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 the U.S. Food
and Drug Administration (FDA) has accepted for review the Company’s
New Drug Application (NDA) for RECORLEV® (levoketoconazole) for the
treatment of endogenous Cushing’s syndrome.
Earlier than anticipated and following
Strongbridge’s reporting of first quarter earnings results on May
12, 2021, the Company received the official Day 74 letter from the
FDA. Within the Day 74 letter, the FDA set a Prescription Drug User
Fee Act (PDUFA) target action date of January 1, 2022, which
reflects a projected 10-month standard review period. The letter
made no mention of a plan to hold an advisory committee
meeting.
“We are pleased with the FDA's acceptance for
filing of the RECORLEV® (levoketoconazole) New Drug Application. We
believe this decision reflects the comprehensive clinical evidence
that went into the NDA submission, including the positive and
statistically significant efficacy and safety results from the
multinational Phase 3 SONICS and LOGICS studies evaluating RECORLEV
as a potential treatment option for adults with endogenous
Cushing’s syndrome. We are advancing our commercial readiness plans
and look forward to potentially bringing a new therapeutic option
to the Cushing's syndrome community in the first quarter of 2022,”
said John H. Johnson, chief executive officer of Strongbridge
Biopharma.
About the SONICS StudySONICS was 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 had 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 the LOGICS StudyThe Phase 3,
multinational, double-blind, placebo-controlled,
randomized-withdrawal study, LOGICS, randomized Cushing’s syndrome
patients with baseline mean urinary free cortisol (mUFC) at least
1.5 times the upper limit of normal (ULN) following completion of a
single-arm, open-label treatment phase of approximately 14 to 19
weeks, with RECORLEV individually titrated according to mUFC
response.
A total of 79 patients were dosed during the
open-label titration-maintenance phase, 7 of whom had previously
received RECORLEV during the SONICS study, and 72 who had not
previously received RECORLEV. At study baseline, the median mUFC
was 3.5 times the ULN, indicative of significant
hypercortisolemia.
A total of 44 patients (39 who had completed the
titration-maintenance phase and five who directly enrolled from the
SONICS study), were randomized to either continue RECORLEV (n=22)
or to have treatment withdrawn by receiving a matching placebo
regimen (n=22) for up to 8 weeks, followed by restoration to the
prior regimen using blinded drug. Of the 44 patients
randomized, 11 patients (25 percent) had previously received
RECORLEV during the SONICS study. Patients who required rescue
treatment with open-label RECORLEV during the randomized-withdrawal
phase were considered to have lost mUFC response at the visit
corresponding to their first dose of rescue
medication. Patients who did not qualify for randomization
were removed from open-label treatment prior to randomization and
excused from the study.
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 two 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 RECORLEVRECORLEV®
(levoketoconazole) is an investigational adrenal steroidogenesis
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 and key secondary 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), an adrenal
steroidogenesis 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 potential advantages of RECORLEV, the anticipated
timing and process for the review of the NDA for RECORLEV and the
potential launch of RECORLEV (if approved), 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, 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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