When the heads of several key RNAi therapeutics firms came together this week for a focus session on the gene silencing technology at this year’s Biotechnology Industry Organization’s CEO and Investor Conference in New York, a discussion over intellectual property laid bare the foundations of a potential IP conflict between Acuity Pharmaceuticals and Sirna Therapeutics.
Acuity is currently developing an siRNA-based drug, Cand5, for age-related macular degeneration. The company’s core patent application covering Cand5, which was published earlier this month, specifically claims the use of siRNAs targeting VEGF and certain related receptors, as well as their use in fighting ophthalmic disorders (see RNAi News, 2/6/2004).
“We have a patent [position] that’s blocking in the field of anti-angiogenesis in general, and more specifically, in the ophthalmic areas for anti-angiogenesis,” Acuity CEO Dale Pfost said during the RNAi session. “Our team was very ophthalmically focused and very early on reduced [its patent claims] to practice in a very clinically relevant fashion.”
Acuity has already had pre-investigational new drug application discussions about Cand5 with US regulators, and is expecting to file an IND in the fourth quarter this year. Pfost told RNAi News last week that a phase I trial of Cand5 could potentially start before year-end (see RNAi News, 2/20/2004). On the sidelines of the conference, Pfost described the overall RNAi IP space as extremely murky, with broad claims about the technology being made by numerous parties. But Acuity, he said, is in a very clear and well-defined place since its key patent application provides specific data supporting the technology’s use as a human therapeutic.
Despite describing Acuity’s IP as “having the ability to block others” in the field, Pfost shied away from answering questions about the possibility of Acuity filing litigation against perceived infringers. He did say, however, that such a scenario was something that would not likely come up for many years, when products near commercialization, and added that the company’s first priority is developing Cand5.
Pfost also noted that discussions have occurred with appropriate companies about the IP situation, but he declined to comment further.
Acuity’s so-called “blocking” IP would appear to put the company at loggerheads with Sirna, which is developing its own siRNA drug for AMD. Sirna CEO Howard Robin said during a presentation that the company has scheduled a pre-IND meeting with the US Food and Drug Administration next week, and is on track to file an IND for its AMD therapy this year.
But Sirna is confident in its IP position, which it touts both in presentations and on its corporate website as dominant. Bharat Chowrira, vice president of legal affairs at Sirna, told RNAi News during the BIO-CEO conference that Acuity will probably be the one found lacking necessary IP.
Firstly, Chowrira said that Acuity is in need of a license to a key piece of IP known as the Tuschl-1 patent application, which describes the use of short interfering RNAs — 21 to 23 nucleotides in length — to induce RNAi in mammalian cells. Sirna and peer Alnylam Pharmaceuticals are the only companies to have rights to the Tuschl-1 patent application (aside from CytRx, which licensed the IP in the areas of obesity, diabetes, cancer, and ALS) and both have ongoing programs designed to facilitate the outlicensing of this IP (see RNAi News, 12/19/2003 and 9/12/2003).
Chowrira also pointed out that Sirna owns a number of patents and patent applications covering methods of manufacturing RNAs, and that many companies in the RNAi space, Acuity included, may find that their operations are limited without licenses to Sirna’s IP.
Finally, Chowrira said that Sirna has its own IP on VEGF-targeting siRNAs, which the company believes sufficiently covers the field.
The strength of Sirna’s IP, he added, is evidenced by the company’s recent oncology deal with Eli Lilly (see RNAi News, 1/30/2004). Given that VEGF is a major target for cancer therapeutics being developed, he said, Lilly conducted extensive due diligence on Sirna’s IP positions, deeming them solid enough to strike the 18-month deal.
“We’ve put a lot of effort in developing what we think is a very broad and powerful intellectual property base,” Robin said during the RNAi focus session. “If you look at the initial investors in Sirna … they put a lot of time in vetting the IP, Lilly put a lot of time into vetting the IP, so it gives me some comfort level that a number of independent third parties have focused on this.”
Robin concluded, however, on a diplomatic note. “There’s a lot of room in the siRNA community for a number of different companies and a number of different drugs,” he said. “In the end, no product is going to be kept off the market because of an IP issue — it’s just a matter of who’s going to pay who[m] [for a license] and who’s going to get the most amount of riches on it.”