In late 2019, Theranexus and BBDF signed an agreement granting Theranexus an exclusive, global license agreement for the development and commercial use of drug candidate BBDF-101 for juvenile Batten disease. Batten disease belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses (NCLs). BBDF funded research aimed at identifying and validating BBDF-101, a proprietary combination of drugs based on the synergistic effect of two active ingredients, like the other Theranexus drug candidates already in clinical development. Theranexus and BBDF have since been awarded U.S. Orphan Drug Designation and European Orphan Medicinal Product designation as well as U.S. Rare Pediatric Disease status, and lately the FDA authorisation for a phase I/II clinical study.
Juvenile form of Batten disease (CLN3):
- Autosomale recessive disease
- Diagnosis in children aged 4 to 8 ans
- Symptoms: blindness, cognitive decline and loss of motor skills
- Approximately 3000 patients worldwide for all CLN mutations (including CLN3 which is the most frequent)
- No treatment available today and few competitors in clinical development