QuatRx Pharmaceuticals, a
privately-held biopharmaceutical company developing compounds for the
treatment of major endocrine, metabolic and cardiovascular diseases,
announced results from two Phase 1 clinical studies with its novel lipid
lowering compound, sobetirome (formerly QRX-431), in development for the
treatment of elevated LDL cholesterol (also known as "bad cholesterol")
levels. The results of the studies indicated LDL lowering with both single
and multiple doses of sobetirome in a healthy volunteer population not
enriched for elevated LDL levels. Sobetirome was generally well tolerated
in both studies. Sobetirome is a new selective thyroid hormone receptor
beta agonist that may activate reverse cholesterol transport.
"We are excited and extremely encouraged by these Phase 1 results
indicating that sobetirome was generally well tolerated and showed lowering
of LDL levels. We look forward to replicating these results in larger Phase
2 and 3 trials. We believe this new compound has potential as monotherapy
or in combination with statins for the many patients who are unable to
tolerate current therapies or who fail to achieve lipid treatment goals
with statins alone," said Robert L. Zerbe, M.D., chief executive officer
and president of QuatRx.
Study Results
Lipid-lowering effects in both single and multiple doses were seen in
the randomized, double-blind, placebo-controlled Phase 1 studies of
sobetirome. In the single dose study of 32 subjects, low-density
lipoprotein (LDL) cholesterol levels decreased by up to 22% in subjects
receiving up to 450 micrograms of sobetirome, compared to 2% in subjects
who received placebo. The greatest reductions were observed 72 hours after
dosing. In the multiple dose study of 24 subjects over two weeks,
reductions in LDL cholesterol levels decreased up to 41% at doses up to 100
micrograms once a day, compared to an overall 5% reduction for placebo.
Subjects, who were not enriched for LDL levels, followed a low-cholesterol
diet starting one week prior to randomization and then received sobetirome
or placebo. Sobetirome was generally well tolerated in both studies.
In published preclinical studies in primates, sobetirome has been shown
to lower LDL cholesterol and Lp(a) and to promote weight loss. Lp(a) is a
lipoprotein that is associated with an increased risk of stroke and
coronary heart disease. In preclinical studies in mice, sobetirome also
stimulated steps of reverse cholesterol transport. This process involves
the transport of cholesterol from peripheral tissues to the liver for
conversion into bile acids and then excretion from the body.
LDL Cholesterol, Cardiovascular Disease and Current Treatments
High LDL cholesterol levels have been shown to increase the risk of
cardiovascular disease including coronary heart disease and stroke.
Approximately 70 million Americans have some form of cardiovascular
disease, according to the American Heart Association. Lowering LDL
cholesterol to reduce cardiovascular risk is a primary focus of currently
available lipid regulating therapies, of which statins are the most
frequently prescribed class. While statins have been shown to lower LDL
levels in many people with high LDL cholesterol, some patients are unable
to tolerate statins due to side effects and many others fail to reach their
LDL treatment targets in treatment involving statins alone. In addition, a
substantial number of patients with elevated LDL levels also have other
cardiovascular risk factors such as obesity, high Lp(a) and high
triglycerides, which are not adequately addressed by statin therapy. In all
of these sizable patient sub-populations, there is a need for new treatment
options beyond statins.
About QuatRx
QuatRx Pharmaceuticals is focused on the discovery, licensing,
development and commercialization of compounds in the endocrine, metabolic
and cardiovascular therapeutic areas. In addition to sobetirome, QuatRx has
three other product candidates in active clinical development and two
preclinical development programs. Ophena(TM) which recently met all
co-primary endpoints in a large, pivotal Phase 3 clinical trial, is a
potential estrogen-free therapy for post-menopausal vaginal syndrome, a
common problem that results from thinning of vaginal tissues associated
with estrogen deficiency of menopause. Fispemifene is a new, selective
estrogen receptor antagonist that is in Phase 2 studies as an oral
treatment for testosterone deficiency and associated disorders in men.
Becocalcidiol, a patented Vitamin D analogue, is in Phase 2 clinical trials
for the treatment of psoriasis through QuatRx's licensee, CollaGenex
Pharmaceuticals, Inc. QuatRx's two preclinical small molecule programs are
designed to address common endocrine disorders in women. For press release
and other Company information, please visit quatrx.
QuatRx Pharmaceuticals
quatrx