THX Pharma and Biocodex Forge Strategic Rare Disease Licensing Agreement

THX Pharma and Biocodex have announced a strategic licensing agreement to support the development and commercialisation of treatments for three rare genetic diseases with high unmet medical needs. Under the agreement, Biocodex secures an exclusive global licence to develop and commercialise Batten-1, a drug candidate targeting juvenile Batten disease, and a regional licence for the United States and Canada to develop and commercialise TX01, a new formulation aimed at treating both Gaucher disease and Niemann-Pick disease type C.

The total deal is valued at up to €173 million, including a €12 million upfront payment, up to €161 million in milestone payments tied to development and sales, and tiered double-digit royalties on future net sales. THX Pharma will lead clinical development with financial and scientific support from Biocodex. Biocodex will handle compassionate use programmes, market access efforts, and commercial activities in the licensed territories. These inherited conditions typically affect children, cause progressive neurological, visceral or haematological impairment, and currently have limited or no approved treatments, making this collaboration a significant step towards bringing potential therapies to patients who lack effective options.