Statement re Start of KRX-101 Phase II/III Trial
21 October 2003 - 11:54PM
UK Regulatory
Keryx Biopharmaceuticals, Inc. Initiates Phase II/III Clinical Program of
KRX-101 (Sulodexide Gelcaps) for Treatment of Diabetic Nephropathy
KRX-101 has FDA Fast-Track Designation To Treat
A Life-Threatening Kidney Condition
Study to be conducted by the Collaborative Study Group, the world's leading
clinical trial group in the field of diabetic kidney disease
NEW YORK, Oct. 21 -- Keryx Biopharmaceuticals, Inc. [Nasdaq: KERX; London
AIM: KRX] announced today the initiation of a multi-center clinical trial,
representing the Phase II portion of its Phase II/III clinical program for the
treatment of diabetic nephropathy. This trial is a randomized, double-blind,
placebo-controlled study, comparing two doses (200mg and 400mg daily) of KRX-
101, the Company's lead drug candidate, versus placebo.
The Phase II/III program is designed to assess the safety and efficacy of
KRX-101 in patients with Type 2 Diabetes who continue to have persistent
microalbuminuria despite treatment with maximum approved doses of
standard-of-care therapy (ACE inhibitors or A2 Receptor Blockers (ARBs)).
This trial will evaluate KRX-101's ability to impact urinary protein (albumin)
excretion (also referred to as proteinuria) in the study population. Patients
will be on treatment for six months, followed by two months of evaluation.
All patients in the study population will continue to receive maximum approved
doses of ACEs or ARBs during the treatment and follow-up periods.
The entire Phase II/III program is expected to enroll between
approximately 750 and 1,000 patients, with the Phase II component enrolling up
to approximately 135 patients. Enrollment in the Phase II component of the
program is expected to take six to nine months, with enrollment for the Phase
III component of the program expected to take an additional twelve to fifteen
months, depending on the final size of the trial and recruitment rates. Data
from the Phase II component of the program is expected to be available as
early as the fourth quarter of 2004.
The KRX-101 Phase II/III clinical program is being conducted by The
Collaborative Study Group (CSG), the world's largest standing renal clinical
trial group, whose execution of the ACE Inhibition trial in Type 1 Diabetic
Nephropathy and the recently completed trial of Irbesartan in Type 2 Diabetic
Nephropathy (I.D.N.T.) both led to FDA approval and the recommendation of
these agents as standards of care by the American Diabetes Association. The
program's lead investigator is Dr. Edmund J. Lewis, Director of Nephrology at
the Rush-Presbyterian-St. Luke's Medical Center in Chicago and co-chairman of
the CSG. Also participating in the Phase II component are approximately 25 of
the most highly regarded U.S. clinical centers for the disease. As the
program moves into its second component, it is anticipated that a significant
number of additional clinical centers will participate in the trial.
"The commencement of this Phase II/III clinical program marks an extremely
important milestone in the development of KRX-101, and a significant
accomplishment for Keryx," commented Michael S. Weiss, Chairman and CEO of
Keryx. "Diabetic nephropathy is the leading cause of End-Stage Renal Disease
(ESRD), a disease that has an approximately 80% 5-year mortality rate and an
annual cost to the healthcare system in excess of $15 billion today that is
expected to grow to nearly $40 billion per year by 2010. This Phase II/III
program represents a major step towards bringing this novel treatment closer
to the three to four million Americans afflicted by the disease and it
represents the first time KRX-101 will be administered to patients in the U.S.
The goal of this therapy is to potentially prevent patients afflicted with
diabetic nephropathy from ever developing ESRD and suffering the hardship of
its significant associated morbidity and mortality."
Dr. Edmund J. Lewis, Co-Chairman of CSG and lead investigator, commented,
"The CSG is very excited to begin this very important clinical work. We are
quite hopeful that this program will corroborate the efficacy KRX-101 has
demonstrated in its previous European clinical trials in which patients
treated with KRX-101 experienced highly statistically significant reductions
in urinary protein levels versus placebo. If successful, sulodexide will
offer an entirely new approach in the treatment of this devastating disease."
Dr. Lawrence Hunsicker, Co-chairman of the CSG, added, "Despite existing
therapies, the number of patients progressing to end-stage renal disease
continues to rise in epidemic proportions. With such a compelling need for a
novel treatment approach to diabetic nephropathy, this program represents one
of the most important developments in treating diabetic nephropathy today.
The CSG is excited to be able to begin enrolling patients, and looks forward
to the rapid conclusion of this important clinical program."
ABOUT KRX-101
KRX-101 (sulodexide), a first-in-class oral heparinoid compound, is being
developed for the treatment of diabetic nephropathy, a progressive and
life-threatening kidney disease which afflicts approximately 3 to 4 million
people with diabetes in the United States alone.
KRX-101 belongs to a proposed new class of nephroprotective (kidney
protecting) drugs, called glycosaminoglycans. A variety of members of this
chemical family have been shown to decrease pathological albumin excretion in
diabetic nephropathy in man. However, these heparin agents all require
therapy by injection and are all potent anticoagulants, which are blood
thinners capable of inducing bleeding. Sulodexide, on the other hand, is
given orally and, in this form, has demonstrated little, if any, anticoagulant
effects to date.
More than 20 studies have been published in leading medical journals
assessing the safety and efficacy of KRX-101 in diabetic nephropathy and other
vascular conditions. Most recently, KRX-101 demonstrated significant efficacy
in treating diabetic nephropathy in a randomized, placebo-controlled,
223-patient Phase II clinical trial (the DiNAS Study) conducted in Europe. In
this study, patients with Type 1 and Type 2 diabetes with diabetic nephropathy
were treated daily for four months with 50, 100- and 200-milligram gel caps of
KRX-101 and showed substantial dose-dependent reduction in proteinuria, with
the highest dose achieving a 74% reduction versus placebo following four
months of treatment. In addition, the data in the DiNAS Study showed that the
therapeutic effect of KRX-101 was additive to ACE-inhibitor treatment,
suggesting that KRX-101 operates under a different mechanism of action than do
ACE inhibitors and Angiotensin Receptor Blockers ("ARBs"), which represent the
existing first line of treatment for the disease. These findings were
published in the June 2002 issue of the Journal of American Society of
Nephrology.
KRX-101 (sulodexide) has a well-established safety profile based upon
nearly twenty years of marketing experience by the Company's licensor and use
by thousands of patients (representing over 50 million patient days of use) in
Italy, Spain, Eastern Europe, Asia, and South America as a cardiovascular
drug.
In 2001, KRX-101 was granted Fast-Track designation for the treatment of
diabetic nephropathy and, in 2002, the Company announced that the FDA had
agreed, in principle, to permit the Company to avail itself of the accelerated
approval process under subpart H.
ABOUT DIABETIC NEPHROPATHY
According to the American Diabetes Association, there are an estimated
17 million people with diabetes in the United States, of which 90%-95% are
people with Type II diabetes. Approximately 20% of all people with diabetes
develop diabetic kidney disease, a condition known as diabetic nephropathy.
Therefore, it is estimated that there are approximately 3 to 4 million people
suffering from the disease in the U.S. alone. Diabetes is now the most common
cause of End Stage Renal Disease, or ESRD, in the U.S. and in many other
developed nations, and represents 44% of all new cases of ESRD in the U.S.
Despite advances in clinical care, including improvements in glycemic (blood
sugar) control and blood pressure control, the number of diabetes-related
cases of ESRD continues to rise. In particular, the incidence of Type II
diabetes-related ESRD is rapidly increasing. Less than 20% of diabetics on
dialysis in the US survive for five years, making the mortality of ESRD in
this group higher than in most forms of cancer. Principally due to age and
concomitant vascular disease factors, renal transplantation is an option for
less than 20% of patients with ESRD from diabetes (as compared to patients
with ESRD caused by other non-diabetes conditions, for whom 40-50% may benefit
from renal transplantation). Thus, despite recent advances, diabetic
nephropathy remains a potentially catastrophic illness for which partial, but
insufficient treatment is available today.
ABOUT KERYX BIOPHARMACEUTICALS, INC.
Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX; London AIM: KRX) is a
biopharmaceutical company focused on the acquisition, development and
commercialization of novel pharmaceutical products for the treatment of
life-threatening diseases, including diabetes and cancer. Keryx is developing
KRX-101 (sulodexide), a novel first-in-class oral heparinoid compound, for the
treatment of diabetic nephropathy, for which Keryx is currently conducting its
U.S.-based Phase II/III clinical program. Keryx also has an active
in-licensing program designed to identify and acquire clinical-stage drug
candidates. Additionally, Keryx is seeking partners for its KinAce(TM) drug
discovery technology and related products. Keryx Biopharmaceuticals is
headquartered in New York City.
Some of the statements included in this press release, particularly those
anticipating future financial performance, timelines for the completion of the
KRX-101 Phase II/III clinical trial program and the release of data from this
program, size of the program, market size estimates for KRX-101, growth and
operating strategies and similar matters, are forward-looking statements that
involve a number of risks and uncertainties. For those statements, we claim
the protection of the safe harbor for forward-looking statements contained in
the Private Securities Litigation Reform Act of 1995. Important factors may
cause our actual results to differ materially, including: our ability to
successfully begin and complete cost-effective clinical trials of KRX-101; and
other risk factors identified from time to time in our SEC reports, including,
but not limited to, the report on Form 10-K for the year ended December 31,
2002, and our quarterly report on Form 10-Q for the quarter ended June 30,
2003. Any forward-looking statements set forth in this news release speak only
as of the date of this news release. We do not intend to update any of these
forward-looking statements to reflect events or circumstances that occur after
the date hereof. This press release and prior releases are available at
www.keryx.com. The information in Keryx's website is not incorporated by
reference into this press release and is included as an inactive textual
reference only.
KERYX CONTACT:
Ron Bentsur
VP Finance and Investor Relations
Keryx Biopharmaceuticals, Inc.
Tel: +1-212-531-5965
E-mail: ron@keryx.com
SOURCE Keryx Biopharmaceuticals, Inc.
-0- 10/21/2003
/CONTACT: Ron Bentsur, VP Finance and Investor Relations of Keryx
Biopharmaceuticals, Inc., +1-212-531-5965, or ron@keryx.com /
/Web site: http://www.keryx.com /
(KERX)
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