SOUTH PLAINFIELD, N.J.,
Oct. 12, 2017 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today
announced that its joint development program in Spinal Muscular
Atrophy (SMA) with Roche and the SMA Foundation (SMAF) transitioned
into the pivotal part of the study evaluating the efficacy and
safety of RG7916 in pediatric and adult Type 2/3 SMA patients.
RG7916 is an oral survival motor neuron 2 (SMN2) splicing modifier.
The study, named SUNFISH, consists of two parts, an exploratory
dose finding part (Part 1) for 12-weeks which has been concluded -
and a confirmatory part (Part 2) for 24-months. The initiation of
Part 2 triggers a $20 million
milestone payment to PTC from Roche. SMA is a rare genetic disorder
that results in neuromuscular disability and is the leading genetic
cause of mortality in infants and young children.
"We are excited to move RG7916 into the pivotal part of the
SUNFISH trial," said Stuart W.
Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics.
"RG7916 resulted in a substantial increase in SMN2 protein
production in SMA patients. We believe that a major advantage of
RG7916 is that it is an oral drug that distributes throughout the
body. This is important because the SMN protein is critical both in
the CNS and peripheral tissues."
The confirmatory part of SUNFISH is a randomized,
double-blinded, placebo-controlled study. It will be conducted in
approximately 168 Type 2 and Type 3 SMA patients for up to 24
months, followed by an open-label extension. The primary objective
of the study is to evaluate the efficacy of RG7916 compared to
placebo after 12 months of treatment.
The U.S. Food and Drug Administration granted orphan drug
designation to RG7916 for the treatment of patients with SMA
earlier this year. RG7916 directly targets the underlying molecular
deficiency of SMA by modulating SMN2 splicing to increase
expression of full-length SMN2 mRNA from the SMN2
gene. An interim analysis from the first part of SUNFISH of the
five cohorts treated with RG7916 for 28 days or longer demonstrated
an exposure-dependent increase in SMN protein. SMA is characterized
by reduced levels of SMN protein, motor neuron loss, and muscle
atrophy. To date, RG7916 remains well-tolerated in patients at all
doses and there have been no drug-related safety findings leading
to withdrawal.
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, Inc.
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's 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; 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.