Alnylam Pharmaceuticals took another step last week toward its publicly stated goal of making its RNA interference intellectual property available to other drug developers, announcing the launch of the InterfeRx licensing program.
According to the company, the program will facilitate the licensing to third parties the rights to develop RNAi therapeutics under Alnylam’s portfolio of issued and pending patents, and will be overseen by Vincent Miles, senior vice president of business development.
“We’re expecting that most of the licensees will be small biotech companies who would have expertise in a particular target or delivery technology” and want to move into the field of RNAi therapeutics, Miles told RNAi News. “We feel that they would need licenses to the IP that we’re holding.
“The potential for RNAi therapeutics is so large, it’s clear that no one company will be able to develop therapeutics for every target,”Miles added. “So, it makes a lot of sense to do this kind of licensing program.
”Miles declined to specify which therapeutic areas the company would consider granting licenses in, other than stating that the company plans to hold onto rights in areas where “we would expect to form alliances with big pharmaceutical companies. It’s likely that the most important targets or key therapeutic areas are ones that we’re going to retain,” he said.
“The potential of the [siRNA] technology is such that there are many examples of targets that would probably fall outside of our strategic focus and/or our capacity to get to them,” Miles said.
As for what exactly falls outside Alnylam’s focus: “We’ll know it when we see it, in the sense that there are certain targets that are probably on everybody’s mind, and there are others that would be driven by a specific area of expertise of a biotech company,” Miles said. For example, he said, the company might outlicense a newly validated target for a disease Alnylam doesn’t expect to pursue.
Alnylam has, however, signaled its specific interests through partnerships it has formed: The company recently announced that it has struck a deal with the Mayo Clinic to develop drugs that suppress the alpha synuclein gene, which has been found to be overexpressed in people with Parkinson’s disease. The company also signed material transfer agreements with researchers at Rockefeller University who are applying siRNAs against hepatitis C.
Additionally, Alnylam CEO John Maraganore has said in the past that the company is looking at targets such as apolipoprotein B for hyperlipidemia and protein-tyrosine phosphatase 1B for diabetes. (See RNAi News, 10/31/2003).
Doing a Deal
Miles said that the terms of each InterfeRx licensing deal will be hammered out on a case-by-case basis, but that “we would imagine a general structure in which there is an upfront payment, milestone payments as things progress, and royalties. We would always expect to participate in any downstream value that is created,” he said.
However, “the specifics will always depend on the target and the situation of the [licensee],” he added.
Miles said that parties interested in the program can contact him directly, adding that he has already received several inquiries. While he declined to say when Alnylam expects to strike a licensing deal, he said that “a good period of time is required to negotiate these types of license agreements … [typically] between six and 12 months.”
The Tuschl IP
Included in the intellectual property Alnylam is planning on outlicensing are the so-called Tuschl-1 and Tuschl-2 patent applications, both of which are likely to be of great interest to companies eyeing the RNAi-as-therapeutics field.
These patent applications cover inventions made by Rockefeller University researcher Thomas Tuschl. The first, Tuschl-1 (US patent application number 20020086356), describes the use of short interfering RNAs, 21 to 23 nucleotides in length, to induce RNAi in mammalian cells. Tuschl-2 (World Intellectual Property Organization patent application number WO 02/44321) in part describes RNAi using siRNAs with two-to-three nucleotide 3’ overhangs on the end.
For a time, Alnylam was the sole licensee of both these patent applications, in February having exclusively licensed Tuschl-1 from three of its four co-owners — the Max-Planck Institute, the Whitehead Institute, and MIT — and Tuschl-2 exclusively from its sole owner, the Max-Planck Institute. During this time, when Alnylam appeared to be in a position of having a say in who does what with siRNA-based RNAi in mammals, the company made no mention in its public statements of being willing to license its IP to potential competitors.
All that changed, however, when rival Sirna Therapeutics picked up an exclusive license to the Tusch-1 IP from its fourth co-owner, the University of Massachusetts Medical School. (See RNAi News 09/12/03). According to UMass, it held off on giving the license to Alnylam along with the other institutions because it wanted to see the technology made widely available; Sirna agreed to sublicense broadly enough to satisfy UMass.
At the time news of the Sirna licensing deal was announced, Maraganore told RNAi News that a policy of outlicensing company IP in areas outside its interest was “a strategy we’ve had for a long time.” Miles also stated that Sirna has little bearing on Alnylam’s decision to launch InterfeRx. He said that when he joined the company in July, “it was already clear that [a plan to outlicense] was what we were talking about.”
The driving factor behind the timing of the launch of the InterfeRx program, Miles said, was Alnylam’s midsummer merger with German RNAi peer Ribopharma, as well as the recent consolidation of the two companies’ IP estates, which marked “a sensible time to start this [outlicensing] exercise.”
As for Sirna, Bharat Chowrira, the company’s vice president of legal affairs, recently told RNAi News that a pricing structure had been established for sublicensing the Tuschl-1 IP, but he declined to comment on it. He had also said that Sirna hoped to sign at least one deal before year-end, and added in an email this week that the company is “working on [the] matter.”