By Doug Macron
MDRNA's top executive this week confirmed that the company has moved its bladder cancer program to the head of its pipeline, and that it would yield an investigational new drug application filing — the firm's first — in the fourth quarter.
At the same time, MDRNA President and CEO Michael French, speaking at this year's BIO-CEO and Investor conference held in New York, said that the company was on track to forge two full-fledged research and development collaborations with a big pharma partner in 2010. Previously, the company had said that it expected to sign only a single deal this year (see RNAi News, 11/19/2009).
After reorganizing last year from intranasal delivery firm Nastech Pharmaceutical into a pure-play RNAi drug shop with a variety of drug-development programs, MDRNA narrowed its focus onto a single indication: liver cancer (see RNAi News, 3/26/2009).
Less than six months later, however, the company announced that it had added bladder cancer to its pipeline, citing positive results in rodent studies and delivery advantages given the ease with which a drug can be locally administered to the bladder (see RNAi News, 8/6/2009).
Since then, the bladder cancer effort has steadily gained steam, and by October, MDRNA was weighing the possibility that the program could enter clinical testing before a liver cancer candidate (see RNAi News, 10/15/2009). Now, it appears that it will, while a second IND, "likely in liver cancer," is slated to be filed with US regulators in the first half of 2011, French said at BIO-CEO.
He added at the conference that MDRNA will select a lead bladder cancer candidate for the IND "in the coming weeks," and that the drug will have two targets. Already, the company has released rodent data demonstrating knockdown of two oncology targets, survivin and polo-like kinase 1, using its proprietary UsiRNA molecules formulated in its DiLA2 liposomal delivery vehicles.
As such, those two are the "top targets" being considered, although the company is evaluating others, French told RNAi News.
At the same time, MDRNA continues to pursue big pharma partnerships, and is poised to meet its previously stated goal of inking one before the end of the second quarter. The company is now also expecting to sign a second before the end of the year.
"These collaborations, we would expect, [will be] somewhere in the $10-to-$20 million up-front range, [and include] R&D funding, downstream milestones, and royalties," French said during the conference.
While alliances with bigger players have a number of benefits for smaller biotech players, such as providing access to scientific expertise, the financial upside is a key consideration, especially for MDRNA, which French said currently has enough cash to last into the second quarter.
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"I'd feel much better if I had cash well through the [middle] of the year to establish these pharma partnerships," he added. Still, "we've got a few tricks up our sleeve" when it comes to securing funding.
"We're not dependent solely on that large pharma collaboration for non-dilutive cash," he stressed to RNAi News. "We have a lot of assets at MDRNA — we still have legacy intranasal assets [from Nastech] and the ability to license pieces and portions of the RNAi discovery platform, as we've done before."
In April, MDRNA sold off manufacturing facilities inherited from Nastech for an undisclosed amount to Par Pharmaceuticals (see RNAi News, 4/2/2009). A few months before, the company licensed certain of its RNAi technology to Roche for use in RNAi drug development (see RNAi News, 2/19/2009).
Still, a big pharma deal could bring in significant funding, and MDRNA is confident that it will be able to find partners, in part because of its efforts to build up its in-house capabilities and efforts to address all the issues that come into play in developing a successful drug.
"Pharma is interested in the whole ball of wax … [and] we're looking at all aspects of our RNAi-based compounds in terms of commercialization," French told RNAi News. "Can they be stored, refrigerated, frozen? … We're looking at all aspects of [the drug-development process] that we think are critical to ultimately delivering RNAi-based therapeutics."
This week, MDRNA released data showing that drugs formulated with its DiLA2 technology maintained stability over a 1-year period under various conditions. Specifically, the compounds retained "in vivo knockdown activity with no observed loss in potency over the course of [a] year-long study" when stored under conditions ranging from -80 degrees Celsius to 4 degrees Celsius.
"In addition, neither change in particle characteristics, such as size or charge, nor loss in siRNA integrity, was observed," the company said.
"It's not just about knocking things down in rodents and showing tolerability in non-human primates. It's all about whether or not you can ultimately come up with a compound that you can take to market," French told RNAi News. "We feel very confident that our delivery technology and the siRNAs that we have encapsulated … are going to be stable under a variety of storage conditions [and] we have the opportunity to advance these to the commercial market.
"Pharma isn't saying, 'We've got to see a year's stability before doing a collaboration with you,' but it's nice for [them] to know you're thinking about that," he added.