Vivet's First Gene Therapy Product, VTX 801 for Wilson's Disease, Receives European and US …

PARIS–()–Vivet Therapeutics, announced today that both the Food and Drug
Administration (FDA) and the European Commission (EC) have granted
Orphan Drug Designation (ODD) for Vivet’s lead gene therapy product,
VTX-801, for the treatment of Wilson’s Disease (WD), a chronically
debilitating and life-threatening condition if not treated.

“This designation validates Vivet’s efforts to treat the copper
metabolism disorder, Wilson’s Disease. The FDA and EC have both
recognized the unmet need for a safe and effective treatment of Wilson’s
Disease by physiological restoration of copper homeostasis and the
potential of VTX 801 to address such need”
stated Jean-Philippe
Combal, CEO of Vivet Therapeutics.

VTX-801 is Vivet’s first gene therapy product to be granted Orphan Drug
Designation. This is an important value driver for Vivet, in particular
through the 10 & 7-years market exclusivity in the EU & US respectively
if VTX-801 is approved for the treatment of Wilson’s Disease. Initiation
of clinical development is planned for Q4 2018 in both the US and the EU.

“Through our strategic collaboration with Fundación para la
Investigación Médica Aplicada (FIMA) at the Centro de Investigación
Medica Aplicada (University of Navarra, Spain), we are building an
unparalleled platform to address key challenges in gene therapy, notably
improving sustainability and immune tolerance, targeting Wilson’s
Disease and other hepatic rare metabolic genetic disorders. A critical
asset to this platform is a new liver targeting AAV-Anc80 serotype,
licensed from Massachusetts Eye and Ear (MEE), a teaching hospital of
Harvard Medical School, Boston” added Jean-Philippe Combal.

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About VTX-801: VTX-801 is an adeno-associated virus (AAV)
containing a gene coding for a functional mini-ATP7B copper transporter,
that is intended to restore copper metabolism through liver cell

About WiIson’s Disease: Wilson’s disease is an inherited orphan
disorder affecting approximately 1:40,000 individuals. In this
condition, the physiological copper biliary elimination pathway is
disrupted, leading to tissue accumulation of toxic copper levels, most
notably in liver and CNS. Onset of symptoms typically occurs in
teenagers or in young adults and left untreated, the condition uniformly
progresses to severely debilitating complications and death. The disease
is due to inactivating mutations in the gene encoding the copper
transporter ATP7B.

About Vivet Therapeutics: Vivet Therapeutics is a gene therapy
biotech company with headquarters in Paris, France, dedicated to the
research, development and future commercialization of gene therapy
products for inherited liver disorders with high medical need.

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