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CLN3 disease investigational therapy given FDA approval for clinical trial

12 September 2021

We are excited to share the news that the Beyond Batten Disease Foundation (BBDF) together with Theranexus have received Investigational New Drug (IND) approval from the FDA. This means they are now able to launch in-human studies of the drug candidate BBDF-101 to investigate the safety, tolerability and efficacy of the drug in the treatment of CLN3 disease. BBDF-101 is a combination therapy of trehalose (administered intravenously) and miglustat (administered orally).
More information on the study can be found here:
Please be assured that BDSRA Australia, along with members of our Medical and Scientific Advisory Board, remain in regular communication with BBDF and Theranexus – as we have done over the past 18 months during the program’s development – and we are strongly advocating for the opportunity for Australian patient involvement in the Phase 3 study.
We will continue to provide updates as new information comes to light.
Additionally, the BBDF will be hosting a live online Research Update webinar for families next Friday 17th September at 7am AEST. You can register to attend here.
If you have any questions in the meantime, please feel free to reach out to our head of Research, Medical and Scientific affairs, Dr Ineka Whiteman, at