SOUTH PLAINFIELD, N.J.,
July 1, 2017 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today announced the presentation
of preliminary clinical data from the company's joint
development program with Roche and the SMA Foundation in spinal
muscular atrophy (SMA) at the Cure SMA Meeting in Orlando, FL. Preliminary results from an early
analysis of Part 1 of the Phase 2 SUNFISH trial evaluating oral
RG7916, a small molecule modifier of Survival Mo tor Neuron 2
(SMN2) splicing, were highlighted in an oral presentation titled
"Clinical Studies of RG7916 in Patients with Spinal Muscular
Atrophy: Study Update." SMA is a rare genetic disorder that results
in neuromuscular disability beginning in infancy and is the leading
genetic cause of mortality in infants and young children.
"The increase in SMN2 full length transcript in SMA patients is
promising and confirms that RG7916 targets the underlying cause of
the disease," said Stuart W. Peltz,
Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "The low
levels of SMN protein in SMA patients affect multiple tissues
throughout the body, including muscles, bones and nerves. We
believe that the ease of administration of an oral therapy and its
broad tissue distribution give RG7916 the potential to address the
complete spectrum of functional deficits observed in SMA patients.
We look forward to advancing RG7916 into pivotal clinical trials in
the second half of the year."
Preliminary results from an early analysis from the ongoing Part
1 of the RG7916 SUNFISH study in Type 2/3 SMA patients demonstrated
a dose-dependent increase in SMN2 full length/Δ7 mRNA ratio of ~
400% versus baseline, as measured in whole blood. These results
provided proof of mechanism for oral small molecule SMN2 splicing
modifiers. No drug-related adverse events leading to
withdrawal have been observed to date for RG7916.
SUNFISH is a two-part phase 2 clinical study. Part 1 is a
double-blind, placebo-controlled, randomized, exploratory
dose-finding study in Type 2/3 pediatric and adult SMA patients.
The primary objective of the first part of the study is to evaluate
the safety, pharmacokinetics, and pharmacodynamics of RG7916 in
patients, and to select the dose for the second part of the study.
The pivotal second part is a double-blind, placebo-controlled,
randomized, confirmatory study in Type 2/3 SMA patients followed by
an open-label extension. The primary objective of the pivotal
second part of the study is to evaluate the safety and efficacy of
RG7916 compared to placebo. SUNFISH is one of three ongoing
clinical trials of RG7916, along with FIREFISH and JEWELFISH.
The SMA program was initially developed by PTC Therapeutics in
partnership with the SMA Foundation in 2006 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 these compounds is being
executed by Roche and overseen by a joint steering committee with
members from PTC, Roche, and the SMA Foundation.
About PTC Therapeutics
PTC is a global biopharmaceutical company focused on the
discovery, development, and commercialization of novel medicines
using our expertise in RNA biology. PTC's internally discovered
pipeline addresses multiple therapeutic areas, including rare
disorders and oncology. PTC has discovered all of its compounds
currently under development using its proprietary technologies.
Since its founding nearly 20 years ago, PTC' mission has focused on
developing treatments to fundamentally change the lives of patients
living with rare genetic disorders. The company was founded in 1998
and is headquartered in South Plainfield, New Jersey. 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:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com
Forward Looking Statements:
All statements, other
than those of historical fact, contained in this press release, are
forward-looking statements, including statements regarding: any
advancement of the joint development program in SMA with PTC,
Roche, and SMAF, in particular as related to the timing of
enrollment, completion and evaluation of the Phase 2 clinical
studies of RG7916 in SMA patients and the period during which the
results of the studies will become available; whether and when a
milestone payment to PTC from Roche may be triggered; the clinical
utility and potential advantages of RG7916, including its potential
to impact every aspect of the disease; the timing and outcome of
PTC's regulatory strategy and process; PTC's strategy, future
expectations, plans and prospects, future operations, future
financial position, future revenues or projected costs; and the
objectives of management. Other forward-looking statements may be
identified by the words "potential," "will," "promise," "expect,"
"plan," "target," "anticipate," "believe," "estimate," "intend,"
"may," "project," "possible," "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,
enrollment, conduct and availability of data from either the
SUNFISH or FIREFISH studies and the outcome of such studies; events
during, or as a result of, these studies that could delay or
prevent further development of RG7916, including future actions or
activities under the SMA joint development program; our
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. As with any pharmaceutical
under development, there are significant risks in the development,
regulatory approval and commercialization of new products,
including with respect to PTC's joint development program in SMA
with Roche and the SMAF. There are no guarantees that any product
candidate under the joint development program will receive
regulatory approval in any territory or prove to be commercially
successful.
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 press release except
as required by law.
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SOURCE PTC Therapeutics, Inc.