Private biotech investment and development firm Accelerator announced last week that it has established a new company called Mirina that will develop microRNA-targeting therapeutics based on minor groove binder technology licensed from Nanogen.
”Working with Accelerator to license our technology to this new venture is an exciting new area for Nanogen,” Nanogen CEO Howard Birndorf said in a statement. “Our MGB technology has been widely licensed for research and diagnostic applications. Extending its utility to therapeutics … will provide the company with new opportunities for revenues from non-competing markets.”
The startup is currently in the process of filling out its staff and expects to be operational within a “couple of weeks,” David McElligott, Mirina’s vice president of research and development, told RNAi News this week.
Still, use of the MGB technology with miRNAs is still in the earliest stages, he noted. As such, Mirina has yet to settle on specific indications or disease areas it will pursue.
“Our [initial] milestones are really associated with proof-of-principle demonstration, and that’s what we’re budgeted and staffed to do,” McElligott said. He added that part of that … work would involve generating animal data around the technology, but declined to elaborate.
Originally developed in the 1960s as potential cancer therapeutics, MGBs are peptide antibiotics that non-covalently bind to the minor grooves of nucleic acid duplexes, according to Merl Hoekstra, Nanogen’s vice president of business development.
“Obviously, there are a lot of people playing in [the miRNA drugs] space right now … [and] we don’t tend to do things because it’s sexy to do so; we actually do things that are a little bit more off-focus. But this was an opportunity we saw in a very exciting area where there is a large pharmaceutical partnering interest.”
While they failed as cancer drugs, researchers from Epic Biosciences, now part of Nanogen, later demonstrated that the compounds could be used to stabilize DNA duplexes, Hoekstra said. Nanogen is currently developing the technology for use in molecular diagnostics, and has licensed it to Applied Biosystems for real-time PCR applications.
Hoekstra said that cell-culture studies conducted a couple of years ago by Nanogen suggested that MGBs could also be used to boost the target selectivity and potency of miRNA antagonists.
Since therapeutics is not part of Nanogen’s focus, Hoekstra said he put together a data package on MGBs and began talks with groups interested in licensing the technology. Having worked with one of Accelerator’s scientific consultants in the past, he said he contacted the investment company and a deal was struck for the exclusive rights to the technology for RNA-based therapeutic uses.
Though the MGB technology is unproven for this application, the preliminary data presented by Nanogen was enough to pique Accelerator’s interest, Accelerator CBO David Schubert told RNAi News.
Accelerator’s “calling card is that we invest very early before there is [a significant body of] proof-of-concept data,” he explained. “We are known for investing in things that don’t have a lot of data around them and … [generating] data to create a more compelling investment thesis for follow-on rounds of investment.”
He said Accelerator created Mirina on its own, and that it has closed an undisclosed Series A round of financing led by Accelerator’s built-in series of venture capital investors including Alexandria Real Estate Equities, Amgen Ventures, ARCH Venture Partners, OVP Venture Partners, and WRF Capital.
“Obviously, there are a lot of people playing in [the miRNA drugs] space right now … [and] we don’t tend to do things because it’s sexy to do so; we actually do things that are a little bit more off-focus,” Schubert said. “But this was an opportunity we saw in a very exciting area where there is a large pharmaceutical partnering interest.”
Indeed, some players in the miRNA drugs space have found big pharma receptive to their technology. Earlier this year, Alnylam Pharmaceuticals and Isis Pharmaceuticals joint venture Regulus Therapeutics announced that it had forged an miRNA drug alliance with GlaxoSmithKline (see RNAi News, 4/17/2008). A few months earlier, GlaxoSmithKline took an option to Santaris Pharma’s miRNA-targeting hepatitis C drug candidate SPC3649 as part of a broader antiviral drug alliance (see RNAi News, 12/20/2007).
Although it has yet to strike any deal, Roche has also expressed interest in miRNA drugs (see RNAi News, 2/14/2008).
Accelerator also believes it has a clear path to operate in the field when it comes to intellectual property.
“We believe there is enough room to navigate some space for Mirina to … develop its products without riding on other people’s IP,” Schubert said. He declined to elaborate.
McElligott said that Mirina currently expects that it will develop single-stranded oligos that have been modified with MGBs as miRNA antagonists. However, the company may pursue other approaches depending on the outcome of its initial proof-of-concept experiments with the technology.
As with all of Accelerator’s companies, Mirina will operate out of the Accelerator facility in Seattle. Accelerator will also handle all non-scientific managerial responsibilities for the new company.
According to Schubert, Mirina will have around four employees, including McElligott, for the next 18 to 24 months. After this time, Accelerator expects that Mirina will have amassed enough proof-of-concept animal data to be able to complete a significant Series B financing round, which will provide the startup with funds enough to hire its own management team and begin to operate independently.
“What we’re looking for is to have the right amount of data in place to have enough confidence to raise a substantially larger amount of financing,” he said. “Then, the company goes off on its own and achieves its own milestones. Then … [we’ll] take a look at where things are at, whether the public markets [have] re-opened or [we’ll try to enter an] an acquisition situation.”