SOUTH PLAINFIELD, N.J.,
Oct. 3, 2017 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today announced data from Part 1
of the SUNFISH trial of RG7916 in Type 2/3 spinal muscular atrophy
(SMA) patients. RG7916 is part of PTC's joint development
program in SMA with Roche and the SMA Foundation (SMAF). The data
were presented at the 22nd International World Muscle
(WMS) Congress in St. Malo, France. An interim analysis 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. It is the leading genetic cause of mortality in infants
and young children. To date, RG7916 remains well-tolerated in
patients at all doses and there have been no drug-related safety
findings leading to withdrawal.
"We are excited to report that in SMA patients who are deficient
in SMN protein, RG7916 treatment led up to a median 2.5-fold
increase in SMN protein," said Stuart W.
Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics.
"SMN protein is a critical factor that is needed both in the
central nervous system and in a number of other tissues and organs
throughout the body. Increasing SMN protein levels throughout the
body has the potential to impact every aspect of this disorder.
These results have allowed us to determine the dose for the pivotal
phase of the SUNFISH trial to evaluate the efficacy and safety of
RG7916 versus placebo."
SUNFISH is a double-blind, two-part, placebo-controlled trial.
Part 1 of SUNFISH enrolled patients with Type 2/3 SMA to evaluate
safety, tolerability, and PK/PD of several RG7916 dose levels. The
pivotal SUNFISH Part 2, in non-ambulant patients with Type 2/3 SMA,
will evaluate safety and efficacy of the RG7916 dose level selected
from Part 1.
The U.S. Food and Drug Administration granted orphan drug
designation and fast track designation to RG7916 for the treatment
of patients with SMA earlier this year. RG7916, an orally
administered small molecule, directly targets the underlying cause
of SMA by modulating SMN2 splicing to increase expression of
full-length SMN2 mRNA and increases SMN protein levels.
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; 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.