Developing Drug Candidates for the Treatment of Rare Neurological Diseases
THX Pharma (ALTHX) is a listed biopharmaceutical company dedicated to discovering and developing treatments for rare neurological disorders. THX pharma has two lead assets TX01 and Batten-1:
TX01 is a proprietary, high-concentration oral solution built on an approved active ingredient, specifically formulated to address specific unmet needs in two rare lysosomal storage disorders: Niemann-Pick disease type C and Gaucher disease. THX Pharma (ALTHX) is currently finalizing its market authorization dossier for submission to both the EMA and FDA before year-end, with regional launches to follow across additional territories by 2027, and a projected revenue milestone of ~€50M by 2032. To accelerate patient access globally, the company has established two strategic licensing partnerships: Exeltis (Insud Pharma Group) holds exclusive commercialization rights across Europe, Latin America, and the MENA region, while Biocodex covers the United States and Canada. THX retains APAC rights and is actively seeking out-licensing partners to complete its global coverage.
Batten-1 is a proprietary, high-concentration oral liquid formulation of miglustat, optimized to meet the specific needs of pediatric and dysphagic patients with Batten disease — a devastating rare neurological disorder. Early clinical evidence is compelling: Phase I/II data demonstrated clear target engagement, reductions in neuronal death markers, and stabilization signals, while real-world data have shown clinically meaningful visual acuity stabilization. Both the FDA and EMA have endorsed a single pivotal Phase 3 pathway, streamlining the path to approval across major markets. THX Pharma (ALTHX) is currently advancing its Phase 3 program alongside an optimized Early Access strategy, underpinned by orphan designation and patent protection extending to ~2047 — providing a durable competitive moat. The global development and commercialization rights have been exclusively licensed to Biocodex, ensuring a well-resourced partner to drive the program through to launch and beyond. Peak revenue is modelled at ~€200M by 2033 across target markets.
Beyond its clinical-stage assets, THX Pharma can leverage a proprietary discovery platform to identify and develop next-generation drug candidates based on antisense oligonucleotide (ASO) technology. Two programs are currently underway:
The first targets TFEB, a transcription factor widely regarded as the master regulator of autophagy — the cellular recycling process by which cells clear damaged proteins and organelles. Dysregulation of autophagy is implicated in a broad spectrum of degenerative conditions, including Parkinson’s disease, Alzheimer’s disease, ALS, and Huntington’s disease, positioning TFEB modulation as a high-value therapeutic axis across neurology and beyond.
The second program is focused on characterizing an undisclosed target with promising therapeutic potential in glioblastoma — the most aggressive and lethal primary brain tumor in adults, with a median survival of under 15 months and fewer than 5% of patients alive at five years; approximately 300,000 new cases are diagnosed worldwide each year. Both programs are conducted in collaboration with leading French academic teams specializing in ASO therapeutics, and the TFEB project has received formal recognition through a France 2030 grant awarded by BPI France — validating both the scientific rationale and the quality of the research partnership.