SOUTH PLAINFIELD, N.J.,
July 28, 2021 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced
that results from Part 2 of the FIREFISH pivotal study were
published in the New England Journal of Medicine. This trial showed
that symptomatic Type 1 spinal muscular atrophy (SMA) infants
treated with Evrysdi™ (risdiplam) showed significant improvements
in survival, motor milestones, and motor function compared to
historical controls.
"It is exciting to see such profound results in infants treated
with Evrysdi," said Stuart W. Peltz,
Ph.D., Chief Executive Officer, PTC Therapeutics. "We are proud
that such a significant treatment for SMA patients was developed
from PTC's splicing platform. We believe that the splicing platform
is a disruptive technology that will continue to drive the
development of new therapies in diseases with no treatment options
available."
The table below depicts the primary and secondary endpoints
achieved across Part 2 of the FIREFISH study after 12 months of
treatment:
Milestone
Achievement
|
Percentage of
Infants
Achieving Milestones After
12 months with Evrysdi
|
Historical
Controls
|
Ability to sit
without support for
at least five seconds
|
29%
(12/41)
|
5%
|
CHOP-INTEND* score of
40 or
more
|
56%
(23/41)
|
17%
|
An increase of at
least 4 points
in the CHOP-INTEND total score
from baseline
|
90%
(37/41)
|
17%
|
HINE-2**
motor-milestone responders
|
78%
(32/41)
|
12%
|
Event free
survival‡
|
85%
(35/41)
|
42%
|
FIREFISH is an open-label, two-part, pivotal clinical trial
designed to evaluate the efficacy, safety, tolerability,
pharmacokinetics and pharmacodynamics in infants with type 1 SMA.
In the dose-finding Part 1 of the study, treatment with Evrysdi led
to an increased expression of SMN protein levels. Part 2 evaluated
the efficacy and safety of Evrysdi in Type 1 SMA.
Roche leads the clinical development of Evrysdi as part of a
collaboration with the SMA Foundation and PTC Therapeutics. Evrysdi
is marketed in the United States
by Genentech, a member of the Roche Group.
* CHOP-INTEND, Children's Hospital of Philadelphia Infant Test
of Neuromuscular Disorders
** HINE-2, Hammersmith Infant Neurological Examination
‡ Event-free survival is defined as alive without need
for permanent ventilation (defined as requiring tracheostomy or
ventilation for ≥16 hours per day continuously for >3 weeks or
continuous intubation >3 weeks, in the absence of, or following
the resolution of, an acute reversible event). Data cut-off:
14 Nov 2019.
About Spinal Muscular Atrophy (SMA)
Spinal muscular
atrophy (SMA) is a severe, progressive neuromuscular disease that
can be fatal. It affects approximately 1 in 10,000 babies and when
untreated is the leading genetic cause of infant mortality. SMA is
caused by a mutation of the survival motor neuron 1 (SMN1)
gene, which leads to a deficiency of SMN protein. This protein is
found throughout the body and is essential to the function of
nerves that control muscles and movement. Without it, nerve cells
cannot function correctly, leading to progressive muscle weakness
over time. Depending on the type of SMA, an individual's physical
strength and their ability to walk, eat or breathe can be
significantly diminished or lost.
About Evrysdi™ (risdiplam)
Evrysdi™ is a survival
motor neuron 2 (SMN2)-directed RNA splicing modifier
designed to treat SMA caused by mutations in chromosome 5q that
lead to SMN protein deficiency. Evrysdi™ is designed to distribute
evenly to all parts of the body, including the central nervous
system (CNS). Evrysdi™ is administered daily at home in liquid form
by mouth or feeding tube. Evrysdi has been approved in over 50
countries including the United
States, the European Union and Japan. Evrysdi™ (risdiplam) is marketed in
the United States by Genentech, a
member of the Roche Group.
About the Evrysdi™ (risdiplam) Clinical Studies
FIREFISH (NCT02913482) is an open-label, two-part
pivotal clinical trial in infants with Type 1 SMA. Part 1 was a
dose-escalation study in 21 infants with the primary objective of
assessing the safety profile of risdiplam in infants and
determining the dose for Part 2. Part 2 is a pivotal, single-arm
study of risdiplam in 41 infants with Type 1 SMA treated for two
years followed by an open-label extension. The primary objective of
Part 2 was to assess efficacy as measured by the proportion of
infants sitting without support after 12 months of treatment, as
assessed in the Gross Motor Scale of the Bayley Scales of Infant
and Toddler Development - Third Edition (BSID-III) (defined as
sitting without support for five seconds). The study met its
primary endpoint.
SUNFISH (NCT02908685) is a two-part, double-blind,
placebo controlled pivotal study in people aged 2 to 25 years with
Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the
confirmatory Part 2. Part 2 (n=180) evaluated motor function using
the Motor Function Measure 32 (MFM-32) scale at 12 months. MFM-32
is a validated scale used to evaluate fine and gross motor function
in people with neurological disorders, including SMA. The study met
its primary endpoint.
RAINBOWFISH (NCT03779334) is an open-label,
single-arm, multi-center study, investigating the efficacy, safety,
pharmacokinetics, and pharmacodynamics of risdiplam in babies
(~n=25), from birth to 6 weeks old (at first dose), with
genetically diagnosed SMA, who are not yet presenting symptoms. The
study is currently recruiting.
JEWELFISH (NCT03032172) is an open-label exploratory
trial designed to assess the safety, tolerability, pharmacokinetics
(PK) and pharmacodynamics (PD) in people with SMA aged 6 months to
60 years who received other investigational or approved SMA
therapies for at least 90 days prior to receiving Evrysdi™. The
study has completed recruitment (n=174).
Clinical Trial Safety Data
The safety profile of
Evrysdi™ was established across FIREFISH and SUNFISH pivotal
trials. The most common adverse reactions in later-onset SMA
(incidence of at least 10 percent of patients treated with Evrysdi™
and more frequently than control) were fever, diarrhea, and rash.
The most common adverse reactions in infantile-onset SMA were
similar to those observed in later-onset SMA patients.
Additionally, the most common adverse reactions (incidence of at
least 10 percent) were upper respiratory tract infection,
pneumonia, constipation, and vomiting.
About PTC
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 and diversified pipeline of transformative
medicines and our mission to provide access to best-in-class
treatments for patients who have an unmet medical need. The
Company's strategy is to leverage its strong scientific expertise
and global commercial infrastructure to maximize value for its
patients and other stakeholders. To learn more about PTC, please
visit us at www.ptcbio.com and follow us on Facebook, on Twitter at
@PTCBio, and on LinkedIn.
For More Information:
Investors:
Kylie
O'Keefe
+1 (908) 300-0691
kokeefe@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:
advancement of PTC's joint collaboration program in SMA, including
the commercialization of any products therein or royalty or
milestone payments; the future expectations, plans and prospects
for PTC, including with respect to the expected timing of clinical
trials and studies, availability of data, regulatory submissions
and responses, licensing or commercialization of its products and
products candidates and other matters; PTC's strategy, future
operations, future financial position, future revenues, projected
costs; and the objectives of management. Other forward-looking
statements may be identified by the words, "guidance", "plan,"
"anticipate," "believe," "estimate," "expect," "intend," "may,"
"target," "potential," "will," "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 outcome of pricing,
coverage and reimbursement negotiations with third party payors for
PTC's products or product candidates that PTC commercializes or may
commercialize in the future; the enrollment, conduct, and results
of ongoing studies under the SMA collaboration and events during,
or as a result of, the studies that could delay or prevent further
development under the program, including any regulatory submissions
and commercialization with respect to Evrysdi; significant business
effects, including the effects of industry, market, economic,
political or regulatory conditions; changes in tax and other laws,
regulations, rates and policies; the eligible patient base and
commercial potential of PTC's products and product candidates;
PTC's scientific approach and general development progress; and the
factors discussed in the "Risk Factors" section 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. There are no guarantees that any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
Evrysdi.
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.