~ Post-Hoc Analysis of Phase 3 Placebo-Controlled
Study of KEVEYIS Showed Adolescents had Similar Improvements in
Short-Term Attack Rates as Adults ~
Strongbridge Biopharma plc, (Nasdaq:SBBP), a global
commercial-stage biopharmaceutical company focused on the
development and commercialization of therapies for rare diseases
with significant unmet needs, today announced that new clinical
analyses for KEVEYIS® (dichlorphenamide), the first and only U.S.
Food and Drug Administration (FDA) approved treatment for
hyperkalemic, hypokalemic, and related variants of primary periodic
paralysis, will be presented at the 70th American Academy of
Neurology (AAN) Annual Meeting from April 21-27 in Los Angeles,
California.
“These new analyses help to validate the overall
clinical profile of KEVEYIS in a variety of patient populations and
disease variants,” said Fredric Cohen, M.D., chief medical officer
of Strongbridge Biopharma. “Primary periodic paralysis is a rare
genetic neuromuscular condition with significant unmet need and we
are encouraged by the consistency of positive data for KEVEYIS and
the potential value it can bring to the underserved periodic
paralysis community.”
In a poster presentation entitled, “Efficacy and
Safety of Dichlorphenamide in Adolescent Patients with Primary
Periodic Paralysis,” Emma Ciafaloni, M.D., lead investigator and
professor of neurology and pediatrics at the University of
Rochester Medical Center, will present new findings from a post-hoc
analysis of a Phase 3, placebo-controlled, cross-over trial
comparing the effects of dichlorphenamide in adolescent and adult
patients with primary periodic paralysis. The results show that
adolescents had similar improvements in short-term attack rates and
similar side effects as adults.
In an oral poster presentation entitled,
“Efficacy of Dichlorphenamide in Primary Periodic Paralysis:
Pooled-Data Analysis of Two Phase 3 Clinical Trials,” Perry B.
Shieh, M.D., Ph.D., lead investigator and associate professor of
the neuromuscular program at the University of California at Los
Angeles, David Geffen School of Medicine, will present a pooled
analysis of efficacy data from two Phase 3 placebo-controlled
trials evaluating dichlorphenamide in 138 patients with primary
periodic paralysis. The results add to the growing body of clinical
evidence for KEVEYIS and supports its efficacy across disease
variants by measuring changes from baseline in weekly attack
rates.
Presentation Details:
• Poster #955
– Efficacy and safety of dichlorphenamide in
adolescent patients with primary periodic
paralysis; Emma Ciafaloni; Poster Session P3,
April 24, 2018 – 05:30 p.m.-7:00 p.m. PT
Detailed findings include: - Dichlorphenamide treatment
resulted in median changes from baseline in weekly attack rate of
−0.96 [CI: ‒1.63, ‒0.88] in adolescents vs ‒0.83 [CI: ‒2.75, ‒0.26]
in adults. - Median changes from baseline in
severity-weighted weekly attack rate were ‒2.25 [CI: −3.50, −1.38]
in adolescents vs ‒1.17 [CI: −3.38, ‒0.63] in adults.
- Three of six adolescents with safety data reported ≥1
solicited side effect with dichlorphenamide treatment; no side
effects were dose-limiting or resulted in study withdrawal.
- The frequency of each side effect reported by the
adolescents was similar in adults.
• Oral Poster #958
– Efficacy of Dichlorphenamide in Primary Periodic
Paralysis: Pooled-Data Analysis of Two Phase 3 Clinical
Trials; Perry B. Shieh; Poster Session P2,
April 23, 2018 – Oral Presentation: 12:25 p.m. - 12:30
p.m. PT / Poster Presentation: 05:30 p.m.-7:00 p.m. PT
Detailed findings include: - Median weekly attack rate
improved with dichlorphenamide vs placebo: Median change from
baseline was −0.75 vs +0.13, P=0.0004; treatment-effect size −1.22
[CI: −3.15, −0.38]. Median relative change from baseline was
−51.67% for dichlorphenamide and 0.00% for placebo, P<0.0001;
treatment-effect size −65.31% [CI: −93.17%, −34.40%].
- Median severity-weighted attack rate also improved with
dichlorphenamide: Median change from baseline was −0.67 vs +0.55,
P=0.0049; treatment-effect size −1.63 [CI: −4.47, −0.09].
Median relative change from baseline was −45.18% vs +16.05%,
P=0.0002; treatment-effect size −60.85% [CI: −88.46%, −23.53%].
Primary periodic paralysis is a rare inherited disorder
characterized by recurrent, progressive, and debilitating episodes
of muscle weakness and temporary paralysis. Primary periodic
paralysis is very rare, affecting about 5,000 to 6,000 individuals
in the United States.
About Strongbridge Biopharma
plcStrongbridge Biopharma is a global
commercial-stage biopharmaceutical company focused on the
development and commercialization of therapies for rare diseases
with significant unmet needs. Strongbridge's commercial portfolio
within its rare neuromuscular and rare endocrine franchises
includes KEVEYIS®(dichlorphenamide), the first and only
FDA-approved treatment for hyperkalemic, hypokalemic, and related
variants of Primary Periodic Paralysis, and MACRILEN™
(macimorelin), the first and only FDA-approved oral drug indicated
for the diagnosis of Adult Growth Hormone Deficiency. KEVEYIS has
orphan drug exclusivity status in the U.S. to August 2022. MACRILEN
has been granted orphan drug designation in the U.S. and has
patents with expiration dates through late 2027. The Company’s rare
endocrine franchise also includes a clinical-stage pipeline of
therapies: RECORLEV™ (levoketoconazole), a
cortisol synthesis inhibitor currently being studied for the
treatment of endogenous Cushing's syndrome, and veldoreotide, a
next-generation somatostatin analog being investigated for the
treatment of acromegaly, with potential additional applications in
other conditions amenable to somatostatin receptor activation. Both
RECORLEV and veldoreotide have received orphan designation from the
U.S. Food and Drug Administration and the European Medicines
Agency. For more information,
visit www.strongbridgebio.com.
About KEVEYIS
KEVEYIS® (dichlorphenamide) is indicated for the treatment of
primary hyperkalemic periodic paralysis, primary hypokalemic
periodic paralysis, and related variants. In clinical studies, the
most common side effects of KEVEYIS were a numbness or tingling,
difficulty thinking and paying attention, changes in taste, and
confusion. These are not all of the possible side effects that you
may experience with KEVEYIS. Talk to your doctor if you have any
symptoms that bother you or do not go away. You are encouraged to
report side effects to Strongbridge Biopharma at 1-855-324-8912, or
to the FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch/.
For additional KEVEYIS important safety information and the
full prescribing information visit www.keveyis.com.
Forward-Looking Statements This press release
contains forward-looking statements that involve substantial risks
and uncertainties. All statements, other than statements of
historical facts, contained in this press release, are
forward-looking statements. These statements relate to future
events and involve known and unknown risks, including, without
limitation, uncertainties regarding Strongbridge's strategy, plans,
anticipated investments, costs and results, product development and
commercialization efforts and objectives of management for future
operations. The words "anticipate," "estimate," "expect," "intend,"
"may," "plan," "potential," "project," "target," "will," "would,"
or the negative of these terms or other similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. These
forward-looking statements are based on current expectations,
estimates, forecasts and projections and are not guarantees of
future performance or development and involve known and unknown
risks, uncertainties and other factors. The forward-looking
statements contained in this press release are made as of the date
of this press release, and Strongbridge Biopharma does
not assume any obligation to update any forward-looking statements
except as required by applicable law.
Contacts:
Corporate and Media Relations Elixir Health
Public Relations Lindsay Rocco +1 862-596-1304
lrocco@elixirhealthpr.com
Investor Relations U.S.: The Trout Group Marcy
Nanus +1 646-378-2927 mnanus@troutgroup.com
Europe: First House Mitra Hagen Negård +47 21
04 62 19 strongbridgebio@firsthouse.no
USA 900 Northbrook Drive Suite 200 Trevose,
PA 19053 Tel. +1 610-254-9200 Fax. +1 215-355-7389
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