Vital Therapies Outlines Plans for New Phase 3 Trial of ELAD(R) in Alcohol-Induced Liver Decompensation

Vital Therapies, Inc.

, a biotherapeutic company developing ELAD®, a cell-based therapy targeting the treatment of liver failure, today outlined plans for a possible new phase 3 clinical trial designed to confirm ELAD's safety and efficacy in alcohol-induced liver decompensation (AILD), including severe acute alcoholic hepatitis, based on trends identified in subset analyses of the Company's recently completed VTI-208 clinical trial. On August 21, 2015, the Company announced that while its first phase 3 trial, VTI-208, did not reach its primary or secondary endpoints, medically pertinent pre-specified subsets based on age and disease severity did show promising trends toward efficacy. Since then, the Company has conducted a series of pre-specified and post-hoc analyses of subsets of the data in order to determine if there is a basis for designing and implementing a new clinical trial to confirm the efficacy and safety signals identified in these subsets. The Company has drafted a preliminary protocol for the proposed new clinical trial, to be known as VTL-308, incorporating limits on age and factors associated with kidney dysfunction (creatinine) and blood clotting dysfunction (international normalized ratio, or INR), which are discrete components of the Model for End Stage Liver Disease (MELD) score. Key features of the preliminary VTL-308 protocol are being reviewed with the Company's Clinical Advisory Board and many proposed clinical investigators. While additional modifications are possible, this preliminary protocol will be used in planning for the new trial. A dialogue with the U.S. Food and Drug Administration (FDA) has been initiated and the Company has been granted a Type C meeting to review the VTL-308 protocol and discuss whether the results, if positive, would be accepted as the basis for a biologics license application, or BLA, filing, potentially supported by the VTI-208 subset analyses. The Company plans to submit the VTL-308 protocol to the FDA early in the fourth quarter, followed shortly thereafter with submissions to various European regulatory authorities. The Company also expects to begin the process of opening clinical sites in the fourth quarter. The clinical sites selected to participate in VTL-308 will be comprised mostly of high enrolling sites from VTI-208, with approximately 35 sites targeted in U.S., U.K., Ireland, Germany and Spain. The Company anticipates enrolling the first subject in VTL-308 during the first half of 2016. A post hoc analysis of VTI-208, which serves as the basis for design of our preliminary VTL-308 protocol, excludes subjects 50 years and older and also those with creatinine 1.3 mg/dL and above and INR above 2.5, in an attempt to avoid subjects with significant kidney and blood clotting dysfunction. In accord with this analysis, the 91-day survival rates were 93.9% for ELAD-treated subjects and 68.4% for controls (N=71; Pearson's Chi-squared P < 0.05). The full Kaplan-Meier curve is reproduced below: Figure 1. Kaplan Meier Curve of the Post Hoc VTI-208 Subset Age <50 years, Creatinine < 1.3 mg/dL and INR ≤ 2.5). (N=71; p<0.05; hazard ratio = 0.343). http://media.globenewswire.com/cache/31568/file/37595.pdf Based on the Company's trial design assumptions, 150 subjects would need to be enrolled for VTL-308 to have 95% power to achieve a statistically significant result. The Company estimates it will require additional capital of about $30 to $40 million to fund this trial to topline data. Of that capital, the Company contemplates raising approximately $20 million over the next six months. The Company also currently contemplates raising the balance through an at-the-market, or ATM, offering over the course of the next couple of years. As a result of the recently announced expense reductions and the expected structure and timing of the VTL-308 clinical trial, the Company believes that its existing cash and cash equivalents of approximately $63 million at the end of August will be sufficient to fund its operations to mid-2017. New Investor Presentation Concurrent with this news release, the Company's new investor presentation has been filed in a Form 8-K with the Securities and Exchange Commission. Investors can obtain further information about the data supporting the VTL-308 trial design from this presentation.