Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease
therapeutics company, proudly supports the Niemann-Pick community
on Global Niemann-Pick Disease Awareness Day and throughout
Niemann-Pick Disease Awareness Month. Patient organizations like
the International Niemann-Pick Disease Alliance (INPDA) and the
National Niemann-Pick Disease Foundation (NNPDF) provide essential
support for people living with Niemann-Pick disease type C (NPC)
and their families and advocate for needed treatments.
“So many members of the advocacy community
around the world work tirelessly to support people who live with
NPC and raise awareness of this devastating disease. At NNPDF, we
welcome the opportunities to collaborate with industry partners
like Zevra that are advancing promising research that can lead to
new treatments that drive change and hope for the NPC community,”
said Joslyn Crowe, Executive Director at NNPDF and Vice President
of INPDA. “Throughout the year and especially during October where
we observe Global Niemann-Pick Awareness Day and Niemann-Pick
Awareness Month, we join with leaders from industry and our
colleagues from the International Niemann-Pick Disease Alliance to
help others understand the challenges associated with the condition
and the future potential to provide needed relief to patients and
families.”
“NPC remains a condition with no approved
treatment options in the U.S.,” said Daniel Gallo, Ph.D., Senior
Vice President of Medical Affairs and Advocacy at Zevra. “It is an
ultra-rare, genetic, relentlessly progressive neurodegenerative
disease impacting approximately 1 in 100,000 live births. At Zevra
we are focused on working closely with the advocacy community for
the benefit of people living with NPC and their families.”
Zevra remains committed to working with INPDA,
NNPDF and the full NPC community to advance therapeutic options for
this rare, neurodegenerative disease and devastating condition.
Zevra also remains committed to patients receiving access to the
Company’s investigational treatment, arimoclomol, through the
ongoing Expanded Access Program (EAP), which is currently active in
the United States and other European countries.
About Niemann-Pick Disease Type C
(NPC):Niemann-Pick disease type C (NPC) is an ultra-rare,
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. 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 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.
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 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, and which can be identified by the use of words such as
"may," "will," "expect," "project," "estimate," "anticipate,"
"plan," "believe," "potential," "should," "continue," "could,"
"intend," "target," "predict," or the negative versions of those
words or other comparable words or expressions, although not all
forward-looking statements contain these identifying words or
expressions. Forward-looking statements are not guarantees of
future actions or performance. These forward-looking statements
include without limitation statements regarding senior leadership
and board member transitions and refreshment, or the timing
thereof, and our strategic and product development objectives, the
potential sale of the Priority Review Voucher, the promise and
potential impact of our preclinical or clinical trial data,
including without limitation the initiation, timing and results of
any clinical trials or readouts, the content, information used for,
timing or results of any IND applications and NDA submissions or
resubmissions for arimoclomol, KP1077, or any other product
candidates for any specific disease indication or at any dosage,
the potential achievement of commercial sales or revenue milestones
for AZSTARYS and the timing thereof, the sufficiency of our cash,
cash equivalents and investments to fund our operating activities
for any specific period of time, 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, 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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