Zevra Therapeutics to Present at the 52nd Child Neurology Society Annual Meeting
05 Outubro 2023 - 8:30AM
Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company),
a rare disease therapeutics company, today announced that it will
present data related to clinical study outcomes and its early
access programs via both oral and poster presentations at the
upcoming 52nd Child Neurology Society (CNS) Annual Meeting. This
meeting will be held October 4-7, 2023, in Vancouver, Canada. The
presentations will focus on arimoclomol, Zevra’s late-stage
pipeline program in development for the treatment of Niemann-Pick
Disease type C (NPC), including 48 months of efficacy and safety
data captured through the double-blind and open-label extension
phases of the registrational trial, as well as methodology related
to establishing an early access program with real-world data
collection capabilities.
“Our team is eager to highlight our arimoclomol
program at the 52nd CNS Annual Meeting and engage with the key
child neurologists who are treating devastating conditions like NPC
where few therapeutic options exist,” said Daniel Gallo, PhD,
Senior Vice President of Medical Affairs and Advocacy for Zevra.
“The arimoclomol data demonstrate the importance of understanding
the long-term efficacy and treatment approach for this potential
new therapy for NPC.”
Oral Presentation Details:
Title: |
Evaluation of the Long-Term Effect of Arimoclomol in NPC – 48
Months Data from CT-ORZY-NPC-002 |
Presenter: |
Marc Patterson, MD, Professor of Neurology, Pediatrics, and Medical
Genetics, Mayo Clinic College of Medicine and Science, Rochester,
MN |
Session: |
Platform 3 |
Time: |
Friday, October 6th at 2:15 - 4:00 p.m. |
Location: |
Ballroom BC |
Poster Presentation Details:
Title: |
Real World Data Collection in Niemann-Pick Disease Type C – Data
from Expanded Access Program with Arimoclomol |
Presenter: |
Elizabeth Berry Kravis, MD, PhD, Professor of Pediatrics,
Neurological Sciences, and Biochemistry, Rush Medical College, Rush
University Medical Center, Chicago, IL |
Poster: |
#100 |
Poster Review: |
Thursday, Oct 5, 2023, at 12:30 - 2:00 p.m. and 5:30 - 7:30
p.m. |
The Zevra team will also be on site throughout the event and
invites attendees to connect at Booth #518 to learn more about how
Zevra is supporting the child neurology community.
About Niemann-Pick disease type C
(NPC):
Niemann-Pick disease type C (NPC) is an
ultra-rare and progressive, neurodegenerative lysosomal storage
disorder characterized by an inability of the body to transport
cholesterol and other lipids within the cell, leading to an
accumulation of these substances in various tissue areas, including
brain tissue. The disease is caused by mutations in the NPC1 or
NPC2 genes which are responsible for making lysosomal proteins and
is an autosomal recessive trait. Both children and adults can be
affected by NPC with varying clinical presentations. Those living
with NPC lose independence due to physical and cognitive
limitations, with key neurological impairments presenting in
speech, cognition, swallowing, ambulation, and fine motor skills.
Disease progression is irreversible and can be fatal within months
or take years to be diagnosed and advance in severity.
About Arimoclomol:
Arimoclomol, Zevra’s orally-delivered,
first-in-class investigational product candidate for the treatment
of NPC, has been granted orphan drug designation, Fast Track
designation, Breakthrough Therapy designation and rare pediatric
disease designation for NPC by the FDA, and orphan medicinal
product designation for the treatment of NPC by the European
Medicines Agency (EMA). The arimoclomol NDA is currently being
prepared for resubmission to the FDA.
About Zevra Therapeutics:
Zevra Therapeutics is a rare disease company
melding science, data, and patient need to create transformational
therapies for diseases with limited or no treatment options. With
unique, data-driven clinical, regulatory, and commercialization
strategies, the Company is overcoming complex drug development
challenges to bring much-needed therapies to patients. With both
regulatory and clinical stage product candidates, the Company is
building its commercial capability to make new therapies available
to the rare disease community.
Early access programs are made available by
Zevra Therapeutics and its affiliates and are subject to the
Company's Early Access Program (EAP) policy as published on its
website at zevra.com. Participation in these programs is subject to
the laws and regulations of each jurisdiction under which each
respective program is operated. Eligibility for participation in
any such program is at the treating physician's discretion.
Caution Concerning Forward-Looking
Statements:
This press release may contain forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Forward-looking statements include all
statements that do not relate solely to historical or current
facts, including without limitation statements regarding upcoming
events or Zevra’s participation at such events. Forward-looking
statements are based on information currently available to Zevra
and its current plans or expectations. They are subject to several
known and unknown uncertainties, risks, 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. These and other important factors are described in
detail in the “Risk Factors” section of Zevra’s Annual Report on
Form 10-K for the year ended December 31, 2022, as updated in
Zevra’s Quarterly Report on Form 10-Q for the quarter ended June
30, 2023, and Zevra’s other filings with the Securities and
Exchange Commission. While we may elect to update such
forward-looking statements at some point in the future, except as
required by law, we disclaim any obligation to do so, even if
subsequent events cause our views to change. Although we believe
the expectations reflected in such forward-looking statements are
reasonable, we cannot assure that such expectations will prove
correct. These forward-looking statements should not be relied upon
as representing our views as of any date after the date of this
press release.
Contacts:
Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com
Janine Bogris+1 (201) 245-6838Janine.Bogris@canalecomm.com
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