Noema Pharma AG

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. 

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.

BASEL, Switzerland, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Noema Pharma, a clinical-stage biotech company targeting debilitating central nervous system (CNS) disorders, today announced the appointment of Volker Knappertz, M.D., as Executive Vice President of Research & Development. “We are excited to welcome Volker to Noema. Volker is a recognized leader in drug development with a proven track record building and leading Research and Development organizations and obtaining market approvals for novel drugs, including Epidiolex for the treatment of rare developmental childhood onset forms of epilepsy,” said Ilise Lombardo, M.D., Chief Executive Officer of Noema Pharma. “His experience and expertise across CNS development will be pivotal as Noema enters a transformational year with readouts of multiple Phase 2 studies across our pipeline.” “I am thrilled to join Noema and look forward to the continued clinical development across the company’s multiple indications including seizures in Tuberous Sclerosis Complex, pain in Trigeminal Neuralgia, Tourette syndrome and vasomotor symptoms plus CNS-mediated symptoms of menopause,” said Dr. Knappertz. “Noema’s commitment to patients and innovative approach to CNS disorders is proven by their late-stage clinical pipeline and I look forward to working with the team to bring these first-in-disease therapeutics to patients in need.” Dr. Volker Knappertz has spent over 25 years in drug development and has been involved in numerous Investigational New Drug (IND) filings and product approvals during his career. Prior to joining Noema, Dr. Knappertz served as Executive Vice President of Research & Development (R&D) at Aurinia Pharmaceuticals, where he led the entirety of R&D across multiple programs targeting autoimmune diseases. Dr. Knappertz also served as Executive Vice President of R&D and Chief Medical Officer at GW Pharmaceuticals (acquired by Jazz Pharmaceuticals). During his time at GW, Dr. Knappertz was instrumental in driving the transformation of GW from a botanical pharmaceutical company to a fully integrated biopharmaceutical company following the FDA approval of Epidiolex for the treatment of rare developmental childhood onset forms of epilepsy. In addition, he played a role in several drug approvals and major business development deals during his tenure in leadership positions at Teva Pharmaceuticals, AstraZeneca and Bayer. Dr. Knappertz received his doctorate and Medical Degree from Cologne University and is a US board certified neurologist. He has served as Adjunct Professor in Neurology at the University of Pennsylvania and Heinrich-Heine University in Düsseldorf. About Noema PharmaNoema Pharma is a clinical-stage biotech company advancing a portfolio of transformative, first-in-disease therapeutics targeting neuroscience-based conditions with high unmet need. Noema has four programs currently in active Phase 2 clinical trials evaluating seizures in Tuberous Sclerosis Complex, pain in Trigeminal Neuralgia, Tourette syndrome and vasomotor symptoms plus CNS-mediated symptoms of menopause with readouts expected in 2025. Noema was founded by leading venture capital firm Sofinnova Partners and is supported by current investors including EQT Life Sciences, Forbion, Gilde Healthcare, Invus, Jeito Capital, Polaris Partners and UPMC Enterprises. Learn more at www.noemapharma.com. Investor ContactArgot Partners Maeve Conneighton 212-600-1902noema@argotpartners.com