SOUTH PLAINFIELD, N.J.,
June 16, 2018 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today
announced the presentation of updated interim clinical data from
Part 1 of the FIREFISH study investigating risdiplam (RG7916) in
babies with Type 1 Spinal Muscular Atrophy (SMA), at the 22nd
Annual SMA Researcher Meeting. The data presented by Dr. Baranello
demonstrated that at Day 182, over 90% of the babies achieved a
greater than 4-point increase in CHOP-INTEND score compared to
baseline. The CHOP-INTEND data were further supported by video
footage presented by Dr Baranello demonstrating antigravity
movements, the ability to control their head, roll, or sit in
babies participating in FIREFISH. Part 2 of the pivotal
FIREFISH study is ongoing. The SMA program is a collaboration
between PTC, Roche, and the SMA Foundation.
"We are delighted that up to 6.5-fold increase of protein
production has translated into clinical impact for these babies in
the FIREFISH study," said Stuart W.
Peltz, Ph.D. Chief Executive Officer of PTC Therapeutics.
"The survival data and CHOP-INTEND scores are very promising, since
babies with Type 1 SMA typically do not experience functional motor
milestone improvement based on natural history. We look forward to
sharing updates for the programs as the data further develop at
upcoming medical meetings."
The Children's Hospital of Philadelphia Infant Test of
Neuromuscular Disorders (CHOP-INTEND) is a test designed to measure
motor milestone development of patients with SMA Type 1. In the
FIREFISH study the median increases in CHOP-INTEND scores were 5.5
points (n=20) at Day 56, 12.5 points (n=16) at Day 119, and 14
points (n=11) at Day 182 of treatment. The proportion of patients
achieving greater than a four-point increase from baseline in
CHOP-INTEND scores was 75% at Day 56 (n=20), 94% at Day 119 (n=16),
and 91% (n=11) at Day 182. In addition, risdiplam has been well
tolerated at all dose levels and to date there have been no
drug-related safety findings leading to withdrawal. Also, no babies
have required a tracheostomy or permanent ventilation since study
initiation and no baby has lost the ability to swallow. Median age
of patients at first dose in the FIREFISH trial is 6.7 months;
oldest patient in the trial is currently 23.8 months old.
"I am impressed by the clinical data and the changes reported by
the patients' families," stated Dr. Giovanni Baranello, Fondazione Istituto
Neurologico Carlo Besta in Milan,
Italy. "Data on motor function seem more encouraging when we
consider that we are seeing motor function improvements and
milestones achievement at this early stage of the study, which was
essentially a dose-finding study and most of the infants included
have received their first dose after the age of 5 months. It is
exciting to see evidence of clinical benefit from a systemic oral
treatment for SMA."
Risdiplam is an investigational splicing modifier targeting the
survival motor neuron 2 (SMN2) RNA, restoring a functional
transcript. Risdiplam is taken orally, crosses the blood brain
barrier, and shows systemic distribution to the organs that are
affected by low levels of SMN protein.
Other presentations included an analysis of data from Part 1 of
the SUNFISH study which demonstrated that risdiplam administration
resulted in a dose-dependent increase in SMN protein levels up to
3.5-fold; pharmacodynamic data from the JEWELFISH trial; and
preclinical data demonstrating SMN protein production and
distribution, as well as the development of the SMA Independence
Scale (SMAIS).
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 the SMA Clinical Trials
FIREFISH: An
open-label, two-part clinical trial. Part 1 was a dose escalation
study in 21 infants. The primary objective of Part 1 was to assess
the safety profile of risdiplam in infants and determine the dose
for Part 2. Part 2 is a single-arm study with the dose selected in
Part 1 in approximately 40 infants with Type 1 SMA for 24 months,
followed by an open-label extension. This study is recruiting
globally.
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
risdiplam dose levels. The pivotal SUNFISH Part 2, in non‐ambulant
patients with Type 2 or 3 SMA, is evaluating safety and efficacy of
the risdiplam dose level selected from Part 1 for 24 months,
followed by an open label extension. This study is recruiting
globally.
JEWELFISH: An ongoing, exploratory, open-label study
to establish the safety and tolerability of risdiplam in people who
have previously participated in a study with another therapy
targeting SMN2 splicing.
About the SMA collaboration
The SMA program was
initially developed by PTC Therapeutics in partnership with the SMA
Foundation in 2006. In November 2011,
Roche gained an exclusive worldwide license to the PTC/SMA
Foundation SMN2 alternative splicing program. The development of
risdiplam RG7916 is being executed globally by Roche, including in
the US through Genentech, a member of the Roche group. The SMA
program is overseen by a Joint Steering Committee with members from
PTC, Roche, and the SMA Foundation.
About PTC Therapeutics
PTC is a science-led, global
biopharmaceutical company focused on the discovery, development and
commercialization of clinically-differentiated medicines that
provide benefits to patients with rare disorders. Founded 20 years
ago, PTC Therapeutics has successfully launched two rare
disorder products and has a global commercial footprint. This
success 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.
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" sections of PTC's most recent Quarterly Report on
Form 10-Q and 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.