BRIGHTON, United Kingdom,
Oct. 4, 2015 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT), today announced that clinical
data from the company's joint development program with Roche and
the SMA Foundation in spinal muscular atrophy (SMA) were presented
at the 20th International Congress of the World Muscle
Society (WMS) in Brighton, U.K.
Results from the first cohort of patients enrolled in the Phase 2
'MOONFISH' trial evaluating oral RG7800, a small molecule modifier
of Survival Motor Neuron 2 (SMN2) splicing, were highlighted in a
late breaking oral session. The presentation is titled "SMN2
splicing modifier RG7800 increases SMN protein in first study in
SMA patients."
"We have now demonstrated, in two independent studies, that
treatment with RG7800 shifts SMN2 splicing toward the production of
full length SMN mRNA. Most importantly, in the MOONFISH trial
we also observed relevant increases in SMN protein levels in whole
blood in patients with SMA," said Stuart W.
Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics,
Inc. "This is important because a two-fold increase of SMN protein
levels has the potential to provide meaningful clinical benefit to
SMA patients. SMA is a devastating disease with no marketed
therapies currently available, and we remain focused on developing
an oral therapy for the treatment of this disorder."
MOONFISH is a Phase 2 randomized, double-blind,
placebo-controlled study investigating the safety, tolerability,
pharmacokinetics and pharmacodynamics of RG7800 with a target
enrollment of approximately 64 adult and pediatric patients with
SMA. Results from the first cohort that included 13 adult and
adolescent SMA patients demonstrated that SMN protein can be
increased with RG7800, providing proof of mechanism for oral small
molecule SMN2 splicing modifiers. Up to three-fold increases
in the ratio of full length SMN2 mRNA to SMN2Δ7 mRNA and up to
two-fold increases in SMN protein were observed versus baseline, as
measured in whole blood. RG7800 was well tolerated over 12 weeks at
a dose of 10 mg once daily.
RG7800 is an orally available small molecule being investigated
for its ability to selectively modify the alternative splicing of
the SMN2 gene, which is present both in healthy individuals and SMA
patients, towards the production of full length mRNA. Preclinical
studies in animal models of SMA demonstrated an increase in
functional full length SMN protein levels with significant efficacy
benefits on survival and motor function. In a Phase 1
clinical study in healthy volunteers, a dose-dependent effect on
SMN2 alternative splicing was observed. Dosing in the Phase 2
MOONFISH trial was suspended in April
2015 as a precautionary measure, while a non-clinical safety
finding observed in a longer-term animal study is investigated.
The SMA program was initially developed by PTC Therapeutics in
partnership with the SMA Foundation. The SMA Foundation was
established in 2003 to accelerate the development of a treatment
for SMA. In November 2011, Roche
gained an exclusive worldwide license to the PTC / SMA Foundation
SMN2 alternative splicing program. The development of RG7800 is
being executed by Roche and overseen by a joint steering committee
with members from PTC, Roche, and the SMA Foundation.
About Spinal Muscular Atrophy (SMA)
Spinal Muscular
Atrophy (SMA) is a genetic neuromuscular disorder that is the
leading genetic cause of mortality in infants and toddlers caused
by a missing or defective survival of motor neuron 1 (SMN1) gene,
which results in reduced levels of SMN protein. The homologous SMN2
gene is predominantly spliced to a shortened mRNA, and only
produces small amounts of functional SMN protein. Insufficient
levels of SMN protein are responsible for the loss of motor neurons
within the spinal cord leading to muscle atrophy and death in
infants and toddlers in its most severe form. It is estimated that
this devastating disease affects 1 in every 11,000 children born.
There are no marketed therapies for SMA.
About PTC Therapeutics, Inc.
PTC is a global
biopharmaceutical company focused on the discovery, development and
commercialization of orally administered, proprietary small
molecule drugs targeting an area of RNA biology we refer to as
post-transcriptional control. Post-transcriptional control
processes are the regulatory events that occur in cells during and
after a messenger RNA, or mRNA, molecule is copied from DNA through
the transcription process. PTC's internally discovered pipeline
addresses multiple therapeutic areas, including rare disorders,
oncology and infectious diseases. PTC has discovered all of its
compounds currently under development using its proprietary
technologies. PTC plans to continue to develop these compounds both
on its own and through selective collaboration arrangements with
leading pharmaceutical and biotechnology companies. For more
information on the company, please visit our website
www.ptcbio.com
For More Information:
Investors:
Emily
Hill
+1(908) 912-9327
ehill@ptcbio.com
Media:
Justine
O'Malley
+1 (908) 912-9551
jomalley@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; our expectations
with respect to the development and regulatory status of our
product candidate and joint development program with Roche and the
SMA Foundation directed against SMA; the timing and conduct of
clinical trials and studies under PTC's SMA collaboration with
Roche and the SMA Foundation, including the Phase 2 MOONFISH study;
our 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 those related to the initiation, conduct
and availability of data from clinical trials and studies;
expectations for regulatory approvals; PTC's scientific approach
and general development progress; and the factors discussed in the
"Risk Factors" section of PTC's most recent Quarterly Report on
Form 10-Q 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.
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/phase-2-data-from-spinal-muscular-atrophy-program-to-be-presented-at-the-20th-international-annual-congress-of-the-world-muscle-society-300153783.html
SOURCE PTC Therapeutics, Inc.