Lead Candidate, EPX-100, Holds Orphan Drug and
Rare Pediatric Disease Designations
EPX-100 in Pivotal Registrational Trial for
Dravet Syndrome; Topline Data Expected in 2026
Plan to Initiate Phase 3 Trial for
Lennox-Gastaut Syndrome in Second Half of 2024
Potential Billion Dollar Plus Market
Opportunity in Rare Epilepsy
PLYMOUTH
MEETING, Pa., April 30,
2024 /PRNewswire/ -- Harmony Biosciences (Nasdaq:
HRMY), today announced the acquisition of Epygenix Therapeutics,
Inc., accelerating its growth strategy by adding a rare epilepsy
franchise to its expanding late-stage pipeline of innovative CNS
assets.
"The acquisition of Epygenix gives us three distinct CNS
franchises in late-stage development, each with a potential US peak
sales opportunity of $1B -
$2B," said Jeffrey M. Dayno, M.D., President and Chief Executive Officer at
Harmony Biosciences. "We have been strategically building a robust
and diverse pipeline of innovative CNS assets for rare diseases
that has transformed our business. This acquisition builds on our
leadership position in sleep/wake and our franchise in
neurobehavioral disorders, allowing us to leverage our expertise in
CNS and internal synergies to deliver new therapies to patients
with unmet medical needs."
"Harmony shares our team's core values, including a commitment
to patients and families living with Dravet syndrome and
Lennox-Gastaut syndrome, where a significant unmet need for
effective therapies remains," said Alex
Yang, Chair and CEO of Epygenix Therapeutics. "Harmony's
demonstrated expertise in drug development and commercialization
will accelerate the full potential of these treatments and make a
real difference in the lives of Dravet and LGS families."
The acquisition includes clemizole hydrochloride (EPX-100), a
potent, oral, centrally acting serotonin (5HT2) agonist. It is
currently in a pivotal registrational clinical trial for the
treatment of Dravet syndrome in children and adults and is poised
to enter Phase 3 for the treatment of Lennox-Gastaut syndrome. The
proven mechanism of action of clemizole could potentially offer an
improved product profile over currently available treatment options
and improve daily functioning in patients living with Dravet
syndrome and Lennox-Gastaut syndrome.
EPX-100 has received Orphan Drug Designation (ODD) and Rare
Pediatric Disease Designation (RPDD) from the FDA for both Dravet
syndrome and Lennox-Gastaut syndrome. A second investigational
product, EPX-200, is a potent, oral, centrally active and selective
5HT2C agonist, and is currently in IND-enabling studies. EPX-200
also received ODD from FDA for Dravet syndrome and Lennox-Gastaut
syndrome as well as RPDD for Lennox-Gastaut syndrome.
Under the terms of the definitive agreement, Harmony paid
$35 million in cash with the
potential for payments of up to $130
million based on development and regulatory milestones. In
addition, there are potential payments of up to $515 million if certain sales milestones are
achieved.
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.1 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.1 Approximately 85% of Dravet syndrome cases
are caused by de novo loss-of-function (LOF) mutations in a
voltage-gated sodium channel gene, SCN1A1.2
DS has an estimated incidence rate of
1:15,7003.
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.2,4 As a
result, patients experience multiple seizure types, including
atonic seizures, and developmental, cognitive, and behavioral
issues.3 LGS affects approximately 48,000 patients in
the U.S5.
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.
About Epygenix Therapeutics, Inc.
Epygenix Therapeutics, Inc. is a precision medicine-based
biopharmaceutical company focused on genetically screening,
discovering, and developing drugs to treat rare and intractable
forms of genetic epilepsy in childhood, such as Dravet syndrome
(DS). Epygenix is currently focused on developing EPX-100 and
EPX-200, drug candidates that were discovered in a zebrafish model
of DS which replicates the genetic mutation that causes DS and
mimics the human pathology. This mechanism of action has been shown
to eliminate both clinical and electrographic seizure activity
which could translate into improved patient functioning.
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 Epygenix, including clemizole hydrochloride. 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 and our other product candidates, including
clemizole hydrochloride; the rate and degree of market acceptance
and clinical utility of WAKIX, pitolisant in additional
indications, if approved, and any other product candidates,
including clemizole hydrochloride, 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 and our other
product candidates, including clemizole hydrochloride; 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 and those that we
have acquired; 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, Epygenix
and another entity or assets that we acquire; 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, as amended, 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 Investor Contact:
Luis Sanay, CFA
445-235-8386
lsanay@harmonybiosciences.com
Harmony Biosciences Media Contact:
Cate McCanless
202-641-6086
cmccanless@harmonybiosciences.com
1 EpyGenix Company Presentation:
https://www.epygenix.com/news
2 EpyGenix
Poster: https://www.epygenix.com/_files/ugd/4ad619_2db63a277738444c85e70a47b816a67c.pdf
3 Wu, E., et. al. (2015). Incidence of Dravet Syndrome
in a US Population. Pediatrics 136(5): 1310-e1315.
doi: 10.1542/peds.2015-1807.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4621800/
4
https://www.epygenix.com/rare-genetic-epilepsy
5
https://www.lgsfoundation.org/about-lgs-2/how-many-people-have-lgs/
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SOURCE Harmony Biosciences