PLYMOUTH
MEETING, Pa., April 3,
2024 /PRNewswire/ -- Harmony Biosciences Holdings,
Inc. ("Harmony") (Nasdaq: HRMY) has initiated its global Phase 3
registrational trial, the TEMPO study, to evaluate the safety and
efficacy of pitolisant as a treatment for excessive daytime
sleepiness (EDS) and behavioral symptoms in patients aged six years
and older with Prader-Willi syndrome (PWS).
The TEMPO study initiation follows a successful End-of-Phase 2
meeting with the U.S. Food & Drug Administration (FDA) and
their recent decision to grant Orphan Drug Designation (ODD) for
pitolisant in PWS. ODD incentivizes the advancement of promising
therapies for rare diseases by providing tax credits for clinical
development, waivers for user fees, and seven years of market
exclusivity following drug approval.
"The initiation of our Phase 3 TEMPO study, a global,
randomized, double-blind, placebo-controlled, multicenter trial
with an open-label extension period, reflects continued positive
momentum across our organization spanning our lifecycle management
programs in PWS and other indications with high unmet medical
need," said Kumar Budur, M.D., M.S.,
Chief Medical Officer of Harmony Biosciences. "With the initiation
of this study and the FDA's recent decision to grant ODD for
pitolisant in PWS, we aim to potentially introduce a new,
non-scheduled treatment option for EDS and the common behavioral
symptoms in patients living with this condition."
Approximately 15,000 – 20,000 people in the U.S. are
living with PWS, the majority experiencing behavioral symptoms and
more than half with EDS. There is currently no FDA-approved
treatment for EDS in this patient population.
"Our progress in expanding the clinical applications of
pitolisant in PWS and in other rare diseases, remains promising.
These efforts are integral to our life cycle management programs,
which, if successful, could benefit over 100,000 patients living
with unmet medical needs," Budur added.
Pitolisant is marketed as WAKIX® in
the U.S. and is FDA approved to treat EDS or cataplexy in
adult patients with narcolepsy. Pitolisant is not approved for use
in patients with PWS and is currently being evaluated as an
investigational agent in this patient population.
About Prader-Willi Syndrome
PWS is an orphan/rare,
genetic neurological disorder with many of the symptoms resulting
from hypothalamic dysfunction. The hypothalamus is the part of the
brain that controls both sleep-wake state stability and signals
that mediate the balance between hunger and satiety, resulting in
the main symptoms in patients with PWS, hyperphagia (an intense
persistent sensation of hunger accompanied by food preoccupations,
an extreme drive to consume food, food-related behavior problems,
and a lack of normal satiety), EDS and behavioral symptoms. Other
features include low muscle tone, short stature, and cognitive
impairment.
About WAKIX® (pitolisant)
Tablets
WAKIX, a first-in-class medication, is approved by
the U.S. Food and Drug Administration for the treatment
of excessive daytime sleepiness or cataplexy in adult patients with
narcolepsy and has been commercially available in
the U.S. since Q4 2019. It was granted orphan drug
designation for the treatment of narcolepsy in 2010, and
breakthrough therapy designation for the treatment of cataplexy in
2018. WAKIX is a selective histamine 3 (H₃) receptor
antagonist/inverse agonist. The mechanism of action of WAKIX is
unclear; however, its efficacy could be mediated through its
activity at H₃ receptors, thereby increasing the synthesis and
release of histamine, a wake promoting neurotransmitter. WAKIX was
designed and developed by Bioprojet (France). Harmony has an exclusive license
from Bioprojet to develop, manufacture and commercialize
pitolisant in the United States.
INDICATIONS AND USAGE
WAKIX is indicated for the
treatment of excessive daytime sleepiness or cataplexy in adult
patients with narcolepsy.
IMPORTANT SAFETY INFORMATION
Contraindications
WAKIX is contraindicated in patients
with known hypersensitivity to pitolisant or any component of the
formulation. Anaphylaxis has been reported. WAKIX is also
contraindicated in patients with severe hepatic impairment.
Warnings and Precautions
WAKIX prolongs the QT
interval; avoid use of WAKIX in patients with known QT prolongation
or in combination with other drugs known to prolong the QT
interval. Avoid use in patients with a history of cardiac
arrhythmias, as well as other circumstances that may increase the
risk of the occurrence of torsade de pointes or sudden death,
including symptomatic bradycardia, hypokalemia or hypomagnesemia,
and the presence of congenital prolongation of the QT interval.
The risk of QT prolongation may be greater in patients with
hepatic or renal impairment due to higher concentrations of
pitolisant; monitor these patients for increased QTc. Dosage
modification is recommended in patients with moderate hepatic
impairment and moderate or severe renal impairment (see full
prescribing information). WAKIX is not recommended in patients with
end-stage renal disease (ESRD).
Adverse Reactions
In the placebo-controlled clinical
trials conducted in patients with narcolepsy with or without
cataplexy, the most common adverse reactions (≥5% and at least
twice placebo) for WAKIX were insomnia (6%), nausea (6%), and
anxiety (5%). Other adverse reactions that occurred at ≥2% and more
frequently than in patients treated with placebo included headache,
upper respiratory tract infection, musculoskeletal pain, heart rate
increased, hallucinations, irritability, abdominal pain, sleep
disturbance, decreased appetite, cataplexy, dry mouth, and
rash.
Drug Interactions
Concomitant administration of WAKIX
with strong CYP2D6 inhibitors increases pitolisant exposure by
2.2-fold. Reduce the dose of WAKIX by half.
Concomitant use of WAKIX with strong CYP3A4 inducers decreases
exposure of pitolisant by 50%. Dosage adjustments may be required
(see full prescribing information).
H1 receptor antagonists that cross the blood-brain barrier may
reduce the effectiveness of WAKIX. Patients should avoid centrally
acting H1 receptor antagonists.
WAKIX is a borderline/weak inducer of CYP3A4. Therefore, reduced
effectiveness of sensitive CYP3A4 substrates may occur when used
concomitantly with WAKIX. The effectiveness of hormonal
contraceptives may be reduced when used with WAKIX and
effectiveness may be reduced for 21 days after discontinuation of
therapy.
Use in Specific Populations
WAKIX may reduce the
effectiveness of hormonal contraceptives. Patients using hormonal
contraception should be advised to use an alternative non-hormonal
contraceptive method during treatment with WAKIX and for at least
21 days after discontinuing treatment.
There is a pregnancy exposure registry that monitors pregnancy
outcomes in women who are exposed to WAKIX during pregnancy.
Patients should be encouraged to enroll in the WAKIX pregnancy
registry if they become pregnant. To enroll or obtain information
from the registry, patients can call 1-800-833-7460. The safety and
effectiveness of WAKIX have not been established in patients less
than 18 years of age.
WAKIX is extensively metabolized by the liver. WAKIX is
contraindicated in patients with severe hepatic impairment. Dosage
adjustment is required in patients with moderate hepatic
impairment.
WAKIX is not recommended in patients with end-stage renal
disease. Dosage adjustment of WAKIX is recommended in patients with
moderate or severe renal impairment.
Dosage reduction is recommended in patients known to be poor
CYP2D6 metabolizers; these patients have higher concentrations of
WAKIX than normal CYP2D6 metabolizers.
Please see the Full Prescribing Information for
WAKIX for more information.
To report suspected adverse reactions, contact Harmony
Biosciences at 1-800-833-7460 or the FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
About Harmony Biosciences
At Harmony Biosciences, we specialize in developing and
delivering treatments for rare neurological diseases that others
often overlook. We believe that where empathy and innovation meet,
a better life can begin for people living with neurological
diseases. Established by Paragon Biosciences, LLC, in 2017 and
headquartered in Plymouth Meeting, PA, our team of experts
from a wide variety of disciplines and experiences is driven by our
shared conviction that innovative science translates into
therapeutic possibilities for our patients, who are at the heart of
everything we do. For more information, please
visit www.harmonybiosciences.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements contained in this press release that do not
relate to matters of historical fact should be considered
forward-looking statements, including statements regarding our
product WAKIX and our future capabilities following the acquisition
of Zynerba. These statements are neither promises nor guarantees,
but involve known and unknown risks, uncertainties and other
important factors that may cause our actual results,
performance or achievements to be materially
different from any future results, performance or
achievements expressed or implied by the
forward-looking statements, including, but not limited to, the
following: our commercialization efforts and strategy for WAKIX;
the rate and degree of market acceptance and clinical utility of
WAKIX, pitolisant in additional indications, if approved, and any
other product candidates we may develop or acquire, if approved;
our research and development plans, including our development
activities with Bioprojet, and plans to explore the
therapeutic potential of pitolisant in additional indications; our
ongoing and planned clinical trials; the availability of favorable
insurance coverage and reimbursement for WAKIX; the timing of and
our ability to obtain regulatory approvals for pitolisant for other
indications as well as any of our product candidates, including
those we are developing with Bioprojet; our failure to achieve
the potential benefits of the 2022 LCA with Bioprojet; our
ability to recognize the intended benefits of our acquisition of
Zynerba Pharmaceuticals; our estimates regarding expenses, future
revenue, capital requirements and needs for additional financing;
our ability to identify additional products or product candidates
with significant commercial potential that are consistent with our
commercial objectives; our commercialization, marketing and
manufacturing capabilities and strategy; significant competition in
our industry; our intellectual property position; loss or
retirement of key members of management; failure to successfully
execute our growth strategy, including any delays in our planned
future growth; our failure to maintain effective internal controls;
the impact of government laws and regulations; volatility and
fluctuations in the price of our common stock; the significant
costs and required management time as a result of operating as a
public company; the fact that the price of Harmony's common stock
may be volatile and fluctuate substantially; statements related to
our intended share repurchases and repurchase timeframe and the
significant costs and required management time as a result of
operating as a public company. These and other important factors
discussed under the caption "Risk Factors" in our Annual Report on
Form 10-K filed with the Securities and Exchange
Commission (the "SEC") on February 22, 2024, and our
other filings with the SEC could cause actual results to
differ materially from those indicated by the forward-looking
statements made in this press release. Any such forward-looking
statements represent management's estimates as of the date of this
press release. While we may elect to update such forward-looking
statements at some point in the future, we disclaim any obligation
to do so, even if subsequent events cause our views to
change.
Harmony Biosciences Media Contact:
Cate McCanless
202-641-6086
cmccanless@harmonybiosciences.com
Harmony Biosciences Investor Contact:
Luis Sanay, CFA
445-235-8386
lsanay@harmonybiosciences.com
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