Gemlapodect

Emalex Biosciences plans to meet with the FDA and other global health authorities to discuss submitting ecopipam for approval later in 2025.\n Emalex Biosciences’ dopamine blocker led to fewer patients with Tourette syndrome relapsing in a phase 3 trial, setting the stage for the Chicago-based firm to submit the drug candidate for approval later this year.The phase 3 study enrolled 167 children and 49 adults, according to a Feb. 25 press release. After a 12-week open-label period, patients who experienced reductions in vocal and motor tics when given the drug were randomized to either continue receiving the drug, called ecopipam, or to switch to placebo for a further 12 weeks. This phase of the trial was double-blind.When continued on ecopipam, 41.9% of children had their symptoms relapse, defined as a 50% loss of the benefit they initially received from the drug in their Yale Global Tic Severity Score. This relapse rate was significantly lower than the 68.1% rate seen in children given placebo, Emalex said in the release. Time to relapse for the pediatric patients was the study’s primary endpoint.Emalex plans to meet with the FDA and other global health authorities to discuss submitting ecopipam for approval later in 2025, the firm said.The most common side effects of ecopipam were drowsiness, insomnia, anxiety, fatigue and headache, according to the release. About 10% of patients experienced drowsiness, also called somnolence, while 7% had insomnia.For the study’s secondary endpoint, Emalex analyzed the time to relapse for children and adult patients together. Here, the relapse rates were similar, with 41.2% of patients relapsing on ecopipam compared to 67.9% on placebo.The phase 3 study builds on Emalex’s prior phase 2 work, which found that ecopipam reduced tic severity by 30% after 12 weeks in 150 children and adolescents with Tourette syndrome.“These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome,” Frederick Munschauer, M.D., Emalex’s chief medical officer, said in the release. The top-line data “show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for pediatric subjects with Tourette syndrome as compared to placebo.” Ecopipam blocks the D1 dopamine receptor, which has been implicated in the repetitive and compulsive behaviors that are characteristic of Tourette syndrome, according to the release. Approved therapies for the disease target the D2 receptor instead.Emalex raised a $250 million series D in 2022 to bring ecopipam into phase 3, a sharp increase in investor interest from the comparatively paltry $35 million series C that preceded it.The Windy City biotech was founded in 2018 by biotech incubator Paragon Biosciences.Swiss biotech Noema Pharma had a Tourette win of its own late last year, when its PDE10A inhibitor gemlapodect—originally developed by Roche—reduced tic severity in a small phase 2 trial that enrolled 15 patients. 

Noema Pharma has successfully closed a Series B extension financing round with an investment from EQT Life Sciences, bringing the total capital raised in the round to CHF 130 million (approx. USD 147 million). With its investment in Noema Pharma, EQT Life Sciences joins the syndicate of previous Series B investors including Forbion, Jeito Capital, Sofinnova Partners, Gilde Healthcare, Polaris Partners, Invus and UPMC Enterprises. The new financing will support Noema Pharma’s four active Phase 2 trials, with key data readouts anticipated in 2025. This includes additional development activities for basimglurant (NOE-101), an mGluR5 negative allosteric modulator currently in Phase 2 trials for severe pain in trigeminal neuralgia (TN) and seizures in tuberous sclerosis complex (TSC); gemlapodect (NOE-105), a PDE10a inhibitor in a Phase 2b trial for Tourette syndrome and under development for childhood-onset fluency disorder (COFD or stuttering); and NOE-115, a broad-spectrum monoamine modulator in a Phase 2 trial for vasomotor symptoms and other symptoms of menopause. Ilise Lombardo, MD, CEO of Noema Pharma, stated, “This latest financing underscores the strong support and confidence from investors in Noema’s vision to treat neuroscience-based conditions. We are thrilled to welcome EQT Life Sciences into this strong syndicate of investors and have Felice join our Board. EQT’s support and expertise alongside our syndicate of investors will be invaluable as we progress our clinical programs and strive to make a meaningful impact on patients’ lives.” Felice Verduyn-van Weegen, Partner at EQT and member of the Board at Noema, commented, “Noema Pharma’s innovative approach to CNS disorders aligns very well with our investment strategy and we are excited to support their late-stage clinical pipeline and transformative therapies. Having worked with Ilise Lombardo on a previous successful investment, her exceptional leadership as a repeat entrepreneur reinforces our confidence in Noema’s potential to deliver meaningful impact to patients in need.” The Series B extension follows the initial Series B financing of CHF 103 million (USD 112 million) announced on March 2023, and a CHF 54 million (approx. USD 60 million) Series A round in December 2020. Noema Pharma was founded in 2019 with seed investment from Sofinnova Partners.