THX Pharma and Biocodex Announce a Strategic Licensing Agreement in Batten, Gaucher and Niemann-Pick Type C Diseases

GENTILLY, France–(BUSINESS WIRE)–THX Pharma (Theranexus) and Biocodex, an independent international pharmaceutical group, today announced the execution of a strategic licensing agreement to advance the development of two drug candidates in three rare diseases with high unmet medical needs: Batten disease with Batten-1, and Gaucher disease and Niemann-Pick type C disease with TX01.

Under the agreement, Biocodex acquires from THX Pharma two licenses: an exclusive worldwide license for the development and commercialization of Batten-1, a drug candidate developed by THX Pharma in juvenile Batten disease (CLN3); and an exclusive license for the United States and Canada for the development and commercial exploitation of TX01, a new formulation of an already approved molecule intended for the treatment of Gaucher disease and Niemann-Pick type C disease.

These genetic diseases, frequently pediatric, cause severe and progressive visceral, hematological or neurological impairments, with a major impact on patients’ quality of life and life expectancy. For some of them, therapeutic options remain nonexistent or very limited, making each development program a considerable scientific and medical challenge. The shared ambition of Biocodex and THX Pharma is to advance therapeutic solutions where needs remain significant, relying on science, partnership and dialogue with all communities, including healthcare professionals and patient organizations.

THX Pharma will receive total payments that may reach €173 million, including an upfront payment of €12 million and up to €161 million in development and commercialization milestone payments, as well as double-digit tiered royalties on net sales. THX Pharma will lead the clinical development of the programs, with financial and scientific support from Biocodex, which will also carry out compassionate access, market access and commercialization activities in the territories covered by the licenses.

Batten-1 is currently in preparation for phase 3 (start planned in 2026). Juvenile Batten disease (CLN3) is an ultra-rare pediatric neurodegenerative disease leading to progressive loss of vision, cognitive and motor functions, followed by death in early adulthood. No treatment is approved to date. Batten-1 aims to become the first available treatment for this condition, with an international launch targeted for 2030. The Batten-1 program was initially developed by the Beyond Batten Disease Foundation.

TX01 is based on an adapted oral formulation of an already approved active substance and is intended for the treatment of two rare lysosomal diseases: Niemann-Pick type C disease and Gaucher disease type 1. This approach aims to improve treatment administration and its suitability for patients’ specific needs.

About Biocodex: Biocodex is an independent French pharmaceutical company present in over 100 countries. Building on decades of commitment in rare diseases, Biocodex works to advance therapies for patients facing high unmet medical needs, alongside its pioneering work in microbiota research and its innovative solutions in women’s health and established products addressing common health needs.

About THX Pharma: THX Pharma (Theranexus) is a biopharmaceutical company specializing in the development of innovative treatments for rare diseases, with a focus on neurological conditions. It is developing Batten-1, targeting the juvenile form of Batten disease, and TX01, a new formulation intended for the treatment of Gaucher disease and Niemann-Pick type C disease.

Contacts

Media:
BIOCODEX / Monet – Anne-Lise Le Vaillant or Florence Desremaux
rpbiocodex@monet-rp.com – +33 1 45 63 12 43