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 secondary
endpoint results from the Phase 3 SONICS study of RECORLEV™
(levoketoconazole) for the potential treatment of endogenous
Cushing’s syndrome were published online in advance of print
publication in the peer-reviewed journal, Pituitary, the
official publication of the Pituitary Society and the Growth
Hormone Research Society.
“People with Cushing’s syndrome often experience
a variety of problematic signs, symptoms, and comorbidities related
to excess cortisol, testosterone or both,” said Fredric Cohen,
M.D., chief medical officer of Strongbridge Biopharma. “The SONICS
study provided important evidence that RECORLEV treatment was
associated with durable improvements in several of the more common
and bothersome signs and symptoms of the syndrome, including those
that most commonly affect women, such as hirsutism and acne, which
are related to increases in testosterone produced by the adrenal
glands.”
The manuscript, entitled
“Levoketoconazole improves clinical signs and symptoms and
patient-reported outcomes in patients with Cushing’s
syndrome,” includes analyses demonstrating that treatment
with RECORLEV led to significant improvements in Cushing’s syndrome
signs and symptoms, patient-reported quality of life (QoL)
outcomes, depression symptoms, and testosterone levels.
-
Significant mean improvements from baseline were noted at end of
the Maintenance phase (Month 6) for acne, hirsutism (females only),
and peripheral edema.
- These
improvements were observed as early as Day 1 of Maintenance for
hirsutism, the end of Month 1 of Maintenance for acne, and Month 4
of Maintenance for peripheral edema.
- By Month
3 of Maintenance, significant mean improvements in patient-reported
QoL outcomes were observed and by Month 6, symptoms of depression
had improved.
- A
reduction in mean free-testosterone in women, consistent with
improvements in clinical signs of hyperandrogenism was observed,
and a modest increase in mean free-testosterone in men was
observed.
- RECORLEV
was generally well-tolerated; the most commonly reported adverse
events during the dose-titration and maintenance phases were nausea
(32%) and headache (28%).
The full manuscript can be accessed online
at: https://www.strongbridgebio.com/therapeutic-focus/key-publications-posters/related-investigational-agent-recorlev/#heading_4
About Cushing’s
SyndromeEndogenous Cushing’s syndrome is a rare, serious
and potentially lethal endocrine disease caused by chronic elevated
cortisol exposure - often the result of a benign tumor of the
pituitary gland. This benign tumor tells the body to overproduce
high levels of cortisol for a sustained period of time, and this
often results in undesirable physical changes. The disease is most
common among adults between the ages of 30 to 50, and it affects
women three times more often than men. Women with Cushing's
syndrome may experience a variety of health issues including
menstrual problems, difficulty becoming pregnant, excess male
hormones (androgens), primarily testosterone which can cause
hirsutism (growth of coarse body hair in a male pattern), oily
skin, and acne. Additionally, the internal manifestations of the
disease are potentially life threatening. These include metabolic
changes such as high blood sugar, or diabetes, high blood pressure,
high cholesterol, fragility of various tissues including blood
vessels, skin, muscle and bone, and psychologic disturbances such
as depression, anxiety and insomnia. Untreated, the five-year
survival rate is only approximately 50 percent.
About the SONICS StudySONICS
is an open-label, Phase 3 study of RECORLEV as a treatment for
endogenous Cushing’s syndrome that enrolled 94 patients at centers
in North America, Europe and the Middle East.
Following a screening phase, SONICS has three treatment phases:
(1) Dose Titration Phase: Patients started
RECORLEV at 150 mg twice daily (300 mg total daily dose) and
titrated in 150 mg increments with the goal of achieving a
therapeutic dose – a dose resulting in mUFC normalization – at
which point titration was stopped; (2) Maintenance Phase: The dose
was fixed and should not have been changed other than for safety
reasons or loss of efficacy. At the end of the six-month
maintenance phase, the mUFC response rate was measured; and (3)
Extended Evaluation Phase: Patients continued on RECORLEV for
another six months to evaluate long-term safety and tolerability
and explore efficacy durability.
About RECORLEVRECORLEV®
(levoketoconazole) is an investigational cortisol synthesis
inhibitor in development for the treatment of patients with
endogenous Cushing’s syndrome, a rare but serious and potentially
lethal endocrine disease caused by chronic elevated cortisol
exposure. RECORLEV is the pure 2S,4R enantiomer of
ketoconazole, a steroidogenesis inhibitor. RECORLEV has
demonstrated in two successful Phase 3 studies to significantly
suppress serum cortisol and has the potential to be a
next-generation cortisol inhibitor. The Phase 3 program
for RECORLEV includes SONICS and LOGICS: two multinational studies
designed to evaluate the safety and efficacy of RECORLEV when used
to treat endogenous Cushing’s syndrome. The SONICS study met its
primary and secondary endpoints, demonstrating a statistically
significant normalization rate of urinary free cortisol at six
months. The LOGICS study, which met its primary endpoint, is a
double-blind, placebo-controlled randomized-withdrawal study of
RECORLEV that is designed to supplement the long-term efficacy and
safety information supplied by SONICS. The ongoing long-term open
label OPTICS study will gather further useful information related
to the long-term use of RECORLEV.
RECORLEV has received orphan drug designation
from the FDA and the European Medicines Agency for the
treatment of endogenous Cushing's syndrome.
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), a cortisol
synthesis inhibitor currently being studied in Phase 3
clinical studies 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 the secondary endpoint data from the SONICS study,
the potential advantages of RECORLEV, Strongbridge’s
strategy, plans, 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, 2019 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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