THX Pharma (Theranexus) entered a strategic licensing agreement with Biocodex to advance two drug candidates across three rare genetic diseases, underscoring continued deal activity in high-need, low-prevalence indications where clinical risk remains high but competitive intensity is limited.

The agreement covers Batten-1, a candidate in development for juvenile Batten disease (CLN3), and TX01, a reformulated version of an already approved molecule intended for Gaucher disease type 1 and Niemann-Pick type C disease.¹ Together, the programs target ultra-rare and rare lysosomal and neurodegenerative disorders that predominantly affect pediatric populations and are associated with progressive neurological, visceral, and hematological decline.¹

Juvenile Batten disease is an ultra-rare pediatric neurodegenerative disorder characterized by progressive loss of vision, cognitive decline, motor impairment, and premature death, typically in early adulthood, with no current treatment options approved.³

Batten-1 is currently being prepared for Phase III development, with trial initiation planned in 2026.² Batten-1, originally developed by the Beyond Batten Disease Foundation, is positioned as a potential first-in-class therapy, with an international launch targeted for 2030.

TX01, by contrast, represents a life cycle and formulation innovation strategy, as the candidate is based on an adapted oral formulation of an already approved active substance and is intended to address Gaucher disease type 1 and Niemann-Pick type C disease, both lysosomal storage disorders with serious, progressive manifestations.¹

The reformulated approach is designed to improve treatment administration and better align with patient-specific needs, potentially enhancing adherence and broadening usability in real-world settings.

What are the details of the agreement?

Under the terms of the agreement, Biocodex is set to acquire two licenses, an exclusive worldwide license for the development and commercialization of Batten-1 and an exclusive license in the United States and Canada for the development and commercial exploitation of TX01.¹THX Pharma retains responsibility for leading clinical development of both programs, with financial and scientific support from Biocodex, who will manage compassionate access, market access, and commercialization activities in the licensed territories.¹

THX Pharma is also eligible to receive total payments of up to $205 million, including $14 million upfront and up to $190 million in development and commercialization milestone payments, including double-digit tiered royalties on net sales.¹

The companies said their shared objective is to advance therapeutic options in diseases where current treatments are nonexistent or limited. In rare disease markets, partnerships that combine specialized development expertise with established commercial infrastructure can be decisive, particularly when global reach and patient engagement are required.

Sources

1. THX Pharma and Biocodex Announce a Strategic Licensing Agreement in Batten, Gaucher and Niemann-Pick Type C Diseases Biocodex February 10, 2026 https://www.businesswire.com/news/home/20260210908813/en/THX-Pharma-and-Biocodex-Announce-a-Strategic-Licensing-Agreement-in-Batten-Gaucher-and-Niemann-Pick-Type-C-Diseases
2. Batten-1 THX Pharma Accessed February 11, 2026 https://www.theranexus.com/en/pipeline/batten-1.html
3. Batten Disease Clevland Clinic September 30, 2024 https://my.clevelandclinic.org/health/diseases/6018-batten-disease