Vivet Therapeutics receives EUR 4.9 million to advance development of a gene therapy for the treatment of cerebrotendinous xanthomatosis

Funding from French Government as part of the France Health Innovation Plan 2030 operated by Bpifrance

Paris, France, February 1, 2024 - Vivet Therapeutics (“Vivet”), a clinical stage biotech company developing novel and long-lasting gene therapies for rare inherited metabolic disorders, announces the financing of EUR 4.9 million received from the French government to advance the development of a gene therapy for the treatment of cerebrotendinous xanthomatosis (CTX), a rare neurodegenerative disease. This funding is from the “Innovations in biotherapies” framework of the France Health Innovation Plan 2030 and specifically the acceleration strategy Biotherapies – Bioproduction in innovative therapies. Coordinated by the French Health Innovation agency, the funding, will be distributed over a three-year period and operated by Bpifrance.

CTX is a rare autosomal recessive genetic disorder that affects the body’s ability to metabolize fats known as cholesterols. Patients with CTX are unable to break down different forms of cholesterol, which build up in certain areas of the body and are characterized by fatty yellow nodules (tendon xanthomas) located in the connective tissues within the brain. These deposits can cause progressive damage to the brain and other areas of the body.  Disease symptomatology is characterized by chronic diarrhea during infancy and cataracts in late childhood. When patients enter adulthood, they begin to demonstrate progressive neurological symptoms, which may include dementia, seizures, hallucinations, depression and difficulty with coordination and speech. At present there is no cure for CTX, only treatment to slow disease progression.

Vivet will use the funds to further develop its gene therapy product VTX-806 as an effective treatment option to stop or reverse disease progression over the long-term, or potentially cure CTX in patients. By reinstating the CYP27A1 gene activity, which is impaired in CTX patients, using an adeno-associated viruses (AAV) vector, Vivet aims to demonstrate a safe and feasible treatment option for CTX disease.

Gloria Gonzalez, Chief Scientific Officer at Vivet Therapeutics, said: “Our commitment to developing treatment options for metabolic disorders is demonstrated by our lead clinical program, VTX-801 which targets Wilson Disease, alongside a second program, VTX-806, which is in development as a potential treatment option for CTX patients. Our advanced pipeline highlights our ability to provide strong translational support for rare genetic disorders and patients’ needs, notably with the help of the CTX Alliance and the Spanish CTX patient association.”

Anne Douar, Chief Operating Officer at Vivet Therapeutics, commented: “CTX is a progressive, neurodegenerative disorder, that if left untreated can lead to debilitating consequences and a poor prognosis effecting a patient’s lifespan. The funding granted as part of France Health Innovation Plan 2030 will enable Vivet to accelerate the development of a transformative and potentially curative treatment with our gene therapy product VTX-806 for patients with CTX.”

The funding is for the CrisTauX program which will cover preclinical research and development activities of VTX-806 and includes the manufacturing process development within medicine good manufacturing practices rules and a clinical study aiming at identifying biomarkers for the effectiveness of treatment in patients under standard of care. CrisTauX will enable accelerated clinical development of VTX-806 upon completion of the program.

About Vivet Therapeutics

Vivet Therapeutics is a private, clinical-stage biotech company developing novel and long-lasting gene therapies for rare inherited metabolic conditions, including Wilson’s Disease. Vivet’s gene therapy platform uses recombinant adeno-associated viruses (rAAVs) as vectors and has initiated two clinical programs and four pre-clinical assets to date. Its most advanced therapy is VTX-801, a novel gene therapy for Wilson’s Disease, with key clinical read-outs expected by the end of 2024. Vivet Therapeutics was founded in 2016 by CEO Dr Jean-Philippe Combal and CSO Dr Gloria Gonzalez-Aseguinolaza and is led by a highly experienced management team with deep expertise developing gene therapies and orphan drugs.

Vivet Therapeutics is backed by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Eurazeo.

For more information, please visit www.vivet-therapeutics.com - Follow us on LinkedIn @Vivet Therapeutics and Twitter @Vivettherapeutics

About France 2030

• Translates a double ambition: to transform key sectors of our economy (energy, automotive, health, aeronautics, space) through technological and industrial innovation, and to position France not only as a participant but as a leader in the world of tomorrow. From fundamental research and the emergence of an idea to the production of a new product or service, France 2030 supports the entire life cycle of innovation until its industrialization.

• Is unprecedented in its scale: 54 billion euros will be invested so that our companies, universities, and research organizations can successfully transition in these strategic sectors. The goal: to allow them to respond competitively to environmental challenges and attractiveness of the world to come, and to emerge as future champions in our fields of excellence, thus strengthening French sovereignty and independence in key sectors. 50% of the expenses will be dedicated to decarbonizing the economy, and 50% will be directed towards emerging actors who are driving innovation without negatively impacting the environment (according to the principle of Do No Significant Harm).
• Will be implemented collectively: the plan is thought out and deployed in consultation with economic, academic, local, and European actors who have contributed to determining strategic orientations and flagship actions. Project leaders are invited to submit their applications through open, demanding, and selective procedures to benefit from support from the State.
• Is led by the General Secretariat for Investment on behalf of the Prime Minister and implemented by the Agency for Ecological Transition (ADEME), the National Research Agency (ANR), Bpifrance and the Caisse des Dépôts et Consignations (CDC).

More information on: france2030.gouv.fr | https://www.gouvernement.fr/agence-innovation-sante | @SGPI_avenir

About-Bpifrance

Bpifrance finances companies at every stage of their development with credit, guarantees and equity. Bpifrance supports them in their innovation and international projects and now also insures their export business through a wide range of products. Consulting, training, networking and acceleration programs for start-ups, SMEs and mid-sized companies are some of the other services it offers to entrepreneurs. Thanks to Bpifrance and its 50 regional offices, entrepreneurs have a single local contact to help them through the challenges they face.

For more information, visit www.bpifrance.fr – Follow us on Twitter: @Bpifrance - @BpifrancePresse