SOUTH PLAINFIELD, N.J.,
Oct. 20, 2016 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that
its joint development program in Spinal Muscular Atrophy (SMA) with
Roche (SIX: RO, ROG; OTCQX: RHHBY) and the SMA Foundation (SMAF)
initiated a Phase 2 study in pediatric and adult Type 2/3 SMA
patients. The study, named SUNFISH, is a two-part study
investigating the safety, tolerability and efficacy of RG7916, an
oral small molecule survival motor neuron 2 (SMN2) splicing
modifier. The first part of the study will evaluate safety and
tolerability through escalating doses of RG7916. After dose
selection, the study will transition into the pivotal second part
evaluating the efficacy of RG7916. Initiation of the pivotal second
part of the study is expected to begin in 2017 and will trigger a
$20 million milestone payment to PTC
from Roche. A similarly designed two-part study to evaluate RG7916
in Type I SMA patients is expected to begin in the coming months.
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.
"We are excited to initiate clinical studies in SMA patients
with RG7916 and advance our Spinal Muscular Atrophy program
forward," said Stuart W. Peltz,
Ph.D., Chief Executive Officer of PTC Therapeutics. "An oral small
molecule splicing modifier has the potential benefit of systemic
exposure to promote SMN protein in all affected organs and tissues.
This attribute, along with an ease of administration, provides
promise for this program to have a meaningful benefit for SMA
patients."
SUNFISH is a two-part clinical study. Part one is a
double-blinded, placebo-controlled, randomized, exploratory
dose-finding study in approximately 36 Type 2 and Type 3 pediatric
and adult SMA patients for a minimum of 12 weeks. 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-blinded, placebo-controlled, randomized,
confirmatory study in approximately 150 Type 2 and Type 3 SMA
patients for up to 24 months, followed by an open-label extension.
The primary objective of the pivotal second part of the study is to
evaluate the efficacy of RG7916 compared to placebo.
A trial in Type I SMA patients, named FIREFISH, is planned to
initiate in the coming months. FIREFISH is also planned to be a
two-part study. The first part is an open-label, dose-escalation
study in at least eight infants for a minimum of four weeks. The
primary objective of part one of the study is to assess the safety
profile of RG7916 in infants and determine the dose for part two.
The second part of FIREFISH is expected to be an open-label,
single-arm study in approximately 40 infants with Type I SMA for 24
months, followed by an open-label extension. The primary objective
of the second part of the study will be to assess the efficacy of
RG7916 at the selected dose after 12 months of treatment.
RG7916 directly targets the underlying molecular deficiency of
SMA by modulating SMN2 splicing to increase expression of
stable full-length SMN2 mRNA from the SMN2 gene. A healthy
volunteer study was recently completed and the preliminary results
demonstrate that RG7916 increased the production of full-length
SMN2 mRNA further demonstrating proof of mechanism for oral small
molecule SMN2 splicing modifiers. RG7916 was also well
tolerated.
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 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 and
oncology. 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:
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
initiation, evaluation, enrollment and completion 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
whether oral small molecule splicing modifier may promote SMN
protein in all affected organs and tissues and whether this
attribute, along with an ease of administration, may have a
meaningful benefit for SMA patients; 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.