Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the
Company), a rare disease therapeutics company, today
announced that new long-term real-world data from the
arimoclomol expanded access program (EAP: NCT04316637) for the
treatment of Niemann-Pick disease type C (NPC) was featured in an
oral presentation during the Society for Inherited Metabolic
Disorders (SIMD) 45th Annual Meeting taking place at the
Sheraton/Le Meridien Charlotte Hotel Complex in Charlotte, NC, from
April 14-17, 2024.
“These real-world data collected in the
arimoclomol US EAP represent the first evidence to support the use
of arimoclomol in adults with NPC, demonstrating a clinically
meaningful slowing of disease progression,” noted Kristina Julich,
M.D., Assistant Professor, Department of Neurology, at The
University of Texas at Austin, Dell Medical School. “NPC presents a
therapeutic challenge, lacking approved treatment options in the
U.S. and invariably leading to progressive loss of independence due
to physical and cognitive impairments. We are very pleased with the
positive new data that brings hope for a community with a high
unmet medical need.”
“These observed long-term clinical benefits add
to the body of evidence for arimoclomol that supports its
tolerability and effectiveness in the treatment of NPC, bringing us
one step closer to helping patients suffering from this relentless
and fatal disease,” remarked Neil F. McFarlane, President and Chief
Executive Officer of Zevra. “We remain committed to continue
collaborating closely with the FDA as it reviews arimoclomol's NDA
ahead of our PDUFA date on September 21, 2024.”
Highlights from the
Presentation:
The results were presented by Kristina Julich,
M.D., Chief, Neurogenetics Center, and Assistant Professor of
Pediatric Neurosciences at Dell Medical School from The University
of Texas.
The first patient in the EAP was enrolled in
June 2020. As of July 19, 2023, 41 participants from 11 U.S.
centers were ≥18 years at arimoclomol initiation; 26 had baseline
Physician-reported 5-domain NPC Clinical Severity Scale (5DNPCCS)
assessments with ≥1 year of follow-up. Among these 26 adults, mean
(standard deviation, SD) age at NPC diagnosis and arimoclomol
initiation was 23.7 (9.0) and 28.5 (6.5) years, respectively.
Patients continued arimoclomol treatment for a mean of 21 months
(range: 12-32) and 69% (18/26) had recorded miglustat use. Adults
treated with arimoclomol, including those with and without
miglustat use, generally had a stable disease course over two years
of treatment and follow-up and the safety profile was consistent
with that observed in the Phase 2/3 study where no new safety
adverse events were identified.
In addition to this scientific presentation,
Marc C. Patterson, MD, Professor of Pediatrics at the Mayo Clinic
College of Medicine and Science, also presented the data
at the 2024 American Academy of Neurology Annual Meeting (AANAM),
during the Society Spotlight: Child Neurology Society session. Dr.
Patterson’s talk was entitled “Evaluation of the Long-Term Effect
of Arimoclomol in NPC - 48 Months Data from CT-ORZY-NPC-002.”
About Niemann-Pick Disease Type C
(NPC)
Niemann-Pick disease type C (NPC) is an
ultra-rare, progressive, and 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.
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 by the FDA, and Orphan Medicinal Product
designation for the treatment of NPC by the European Medicines
Agency (EMA). The FDA has accepted the resubmission of the NDA for
arimoclomol and has set a user fee goal date (PDUFA date) of
September 21, 2024.
About Zevra Therapeutics
Zevra Therapeutics is a rare disease company
combining science, data, and patient needs to create
transformational therapies for diseases with limited or no
treatment options. Our mission is to bring life-changing
therapeutics to people living with rare diseases. With unique,
data-driven development and commercialization strategies, the
Company is overcoming complex drug development challenges to make
new therapies available to the rare disease community.
Expanded access programs are made available by
Zevra Therapeutics and its affiliates and are subject to the
Company's Expanded Access Program (EAP) policy as published on its
website at www.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.
For more information, please visit www.zevra.com
or follow us on X (formerly Twitter)
and LinkedIn.
Cautionary Note 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 the
presentation of data at conferences, the promise and potential
impact of our preclinical or clinical trial data, the initiation,
timing and results of any clinical trials or readouts, the content,
information used for, timing or results of any NDA submissions or
resubmissions for arimoclomol or any other product candidates for
any specific disease indication or at any dosage, the potential
launch or commercialization of any of product candidates or
products, and our strategic and product development objectives,
including with respect to becoming a leading, commercially focused
rare disease company. 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, 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.
Zevra Contact
Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com
Russo Partners Contacts
Adanna G. Alexander, Ph.D.+1 (646)
942-5603adanna.alexander@russopartnersllc.com
Ignacio Guerrero-Ros, Ph.D.+1 (646)
942-5604ignacio.guerrero-ros@russopartnersllc.com
Zevra Therapeutics (NASDAQ:ZVRA)
過去 株価チャート
から 4 2024 まで 5 2024
Zevra Therapeutics (NASDAQ:ZVRA)
過去 株価チャート
から 5 2023 まで 5 2024