Backed by Bill Gates, low-profile Exicure steps into spotlight with a $42M R&D gamble

Exicure CEO David Giljohann

For a biotech that's raised $42 million from a group of deep-pocketed players that includes Bill Gates, Exicure is one of the most interesting drug developers you've never heard of.

Partly, that's because the company doesn't always rush out a press release with every raise. Partly, it's because it's based in Chicago, which doesn't have the same kind of media visibility which is given to new companies in a hot and happening place like Cambridge/Boston.

I accidentally ran into Exicure CEO David Giljohann in San Francisco, after I ducked into MacDougall's lounge between interviews at J.P. Morgan in San Francisco. Giljohann introduced himself, we chatted for a few minutes, and as I headed out I asked for a follow-up call.

A spinout from the lab of Northwestern's Chad Mirkin, Exicure--formerly AuraSense--is developing a portfolio of drugs based on its spherical nucleic acid nanotechnology. Taking an artificial nanosphere as a scaffold, it assembles single- and double-stranded nucleic acids on the surface--creating a "third form" of nucleic acids which can easily slip into cells without triggering an immune response.

Illustration of Exicure's SNA platform--Courtesy of Exicure

"The key for us, the magic, is that when you create these structures they naturally go into cells," says a youthful Giljohann, who got involved doing graduate work in Mirkin's lab.

Then the SNAs can use antisense or RNAi pathways to do its duty in regulating gene expression. (It's a complex science, and the company put together a very handy, very short video to show how it works. You can see it here.)

This particular technology works well with skin cells, says the CEO, which is why Exicure decided to focus its initial proof-of-concept work on psoriasis, with TNF-alpha as the target. That helps explain why AbbVie--which markets the megablockbuster Humira--appears as one of the biotech's investors.

"We just filed a regulatory package in Germany for a first-in-human Phase I trial," says Giljohann, set to begin at the end of this month. Germany is a particularly good spot, he explains, because regulators will allow the biotech to use a variety of topical doses on one patient, with each patient providing multiple data points to track for any future studies.

If it works, and that's always the big question about any startup, Exicure has another 230 or so dermatology targets to consider. Giljohann says he's waiting for that POC data before he gets serious about partnering. And there's a further, far more daunting challenge that lies ahead in applying this tech to the booming field of cancer immunotherapy.

While Giljohann hasn't had a ton of exposure in the industry, he's made a convincing case to an eclectic group of high-profile, deep-pocketed investors, those high-net-worth individuals you hear about a lot in biotech, but rarely see.

Billionaire Gates came in after Craig Mundie, a senior adviser to the software mogul, stepped in to back the company. They were joined by Pat Ryan, the founder of Aon; David Walt, co-founder of Illumina and a company director; and Boon Hwee Koh, director of Agilent Technologies and former Chairman of Singapore Airlines as well as the Rathmann Family Foundation.

It's a high-stakes gamble whether or not Exicure--with a staff of 25--can succeed. But it's well worth staying tuned in for the journey.

Read more on