DUBLIN, April 26, 2017 /PRNewswire/ -- Jazz
Pharmaceuticals plc (Nasdaq: JAZZ) today announced positive
efficacy results from the global multicenter study of JZP-110 in
adult patients with excessive sleepiness associated with
narcolepsy. Based on the preliminary safety analysis, the
most commonly reported adverse events (AEs) in this study were
generally consistent with those previously observed in the Phase 2
clinical studies evaluating JZP-110 in narcolepsy.
"Excessive sleepiness in narcolepsy remains an important unmet
medical need, and we believe that the statistically and clinically
significant effect observed in this study indicates that JZP-110
could be an important treatment option for patients," said
Karen Smith, M.D., Ph.D., global
head of research and development and chief medical officer of Jazz
Pharmaceuticals. "Subject to completion of the data
analysis and our regulatory discussions with FDA, we expect to
submit the JZP-110 NDA for excessive sleepiness in obstructive
sleep apnea and narcolepsy in late 2017. We are grateful to the
patients and investigators who contributed to this successful
study. This study exemplifies our ongoing and increasing
commitment to R&D, as demonstrated by the large number of
important clinical and regulatory activities that we have underway
to bring new therapies to patients."
"Patients with narcolepsy struggle with excessive sleepiness,
which is the symptom of narcolepsy that patients report as having
the most significant impact on their daily lives," said
Michael Thorpy, M.D., Director of
the Sleep-Wake Disorders Center at the Montefiore Medical Center,
Bronx, New York. "Excessive
sleepiness occurs in all patients with narcolepsy and when combined
with frequent irresistible sleep attacks, can lead to a
considerable burden of illness and adverse effect on patients'
health."
The Treatment of OSA and Narcolepsy Excessive Sleepiness (TONES)
Phase 3 program is comprised of four studies, two in obstructive
sleep apnea (OSA) (TONES 3 and TONES 4 studies), one in narcolepsy
(TONES 2 study) and one open-label, long-term safety and
maintenance of efficacy study. The TONES 2 study enrolled 240
patients. The company plans to present the results from
the TONES 2, TONES 3 and TONES 4 studies assessing the effect of
JZP-110 on excessive sleepiness in narcolepsy and OSA at the APSS
meeting in June.
Efficacy and Preliminary Safety Results of TONES 2
Study
The TONES 2 study, or 14-002, is a 4-arm, parallel-group study
evaluating three doses of JZP-110 (300 mg, 150 mg, and 75 mg)
compared to placebo for a 12-week period.
In TONES 2, JZP-110 demonstrated highly statistically
significant improvement in the co-primary endpoints of the
Maintenance of Wakefulness Test (MWT) and the Epworth Sleepiness
Scale (ESS) and in the key secondary endpoint of the Patient Global
Impression of Change (PGIc) scale at the 300 mg and 150 mg doses
compared to placebo. These effects were maintained
throughout the course of the study. The JZP-110 75 mg dose
reached statistical significance on the co-primary endpoint of MWT,
but did not reach statistical significance on the co-primary
endpoint of ESS. The endpoints of MWT and ESS measure
patients' ability to stay awake and patients' subjective levels of
sleepiness, respectively.
Based on a preliminary safety analysis, the most commonly
reported adverse events across all doses in TONES 2 were headache,
nausea, decreased appetite, nasopharyngitis, dry mouth, and
anxiety. There was one patient with two serious adverse
events (SAEs) on JZP-110 that were considered not treatment related
as assessed by the investigator. The open-label, long-term
safety and maintenance of efficacy study is ongoing, and the
results will be included in the planned New Drug Application
(NDA) submission.
About the Global Phase 3 TONES 2 Study
TONES 2 is a 12-week, 4-arm, parallel-group, double-blind,
placebo-controlled, randomized Phase 3 study evaluating the safety
and efficacy of JZP-110 in adults with excessive sleepiness in
narcolepsy. Patients were randomized 1:1:1:1 to JZP-110 300
mg, 150 mg, 75 mg and placebo. The co-primary endpoints are
the change in mean sleep latency on the MWT and the change in the
ESS score from baseline to week 12. The key secondary
endpoint is the percentage of patients who report improvement on
the PGIc scale, a patient-reported measure of improvement,
worsening, or no change in overall condition from baseline to week
12. The TONES 2 study enrolled 240 patients.
For information about the TONES 3 and TONES 4 studies, refer to
the company's press release dated March 20,
2017 at www.jazzpharmaceuticals.com.
About Narcolepsy
Narcolepsy is a debilitating neurological disorder characterized by
excessive sleepiness, and the inability to regulate sleep-wake
cycles normally. It affects an estimated one in 2,000 people
in the United States, with
symptoms typically appearing in early adulthood. It is
estimated that 50 percent or more patients with narcolepsy have not
been diagnosed. Studies have shown it may take 10 years or
more for people with narcolepsy to receive a correct
diagnosis. Excessive sleepiness is the primary symptom of
narcolepsy and is present in all people with the disorder.
Excessive sleepiness is characterized by the inability to stay
awake and alert during the day resulting in unplanned lapses into
sleep or drowsiness.
About JZP-110
JZP-110 is a selective dopamine and norepinephrine reuptake
inhibitor (DNRI) in late-stage development for treatment of
excessive sleepiness in adult patients with narcolepsy or
OSA. In 2014, Jazz Pharmaceuticals plc acquired a world-wide
license (except in certain countries in Asia) to develop and commercialize JZP-110
from SK Biopharmaceuticals, who discovered the compound. Jazz
Pharmaceuticals has worldwide development, manufacturing, and
commercialization rights to JZP-110, excluding certain
jurisdictions in Asia. SK Biopharmaceuticals maintains rights
in Korea, Japan, China, Taiwan, Singapore, Indonesia, India, Philippines, Thailand, Malaysia, Vietnam and Hong Kong. JZP-110 has
orphan drug designation in the United
States for narcolepsy.
Across the entire JZP-110 development program, over 2,000
subjects have enrolled in 20 studies. The JZP-110 Phase 3
clinical program includes two studies evaluating excessive
sleepiness in adult patients with OSA (TONES 3 and TONES 4), one
study evaluating excessive sleepiness in adult patients with
narcolepsy (TONES 2) and an open-label, long-term safety and
maintenance of efficacy study. Enrollment is complete in all
studies that are expected to support the planned JZP-110 NDA
submission to the U.S. Food and Drug Administration (FDA).
About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is an international
biopharmaceutical company focused on improving patients' lives by
identifying, developing and commercializing meaningful products
that address unmet medical needs. The company has a diverse
portfolio of products and product candidates, with a focus in the
areas of sleep and hematology/oncology. In these areas, Jazz
Pharmaceuticals markets Xyrem® (sodium oxybate) oral solution,
Erwinaze® (asparaginase Erwinia chrysanthemi) and Defitelio®
(defibrotide sodium) in the U.S. and markets Erwinase® and
Defitelio® (defibrotide) in countries outside the U.S. For more
information, please visit www.jazzpharmaceuticals.com.
"Safe Harbor" Statement under the Private Securities
Litigation Reform Act of 1995
This press release contains
forward-looking statements, including, but not limited to,
statements related to JZP-110 as a potential treatment for
excessive sleepiness in adult patients with narcolepsy, the
expected presentation of JZP-110 study results at APSS, the
company's plans for submission of an NDA for JZP-110 with the FDA,
the timing of such events and activities, and other statements that
are not historical facts. These forward-looking statements
are based on the company's current plans, objectives, estimates,
expectations and intentions and inherently involve significant
risks and uncertainties. Actual results and the timing of
events could differ materially from those anticipated in such
forward-looking statements as a result of these risks and
uncertainties, which include, without limitation, risks and
uncertainties associated with: pharmaceutical product development
and clinical success thereof; the regulatory approval process,
including the risk that the company may be unable to obtain
approval by the FDA for JZP-110 in a timely manner or at all; and
effectively commercializing JZP-110; and other risks and
uncertainties affecting the company and its development programs,
including those described from time to time under the caption "Risk
Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and
Exchange Commission filings and reports (Commission File No.
001-33500), including the company's Annual Report on Form 10-K for
the period ended December 31, 2016
and future filings and reports by the company. Other risks
and uncertainties of which the company is not currently aware may
also affect the company's forward-looking statements and may cause
actual results and the timing of events to differ materially from
those anticipated. The forward-looking statements herein are
made only as of the date hereof or as of the dates indicated in the
forward-looking statements, even if they are subsequently made
available by the company on its website or otherwise. The
company undertakes no obligation to update or supplement any
forward-looking statements to reflect actual results, new
information, future events, changes in its expectations or other
circumstances that exist after the date as of which the
forward-looking statements were made.
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SOURCE Jazz Pharmaceuticals plc