PLYMOUTH
MEETING, Pa., Dec. 5, 2023
/PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or
the "Company") (Nasdaq: HRMY), a pharmaceutical company dedicated
to developing and commercializing innovative therapies for patients
with rare neurological diseases, will present caregiver-reported
impacts of 22q11.2 deletion syndrome (22q) and data from the Phase
2 INSPIRE trial at the American College of Neuropsychopharmacology
Annual Meeting from December 3-6,
2023.
The INSPIRE trial evaluated ZYN002 as a potential treatment for
anxiety and other irritable behavioral symptoms associated with
22q. Qualitative behavioral analyses of patients and their
caregivers, both from INSPIRE and an independent panel, as well as
prospective post-hoc analyses of the INSPIRE trial, informed the
creation of a conceptual framework for the selection of
patient-centered assessments for future clinical trials. This
framework proposes endpoint measures to assess the treatment of
behavioral symptoms associated with the condition, which currently
lacks FDA-approved treatments and affects approximately 80,000
individuals in the U.S. and 129,000 in the EU and UK.
"Harmony is dedicated to finding effective treatments for
children and their families living with 22q and other
rare neuropsychiatric conditions that currently lack approved
therapies," said Kumar Budur, M.D.,
M.S., Chief Medical Officer at Harmony Biosciences. "Through our
recent acquisition of Zynerba, we are making meaningful strides
toward developing new treatments for individuals with the
behavioral symptoms associated with 22q and Fragile X syndrome
(FXS). The development of endpoint measures for future clinical
trials is an important step forward in our work for 22q patients
who have high unmet medical needs."
Poster: Anxious and Irritable Behaviors in Children with
22q11.2 Deletion Syndrome: A Qualitative Interview Study and
Development of a Conceptual Framework
- Poster Session II: Tuesday, December
5, 5PM – 7PM (ET)
ZYN002 is being evaluated as an investigational treatment for
22q. It is not approved for commercial distribution by government
regulatory bodies, including the U.S. Food and Drug Administration
(FDA).
About the INSPIRE Trial
The 38-week INSPIRE trial was an open-label, Phase 2 clinical trial
designed to evaluate the safety, tolerability and effectiveness of
ZYN002 in children and adolescents (ages four through 15) with
genetically confirmed 22q11.2 deletion syndrome. Enrolled patients
received weight-based doses of 250 mg or 500 mg daily of ZYN002.
Patients were allowed to increase the daily dose after six weeks of
treatment to 500 mg and 750 mg if the investigator felt such an
increase was appropriate. At the completion of the first 14-week
period of treatment, patients who demonstrated an improvement in
symptoms of irritability continued ZYN002 for an additional six
months, for a total of 38 weeks of treatment.
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 22q11.2 Deletion Syndrome
22q11.2 deletion
syndrome (22q) is a disorder caused by a small missing piece of the
22nd chromosome. The deletion occurs near the middle of the
chromosome at a location designated q11.2. It is considered a
mid-line condition, with physical symptoms including characteristic
palate abnormalities, heart defects, immune dysfunction, and
esophageal / GI issues, as well as debilitating neuropsychiatric
and behavioral symptoms, including anxiety, social withdrawal,
ADHD, cognitive impairment and autism spectrum disorder. It is
estimated that 22q occurs in one in 4,000 live births, suggesting
that there are approximately 80,000 people living with 22q in the
U.S. and 129,000 in the European Union and the UK. Patients with
22q deletion syndrome are managed by multidisciplinary care
providers, and there are currently no FDA approved treatments for
this disorder.
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,
affecting 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000
females. 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. There are approximately 80,000 people
in the U.S. and approximately 121,000 people in the European
Union and UK living with FXS. 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 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 Statement
This press release contains
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. These statements are
based on the current beliefs and expectations of Harmony's
management and are subject to significant risks and uncertainties.
Actual results may differ materially from those described in the
forward-looking statements. All statements contained in this press
release that do not relate to matters of historical fact, including
statements about our beliefs and expectations, should be considered
forward-looking statements. Forward-looking statements include
information concerning possible or assumed future results of
operations, including descriptions of our business plans and
strategies. These statements often include words such as
"anticipate," "expect," "guidance," "suggest," "plan," "believe,"
"intend," "estimate," "target," "project," "should," "could,"
"would," "may," "will," "forecast," "outlook," "potential,"
"continues," "seeks," "predicts," or the negative of these words
and other similar expressions. Factors that could cause actual
results to differ materially from those described in the
forward-looking statements include: risks related to the
distraction of management from ongoing business operations and
other opportunities due to recent acquisitions; our ability to
acquire businesses, successfully secure financing for our
acquisitions and timely consummate such acquisitions; the
possibility that we will not successfully integrate the operations
of our acquisitions, control the costs of integrating our
acquisitions or realize the intended benefits of such acquisitions,
including our recent Zynerba acquisition; government regulation and
changes in the regulatory environment; litigation or regulatory
proceedings; our ability to effectively manage our costs; our
ability to retain or renew existing agreements with large or
long-term customers; our ability to manage and expand our
operations and keep up with rapidly changing technologies; our
ability to maintain the security and integrity of our data; losses
against which we do not insure; our ability to make timely payments
of principal and interest on our indebtedness; our ability to
satisfy covenants in the agreements governing our indebtedness; our
ability to maintain our liquidity; our reliance on key management
personnel; and other one-time events and other factors that can be
found in our Annual Report on Form 10-K for the year ended
December 31, 2022, and any subsequent
Quarterly Report on Form 10-Q or Current Report on Form 8-K, which
are filed with the Securities and Exchange Commission. Many of
these factors are beyond our control. The forward-looking
statements contained in this earnings release speak only as of the
date of this earnings release. We undertake no obligation to
publicly release the result of any revisions to these
forward-looking statements to reflect the impact of events or
circumstances that may arise after the date of this earnings
release.
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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