an equity stake in the startup it created around the work, Ataxion (the seller would also get a “small” royalty on any drug candidates that ever hit the market, according to Resnick).
The startup is essentially a virtual company. Resnick serves as the CEO, while Martin Jefson, an entrepreneur-in-residence at Atlas and the former head of neuroscience at Pfizer, is its chief scientific officer. Those two are Ataxion’s only full-time employees.; the rest of the work is being contracted out. Saniona, for example, is doing a lot of the preclinical research and discovery work. When it’s time to run a clinical trial, Ataxion will design it and pay a CRO to run it. Resnick says Ataxion might make one or two hires as it progresses into clinical development, but that would likely be it.
Resnick declined to say exactly which biological targets the company plans to go after with a drug, but the underlying scientific idea is this: In a number of ataxias, Purkinje cells tend to fire irregularly, or improperly, leading them to send out a discombobulated set of signals, which results in a loss of coordination. So Ataxion is trying to develop an oral, small-molecule drug that hits a target that is directly responsible for the signaling of the Purkinje cells. Taken chronically, in theory, the drug would “restore normalcy” to those cells, and along with it, cause the cerebellum to function properly, according to Resnick.
“We don’t know of anybody else who is going after this biology at this point,” he says.
Resnick surmises that this is partly because there has been a “bias towards disease modification,” meaning companies have been much more focused on trying to get at the root cause of certain genetically based ataxias by replacing a missing protein, silencing one that’s toxic, or otherwise. That’s why there have been several attempts to reverse the course of Friedreich’s ataxia, for instance, which is caused by a specific genetic defect.
“What’s lost focus in that is you can achieve a lot by treating the symptoms and by treating the underlying biology without creating a ‘cure,’” he says. “As we’re talking to people, particularly strategics, about this program, there’s an increasing understanding and appreciation for the relevance of symptomatic treatment in neurodisease even if you’re not achieving a genetic or protein-based cure.”
Of course Ataxion has a ways to go before that vision is a reality. So far, the startup only has data showing that it’s able to improve the firing of Purkinje cells in mice, and that doing so helps restore normal function. But it hasn’t yet mapped out a path to get from animal studies to human trials other than deciding to initially focus on a set of a few hereditary (rather than acquired) ataxias before looking at more ataxias, and other CNS disorders as well. That’s part of the reason that Biogen is involved. Biogen executive vice president of corporate development Steven Holtzman and senior VP of research and development Kevin Koch are joining Ataxion’s board of directors as part of the financing (joining Atlas’ David Grayzel and Jean-Francois Formela, and Sanonia chairman Claud Braestrup), and the big firm will give some insight into which clinical path it would be most interested in as a potential acquirer. Ataxion can then design the clinical program and go from there.
“That’s one of the advantages of the option structure,” Resnick says. “By moving collaboratively from the early stages here that lets us design this towards the optimal clinical path.”
Resnick declined to project just when Ataxia’s first program would be ready for a trial, or how long that study might take. But because the ataxia population is small—Resnick estimates the group consists of about 15 to 20 out of every 100,000 people—and a treatment effect should be seen relatively quickly, it means Ataxion wouldn’t be left in a lurch if Biogen were to walk away.
“The amount of capital to take it to a very solid proof of concept is not an unreasonable amount of capital,” Resnick says.
