Mirum Pharmaceuticals and Vivet Therapeutics Enter into Exclusive Worldwide Option and License Agreement for Vivet’s Gene Therapy Programs Targeting Progressive Familial Intrahepatic Cholestasis
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Vivet Therapeutics (“Vivet”) announced today 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 (PFIC), subtypes 3 and 2. The two programs, VTX-803 and VTX-802, are currently being evaluated in preclinical studies by Vivet, a privately-held gene therapy biotechnology company.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210412005354/en/
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.
“This gene therapy collaboration will work to address the root cause of PFIC3 and PFIC2 and provide an option for patients who do not respond to ASBT inhibition,” said Chris Peetz, president and chief executive officer at Mirum. “We are encouraged by the expertise and dedication of the Vivet team to develop next-generation gene therapies that have the potential to transform the lives of patients and their families. There are clear synergies in our combined missions to help the people who need it the most.”
“Mirum’s dedication to the treatment of cholestatic liver diseases and those rare diseases for which there are limited medications make them a great partner to potentially develop and bring to market VTX-803 and VTX-802,” said Jean-Philippe Combal, chief executive officer and co-founder of Vivet. “Their leadership in rare liver disease and in particular PFIC, is important as they not only understand the urgent need for patients but also that today’s treatment options will never be enough. Gene therapy is a transformative approach, potentially benefiting more patients.”
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 (PFIC), subtypes 3 and 2. It is thought that successful correction by gene therapy of the defective MDR3 transporter and bile salt export pump (BSEP) 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 U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). 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.
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. 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 (ALGS), progressive familial intrahepatic cholestasis (PFIC), 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. For more information, visit MirumPharma.com.
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.
About Vivet Therapeutics
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 (rAAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada (CIMA), 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 (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damages, neurologic symptoms and potentially death.
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.
Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive), and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe. Six types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease The PFIC2 patient population accounts for approximately 60% of the PFIC patient population. PFIC2 is caused by a mutation in the ABCB11 gene, which normally encodes a bile salt export pump protein that moves bile acids out of the liver.
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the option and license agreement between Mirum and Vivet and the potential development of VTX-802 and VTX-803. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “plans,” “will,” ”may,” “anticipates,” “expects,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
Ian Clements, Ph.D.
Vivet Therapeutics Contacts:
About Business Wire
(c) 2018 Business Wire, Inc., All rights reserved.
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Greece’s Eurobank Selects Temenos to Reimagine Wealth Management for the Digital Age6.5.2021 08:30:00 CEST | Press release
Temenos (SIX: TEMN), the banking software company, today announces that Eurobank in Greece has selected Temenos Wealth to strengthen its position in wealth management and affluent segment services. Eurobank, the third largest bank in Greece, will use Temenos Wealth to create a fully-digital investment portfolio management service offering Robo-Advisor and Goal-Based Investing apps to target the mass affluent and affluent market. These growing market segments want personalized offerings and are used to convenience through digital channels. With the Temenos solution, Eurobank combines an efficient back-office and product-building engine with modern omnichannel customer experiences powered by AI to meet the segments’ high expectations. The new Robo-Advisor app will allow Eurobank’s customers to create or adjust a desired portfolio, based on personalized criteria such as risk tolerance, target dates and asset size, by themselves or with an advisor. The solution manages the investments auto
Olympus Targets Carbon Neutrality by 20306.5.2021 08:00:00 CEST | Press release
Olympus Corporation (”Olympus” – Director, Representative Executive Officer, President and CEO: Yasuo Takeuchi) announced today it is targeting carbon neutrality by 2030, while also agreeing with recommendations by the Task Force on Climate-Related Financial Disclosures (TCFD), and adding carbon neutrality and enabling a circular economy to its environmental, societal and governance (ESG) enhancement measures. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210505006248/en/ Olympus has in place a variety of environmental initiatives across its global operations. However, the company is acutely aware of the damaging effects of climate change on natural ecosystems. For this reason, it has added carbon neutrality and enabling a circular economy as an additional ESG materiality criterion. In its corporate strategy announced in November 2019, Olympus listed four distinct ESG criteria as material ESG enhancement measures: (1) healt
Chestertons Global Expands Footprint Including New Offices Across the Caribbean, Europe and North Africa6.5.2021 08:00:00 CEST | Press release
Global estate agency, Chestertons Global Franchise Holdings Limited, today announced that Chestertons Cyprus has joined its expanding network, which has seen rapid growth over the past year. Since Chestertons Ionian, headquartered in Corfu, was welcomed on board as the first Global Franchise Partner in early March 2020, the business has gone from strength to strength, with new teams joining the Chestertons network in the following locations: Barbados, Antigua & Barbuda, Portugal, Morocco, the Costa del Sol, Gibraltar and now Cyprus. Chestertons Cyprus, headed up by Principal Andrew Constantinou, has launched its first office in Paphos, with plans for additional offices in the works. With the Portugal and Costa del Sol openings bringing five offices apiece to the network, the new franchise offering has secured 16 offices in total to date, increasing the number of property listings on the global business’ books by 300%. The expansion of the franchise business means Chestertons’ geographi
SES Announces EUR 100 million Share Buyback Programme6.5.2021 07:30:00 CEST | Press release
SES S.A. today announces a share buyback programme of up to EUR 100 million to be executed by 31 December 2021 under the authorisation given by the Annual General Meeting of shareholders held on 1 April 2021. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210505006251/en/ SES announces EUR 100 million share buyback programme (Photo: Business Wire) The company will purchase up to 12 million A-shares and up to 6 million B-shares in equal proportion to maintain the ratio of two A-shares to one B-share, as required by the Articles of Association. The aggregate value of the programme shall not exceed EUR 100 million and the shares acquired under the programme are intended to be cancelled to reduce the total number of voting and economic shares. SES affirms its commitment to maintain balance sheet metrics consistent with investment grade ratios. “The share buyback programme that we are announcing today reflects our confidence in t
SES: Q1 2021 Results6.5.2021 07:30:00 CEST | Press release
SES S.A. announces financial results for the three months ended 31 March 2021. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210505006250/en/ SES Q1 2021 Results (Photo: Business Wire) Strong execution delivering revenue of €436 million and Adjusted EBITDA(1) of €268 million Improving trajectory in Video from -8.0% YOY(2,3) in FY 2020 to -4.6% YOY(2,3) in Q1 2021 Solid Networks performance (flat YOY(2,3)) in the COVID environment with strong prospects for future growth 7% YOY(3) reduction in recurring Operating Expenses, reflecting S&A(4) gains, supporting higher YOY Adjusted EBITDA margin (61%) Solid cash flow generation and financial discipline supporting lower YOY leverage ratio(5) of 3.1 times at 31 March 2021 Adjusted Net Profit up 42% YOY to €75 million On track to deliver 2021 outlook. Growth investments and C-band proceeds supporting future growth and value creation Over 85% of 2021 revenue outlook (€1,760-1,820 mil
AMD President & CEO Dr. Lisa Su to Keynote at COMPUTEX 2021 on High-Performance Computing Ecosystem6.5.2021 07:08:00 CEST | Press release
TAITRA (Taiwan External Trade and Development Council) announced today that Dr. Lisa Su, President and CEO of AMD, is invited back to deliver a keynote address at COMPUTEX 2021. This digital keynote will be on Tuesday, June 1, at 10:00 AM Taipei time, with the keynote theme “AMD Accelerating – The High-Performance Computing Ecosystem”. COMPUTEX displays will be digital this year, with keynotes and forums running on hybrid. “It has been a year unlike others. Technology has gotten us through some of the most challenging times,” said James Huang, TAITRA Chairman. “We will continue to transform our exhibition models and practices to meet the evolving needs of our exhibitors, visitors, and media, without losing the most essential element of a trade show – connection.” Dr. Lisa Su is proud to join COMPUTEX once again in 2021. “The past year has shown us the important role high-performance computing plays in our daily lives – from the way we work to the way we learn and play. At this year’s C
Deenova Keeps Smashing New Sales Records in 2022 France’s Pharmacy Automation Market6.5.2021 07:00:00 CEST | Press release
Deenova announced today it keeps its record-breaking market momentum started in 2021, by winning competitive public tenders at Resah for 6 D3 ACCED Robots and multiple D3 Astus medication solutions to be operated around the clock at Nimes University Hospital, Centre Hospitalier Intercommunal Eaubonne Montmorency-Simone Veil in Sud Val d’Oise, and finally Centre Hospitalier Sainte-Marie Le Puy-en-Velay. Christophe Jaffuel, Deenova Chief Commercial Officer, stated: “I am very proud France continues to lead Deenova’s international growth for 2 years in a row. Our team latest 3 competitive wins highlight Deenova main differentiating characteristics over our competition, especially in France: Patient Care Crafted for Better Safety and Greater Therapy Compliance, as well as 24/7 availability, a key component of customer needs”. Loïc Bessin, Managing Director of Deenova in France stated: “I am delighted to welcome 3 new groups of hospital customers to the fast-growing Deenova family in Europe