues to anticipate ending 2008 with more than $500 million in cash, excluding the $20 million yet to be received from Cubist.

Based on an anticipated non-GAAP cash net operating loss of between $35 million and $45 million, and assuming between $20 million and $30 million in other cash payments including further investments in its microRNA drugs joint venture Regulus Therapeutics, Alnylam said that it will likely end 2009 with more than $435 million in cash.

At the same time, Alnylam expects it will form at least "two additional major new alliances" this year, which could take the form of deals for its technology platform or for specific products, deals between Regulus and other companies, or the formation of new business ventures.

The company also continues to expect it will file an investigational new drug application for a new product in its pipeline, such as the preclinical candidates ALN-PCS, which targets proprotein convertase subtilisn/kexin type 9 as a treatment for high cholesterol; ALN-TTR, which targets transthyretin as a treatment of TTR amyloidosis; and ALN-HTT, an investigational Huntington's disease drug targeting the huntingtin gene.

"Last year, we announced our … RNAi 2010 objectives highlighting key goals that we expect to achieve by the end of 2010," Alnylam's Greene said in a statement (see RNAi News, 1/10/2008). "These included achievement of major breakthroughs in delivery, advancement of four or more RNAi therapeutic clinical programs, and completion of four or more new major business alliances. We're extremely pleased that we are well on track to meet or exceed these objectives."