Theranexus and BBDF receive ‘favourable’ FDA opinion for drug trial

The Beyond Batten Disease Foundation (BBDF) and biopharma company, Theranexus, announced that they had received a ‘favourable opinion’ from the Food and Drug Administration (FDA) for the continuation of the partnership’s preclinical development plans, and its bid to achieve IND status and commence clinical trials of its BBDF-101 drug.

The ‘binding opinion’ was issued by the FDA in writing during a type C meeting, which approves Theranexus’ proposals to continue the preclinical development of BBDF-101. Speaking on the news, Theranexus Chairman and CEO, Franck Mouthon, commented:

“We would like to thank the FDA for its interest and the quality of regular contacts regarding development of the drug candidate BBDF-101 for Batten disease. We are delighted about this latest positive meeting with the FDA. The result is the continuation of an optimized preclinical development plan to initiate clinical trials in patients with Batten disease starting in 2021”

BBDF Chair of the Board, Craig Benson, added: “We are very pleased by the FDA’s recent approval of the preclinical development plan for BBDF-101, as this paves the way for the initiation of clinical trials which we all hope will deliver a therapeutic response for children and young adults with this terrible disease.”

Towards the end of 2019, Theraxnus and BBDF signed an agreement granting Theraxnus an exclusive licence to develop and use the BBDF-101 drug, for juvenile Batten disease. BBDF funded research aimed at validating BBDF-101, which the partnership describes as a “a proprietary combination of drugs based on the synergistic effect of two active ingredients, like the other Theranexus drug candidates already in clinical development”.

Since working together, the two organisations have been awarded US Orphan Drug Designation, European Orphan Medicinal Product Designation, and US Rare Paediatric Disease status.