THX Pharma and Biocodex Sign Strategic Licensing Agreement for Rare Disease Drug Candidates
Wednesday, February 11, 2026
THX Pharma and Biocodex, an independent international pharmaceutical group, have entered into a strategic licensing agreement to advance two drug candidates targeting three rare diseases with high unmet medical need. The agreement covers Batten disease with Batten-1, and Gaucher disease and Niemann-Pick type C disease with TX01.
Under the terms of the deal, Biocodex has obtained two licences from THX Pharma. These include an exclusive worldwide licence for the development and commercialisation of Batten-1 for juvenile Batten disease (CLN3), and an exclusive licence for the United States and Canada for the development and commercial use of TX01, a new formulation of an already approved molecule intended to treat Gaucher disease and Niemann-Pick type C disease.
These rare genetic disorders, many of which affect children, can cause severe and progressive neurological, visceral and blood-related complications. They have a major impact on quality of life and life expectancy. In several cases, treatment options are limited or not available.
THX Pharma is eligible to receive total payments of up to €173 million under the agreement. This includes an upfront payment of €12 million and up to €161 million in development and commercial milestone payments, along with double-digit tiered royalties on net sales. THX Pharma will continue to lead clinical development, supported financially and scientifically by Biocodex. Biocodex will manage compassionate use programmes, market access and commercial activities in the licensed territories.
Batten-1 is currently being prepared for Phase 3 clinical development, with the trial start planned for 2026. Juvenile Batten disease is an ultra-rare paediatric neurodegenerative disorder that leads to progressive vision loss, decline in cognitive and motor function, and premature death in early adulthood. There are currently no approved treatments. Batten-1 is being developed with the aim of becoming the first approved therapy for this condition, with a potential international launch targeted for 2030. The programme was originally initiated by the Beyond Batten Disease Foundation.
TX01 is an adapted oral formulation of an already approved active substance. It is intended for the treatment of Niemann-Pick type C disease and Gaucher disease type 1, with the goal of improving ease of administration and better meeting patient needs.
Source: businesswire.com
