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Dicerna grabs $60M round to test RNAi tech on prime cancer suspect

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Dicerna Pharmaceuticals has topped up its financial reserves with a $60 million C round earmarked to gather proof-of-concept data on a pair of RNAi programs headed for the clinic. And alongside the traditional venture investors who came back for this round, Dicerna also attracted a group of public company investors lured by the prospects of a biotech company keeping a close watch on the current groundswell of IPOs popping up in the industry.

The Watertown, MA-based biotech's last round in 2010 wound up at $29 million. This third investment in its gene-silencing technology brings its total raised to a whopping $110 million. And the CEO says the substantial fundraising offers some solid proof that after running from hot to cold, RNAi is now gaining renewed interest from some deep-pocketed investors as programs make the leap into the clinic.

"This is the money that drives us into the clinic," CEO Doug Fambrough tells FierceBiotech. He expects a Phase I study of the company's experimental therapy for hepatocellular carcinoma to start in early 2014 with data due in 2015, to run in tandem with another Phase I study on solid tumors. And a shorter study will follow for genetically defined targets of the liver, which Dicerna is not detailing right now. Data from that second program should also arrive in 2015.

Dicerna's cancer program is focused on the Myc gene, one of the "original oncogenes discovered in the 1970s" which has remained undruggable to date, immune to both antibodies and small molecules, says the CEO. But Fambrough believes Dicerna's approach to gene silencing, touted as better designed than many of the first generation of RNAi drugs that made it to the clinic, makes it the perfect program to provide solid early evidence of efficacy.

"We've aimed it right where we can have a competitive advantage," says Fambrough, adding that "we think we can really knock (Myc) down in patients."

Dicerna was named a Fierce 15 company in 2010, just as Big Pharma companies were beginning to shun the field, worried that it was still too early in the discovery process to justify a big investment in a new platform technology. Now Fambrough says the market chill has clearly thawed, with Alnylam's ($ALNY) hot stock offering some added proof. And he thinks Dicerna has a solid shot at winning a pharma collaboration on the cancer program, possibly using its current partnership with Kyowa Hakko Kirin as a model, in which Dicerna retained a substantial commercial opportunity in the U.S. market.

An IPO could also offer some fresh funds to build the company, which has 25 staffers.

"With this financing and the investor group that has now come into the company, (including RA Capital with contributions from Brookside Capital, Deerfield, and Omega Funds), we're well positioned to have an IPO as an option," says the CEO, though just when that will be remains up in the air.

The round includes all 5 of Dicerna's existing institutional investors: Abingworth Management, Domain Associates, Oxford Bioscience Partners, Skyline Ventures and SR One.

- here's the press release

Special Reports: Dicerna: DsiRNAs - 10 Next-Gen Biologics Platforms | Dicerna Pharmaceuticals - 2010 Fierce 15

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