13 April 2021 - - US-based biopharmaceutical company Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) and France-based biotechnology company Vivet Therapeutics have entered into an agreement whereby Mirum has the exclusive option to develop and subsequently commercialize Vivet's two proprietary AAV gene therapy programs for progressive familial intrahepatic cholestasis, subtypes 3 and 2, the companies said.

The two programs, VTX-803 and VTX-802, are currently being evaluated in preclinical studies by Vivet, a privately-held gene therapy biotechnology company.

Under the terms of the agreement, Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively.

Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs.

Until that time, Mirum will provide funding to support the continued research and development costs associated with the two gene therapy programs.

VTX-803 and VTX-802 are two proprietary AAV gene therapy programs of Vivet currently being evaluated in preclinical studies for progressive familial intrahepatic cholestasis, subtypes 3 and 2.

It is thought that successful correction by gene therapy of the defective MDR3 transporter and bile salt export pump functions for PFIC3 and PFIC2, respectively, may ultimately provide a cure for patients living with these rare liver diseases.

Such an approach carries the potential to overcome the main limitations of current standard of care for PFIC3 and PFIC2.

It may provide long-lasting benefits by restoring physiological bile secretion and preventing severe hepatic complications of the diseases and outweighing its related significant costs.

Vivet has received Orphan Drug Designation for VTX-803 by the US Food and Drug Administration and the European Medicines Agency.

Additionally, preclinical proof-of-concept studies highlighting VTX-803 were published in Nature Communications in 2019.

The data demonstrated sustained and significant reversal of PFIC3 disease biomarkers in a model of PFIC3.

Mirum Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.

Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome, progressive familial intrahepatic cholestasis, and biliary atresia.

Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021.

Additionally, Mirum's marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency.

Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.

To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND/CTA-enabling studies.

Vivet Therapeutics is a clinical stage emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.

Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada, a not-for-profit foundation at the Centro de Investigación Medica Aplicada, University of Navarra based in Pamplona, Spain.

Vivet's lead program, VTX-801, currently under IND clinical development with the GATEWAY clinical trial, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation by the Food and Drug Administration and the European Commission.

Vivet's second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020.

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