Biopharmaceutical company THX Pharma (Theranexus) (Euronext Growth Paris:ALTHX) and the Beyond Batten Disease Foundation (BBDF) announced on Wednesday that the European Patent Office has granted European patent No. EP 4 188 371, protecting the use of miglustat, the active ingredient in Batten-1, for treating juvenile Batten disease (CLN3).
The patent is jointly owned by BBDF, Baylor College of Medicine and Cardiff University and is exclusively licensed worldwide to THX Pharma under an agreement signed in 2019. It covers 38 European Patent Convention contracting states and provides protection through to July 2041.
Research funded by BBDF demonstrated in cellular and animal models that Batten-1 can reduce lysosomal accumulation of glycosphingolipids, neuroinflammation and neuronal death.
This patent adds to THX Pharma's proprietary formulation patent covering a high-concentration, palatable oral solution of miglustat designed for paediatric administration, as well as orphan drug designations in Europe and the United States. Subject to marketing authorisation, the orphan drug designations provide 10 years of market exclusivity in the European Union and seven years in the United States, independently of patent protection.
The three protection layers are intended to support Batten-1's exclusivity beyond 2040 and form part of the global licensing agreement with Biocodex signed in February 2026.
Regulatory applications for the Batten-1-02 Phase 3 clinical trial have been submitted to the FDA, the MHRA and the European Union's Clinical Trials Information System.
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