THX Pharma (formerly Theranexus) has unveiled its new corporate and brand identity, marking a new milestone in its development toward the registration and commercialisation of its drug candidates for rare neurological diseases.
Theranexus has been committed to discovering and developing innovative solutions for patients with rare neurological diseases.
By becoming THX Pharma, the company signals its evolution from a biotechnology company to a pharmaceutical company focused on regulatory approvals and market access for its lead drug candidates across multiple geographies.
At the heart of this transformation are TX01, a new formulation of an already approved drug redesigned for paediatric populations with two rare diseases and Batten-1, the most advanced drug candidate worldwide for the treatment of Batten disease.
"THX Pharma embodies the evolution of our company: from laboratory innovation to bringing treatments to market for the benefit of patients."
"Our new identity reflects our ambition: moving up the value chain by rapidly commercialising innovative solutions in three rare diseases with high unmet medical needs."
"The commercial development of TX01 will be carried out both by Exeltis and THX Pharma. Batten-1 will follow a streamlined, less capital-intensive development plan, thanks to the extremely encouraging efficacy data collected so far."
"Our goal is to reach profitability by 2028. We continue to explore financing and partnership options to support the execution of our programmes and accelerate our international commercial development," said Mathieu Charvériat, Chief Executive Officer of THX Pharma.
THX Pharma’s roadmap is now structured around three strategic pillars: the TX01 candidate, the Batten-1 candidate and an antisense oligonucleotide platform tailored to rare diseases.
TX01 is the first formulation suitable for children of an already approved drug, currently only available in solid form. The company is preparing its commercialisation in the following regions:
- Europe, the UK, Latin America and selected Middle Eastern countries: THX Pharma is relying on its partnership with Exeltis, which will soon initiate the registration process for TX01 in Gaucher disease and Niemann-Pick type C disease, with the joint objective of launching commercialisation in 2027
- United States, Canada and Australia: THX Pharma will directly handle the registration of TX01 for Gaucher disease and Niemann-Pick type C disease by 2027, with plans to secure distribution agreements after marketing authorisation.
With this commercialisation strategy, THX Pharma targets €50m in annual peak revenues by 2031-2032.
Batten-1 is the most advanced drug in development for the juvenile form of Batten disease, a neurodegenerative condition affecting 2000 children worldwide with no available treatment to date.
THX Pharma recently published real-world efficacy data on the same primary endpoint required by regulatory agencies FDA and EMA.
This data on visual function enables the company to implement an optimised clinical development plan through to registration and commercialisation in 2028.
The company will run two parallel programmes in the US and Europe to generate efficacy data to support the regulatory submissions to the FDA and EMA.
Additional registrations in Australia and Canada are also expected, targeting a broad population of Batten disease patients.
The company estimates €4m in dedicated expenses for these steps between 2026 and 2028, with market launch expected in 2028.
In this indication, following a commercialisation strategy similar to TX01, THX Pharma aims to generate €200m in additional revenues by 2032-2033.
THX Pharma’s scientific team is also developing an antisense oligonucleotide platform tailored to rare diseases, initially focusing on one candidate targeting autophagy activation in lysosomal diseases and a second programme for glioblastoma, a market estimated at more than €3bn.
This platform benefits from decisive support from Bpifrance as part of the PickASO project.