Gates-backed Exicure wraps 2016 with its first major collaboration and a plan for the year ahead

Exicure CEO David Giljohann

Exicure CEO David Giljohann

Exicure has crossed a few key items off the to-do list that dominates the existence of every upstart biotech. And it’s wrapping up 2016 with a rite of passage that helps position it for a New Year filled with pipeline building plans and new partnership hunts.

This morning the Chicago-based biotech is announcing that it has completed its first industry collaboration, passing along its lead drug that has just successfully navigated a small proof-of-concept study in psoriasis. Purdue Pharma is stepping in with a pact — worth up to $790 million if all the cards turn in its favor — that will give it control of a Phase II-ready psoriasis drug along with three more programs to be decided later.

“This is a new thing for them,” says Exicure CEO David Giljohann about his new partner. Purdue, which has been a central player in the public outcry over Oxycontin abuse, has been actively building its own in-house R&D expertise with an eye to striking a slate of deals this year. And this new venture gives Purdue its first asset for a dermatology portfolio.

I first crossed paths with Giljohann at JP Morgan last January, somewhat accidentally, but was intrigued that the company had kept a low profile after raising $42 million from a group of deep-pocket investors, which included Bill Gates.

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. The SNAs can use antisense or RNAi pathways to do their duty in regulating gene expression.

It’s the sort of technology that makes a lot of sense in dermatology, where Exicure can look to develop topical formulations of new drugs that can be designed to compete with a new wave of biologics that are just now breaking into the market. Exicure is much, much further behind, but the attractions of a potential topical drug positioned against biologics is obvious.

In its two-week PoC study, Exicure says it got the safety data that it needed to press ahead with a larger mid-stage program. But they also did punch assays to assess messenger RNA levels.

“From the punches we saw we were able to regulate genes in the skin,” says Giljohann.

That’s the first snapshot of activity for a platform that will now be focused on programs for IL-17 in psoriasis, IL-4 in atopic dermatitis and IL-1 beta for rare cases of epidermolysis bullosa.

New human studies should get underway next year, says the CEO, who’s looking forward to building an internal pipeline while adding new players to its list of partners.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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