THX Pharma is a biopharmaceutical company specializing in the development and commercialization of innovative treatments for rare neurological diseases and central nervous system (CNS) disorders. The company has a proprietary therapeutic platform that targets both neurons and glial cells through combinations of distinct drugs, aiming to treat severe CNS conditions. Its main products include TX01, a treatment candidate for Niemann-Pick type C and Gaucher diseases, and Batten-1, a proprietary drug candidate for the juvenile form of Batten disease, a rare neurodegenerative disorder with no current approved treatment. THX Pharma also develops an antisense oligonucleotide platform addressing rare diseases and glioblastoma.
Founded as Theranexus and headquartered in Lyon, France, THX Pharma recently rebranded from Theranexus to reflect a strategic shift toward regulatory approval, early market access, and international commercialization of its medicines. The company completed a successful €7.8 to €8 million capital increase in 2025 to support continued development and expansion. THX Pharma plans regulatory submissions to FDA and EMA and aims for market launches of TX01 and Batten-1 by 2027 and 2028, respectively, targeting peak revenues of €50 million by 2031-2032 for TX01 and €200 million by 2032-2033 for Batten-1. The company collaborates with partners such as Exeltis for European commercialization and pursues registrations in additional markets including Australia and Canada. It also pursues innovative projects like the PickASO antisense oligonucleotide program, supported by Bpifrance, targeting lysosomal diseases and glioblastoma.