WAKIX® (pitolisant) Preliminary Net Revenue of
~$201 Million for Fourth Quarter and ~$714 Million for Full Year
2024; Representing Growth of ~23% in Year-Five of Launch
2025 WAKIX Net Revenue Guidance Between $820 -
$860 Million; On Track to a Potential $1 Billion+ Opportunity
Value-Creating Catalysts Anticipated Every
Quarter in 2025:
Q1 – FDA Decision on File Acceptance of
Pitolisant sNDA for Idiopathic Hypersomnia; Potential Approval in
2025 Q2 – Orexin/BP1.15205 Data Presentation at SLEEP 2025
Conference Q3 – Pivotal Phase 3 Topline Data for ZYN002 in Fragile
X Syndrome Q4 – Initiation of Pitolisant-HD Pivotal Phase 3 Trial
in Narcolepsy
Robust, Catalyst-Rich Pipeline Includes Up to
Six Phase 3 Clinical Programs by Year End 2025
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today
announced strong preliminary, unaudited net product revenues for Q4
and full year 2024 of $201 million and $714 million, respectively.
Building on this performance, the company is well positioned for a
catalyst-rich 2025, with value-creating milestones anticipated
every quarter. Harmony will be highlighting its catalysts for 2025
and other key milestones during its presentation at the 43rd Annual
J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025,
at 11:15 a.m. PT / 2:15 p.m. ET.
“2025 is a pivotal year for Harmony Biosciences, as we focus on
unlocking the full potential of our catalyst-rich neuroscience
pipeline,” said Jeffrey M. Dayno, M.D., President and Chief
Executive Officer of Harmony Biosciences. “Building upon our
foundation of strong and continued revenue growth for WAKIX, our
multi-franchise pipeline has come into focus with value-creating
catalysts expected each and every quarter this year. We are
entering 2025 with strong momentum and a clear path toward
long-term growth, building a company with the potential to deliver
over $3 billion in net revenue going forward."
Fourth Quarter and Full Year 2024 Net Product Revenue for
WAKIX (Preliminary and Unaudited)
- Preliminary, unaudited net product revenue for the quarter
ended December 31, 2024, was approximately $201 million, compared
to $168.4 million for the same period in 2023
- Preliminary, unaudited net product revenue for the full year
ended December 31, 2024, was approximately $714 million, compared
to $582 million for the same period in 2023, representing ~23%
growth in its fifth year on the market
- The average number of patients on WAKIX increased by
approximately 300 sequentially to approximately 7,100 for the
quarter ended December 31, 2024
2025 Net Revenue Guidance
- Net revenue projected between $820 to $860 million
Key Catalysts for 2025
- Q1 – FDA decision on file acceptance of pitolisant sNDA
for Idiopathic Hypersomnia (IH)
- IH sNDA for pitolisant submitted in Q4 2024
- Potential FDA approval in 2025
- Q2 – Preclinical data for BP1.15205 (OX2R agonist) to be
presented at SLEEP 2025
- Data to include preclinical safety and efficacy
- BP1.15205 on track for first-in-human study in Q3 2025
- Q3 – Topline data readout for ZYN002 from pivotal Phase
3 trial in Fragile X syndrome (FXS)
- Potential for first and only approved treatment for patients
with FXS
- Estimated 80,000 people living with FXS in the US; worldwide
rights
- Q4 – Initiation of Pitolisant-HD Pivotal Phase 3 trial
in Narcolepsy
- Designed to address the largest unmet need in patients with
narcolepsy by providing greater efficacy for both excessive daytime
sleepiness and cataplexy
- Program to include novel endpoint to assess narcolepsy-related
fatigue
- Preliminary IP filed out to 2044 to extend the pitolisant
franchise
Robust, Multi-Franchise Pipeline
- Anticipates up to six phase 3 clinical development programs by
year end 2025
- Initiated pivotal phase 3 study in Lennox-Gastaut syndrome
(LGS) for EPX-100 in Q4 2024
- EPX-100 is the most advanced 5HT2 (serotonin) agonist in
clinical development
- Initiation of Pitolisant-GR pivotal bioequivalence study in Q1
2025 with readout anticipated Q3 2025
- Pipeline poised to deliver at least one new product or
indication approval every year for the next four years (2028)
- Current pipeline has the potential to generate over $3B in net
revenue
- Additional program updates to be provided during the Management
presentation at the upcoming J.P. Morgan Healthcare Conference on
January 15, 2025, at 11:15 a.m. PT / 2:15 p.m. ET. Presentation
slides are available on the investor page of the Harmony
Biosciences website: https://ir.harmonybiosciences.com/
The financial information included in this press release is
preliminary, unaudited, and subject to change. It does not present
all the information necessary for an understanding of the company's
financial results for the fourth quarter or full year 2024.
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 (EDS) or cataplexy in adult patients with narcolepsy and
for the treatment of EDS in pediatric patients 6 years of age and
older with narcolepsy. 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 (EDS) or cataplexy in adult patients with narcolepsy and
for the treatment of excessive daytime sleepiness (EDS) in
pediatric patients 6 years of age and older 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. WAKIX is
contraindicated in patients with severe hepatic impairment and 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.
In the placebo-controlled phase of the clinical trial conducted
in pediatric patients 6 years and older with narcolepsy with or
without cataplexy, the most common adverse reactions (≥5% and
greater than placebo) for WAKIX were headache (19%) and insomnia
(7%). The overall adverse reaction profile of WAKIX in the
pediatric clinical trial was similar to that seen in the adult
clinical trial program.
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.
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. WAKIX may reduce
the effectiveness of sensitive CYP3A4 substrates, including
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.
Use in Specific Populations
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
for treatment of excessive daytime sleepiness in pediatric patients
less than 6 years of age with narcolepsy.
The safety and effectiveness of WAKIX have not been established
for treatment of cataplexy in pediatric patients with
narcolepsy.
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
eGFR <60 mL/minute/1.73 m2.
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 Narcolepsy
Narcolepsy is a rare, chronic, debilitating neurological disease
of sleep-wake state instability that impacts approximately 170,000
Americans and is primarily characterized by excessive daytime
sleepiness (EDS) and cataplexy – its two cardinal symptoms – along
with other manifestations of REM sleep dysregulation
(hallucinations and sleep paralysis), which intrude into
wakefulness. EDS is the inability to stay awake and alert during
the day and is the symptom that is present in all people living
with narcolepsy. In most patients, narcolepsy is caused by the loss
of hypocretin/orexin, a neuropeptide in the brain that supports
sleep-wake state stability. This disease affects men and women
equally, with typical symptom onset in adolescence or young
adulthood; however, it can take up to a decade to be properly
diagnosed.
About Idiopathic Hypersomnia
Idiopathic Hypersomnia (IH) is a rare and chronic neurological
disease that is characterized by excessive daytime sleepiness (EDS)
despite sufficient or even long sleep time. EDS in IH cannot be
alleviated by naps, longer sleep or more efficient sleep. People
living with IH experience significant EDS along with the symptoms
of sleep inertia (prolonged difficulty waking up from sleep) and
'brain fog' (impaired cognition, attention, and alertness). The
cause of IH is unknown, but it is likely due to alterations in
areas of the brain that stabilize states of sleep and wakefulness.
IH is one of the central disorders of hypersomnolence and, like
narcolepsy, is a debilitating sleep disorder that can result in
significant disruption in daily functioning.
About ZYN002
ZYN002 is the first-and-only pharmaceutically manufactured
synthetic cannabidiol devoid of THC and formulated as a
patent-protected permeation-enhanced gel for transdermal delivery
through the skin and into the circulatory system. The product is
manufactured through a synthetic process in a cGMP facility and is
not extracted from the cannabis plant. ZYN002 does not contain THC,
the compound that causes the euphoric effect of cannabis, and has
the potential to be a nonscheduled product if approved.
Cannabidiol, the active ingredient in ZYN002, has been granted
orphan drug designation by the United States Food and Drug
Administration (FDA) and the European Medicines Agency (EMA) for
the treatment of FXS and for the treatment of 22q. Additionally,
ZYN002 has received FDA Fast Track designation for the treatment of
behavioral symptoms in patients with FXS.
About Fragile X Syndrome
Fragile X syndrome (FXS) is a rare genetic disorder that is the
leading known cause of both inherited intellectual disability and
autism spectrum disorder. The disorder negatively affects synaptic
function, plasticity and neuronal connections, and results in a
spectrum of intellectual disabilities and behavioral symptoms, such
as social avoidance and irritability. While the exact prevalence is
unknown, upwards of 80,000 patients in the U.S. and 121,000
patients in the European Union and the UK are believed to have FXS,
based on FXS prevalence estimates of approximately 1 in 4,000 to
7,000 in males and approximately 1 in 8,000 to 11,000 in females.
There is a significant unmet medical need in patients living with
FXS as there are currently no FDA-approved treatments for this
disorder.
FXS is caused by a mutation in FMR1, a gene which modulates a
number of systems, including the endocannabinoid system, and most
critically, codes for a protein called FMRP. The FMR1 mutation
manifests as multiple repeats of a DNA segment, known as the CGG
triplet repeat, resulting in deficiency or lack of FMRP. FMRP helps
regulate the production of other proteins and plays a role in the
development of synapses, which are critical for relaying nerve
impulses, and in regulating synaptic plasticity. In people with
full mutation of the FMR1 gene, the CGG segment is repeated more
than 200 times, and in most cases causes the gene to not function.
Methylation of the FMR1 gene also plays a role in determining
functionality of the gene. In approximately 60% of patients with
FXS, who have complete methylation of the FMR1 gene, no FMRP is
produced, resulting in dysregulation of the systems modulated by
FMRP.
About Clemizole Hydrochloride (EPX-100)
EPX-100, clemizole hydrochloride, is under development for the
treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome
(LGS). EPX-100 acts by targeting central 5-hydroxytryptamine
receptors to modulate serotonin signaling. The drug candidate is
administered orally twice a day in a liquid formulation and has
been developed based on a proprietary phenotype-based zebrafish
drug screening platform. DS is caused by a loss of function
mutation in the SCN1A gene, and scn1 mutant zebrafish replicate the
genetic etiology and phenotype observed in the majority of DS
patients. The scn1Lab mutant zebrafish model that expresses voltage
gated sodium channels has been used for high-throughput screening
of compounds that modulate Nav1.1 in the central nervous
system.
About Dravet Syndrome
Dravet syndrome (DS) is a severe and progressive epileptic
encephalopathy that begins in infancy and causes significant impact
on patient functioning. DS begins in the first year of life and is
characterized by high seizure frequency and severity, intellectual
disability, and a risk of sudden unexpected death in epilepsy.
Approximately 85% of Dravet syndrome cases are caused by de novo
loss-of-function (LOF) mutations in a voltage-gated sodium channel
gene, SCN1A1. DS has an estimated incidence rate of 1:15,700.
About Lennox-Gastaut Syndrome
Lennox-Gastaut syndrome (LGS) is a rare and drug-resistant
epileptic encephalopathy characterized by onset in children between
3-5 years of age. The underlying cause of LGS is unknown and can be
related to a wide range of factors including genetic differences
and structural differences in the brain. As a result, patients
experience multiple seizure types, including atonic seizures, and
developmental, cognitive, and behavioral issues. LGS affects
approximately 48,000 patients in the U.S.
About Harmony Biosciences
Harmony Biosciences is a pharmaceutical company dedicated to
developing and commercializing innovative therapies for patients
with rare neurological diseases who have unmet medical needs.
Driven by novel science, visionary thinking, and a commitment to
those who feel overlooked, Harmony Biosciences is nurturing a
future full of therapeutic possibilities that may enable patients
with rare neurological diseases to truly thrive. Established by
Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth
Meeting, Pa., we believe that when empathy and innovation meet, a
better future can begin; a vision evident in the therapeutic
innovations we advance, the culture we cultivate, and the community
programs we foster. 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 full
year 2024 net product revenue, expectations for the growth and
value of WAKIX, plans to submit an sNDA for pitolisant in
idiopathic hypersomnia; our future results of operations and
financial position, business strategy, products, prospective
products, product approvals, the plans and objectives of management
for future operations and future results of anticipated products.
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 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 plans to explore the therapeutic
potential of pitolisant in additional indications; our ongoing and
planned clinical trials; our ability to expand the scope of our
license agreements with Bioprojet Société Civile de Recherche
(“Bioprojet”); 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 other product candidates; our estimates regarding expenses,
future revenue, capital requirements and additional financing
needs; our ability to identify, acquire and integrate 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20250113795547/en/
Harmony Biosciences Investor: Brennan Doyle 484-539-9700
bdoyle@harmonybiosciences.com Harmony Biosciences Media:
Cate McCanless 202-641-6086 cmccanless@harmonybiosciences.com
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