Emalex Biosciences, Inc.

Among the 49 adult patients recruited for the trial, 41.2% of patients dosed with Ecopipam experienced some form of relapse. Credit: Shutterstock / Studio Romantic Emalex Biosciences is eyeing approval for its Tourette syndrome therapy after it achieved its primary and secondary endpoints in a Phase III study. The US company’s central nervous system (CNS) disorder drug ecopipam, a dopamine-1 receptor antagonist treatment, met both primary and secondary endpoints in both paediatric and adult patients living with the condition that can lead, in some cases, to involuntary tics and vocalisations. The drug achieved a 41.9% of paediatric patients relapse against the placebo group’s 68.1%. Following the positive data, Emalex Biosciences said it will be meeting with the US Food and Drug Administration (FDA) and other global health authorities to discuss the submission of a New Drug Application (NDA) later in 2025. The DIAMOND registrational study (NCT05615220) examined a total of 167 paediatric subjects and 49 adult subjects with Tourette syndrome. The trial saw a clinically meaningful reduction in vocal and motor tics in paediatric patients receiving ecopipam during a 12-week open-label period. Patients were then randomised to remain on the study drug or be moved to a placebo for a 12-week double-blind withdrawal period. Among the 49 adult patients recruited for the trial, 41.2% of patients dosed with ecopipam experienced some form of relapse against the placebo arm’s 67.9% of subjects. Frederick Munschauer, chief medical officer for Emalex Biosciences, said: “These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome. “The topline data from our large, multi-national, randomised withdrawal study show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for paediatric subjects with Tourette syndrome as compared to placebo.” Research by GlobalData uncovered that in 2024 the global market for Tourette syndrome therapies stood at approximately $248m, with that figure expected to plummet down to just $84m by the end of 2030. Whilst there are many marketed drugs, Otsuka Holdings’ Abilify (aripiprazole) is the most commonly used antipsychotic therapy and is expected to bring in around $192m by the end of 2025. GlobalData is the parent company of Clinical Trials Arena. The US Centers for Disease Control (CDC) estimates that across the US one out of every 333 (0.3%) children between the ages of three and 17 has been diagnosed with the condition. Additionally, 44% of those children have been diagnosed with moderate or severe Tourette syndrome, with boys three times more likely to live with the condition than girls. Previously in results taken from a Phase IIb trial , ecopipam was able to reduce the number of tics experienced by a paediatric patient by 30% compared with placebo. Eric Messner, Emalex Biosciences’ CEO, added: “The Emalex team worked closely with physician investigators and patient advocates throughout the drug development process and we are hopeful that ecopipam can provide symptomatic relief from the tics suffered by patients with Tourette syndrome. We’re entering a new era of progress for people with central nervous system conditions with limited or no treatment options.” Elsewhere in the market of Tourette therapies, Israeli pharmaceutical company SciSparc has received approval from the US Food and Drug Administration (FDA) to begin a Phase IIb clinical trial of SCI-110 in adults living with the condition.

Emalex Biosciences said its daily oral tablet helped reduce relapses for children and adults with Tourette syndrome in a Phase 3 study, and now plans to take the drug to regulators for approval. The study put all patients on the drug, called ecopipam, for a 12-week open-label period. If patients had clinically meaningful reductions in vocal and motor tics during that time, they were then randomized to get either ecopipam or a placebo in a double-blind, 12-week withdrawal period. The trial included 167 pediatric participants and 49 adults in the US, Canada and European Union. In the trial’s primary endpoint, 41.9% of pediatric participants on Emalex’s drug relapsed and 68.1% of pediatric patients on placebo relapsed, Emalex said. A secondary endpoint looked at adult and pediatric patients, and showed similar results: 41.2% of people on the study drug relapsed, whereas 67.9% on placebo relapsed. Emalex said both endpoints were statistically significant. On safety, the company said its drug was “generally well tolerated.” The most common side effects were somnolence (drowsiness or sleepiness), insomnia, anxiety, fatigue and headache. The Chicago-based biotech said it next plans to meet with the FDA and other unnamed global health regulators with the goal of submitting a new drug application for the experimental medicine “later this year.” “We are hopeful that ecopipam can provide symptomatic relief from the tics suffered by patients with Tourette syndrome,” CEO Eric Messner said in a statement. The company didn’t respond to requests for more details about the trial. Tourette syndrome is a neurodevelopmental disorder that can start in childhood and lead to chronic motor and speech tics. Neurocrine and other companies have attempted to make medicines for it, and Noema Pharma is in Phase 2b testing with a different approach. Emalex bought ecopipam from Psyadon in 2018 after the Maryland-based biotech took the investigational treatment through Phase 2b . The drug aims to block dopamine at the D1 receptor. The biotech brought on blue-chip investors in the fall of 2022 to fund the registrational trial after looking at the potential of going public, executives previously told Endpoints News . That year, Bain Capital Life Sciences led a $250 million Series D alongside Emalex’s founder Paragon Biosciences, Valor Equity Partners, Fidelity and “several” family offices. At the time of the Series D, Emalex leaders told Endpoints the funding would take it through the Phase 3 and help support manufacturing and certain pre-commercialization activities. Paragon, a Midwest biotech incubator and investor run by former Marathon Pharmaceuticals leader Jeff Aronin, founded Emalex in 2018 to create new medicines for CNS disorders. Paragon has also backed skin cell therapy company Castle Creek Biosciences , daytime sleepiness drugmaker Harmony Biosciences , AI-driven protein maker Evozyne , and eye and brain disease biotech CiRC Biosciences.

Asarina Pharma revealed in April 2023 that its Tourette syndrome therapy reduced tic severity at 12 weeks by 28%. After reaching out to more than 200 companies to partner a Tourette syndrome therapy that showed the ability to beat standard of care last year, Asarina Pharma has come up empty and will fold. The company asked shareholders to vote to liquidate in a notice posted Monday, the culmination of more than a year of effort to find a savior for the treatment called sepranolone. The Swedish company revealed in April 2023 that the therapy reduced tic severity at 12 weeks by 28% according to a common rating scale of disease severity called the Yale Global Tic Severity Scale (YGTSS), compared to 12.6% in patients who received standard of care. The phase 2a study also hit key secondary endpoints, including improving quality of life, and there were no systemic side effects observed. The open-label study randomized 28 patients to receive the experimental medicine or standard of care, with 17 receiving sepranolone. But those results were not enough to secure a partner, despite a grand effort from the Asarina team. In a proposal to liquidate issued July 18, the company said 200 parties had been contacted with 20 entities expressing interest in a potential in-licensing or acquisition deal. Several went as far as conducting due diligence on the clinical data. But none of those talks resulted in an offer. Asarina also explored a capital raise “but unfortunately has been forced to conclude that conditions for this are missing,” according to the notice. The company currently has equity of -635,000 Swedish kronor (-$59,000). “In light of the company's financial and commercial situation … the board of directors sees no alternative but to propose a winding up of the company's operations in an orderly manner, which can be done through a liquidation,” the notice explained. A meeting will be held in August to consider the plan to wrap up, with a liquidation date slated for Dec. 1. ”After more than 15 years of R&D development and more than 15 months of partnering activities, it is disappointing that we have not been able to find a new home for sepranolone. We still believe that the compound has the potential to be an effective drug for Tourette's syndrome and other neurological disorders,” said board Chairman Paul De Potocki in a statement. While drug development in Tourette syndrome has not seen a lot of action in recent years, at least one biotech is working on it. Emalex Biosciences published phase 2b data last year for a candidate called ecopipam showing a 30% reduction on the YGTSS. The company did not detail placebo results but said the 30% value represented a significant reduction in the total number of tics compared to placebo.  Ecopipam also had a different safety profile, showing adverse events including headache in 15% of recipients, insomnia in 15%, fatigue in 8% and drowsiness in 8%.  Emalex raised a massive $250 million in series D funds in 2022, which was to be used to fund a phase 3 test. That trial is now underway as of March 2023.