NEA, Genzyme Join $18M Round For Rare Disease Startup Edimer

(Page 2 of 2)

female carriers of the disorder who either are pregnant or want to get pregnant who have had affected children in the past. Once Edimer knows the genotype of the mother, it tries to find the genotype either of the fetus, in utero, using amniocentesis, or the baby within 72 hours of its birth. Edimer has three study sites in the U.S. (one on the East Coast, West Coast, and in the Midwest, respectively), and three it’s getting up and running in the U.K., France, and Germany to do this.

This, of course, presents a lot of logistical issues. As Kirby points out, not everyone wants to have amniocentesis—an invasive procedure that comes with a small risk of miscarriage— and this leaves Edimer in a “genetic crapshoot” of sorts tracking pregnancies and waiting for births of children with the disorder. Its goal is to get six to 10 newborns on the drug to fill up its study.

“It’s not an insignificant task,” Kirby says.

The payoff, however, could be massive. Edimer’s plan isn’t to turn this into a chronic therapy in the way that patients with other rare diseases, for example, take enzyme replacement therapy to keep getting the key protein they need. Rather, Edimer has found, in its animal studies, indications that its drug could wipe out the disorder altogether after a few doses at the right time. Specifically, by administering five separate doses of the engineered protein at a key window—within the first two weeks after a child with the mutated gene is born—a switch is, in effect, turned on that triggers normal development.

“If you give a short-course therapy of five doses—at least in the animals—early in life, you can correct the entire phenotype of the disease for life,” Kirby says. “It’s almost like a trigger that needs to be there—if it’s not there at the right time of development, these appendages [like] sweat glands, hair, teeth, other glandular structures, do not form.”

Thus, Edimer’s plan in its trial is to dose these infants with the drug five times during their first two weeks of life, and track their results over the next six months. Edimer will look for things like impact on respiratory disease, number of hospitalizations, sweating, and sweat pore formulation. A follow-up study will track the patients for “as long as we need to,” according to Kirby, or maybe “as long until we get approved.”

“The idea there is to just keep gathering these longer term endpoints, because not all of them are going to be apparent in the first six months of life,” he says.

The FDA and European Medicines Agency have already granted EDI200 an orphan drug designation, which gives it longer market exclusivity. The FDA has also given the drug fast-track status, giving Edimer a quick roadmap should it get the results it’s hoping for.

“If we are as successful in this Phase 2 as we have been in treating dogs, for example, I think there is a pretty good case to be made then to add a few more patients and then look towards an accelerated approval pathway,” he says, before quickly adding, “but it all depends on the data.”

The cash gives Edimer a minimum of a three-year runway, and in addition to funding both the trial and its research activities, will help the company make two key hires: a business development/commercial specialist, and another to help drive patient recruitment in its study.

Plus, given the scalding hot IPO market, Edimer could be headed in a direction it hadn’t originally considered. Kirby says the company’s initial plan was to usher EDI200 through a proof of concept study before finding a big partner to help commercialize it—but that things may change now.

“I think with a little bit more dry powder and what we have in front of us, I think it might be worth having a conversation about the strategy going forward,” he says. “It’s certainly something we’re going to be talking about as the new board gets together.”

Single PageCurrently on Page: 1 2 previous page

Ben Fidler is Xconomy's Deputy Editor, Biotechnology. You can e-mail him at Follow @benthefidler

Trending on Xconomy