Preclinical Data for VTX-806 Demonstrates Normalization of Metabolic Parameters
Vivet Therapeutics, a clinical-stage biotechnology company developing a novel gene therapy for a rare liver metabolic disease, announced that the European Commission has granted Orphan Drug Designation (ODD) to its gene therapy product VTX-806, intended to treat cerebrotendinous xanthomatosis (CTX).
Preclinical studies of VTX-806, an adeno-associated viral (AAV) vector encoding human CYP27A1, have shown reduced hepatomegaly and normalized compensatory enzymes and bile acids in mice compared with current therapies.
This data, coupled with the debilitating effects of CTX, supported the EU Commission’s decision to grant ODD to VTX-806, providing significant benefit to CTX patients in the EU.
“Early diagnosis and treatment of CTX is essential to halt disease progression. Currently, existing standard of care can only slow or stabilize the progression of leukodystrophy, but there is no cure. Preclinical data supports the potential of VTX-806 as an alternative treatment option to potentially halt or reverse disease progression in the long term, or even cure patients with CTX,” said Jean-Philippe Combal, PhD, co-founder and CEO of Vivet Therapeutics.
He added: “Receiving the ODD from the European Commission provides Vivet with certain benefits in its development and commercialization activities and allows it to move forward with its planned submission of the ODD to the U.S. FDA in 2024 and preparation to enter clinical development in the second half of 2025.”
VTX-806’s ODD has received €4.9 million in funding from the French government to advance its development.
The funding will cover preclinical research, manufacturing process development, and clinical studies to identify neurological biomarkers of treatment efficacy.
