Pre-clinical data of VTX-806 in demonstrated normalisation of metabolic parameters
Vivet Therapeutics, a clinical-stage biotech company developing novel gene therapies for rare liver metabolic disorders, has announced that the European Commission has granted Orphan Drug Designation (ODD) to VTX-806, its gene therapy product for the treatment of Cerebrotendinous Xanthomatosis (CTX).
Pre-clinical studies of VTX-806, an Adeno-associated viral (AAV) vector encoding human CYP27A1, showed a reduction of hepatomegaly in mice and normalization of compensatory enzymes and bile acids compared to current treatments.
This data, along with the debilitating effects of CTX, supported the EU Commission’s decision to grant ODD to VTX-806, offering significant benefits to CTX patients in the EU.
Dr Jean-Philippe Combal, Co-Founder & CEO at Vivet Therapeutics, reflected: “Early diagnosis and treatment of CTX is crucial to halting disease progression. At present, existing standard of care treatments can only slow or stabilise leukodystrophy, with no cure available. Preclinical data supports VTX-806’s potential as an alternative treatment option to stop, or reverse, disease progression over the long-term, or possibly even cure CTX patients.
He added: “Receiving ODD from the EU Commission provides Vivet with certain benefits during development and commercialization activities as we press forward with the anticipation of submitting ODD to the US FDA in 2024 and plan to begin preparations to enter clinical development in late 2025.”
VTX-806’s ODD follows EUR 4.9 million funding from the French government to advance its development.
The funding covers preclinical research, manufacturing process development and a clinical study to identify neurological biomarkers for treatment effectiveness.