Vital Therapies, Inc.
(VTI) announced today that it has completed the interim analysis of the
first 45 patients enrolled in its controlled, randomized, two-center,
single-treatment trial in China for the ELAD(R) (Extracorporeal Liver
Assist Device) System. The trial is studying a bioartificial liver
treatment in patients with chronic liver disease at Beijing Youan and 302
Military Hospitals in Beijing. The preliminary data indicate that ELAD has
already achieved statistical significance in the endpoint of
transplant-free survival.
"We have been very pleased with the clinical response we have seen in
patients suffering from an acute exacerbation of chronic liver disease,"
said Duan Zhong-Ping, M.D., lead investigator and professor of Beijing
Artificial Liver Treatment & Training Center and vice president, Beijing
Youan Hospital. "Our center has treated a large number of patients with
other mechanical and bioartificial liver treatments, and although the data
with ELAD is preliminary, none has looked more promising than ELAD to
impact a patient's long-term survival from a single treatment. The response
of ELAD treated patients is best explained by regeneration of the liver
since the improvement continues long after ELAD is discontinued."
ELAD augments the metabolic functions of a patient's liver, thereby
enabling a bridge to transplant or liver recovery. The system differs from
the mechanical filtration systems in development by using an immortalized
line of human liver cells which, in addition to processing and removing
blood toxins, can also supply to the patient metabolites synthesized by the
liver, such as blood clotting proteins.
The statistics for the 42 evaluable patients showed that, at this point
in time, the endpoint of transplant-free survival has already achieved
statistical significance. The Kaplan Meier survival curves showed a p value
for the difference between the treated and control groups of 0.03 (Wilcoxon
basis). A p value of 0.05 and below is generally accepted as the standard
for statistical significance in such a trial.
"While the p values will change as data continues to be generated, it
is encouraging to be achieving these kinds of values halfway through the
trial," added Dr. Duan.
Consistent with the recoveries that have been noted by the physicians
among the patients in the trial, other measures also indicate the success
of the trial to date, such as:
8 of 14 (57 percent) controls have either died or had to be transplanted
versus 7 of 28 (25 percent) treated patients.
Biochemistry measures such as bilrubin, INR, Albumin and MELD scores, and
clinical observations such as ascites, appetite and fatigue are also
showing expected improvements in treated patients versus controls.
"VTI is grateful to its outstanding staff in San Diego and in China for
the smooth implementation of the trial," said Terry E. Winters, Ph.D., VTI
chairman and CEO. "We are also grateful to the tireless staff at the
hospitals for treating the patients and to the Chinese authorities for
their cooperation in importing ELAD into China to treat these very sick
patients. We have received positive clinical response and strong support
from our clinical trial physicians as well as our Clinical Advisory Board.
Additionally, we are thrilled at the flawless scale up of manufacturing and
the logistics of providing ELAD cartridges to China. This has given us the
confidence to be able to provide commercial quantities of ELAD cartridges
to China from our plant in San Diego starting with the planned market
launch in late 2007."
These data were reviewed internally with the Vital Therapies Clinical
Advisory Board (CAB), which includes eight prominent U.S. and European
clinical experts in the field of hepatology and liver transplant surgery.
CAB Chairman, J. Michael Millis, M.D., professor of Surgery and chief,
Section of Transplantation University of Chicago, said: "This is clearly
the most promising data on artificial livers ever generated. This
experience in China and these data will provide the knowledge to enable the
CAB to assist in developing an excellent protocol for the phase 3 U.S. FDA
studies, which the company plans to begin in late 2007."
VTI is now seeking a Series C private equity financing in the United
States and China for both the commercial launch in China as well as the
implementation of phase 3 studies in the United States.
About the Study
This study is a controlled, randomized, two-center, single-treatment
trial in China for the ELAD(R) (Extracorporeal Liver Assist Device) System.
The trial is being run at Beijing Youan and 302 Military Hospitals in
Beijing and will continue enrolling until the planned 90 patient total is
achieved. The first patient was enrolled in March, and the analysis covers
the nine months through November 30. At that time, 26 of the 42 patients
had either completed the 84 day trial, died or been transplanted. Patient
enrollment criteria included seriously ill patients with acute on chronic
liver failure who were estimated to have a 50 percent probability of
mortality in 30 days.
There have been no significant safety issues attributable to ELAD. The
sole serious adverse event was not attributed to ELAD. Thrombocytopenia,
which was expected in this patient group, was handled with platelet
transfusions. The live C3A cell cartridges for this trial have been grown
at VTI's production facility in San Diego and shipped to China within a 24
hour window to treat each patient.
This trial has been conducted in China under an allowance from the
State Food and Drug Administration (SFDA). At the conclusion of the trial,
the results will be submitted to the SFDA and may also be submitted to
regulatory authorities in other countries as part of the basis for
allowance of pivotal trials.
About Liver Disease
According to the World Health Organization (WHO), severe liver disease
annually affects approximately 12 million patients worldwide. In China,
hepatitis is the third most prevalent disease, over 140 million people are
infected with a hepatitis virus and 32 million people have active viral
liver disease. In China, over 400,000 people die each year from liver
disease. Official estimates suggest that China's yearly medical expenses
for liver disease infections are more than $12 billion.
About ELAD(R)
The ELAD(R) system provides important metabolic support for patients
with severe liver failure, thereby enabling a bridge to recovery or
transplant depending on the type and severity of liver disease. The system
consists of four cartridges containing cells that function like a normal
human liver by metabolizing toxins and removing waste products while
delivering essential proteins back into the plasma. These cartridges are
incorporated into a blood pumping system at the patient's bedside and
enable continuous patient treatment for up to 12 days. The key to the
performance of ELAD is its utilization of a proprietary human hepatocyte
cell line, C3A, an immortalized cell line that can be grown, stored and
shipped worldwide.
About Vital Therapies, Inc.
Vital Therapies, Inc. (VTI) is a private San Diego-based liver therapy
company. VTI is developing the first human-liver cell-based system, ELAD(R)
(Extracorporeal Liver Assist Device), which provides important metabolic
support for patients with severe liver failure. For more information,
please visit: vitaltherapies/.
Vital Therapies, Inc.
vitaltherapies/