SAN DIEGO, CA--(Marketwire - Sep 27, 2012) - Vital Therapies, Inc., a development stage biotechnology company focused on human allogeneic cellular therapy for life-threatening acute liver failure, today announced the first closing of $16 million from a multi-stage commitment to invest $76 million in the company led by Vital Therapies' existing private investors.
The investment is designed to fund three pivotal phase 3 trials of the company's bio-artificial liver therapy, ELAD, in two specific forms of acute liver failure. The three separate randomized, controlled, open-label trials are planned to enroll a total of 375 subjects in the USA, EU and Australia/New Zealand and will be the largest such trials done in the field. The company has been working with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) on the design of these trials and has received written guidance from both regulatory agencies. All three trials are measuring survival as their primary endpoint. Details will be posted on www.clinicaltrials.gov soon.
The first trial, in acute alcoholic hepatitis, is expected to involve over 40 clinical sites, mostly in the USA. The trial is now open for enrollment and the first subject is expected to be enrolled in the fourth quarter of 2012. The second and third trials, in subjects with acute alcoholic hepatitis who fail steroid therapy and in subjects with fulminant hepatic failure, respectively, are in the protocol design stage and are anticipated to begin enrolling patients in early 2013. The steroid-failure trial is expected to enroll patients primarily in Europe while the fulminant trial subjects should be enrolled in all the locations. Sites in Australia and New Zealand are also being explored for participation in all three trials.
The trials are expected to be complete by the end of 2015. If the results are successful and the marketing application is granted priority review and approved in both the USA and EU, market launch could occur on both continents before the end of 2016. Vital Therapies retains all rights to ELAD and has no corporate partners.
Dr. Michael Millis, Head of Transplant at the University of Chicago and a member of Vital Therapies Board of Directors, said, "We are delighted to have secured funding to begin the largest pivotal program involving artificial liver support ever implemented. Acute liver failure represents a clear, unmet need and, if this program is successful and regulatory approvals can be obtained, ELAD will hopefully provide a life-saving alternative to these patients for whom so few options exist."
Dr. Duane Nash, Vital Therapies' Chief Business Officer and Medical Director, said, "We are all deeply grateful to our investors for their generous support, particularly in this time of great uncertainty for smaller biotech companies. We are very excited to build on what we believe to be particularly promising clinical data sets for ELAD, and look forward to an expeditious start to our pivotal program."
In separate news, the company received on September 7 written confirmation from the FDA that the company's Orphan Designation for ELAD in acute liver failure, originally received in July 2004 with respect to fulminant hepatic failure, also extends to acute alcoholic hepatitis. "The timing of this notification is particularly welcome, just as we are about to begin our first phase 3 trial. We are now in the fortunate position to have Orphan Designation in both indications targeted by our pivotal program," noted Dr. Nash.
The FDA may grant orphan designation to drugs intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the United States. Orphan drug designation must be requested before submission of a Biologics Licensing Application, or BLA. If the FDA grants orphan drug designation, the generic identity of the therapeutic agent and its potential orphan use are disclosed publically by the FDA. Orphan drug designation does not convey any advantage in, or shorten the duration of, the regulatory review and approval process. If a product that has orphan designation subsequently receives FDA approval for the indication for which it has such designation, the product is entitled to, among other things, orphan exclusivity, which means the FDA may not approve any other application to market the same product for the same indication, except in very limited circumstances, for up to seven years after receiving FDA approval. Independent of Orphan Designation, if ELAD is approved, it will also be eligible for 12 years of data exclusivity under the Patient Protection and Affordable Care Act of 2010.
About Vital Therapies Inc.
Vital Therapies, Inc. is a private development-stage biotechnology company based in San Diego, California. The company is developing ELAD®, an extracorporeal liver support system which functions by processing toxins and synthesizing proteins and metabolites that are the key processes of normal human liver function, and represents the first human allogeneic cellular therapy for life-threatening acute liver failure. Vital Therapies has completed seven clinical trials in US, EU and Asia. A US/EU pivotal trial plan to secure BLA and MAA approval has received written guidance from FDA and EMA. The first protocol under this plan, for a survival trial in acute alcoholic hepatitis, has been allowed by FDA and is open for enrollment.
ELAD is a bedside system whose central component is four cartridges containing 440 grams of immortalized human liver cells and 32,000 hollow fibers. The patient's plasma flows inside of the hollow fibers to allow two-way transfer of metabolites. During ELAD therapy the cells metabolize toxins and synthesize proteins and other liver specific products essential for life. The ELAD cell cartridges are produced at VTI's GMP-compliant facility in San Diego, California.
ELAD is a trademark of Vital Therapies, Inc.
This press release may contain forward-looking statements, including statements regarding anticipated future closings of the staged financing, our planned phase 3 trials and ELAD's market launch. These statements relate to future events and are subject to risks, uncertainties and assumptions about Vital Therapies. These statements are only predictions based on our current expectations and projections about future events. You should not place undue reliance on these statements. Actual events or results may differ materially. Many factors may cause our actual results to differ materially from any forward-looking statement. We do not undertake to update any forward-looking statements.