Thanks to Theranexus for sharing this update, and also for being one of our gold sponsors at the BDFA family conference in September.
Theranexus is committed to support patients with rare neurological disorders by focusing all its research efforts on these diseases, starting with CLN3. In this context, the project Batten-1 we develop together with Beyond Batten Disease Foundation (BBDF), is our flagship project and we have decided to dedicate all our resources and efforts to make this project a success to the benefit of the CLN3 disease community.
Following the initiation of the ongoing P1/2 clinical study involving 6 young adult patients in the United States, we are now in the process of finalizing our discussions with regulatory agencies on the protocol of an international, multicentric phase 2/3 clinical trial that is to start in the first half of 2023. If successful, this trial may allow to have Batten-1, the BBDF/Theranexus’ drug candidate, approved towards the end of 2026.
Miglustat the active compound in Batten-1 is a known substrate reduction therapy that is used for more than 10 years in Niemann Pick C disease and Gaucher disease, two other lysosomal storage disorders. It acts by inhibiting glucosylceramide synthase or GCS an enzyme acting upstream of the production of complex glycosphingolipids.
In the case of juvenile Batten disease, our academic collaborators in the Baylor College of Medicine and Cardiff University have recently shown there is a specific accumulation of Gb3 glycosphingolipids in the lysosomes in various models of juvenile Batten disease, with this accumulation being responsible for lysosomal dysfunction and eventually neuronal cell death.
Inhibiting GCS with miglustat has been shown to prevent the production of these toxic Gb3 glycosphingolipids and results in a decreased lysosomal burden, a decreased neuroinflammation and thereby, a decrease in neuronal death.
This is based on these sound scientific rational and data that Theranexus and BBDF decided to move this product forward in the development process also developing a new formulation more adapted to the population of young patients who are to be treated with Batten-1.