Theranexus rebrands as THX Pharma signals, shift to commercial pharmaceutical development
French pharmaceutical company Theranexus has announced its transformation into THX Pharma, marking a strategic pivot from biotechnology research towards regulatory approval and commercial deployment of treatments for rare neurological diseases. The rebranding of the Lyon-based company positions it to advance two lead drug candidates through registration and international market access.
Strategic evolution centres on two key therapeutic programmes
The rebranding reflects the company’s transition from laboratory innovation to market-ready pharmaceutical products, with particular focus on TX01 and Batten-1. TX01 represents a paediatric formulation of an existing approved drug, currently available only in solid form, whilst Batten-1 has been identified as the most advanced therapeutic candidate worldwide for treating Batten disease.
Mathieu Charvériat, chief executive officer of THX Pharma, said: “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.”
TX01 targets dual rare disease indications with global commercialisation strategy
The company has structured a comprehensive geographical approach for TX01 development. In Europe, the United Kingdom, Latin America, and selected Middle Eastern territories, THX Pharma will leverage its partnership with Exeltis to initiate registration processes for Gaucher disease and Niemann-Pick type C disease, targeting 2027 for commercial launch. For the United States, Canada, and Australia, THX Pharma plans direct registration by 2027, with subsequent distribution agreements following marketing authorisation.
The company projects annual peak revenues of €50 million for TX01 by 2031-2032, based on this multi-regional commercialisation framework.
Batten-1 programme advances with dual-track clinical development THX Pharma’s second major programme addresses juvenile Batten disease, a neuro-degenerative condition affecting approximately 2,000 children globally with no currently available treatment. The company recently published real-world efficacy data measuring the primary endpoint specified by the Food and Drug Administration and European Medicines Agency, focusing on visual function outcomes.
The development strategy comprises two parallel programmes. In the United States, an early access programme will provide treatment to approximately 50 patients, largely funded through patient insurance mechanisms in accordance with recent FDA guidance. Simultaneously, a Phase 3 open-label clinical trial will enrol around 20 patients in Europe for a 12-month treatment period.
Both programmes are designed to generate efficacy data supporting regulatory submissions to FDA and EMA, with additional registrations planned for Australia and Canada. THX Pharma estimates €4 million in dedicated expenses for these development phases between 2026 and 2028, targeting market launch in 2028. The company projects €200 million in additional revenues from this indication by 2032-2033.
Antisense oligonucleotide platform expands therapeutic pipeline
Beyond its two lead candidates, THX Pharma continues developing an antisense oligonucleotide platform tailored for rare diseases. Current work includes a candidate targeting autophagy activation in lysosomal diseases and a second programme for glioblastoma, addressing a market valued above €3 billion. This platform receives support from Bpifrance through the PickASO project. The company aims to achieve profitability by 2028 whilst exploring financing and partnership options to support programme execution and accelerate international commercial development. THX Pharma is listed on Euronext Growth Paris under ticker ALTHX.
For more information, visit: http://www.thxpharma.com
