THX Pharma and Exeltis announce first milestone achieved for neurological disease treatment
Charlie Blackie-Kelly | October 23, 2025 | News story | Research and Development | Neurological Diseases, THX Pharma, Theranexus
THX Pharma (Theranexus), a pharma company focusing on developing treatments for rare neurological disease, and Exeltis, a pharma company within the Insud Pharma group, have announced that the first milestone has been achieved in their licence and supply agreement for TX01, a treatment for rare neurological diseases.
TX01 is a novel formulation of an already approved compound indicated for Gaucher and Niemann-Pick type C diseases, as well as other rare neurological conditions. It is currently undergoing clinical development and commercialisation processes globally, including in the US, Canada and Australia, with the partnership between THX Pharma and Exeltis driving its commercialisation in Europe.
Following the completion of this first clinical milestone, the partners confirmed they remain on track to submit Marketing Authorisation Applications, with European commercialisation planned for 2027, followed by launches in Latin America and selected Middle Eastern countries.
Mathieu Charvériat, CEO of THX Pharma, commented: “The timely completion of this milestone demonstrates our commitment to advancing TX01’s regulatory journey. This step brings us closer to the global commercialisation of TX01, delivering a transformative therapy for patients with Niemann-Pick type C and Gaucher diseases.”
Salustiano Perez, general manager of Exeltis, added: “We are pleased to reach this milestone with THX Pharma, the result of a fruitful collaboration. We can now confidently proceed with the plan to apply for TX01’s registration in the first quarter of 2026 and, commercialisation in Europe expected as early as 2027.”
In addition to the upfront payment received at the start of the partnership, THX Pharma is eligible for further regulatory and milestone payments, as well as royalties based on TX01’s net sales. Exeltis has also announced it will not exercise its right to reimbursement, which was in place should THX Pharma be unable to provide the required documentation by the end of October 2025.
