SOUTH PLAINFIELD, N.J.,
Nov. 11, 2019 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today
announced positive results from part 2 of SUNFISH demonstrating
that the study met its primary endpoint of change from baseline
after 1 year of treatment with risdiplam compared to placebo as
measured by the Motor Function Measure 32, a scale monitoring
severity and progression of fine and gross motor function in
patients with a neuromuscular disease such as spinal muscular
atrophy (SMA). Risdiplam has been well tolerated and no
treatment-related safety findings leading to withdrawal have been
seen in any risdiplam trial to date. Data from part 2 of the
SUNFISH study will be presented at an upcoming medical
congress.
"We are very pleased with the results of the SUNFISH study and
are excited to move one step closer to bringing risdiplam globally
to all patients living with SMA. The SUNFISH trial shows that
risdiplam continues to demonstrate its disease-modifying properties
and compelling safety profile," said Stuart
W. Peltz, Ph.D., Chief Executive Officer of PTC
Therapeutics. "The results from the study will be shared first with
regulators globally and then will be presented at a SMA conference
early next year. We believe that risdiplam has the potential to
enter the market with a best-in-class profile for patients with all
SMA types. We are particularly grateful to the SMA community, the
patients and investigators who participated in the trials."
Risdiplam (RG7916), is an investigational, oral, first-in-class,
mRNA splicing modifier for the treatment of SMA. SUNFISH is a
double‐blind, two‐part, placebo‐controlled trial. Part 1 enrolled
patients with type 2 or 3 SMA to evaluate the safety, tolerability,
and PK/PD of several risdiplam dose levels. The pivotal SUNFISH
part 2, in non‐ambulant patients with Type 2 or 3 SMA, evaluated
safety and efficacy of the risdiplam dose level selected from part
1 for 24 months, followed by an open label extension. Patients from
2-25 years of age were enrolled in the study, representing the
broad real-world spectrum of patients living with SMA. The SMA
program is a collaboration between PTC, the SMA Foundation, and
Roche.
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 truncated 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 its
most severe form. It is estimated that this devastating disease
affects 1 in every 11,000 children born.
About risdiplam
Risdiplam is an investigational
medicine being studied in a broad range of patients with SMA from
birth to 60 years of age. It is designed to provide sustained
increase in SMN protein centrally and peripherally through daily
dosing and is being evaluated for its potential ability to help the
SMN2 gene produce more functional SMN protein throughout the body.
Risdiplam is also being studied in a clinical trial for patients
with type 1 SMA, called FIREFISH, in pre-symptomatic babies,
RAINBOWFISH and in patients who have been in previous clinical
trials for SMA, JEWELFISH.
About PTC Therapeutics, Inc.
PTC is a
science-driven, global biopharmaceutical company focused on the
discovery, development and commercialization of
clinically-differentiated medicines that provide benefits to
patients with rare disorders. PTC's ability to globally
commercialize products 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. To learn more about PTC, please visit us
on www.ptcbio.com and follow us on Facebook, on
Twitter at @PTCBio, and on LinkedIn.
For More Information:
Investors:
Alex
Kane
+1 (908) 912- 9643
akane@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 contained in this release, other than statements of
historic fact, 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 clinical studies of
risdiplam in SMA patients and the period during which the results
of the studies will become available; the clinical utility and
potential advantages of risdiplam, including its potential to
impact every aspect of the disease; the timing and outcome of the
regulatory strategy and process for risdiplam, including any
potential regulatory submissions; 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 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 risdiplam, 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" sections 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.
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.