Emalex Biosciences is closing in on the finish line for a novel Tourette syndrome therapy after revealing positive topline data from its phase 3 clinical trial late last month.
Often characterized by involuntary motor or vocal tics, Tourette syndrome typically appears in childhood and affects up to 450,000 Americans. While symptoms fade for some patients, the tics can persist into adulthood for others.
The current treatment landscape for Tourette is “limited,” according to Emalex CEO Eric Messner, with medication options falling into two categories: antipsychotics and alpha agonists. Treatments in both classes of drugs have been approved for decades, and alpha agonists are often being used off-label to treat Tourette syndrome, Messner said. But the drugs can also come with efficacy challenges and unpleasant side effects, leaving the Tourette community eager for new mechanisms of actions, Messner said.
If Emalex’s drug makes it to market, it will be the first new type of treatment for Tourette syndrome to win approval in 56 years, the company’s chief medical officer recently said.
A novel approach
Emalex’s lead drug candidate, ecopipam, blocks dopamine at the D1 receptor, rather than the D2 receptor alpha agonists currently target.
“It's the only D1 receptor antagonist in late stage development, and, to our knowledge, there are no other D1 antagonists approved for any indication anywhere in the world,” Messner said of ecopipam. “It does affect dopamine, but through a totally different mechanism than the D2 antagonists.”
Topline data from its phase 3 study of the drug among more than 200 children and adults found a “statistically significant benefit for ecopipam” in maintaining reductions in vocal and motor tics among children compared to placebo, Dr. Frederick Munschauer, Emalex’s chief medical officer, said in a statement.
The results were so positive Emalex is aiming to meet with the FDA later this year to discuss approval submission plans. Messner hopes ecopipam will be considered a first line treatment, “depending on what the FDA says,” he noted.
Emalex, which was born out of Paragon Bioscience, picked up ecopipam when the biopharma acquired Psyadon Pharmaceuticals in 2018. The company was on the hunt for “innovative products and categories with high unmet medical need,” Messner said. Since then, Emalex has advanced the drug with the help of a $250 million Series D funding round in 2022.
The company is among a handful investigating new Tourette syndrome drugs. SpiSparc, an Israel based pharma, is looking at a combination synthetic THC and endocannabinoid, while Noema Pharma is in phase 2 testing for a PDE10A inhibitor it picked up from Roche.
And there’s currently plenty of room in the market for a drug like ecopipam, Messner said..
“We think that ecopipam will fit in quite nicely to the treatment paradigm,” Messner said. “What we see in our phase 3 study is good efficacy that's both clinically meaningful and statistically significant. There are adverse events with any drug, but they're generally mild, and it's tolerable over the long term. We think that that's one of the benefits of this product — that patients are able to stay on it for the long term. That's one of the ways it will distinguish itself from the other products.”
The road to market
Emalex plans to apply for a pre-NDA meeting with the FDA, likely in the second quarter, and then for a new drug application toward the end of the year, "depending on the outcome of that meeting,” Messner said. If all goes well, Emalex could be looking at a subsequent launch in the second half of 2026.
Behind the scenes, the commercialization strategy is already underway.
“Right now, we're a fairly small company,” Messner said. “We are quickly expanding throughout 2025 and adding new people to Emalex in the appropriate areas.”
This year, the company will hire personnel for its medical affairs team, which will interact with physicians, as well as manufacturing and quality staff, before adding to its commercial team toward the end of the year and into 2026, Messner said.
The company is also aiming to educate physicians on the new mechanism of action and lean on its data to make the case that ecopipam should be a first line treatment.
“Some physicians are fast to try new mechanisms of actions. Others take a wait-and-see approach,” Messner said. “Physicians are quite familiar with the D2 receptor antagonist because there's many of them out there, and they've been approved for quite a long time. They know how to use them, they know how to dose them, they know what the side effects are, they know what the safety is. And we'll have to build up all that knowledge for our D1 receptor antagonist.”
While the company hasn’t released the full phase 3 study results, Messner said the impact of side effects was “relatively low.”
“One of the things that plagues the D2 receptor antagonists is the prevalence of movement related disorders and metabolic disorders, things like high blood sugar and weight gain,” Messner said. “We hope that we can draw a difference between our product and the other products based on some of those parameters.”
In addition, Emalex is gunning to extend its cash runway, which will take it through 2025. The pre-NDA meeting with the FDA later this year will be key.
“Assuming that meeting goes as we expect it to go, we'll communicate our plans to potential investors and seek to raise money to take us through the launch and beyond,” Messner said.
