THX Pharma (Theranexus) and Biocodex have entered a strategic licensing agreement to further the development of two drug candidates, Batten-1 and TX01, targeted at the Batten, Gaucher, and Niemann-Pick type C rare diseases.

Under the agreement, Biocodex will hold an exclusive worldwide licence to develop and commercialise Batten-1, which has been developed by THX Pharma for juvenile Batten disease (CLN3).

Additionally, Biocodex will receive exclusive rights in Canada and the US to develop and commercialise TX01, a new formulation of an existing molecule for treating Niemann-pick type C and Gaucher diseases.

These are genetic disorders that result in severe neurological, visceral or haematological complications.

THX Pharma is set to receive total payments up to €173m ($206.1m). This includes an upfront payment of €12m and as much as €161m in milestone payments associated with development and commercialisation achievements.

Furthermore, the company will receive double-digit tiered royalties on net sales. It will lead clinical development with financial and scientific support from Biocodex, which will also handle market access and commercialisation within the licensed territories.

Batten-1 is being prepared for a Phase III trial scheduled to commence in 2026. Juvenile Batten disease (CLN3) is an ultra-rare neurodegenerative condition leading to cognitive decline, progressive vision loss, motor impairment, and early mortality. Currently, there are no approved treatments.

The goal is for Batten-1 to become the first available therapy for this indication, with a planned international launch by 2030. The Batten-1 programme originated with the Beyond Batten Disease Foundation.

TX01 utilises an adapted oral formulation of an already approved substance for Niemann-Pick type C disease and Gaucher disease type 1. The approach aims to enhance administration methods and better meet patients’ needs.