--Safety, Tolerability, and Reductions in LDL-C Observed--
Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage drug
development company built on a pathway pharmacology technology platform,
today announced positive top-line Phase 1 clinical trial data for
CAT-2054, the Company’s product candidate targeting the Sterol
Regulatory Element-Binding Protein (SREBP) pathway for the treatment of
hypercholesterolemia. CAT-2054 was well-tolerated with no serious
adverse events (AEs) observed in either the single or multiple ascending
dose arms of the double-blind, randomized clinical trial. In the
multiple ascending dose study, decreases in median LDL-C levels of up to
20% were observed at day 21 in healthy volunteers. Importantly, CAT-2054
was also found to be well-tolerated in combination with atorvastatin,
the statin drug most commonly used in the treatment of
hypercholesterolemia, and there was no evidence for impact of CAT-2054
on the pharmacokinetics of atorvastatin.
“CAT-2054 is an oral agent that targets SREBP, a key regulator of lipid
metabolism, which may be an effective means to lower LDL-C. It also has
potential effects on other metabolic parameters such as triglycerides,
glucose, and liver fat in patients. The Phase 1 data of LDL-C efficacy
signal combined with no safety issues support the continued clinical
development of CAT-2054,” said Evan A. Stein, M.D., Ph.D., Director
Emeritus of the Metabolic & Atherosclerosis Research Center, Cincinnati,
Ohio.
“Based on these data, we intend to initiate a Phase 2a trial in patients
with hypercholesterolemia in the fourth quarter of 2015,” said Jill C.
Milne, Ph.D., chief executive officer of Catabasis.
The Phase 1 clinical trial was designed to assess the safety,
tolerability and pharmacokinetics of single and multiple ascending oral
doses of CAT-2054 in 118 healthy volunteers. In the single ascending
dose portion of the trial, CAT-2054 was well-tolerated and no serious
AEs were reported. No safety signals were observed in laboratory, vital
sign or electrocardiogram results following CAT-2054 administration. The
observed AEs occurring under fed and fasted conditions at doses up to
500 mg were similar for CAT-2054 and placebo. All reported AEs were mild.
In the multiple ascending dose portion of the Phase 1 trial, healthy
volunteers received CAT-2054 or placebo at total daily doses ranging
from 100 to 750 mg given orally once or twice per day for 14 days.
CAT-2054 was also given concurrently with atorvastatin in one cohort.
CAT-2054 was well-tolerated with no serious AEs reported. No safety
signals were observed in laboratory, vital signs or electrocardiogram
results following CAT-2054 administration, and all subjects completed
dosing. At the highest doses, the most common AEs were GI-related, all
of which were mild. CAT-2054 was also well-tolerated with no safety
signals in subjects receiving atorvastatin. There was no evidence of
clinically significant changes in atorvastatin pharmacokinetics when
co-administered with CAT-2054. Catabasis plans to submit the Phase 1
data for presentation at an upcoming medical meeting.
About CAT-2054
CAT-2054 is an investigational oral drug initially being developed for
the treatment of hypercholesterolemia in patients for whom existing
therapies are insufficient. By modulating the SREBP pathway, CAT-2054
may inhibit production of important cholesterol metabolism proteins such
as PCSK9, HMG-CoA reductase, ATP citrate lyase and NPC1L1. If approved,
CAT-2054 may have the potential to be the first therapy to
simultaneously modulate cholesterol synthesis, clearance and absorption.
About Catabasis
Catabasis Pharmaceuticals is a clinical-stage biopharmaceutical company
focused on the discovery, development and commercialization of novel
therapeutics using its proprietary Safely Metabolized And Rationally
Targeted, or SMART, linker technology platform. The Company's SMART
linker technology platform is based on the concept of treating diseases
by simultaneously modulating multiple targets in one or more related
disease pathways. The Company engineers bi-functional product candidates
that are conjugates of two molecules, or bioactives, each with known
pharmacological activity, joined by one of its proprietary SMART
linkers. The SMART linker conjugates are designed for enhanced efficacy
and improved safety and tolerability. The Company’s focus is on
treatments for rare diseases. The Company is also developing other
product candidates for the treatment of serious lipid disorders. For
more information on the Company's technology and pipeline of drug
candidates, please visit www.catabasis.com.
Forward Looking Statements
Any statements in this press release about future expectations, plans
and prospects for the Company, including statements about future
clinical trial plans and other statements containing the words
“believes,” “anticipates,” “plans,” “expects,” and similar expressions,
constitute forward-looking statements within the meaning of The Private
Securities Litigation Reform Act of 1995. Actual results may differ
materially from those indicated by such forward-looking statements as a
result of various important factors, including: uncertainties inherent
in the initiation and completion of preclinical studies and clinical
trials and clinical development of the Company’s product candidates;
availability and timing of results from preclinical studies and clinical
trials; whether interim results from a clinical trial will be predictive
of the final results of the trial or the results of future trials;
expectations for regulatory approvals to conduct trials or to market
products; availability of funding sufficient for the Company’s
foreseeable and unforeseeable operating expenses and capital expenditure
requirements; other matters that could affect the availability or
commercial potential of the Company’s product candidates; and general
economic and market conditions and other factors discussed in the “Risk
Factors” section of the Company’s Quarterly Report on Form 10-Q for the
three months ended June 30, 2015, which is on file with the Securities
and Exchange Commission, and in other filings that the Company may make
with the Securities and Exchange Commission in the future. In addition,
the forward-looking statements included in this press release represent
the Company’s views as of the date of this press release. The Company
anticipates that subsequent events and developments will cause the
Company’s views to change. However, while the Company may elect to
update these forward-looking statements at some point in the future, the
Company specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as representing the
Company’s views as of any date subsequent to the date of this release.
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