Press Release
FOR IMMEDIATE RELEASE
ELAD®
Bioartificial Liver Trial in China Achieves Significance at
Halfway Point
San
Diego, USA, and Beijing, China, January 4, 2007
- 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® (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.”
– end –
About the Study
This study is a controlled, randomized, two-center,
single-treatment trial in China for the ELAD®
(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®
The ELAD® 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® (Extracorporeal Liver
Assist Device), which provides important metabolic support
for patients with severe liver failure.
CONTACTS:
Investors:
Aron Stem
Vital Therapies, Inc.
858-673-6840
Email:
astern@vitaltherapies.com
MEDIA:
Kelly McKenna
MS&L
415-254-6239
Email:
kelly.mckenna@mslpr.com
