– SAT-3247 is a proprietary, oral small
molecule drug being developed by Satellos as a novel treatment to
regenerate skeletal muscle which is lost in Duchenne muscular
dystrophy (DMD)
– DMD is a rare genetic disorder characterized
by progressive muscle degeneration and weakness
– Satellos has also received Orphan Drug
Designation from the FDA for SAT-3247
– Initiation of a Phase 1 clinical trial of
SAT-3247 anticipated in Q3 2024
Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX:
MSCL, OTCQB: MSCLF), a public biotech company developing new small
molecule therapeutic approaches to improve the treatment of muscle
diseases and disorders, announced today that the U.S. Food and Drug
Administration (FDA) has granted Rare Pediatric Disease Designation
to SAT-3247 for the potential treatment of Duchenne muscular
dystrophy (“Duchenne” or “DMD”) after receiving Orphan Drug
Designation earlier this year. SAT-3247 is a first-in-class oral
small molecule therapeutic designed to restore the innate muscle
regeneration and repair process, independent of dystrophin and
regardless of exon mutation status.
“Obtaining the Rare Pediatric Disease and Orphan Drug
Designations for SAT-3247 from the FDA are important milestones for
Satellos as we continue to progressively build value in our DMD
program,” said Frank Gleeson, CEO and Co-founder of Satellos. “The
Rare Pediatric Disease Designation for SAT-3247 highlights the
continued need for new disease modifying therapeutic options for
pediatric patients with Duchenne, a need which we believe SAT-3247
has the potential to address.”
The FDA grants Rare Pediatric Disease Designation for serious
and life-threatening diseases that primarily affect children ages
18 years or younger and fewer than 200,000 people in the United
States. The Rare Pediatric Disease Priority Review Voucher Program
is intended to address the challenges that drug companies face when
developing treatments for these unique patient populations. Under
this program, a sponsor who receives an approval for a drug or
biologic for a “rare pediatric disease” may be eligible for a
voucher that can be redeemed to receive priority review of a
subsequent marketing application for a different product or sold to
another sponsor for priority review of their marketing
application.
The FDA grants Orphan Drug Designation to support development of
medicines for underserved patient populations, or rare disorders,
that affect fewer than 200,000 people in the U.S. Orphan Drug
Designation provides certain benefits, including the potential for
a seven-year market exclusivity upon regulatory approval, exemption
from FDA application fees, tax credits for qualified clinical
trials, and a priority review voucher.
About SAT-3247 SAT-3247 is an oral, small molecule drug
candidate designed to target the root cause of muscle loss in
degenerative diseases, initially in Duchenne. SAT-3247 presents a
novel mechanism of action to restore impaired muscle regeneration
caused by the absence of functional dystrophin.
About Duchenne Muscular Dystrophy Duchenne muscular
dystrophy is an inherited disease caused by mutations in the
dystrophin gene that no longer allow the dystrophin protein to
function properly. Consequently, as discovered by Satellos, muscle
repair and regeneration are impaired. Satellos designed SAT-3247 to
restore the process of muscle repair and regeneration by regulating
a dystrophin-independent pathway with the goal of increasing muscle
function. SAT-3247 is intended to work as a standalone therapeutic
without regard to a patient’s genetic mutation or ambulatory
status. Our approach has the potential to complement approaches
designed to restore dystrophin production.
About Satellos Bioscience Inc. Satellos is a publicly
traded biotechnology company dedicated to developing life-improving
medicines to treat degenerative muscle diseases. Satellos has
incorporated breakthrough research in muscle stem cell polarity
into a proprietary discovery platform, called MyoReGenX™, to
identify degenerative muscle diseases where deficits in this
process affect muscle regeneration and are amenable to therapeutic
intervention. With this platform, Satellos is building a pipeline
of novel therapeutics to correct muscle stem cell polarity and
promote the body’s innate muscle repair and regeneration process.
The Company’s lead program is an oral, small molecule drug
candidate in development as a potential disease-modifying treatment
for Duchenne muscular dystrophy. Satellos is headquartered in
Toronto, Ontario. For more information, visit www.satellos.com.
Notice on Forward-Looking Statements This press release
includes forward-looking information or forward-looking statements
within the meaning of applicable securities laws regarding Satellos
and its business, which may include, but are not limited to,
statements regarding the value of our DMD program; the advancement
of our lead drug candidate into clinical trials; the general
benefits of modulating stem cell polarity by administering small
molecule drugs; its/their prospective impact on Duchenne patients,
patients with other degenerative muscle disease or muscle injury or
trauma, and on muscle regeneration generally; the utility of
regenerating muscle by modulating polarity; adoption of Satellos’
approach by the medical community; and Satellos’ technologies and
drug development plans. All statements that are, or information
which is, not historical facts, including without limitation,
statements regarding future estimates, plans, programs, forecasts,
projections, objectives, assumptions, expectations or beliefs of
future performance, occurrences or developments, are
“forward-looking information or statements.” Often but not always,
forward-looking information or statements can be identified by the
use of words such as “shall”, “intends”, “anticipate”, “believe”,
“plan”, “expect”, “intend”, “estimate”, “anticipate”, “potential”,
“prospective” , “assert” or any variations (including negative or
plural variations) of such words and phrases, or state that certain
actions, events or results “may”, “might”, “can”, “could”, “would”
or “will” be taken, occur, lead to, result in, or, be achieved.
Such statements are based on the current expectations and views of
future events of the management of the Company. They are based on
assumptions and subject to risks and uncertainties. Although
management believes that the assumptions underlying these
statements are reasonable, they may prove to be incorrect. The
forward-looking events and circumstances discussed in this release,
may not occur and could differ materially as a result of known and
unknown risk factors and uncertainties affecting the Company,
including, without limitation, risks relating to the pharmaceutical
and bioscience industry (including the risks associated with
preclinical and clinical trials and regulatory approvals), and the
research and development of therapeutics, the results of
preclinical and clinical trials, general market conditions and
equity markets, economic factors and management’s ability to manage
and to operate the business of the Company generally, including
inflation and the costs of operating a biopharma business, and
those risks listed in the “Risk Factors” section of Satellos’
Annual Information Form dated March 26, 2024 (which is located on
Satellos’ profile at www.sedarplus.ca). Although Satellos has
attempted to identify important factors that could cause actual
actions, events or results to differ materially from those
described in forward-looking statements, there may be other factors
that cause actions, events or results to differ from those
anticipated, estimated or intended. Accordingly, readers should not
place undue reliance on any forward-looking statements or
information. No forward- looking statement can be guaranteed.
Except as required by applicable securities laws, forward-looking
statements speak only as of the date on which they are made and
Satellos does not undertake any obligation to publicly update or
revise any forward-looking statement, whether resulting from new
information, future events, or otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20240808320005/en/
Investors: Liz Williams, ir@satellos.com Business Development:
Ryan Mitchell, Ph.D., bd@satellos.com Media: Jessica Yingling,
Ph.D., jessica@litldog.com, +1.858.344.8091
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