Strongbridge Biopharma plc Announces Publication of Long-term Efficacy and Safety Results for KEVEYIS® (dichlorphenamide) fo...
July 13 2021 - 7:30AM
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 post hoc
analyses from a one year open-label study evaluating daily use of
KEVEYIS® (dichlorphenamide) for the treatment of Primary Periodic
Paralysis (PPP) following a nine-week randomized, controlled study
were published in the peer-reviewed journal, Muscle & Nerve.
The results confirmed that long-term treatment with KEVEYIS is safe
and effective for chronic use.
“The published results from the post hoc
analyses of the HYPHOP study extend the positive findings from the
original KEVEYIS® (dichlorphenamide) HYPHOP study by demonstrating
that efficacy was maintained over the entire 61-week study with no
evidence of waning over time,” said Fredric Cohen, M.D., chief
medical officer of Strongbridge Biopharma. “Moreover, there was
evidence of further reduction in median weekly attacks among
continuous KEVEYIS users to nearly none by the end of the study,
and patients who were switched from placebo to open-label KEVEYIS
after week nine had reduced attack rates that were similar in
frequency at the end of the study to those who had been
continuously treated with KEVEYIS over the entire study.
Collectively, these results reinforce the utility of KEVEYIS as an
effective option in treating PPP, a life-long rare, hereditary
skeletal muscle disorder that leads to debilitating attacks of
muscle weakness.”
The open label extension results of HYPHOP add
to previously reported results from the short-term randomized,
double-blind, placebo-controlled phase of the study, with
hyperkalemic and hypokalemic PPP sub-studies, which demonstrated
the effectiveness of KEVEYIS to prevent attacks of muscle
weakness.
Adverse event analyses indicated no new safety
signals during the final 52 weeks of the study versus the first
nine weeks, with the common adverse events of paresthesia and
cognition impairment, reported at lower frequencies during the
extension as compared with the first 9 weeks. Clinicians can advise
patients starting KEVEYIS that most patients who had one of these
events initially reported it within the first month of treatment.
Temporary dose reduction appeared to be a reasonable approach to
manage bothersome paresthesia or cognition-related AEs, as patients
with these AEs who were managed with dosage reduction often had
subsequent symptom resolution.
The manuscript, entitled “Long-term efficacy and
safety of dichlorphenamide for treatment of primary periodic
paralysis,” can be accessed here.
About KEVEYISKEVEYIS® (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.
About Strongbridge
BiopharmaStrongbridge 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 rare endocrine
franchise includes RECORLEV® (levoketoconazole), an adrenal
steroidogenesis inhibitor with a New Drug Application that is
currently under review by the FDA for the treatment of endogenous
Cushing’s syndrome, and veldoreotide extended release, a
pre-clinical next-generation somatostatin analog being investigated
for the treatment of acromegaly and potential additional
applications in other conditions amenable to somatostatin receptor
activation. Both RECORLEV and veldoreotide have received orphan
drug designation from the FDA and the European Medicines Agency.
The company’s rare neuromuscular franchise includes
KEVEYIS® (dichlorphenamide), the first and only FDA-approved
treatment for hyperkalemic, hypokalemic, and related variants of
primary periodic paralysis. KEVEYIS has orphan drug exclusivity in
the United States.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of the
federal securities laws. 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. All statements, other than statements of
historical facts, contained in this press release, are
forward-looking statements, including statements
related to Strongbridge’s strategy, plans, results of
clinical trials, outcomes of product development efforts and
objectives of management for future operations. Forward-looking
statements involve risks and uncertainties that could cause actual
results to differ materially from those expressed in such
statement, including risks and uncertainties associated with
clinical development and the regulatory approval process, the
reproducibility of any reported results showing the benefits of
RECORLEV, the adoption of RECORLEV by physicians, if approved, as
treatment for any disease and the emergence of unexpected adverse
events following regulatory approval and use of the product by
patients. Additional risks and uncertainties relating to
Strongbridge and its business can be found under the heading “Risk
Factors” in Strongbridge’s Annual Report on Form 10-K for the year
ended December 31, 2020 and its subsequent Quarterly
Reports on Form 10-Q, as well as its other filings with
the SEC. 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 RelationsElixir Health
Public RelationsLindsay Rocco+1
862-596-1304lrocco@elixirhealthpr.com
Investor RelationsSolebury TroutMike Biega+1
617-221-9660mbiega@soleburytrout.com
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