Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage drug
development company built on a pathway pharmacology technology platform,
today announced that the U.S. Food and Drug Administration (FDA) has
granted Rare Pediatric Disease Designation to CAT-1004 for the treatment
of Duchenne muscular dystrophy (DMD). CAT-1004 is designed to inhibit
activated NF-kB, a protein that plays an important role in muscle
health. CAT-1004 has the potential to reduce muscle inflammation and
degeneration and promote muscle regeneration in patients with DMD
regardless of the underlying dystrophin mutation. The FDA has previously
granted Fast Track and Orphan Drug Designations for CAT-1004 for the
treatment of DMD.
“We are pleased that the FDA's Office of Orphan Products Development has
granted CAT-1004 a Rare Pediatric Disease Designation. CAT-1004 has the
potential to make a meaningful impact for the patients and families
affected by DMD through a disease-modifying mechanism that could be
applicable to all patients with this disorder,” said Jill C. Milne,
Ph.D., co-founder and chief executive officer of Catabasis. “We look
forward to continuing to advance this product candidate in our Phase 1 /
2 MoveDMD clinical trial, which is currently enrolling.”
About Rare Pediatric Disease Designation
The FDA defines a “rare pediatric disease” as a disease that primarily
affects individuals aged from birth to 18 years and affects fewer than
200,000 individuals in the U.S. Under the FDA’s Rare Pediatric Disease
Priority Review Voucher program, upon the approval of a qualifying new
drug application (NDA) or biologics license application (BLA) for the
treatment of a rare pediatric disease, the sponsor of such an
application would be eligible for a Rare Pediatric Disease Priority
Review Voucher that can be used to obtain priority review for a
subsequent NDA or BLA. The Priority Review Voucher may be sold or
transferred an unlimited number of times.
About CAT-1004
CAT-1004 is an oral small molecule that inhibits activated NF-kB, a
protein that coordinates cellular response to muscular damage, stress
and inflammation and plays an important role in muscle health. In
skeletal muscle, activated NF-kB drives muscle degeneration and
suppresses muscle regeneration. In animal models of DMD, CAT-1004
inhibited activated NF-kB, reduced muscle inflammation and degeneration
and increased muscle regeneration. In Phase 1 clinical trials, CAT-1004
inhibited activated NF-kB and was well-tolerated with no observed safety
concerns. The FDA has granted CAT-1004 orphan drug and fast track
designations for the treatment of DMD. Catabasis is currently conducting
the MoveDMD Phase 1 / 2 trial of CAT-1004 in 4-7 year-old boys with DMD.
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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