BBDF Blog

Theranexus and BBDF win FDA approval on efficacy endpoints for the Phase III trial to evaluate Batten-1 in CLN3 Batten disease

• Phase III’s primary endpoint will be visual acuity, with secondary endpoints including assessment of cognitive and motor functions,
• The FDA confirmed that this sole Phase III trial would secure Batten-1 approval for Batten CLN3 disease.

Lyon, France – Austin, Texas, United States – 9 May 2023 – 7.30am CEST – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and the Beyond Batten Disease Foundation (BBDF), have today announced receipt of approval from the Food and Drug Administration (FDA) for the design and primary and secondary endpoints of the pivotal Phase III trial for Batten disease CLN3, at a meeting with the Division of Rare Diseases and Medical Genetics (DRDMG) held in mid-April.

“We are delighted with the constructive discussions we had with the FDA on defining the endpoints of Batten-1 and the design of our pivotal Phase III trial. Its valuable guidance allows us to get fully prepared for the trial launch,” explained Theranexus’ Chief Medical Officer Marie Sebille.

“We would like to thank the FDA for its support, which is fundamental to the development of our Batten-1 drug candidate, the only asset in active clinical development for Batten disease (CLN3). Our pivotal Phase III trial will enable us to gain approval for Batten-1, and also deliver rich news flow throughout the duration of the Phase III trial thanks to the parallel open-label patient cohort. More generally, we have no doubt that we can deliver a therapeutic response for patients suffering from Batten disease,” added Theranexus’ CEO Mathieu Charvériat.

From 22 to 26 February 2023 in Orlando, Florida 

▪ Presentation of preclinical data demonstrating how Batten-1 drastically reduces the buildup of toxic glycosphingolipids in Batten disease and a poster describing for the first time the burden of Batten disease. 

Lyon, France – Austin, Texas, United States – Cardiff, UK – 20 February 2023 – 6pm CET – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases and pioneer in the development of drug candidates modulating the interaction between neurons and glial cells, and its partners Beyond Batten Disease Foundation (BBDF) and Cardiff University, today announced several presentations at WORLDSymposium 2023, an international scientific event focused on lysosomal diseases (22 to 26 February in Orlando, Florida – United States).