SOUTH PLAINFIELD, N.J.,
Jan. 23, 2018 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that
updates and preliminary data from ongoing clinical trials of
RG7916, an oral survival motor neuron 2 (SMN2) splicing modifier in
SMA patients will be presented at the International Scientific
Congress on Spinal Muscular Atrophy at Jagiellonian University,
Kraków, Poland from January 25-27th. The first interim
analysis of survival and real-life experiences from the FIREFISH
clinical trial in infants with Type 1 SMA will be presented, as
well as poster presentations on SMN protein production and the
safety and pharmacodynamic effects from the clinical trials in Type
2/3 SMA patients. The three ongoing clinical trials, FIREFISH,
SUNFISH, and JEWELFISH, are being conducted in Type 1 and Type 2/3
SMA patients respectively. SMA is a rare genetic disorder that
results in neuromuscular disability beginning in infancy and is the
leading inherited cause of mortality in infants and young children.
The SMA program is a joint collaboration with Roche and the SMA
Foundation.
Presentation Details:
- "FIREFISH, a multi-center, open-label trial to investigate the
safety and efficacy of RG7916 in babies with Type 1 SMA: Study
update and real-life experience of study implementation" on
Saturday, Jan. 27 at 12:00 p.m. CET
Poster Presentation Details:
- "Updated pharmacodynamic and safety data from SUNFISH Part 1, a
study evaluating the oral SMN2 splicing modifier RG7916 in patients
with Type 2 or 3 spinal muscular atrophy" on Thursday, Jan. 25 at 5:30
p.m. CET
- "Relationship Between Central and Peripheral SMN Protein
Increase Upon Treatment with RO7034067 (RG7916)" on Thursday, Jan. 25 at 5:30
p.m. CET
- "Preliminary evidence for pharmacodynamics effects of RG7916 in
JEWELFISH, a study in patients with spinal muscular atrophy who
previously participated in a study with another SMN2-splicing
targeting therapy" on Friday, Jan. 26
at 3:30 p.m. CET
For more information on the conference, visit
https://krakow2018.sma-europe.eu
About the SMA Clinical Trials
FIREFISH: An
open-label, two-part clinical trial. Part 1 is a dose escalation
study in at least 8 infants for a minimum of 4 weeks. The primary
objective of Part 1 is to assess the safety profile of RG7916 in
infants and determine the dose for Part 2. Part 2 is a single-arm
study in approximately 40 infants with Type 1 SMA for 24 months,
followed by an open-label extension.
SUNFISH: A double‐blind, two‐part, placebo‐controlled
trial. Part 1 enrolled patients with Type 2 or 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 or 3
SMA, will evaluate safety and efficacy of the RG7916 dose level
selected from Part 1.
JEWELFISH: An exploratory, open-label study to establish
the safety and tolerability of RG7916 in people who have previously
participated in a study with another therapy targeting SMN2
splicing.
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 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.