SOUTH PLAINFIELD, N.J.,
April 4, 2018 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) and the CHDI
Foundation, Inc. (CHDI) today announced a research collaboration to
advance PTC's Huntington's disease program. The program is
optimizing small-molecule compounds—identified using PTC's splicing
technology platform—that decrease the production of huntingtin
protein. The current compounds have been shown to be orally
bioavailable in animals, blood-brain barrier penetrant, and
effective in decreasing the amount of huntingtin protein in a mouse
model that has the expanded human huntingtin transgene.
"We are excited to collaborate with CHDI to advance our
small-molecule huntingtin-lowering program for the treatment of
Huntington's disease," said Stuart W.
Peltz, Ph.D., president and chief executive officer of PTC
Therapeutics. "Finding a potential treatment for Huntington's
disease fits with our mission to focus on treatments for disorders
that have an urgent need for therapeutic options. We are proud that
this program utilizes our proprietary splicing platform developed
utilizing cutting edge technology to advance the identification of
new therapeutics for patients suffering from rare diseases."
"After following PTC's Huntington's disease small-molecule
program for several years, we are pleased to start working with the
company more directly," said Robi
Blumenstein, president of CHDI Management. "It's great that
PTC's pre-mRNA splicing expertise has been applied to Huntington's
disease with promising results. A pill that lowers the amount of
huntingtin protein and treats the underlying cause of the disease
holds the promise of improving the quality of life of people with
Huntington's disease and their families. We look forward to
contributing to and accelerating this program."
The discovery and development of small-molecule
huntingtin-expression inhibitors has been enabled by the PTC
pre-mRNA splicing platform, a technology that previously has been
successfully used to identify the SMN2 splicing modifier, RG7916.
PTC, F. Hoffmann-La Roche AG, and the SMA Foundation have a
collaboration using RG7916, which is currently in pivotal clinical
trials for the treatment of SMA. The splicing technology platform
is also being used in a number of other programs, including a
program in late-stage chemical optimization, in collaboration with
the Massachusetts General Hospital, to identify correctors of
IKBKAP splicing to treat familial dysautonomia.
About Huntington's Disease
Huntington's disease (HD)
is a rare inherited neurodegenerative disorder caused by a mutation
in the huntingtin gene. The disorder results in behavioral,
cognitive, and motor impairments. These symptoms progressively
reduce an individual's quality of life, and ultimately lead to
death within 15 to 25 years of overt clinical motoric onset. Each
child of a parent with a mutation in the huntingtin gene has
a 50% chance of inheriting the mutation. It is estimated that
around one person in 10,000 carries the mutated huntingtin gene.
Current HD therapies manage the severity of the symptoms, but there
are currently no therapeutics approved that slow the progression of
the disease.
About CHDI
CHDI Foundation, Inc., is a privately
funded nonprofit biomedical research organization that is
exclusively dedicated to rapidly developing therapies that slow the
progression of Huntington's disease. As a collaborative enabler,
CHDI seeks to bring the right partners together to identify and
address critical scientific issues and move drug candidates to
clinical evaluation as quickly as possible. CHDI scientists work
closely with a network of more than 700 researchers in academic and
industrial laboratories and clinical sites around the world in the
pursuit of these novel therapies, providing strategic scientific
direction to ensure that our common goals remain in focus. More
information about CHDI can be found at www.chdifoundation.org.
About PTC Therapeutics
PTC is a science-led, global
biopharmaceutical company focused on the discovery, development and
commercialization of clinically-differentiated medicines that
provide benefits to patients with rare disorders. Founded 20 years
ago, PTC Therapeutics has successfully launched two rare disorder
products and has a global commercial footprint. This success is the
foundation that drives investment in a robust pipeline of
transformative medicines and our mission to provide access to
best-in-class treatments for patients who have an unmet medical
need.
For More Information:
Investors:
Emily
Hill
+ 1 (908) 912-9327
ehill@ptcbio.com
Media:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com
Forward Looking Statements:
This press release
contains forward-looking statements within the meaning of The
Private Securities Litigation Reform Act of 1995. All statements,
other than those of historical fact, contained in this release, are
forward-looking statements, including statements regarding the
future expectations, plans and prospects for PTC; timing, results
and conduct of PTC's collaboration with CHDI; PTC's alternative
splicing program; timing and results of PTC's collaboration with
Roche and the SMA Foundation; timing and results of PTC's
collaboration with Massachusetts General Hospital; PTC's strategy,
future operations, future financial position, future revenues or
projected costs; and objectives of management.
Other forward-looking statements may be identified by the words
"plan," "guidance," "anticipate," "believe," "estimate," "expect,"
"intend," "may," "predict," "project," "target," "potential,"
"will," "would," "could," "should," "continue," and similar
expressions. PTC's actual results, performance or achievements
could differ materially from those expressed or implied by
forward-looking statements it makes as a result of a variety of
risks and uncertainties, including the factors discussed in the
"Risk Factors" section of PTC's most recent Annual Report on Form
10-K as well as any updates to these risk factors filed from time
to time in PTC's other filings with the SEC. You are urged to
carefully consider all such factors. The forward-looking statements
contained herein represent PTC's views only as of the date of this
press release and PTC does not undertake or plan to update or
revise any such forward-looking statements to reflect actual
results or changes in plans, prospects, assumptions, estimates or
projections, or other circumstances occurring after the date of
this release except as required by law.
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SOURCE PTC Therapeutics, Inc.