Theranexus
- Biotech or pharma, therapeutic R&D
Theranexus (ALTHX) is a listed biopharmaceutical company dedicated to discovering and developing treatments for rare neurological disorders. Its lead asset, TX01 targets Niemann-Pick type C and Gaucher diseases. In December 2024, Theranexus licensed TX01 to Exeltis (Insud Pharma), granting them exclusive marketing and distribution rights in the European, LATAM and MENA markets.
Another key program is Batten-1, an exclusive, high-concentration oral liquid formulation of miglustat developed for juvenile Batten disease (CLN3). In partnership with the Beyond Batten Disease Foundation, Batten-1 has demonstrated a strong safety profile and positive efficacy data in a Phase I/II trial. Notably, real-world data indicate a statistically significant and clinically meaningful preservation of visual acuity over 12 months in treated patients. The FDA and EMA have endorsed a single pivotal Phase III trial design, positioning Batten-1 for potential approval in both the U.S. and Europe.
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