Emalex Closes $250M Series D to Fund Phase III Trial in Tourette Syndrome

03 Nov 2022
Gene TherapyOrphan DrugFirst in ClassFast Track
Emalex Biosciences closed an upsized and oversubscribed Series D funding round Thursday, counting $250 million in earnings, much of which will bankroll a Phase III trial of ecopipam for Tourette syndrome. The funding round was led by Bain Capital Life Sciences. Paragon Biosciences, which founded Emalex in 2018, participated in the financing push alongside Valor Equity Partners and Fidelity Management & Research Company. In a statement, Emalex founder and Paragon CEO Jeff Aronin said that the Chicago-based neuroscience biotech was created specifically to address neurological conditions with high unmet need, like Tourette Syndrome. He added that Emalex recognizes “that drug development for neurologic conditions is exceptionally difficult and few companies are willing to invest in bringing new options to these patients.” With an enrollment target of over 220 patients across some 90 sites, Emalex is aiming for the largest Tourette trial ever conducted in North America. The Phase III study will assess the company’s ecopipam, a first-in-class investigational compound that disrupts the interaction of the neurotransmitter dopamine with the D1 receptor. Current therapies for Tourette target the D2 family of dopamine receptors. Ecopipam targets the D1 family, the super-sensitivity of which has been named in recent studies as a potential contributor to Tourette tics. The Phase IIb D1AMOND clinical trial confirmed ecopipam’s mode of action, showing that treatment with the candidate led to reduced motor and phonic tics as compared with placebo. The investigational drug was also well-tolerated, with the most common side effects being headache, fatigue and somnolence. There was no evidence of movement-related or metabolic adverse events. The FDA has granted ecopipam the Orphan Drug and Fast Track designations. Big Bucks in Brain Diseases Emalex’s oversubscribed financing round is only the latest development in the high-value neuroscience therapeutic space. In early October, Neumora Therapeutics raised $112 million in its Series B financing round to help it develop its neuroscience portfolio further. In particular, the company plans on using the proceeds to advance its lead candidate NMRA-140, a kappa opioid receptor antagonistkappa opioid receptor antagonist for major depressive disorder. The money will also be channeled into Neumora’s other precision medicine programs for neuropsychiatric diseases. The company had previously raised $500 million in 2021. Soon after Neumora’s payday, Astellas put forth $50 million in a strategic equity investment and licensing deal, acquiring 15% of Taysha Gene Therapies. The agreement gives Astellas the exclusive option to license Taysha’s TSHA-102, a gene therapy for the severe genetic neurodevelopmental disorder Rett syndrome. The deal also includes TSHA-120, also a gene therapy under investigation for giant axonal neuropathy.
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