Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the
Company), a rare disease therapeutics company, today
announced the presentation of five posters at the Society for
the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual
Symposium. Four posters focused on data from multiple studies
showing the efficacy and safety of arimoclomol as a treatment for
people living with Niemann-Pick disease type C (NPC), and one
poster highlighted data from pharmacokinetic modeling studies of
OLPRUVA®, a therapy for the long-term management of certain adult
and pediatric patients with urea cycle disorders (UCDs) involving
deficiencies of carbamylphosphate synthetase (CPS), ornithine
transcarbamylase (OTC) or argininosuccinic acid synthetase (AS).
“The data collected during the Phase 2/3 study
of arimoclomol, including long-term data from Open Label Extension
(OLE) and Early Access Program (EAP) participants, add to the large
body of evidence that demonstrates arimoclomol’s clinical efficacy
and safety as a treatment for people living with NPC,” said Adrian
Quartel, M.D., FFPM, Chief Medical Officer of Zevra. “Additionally,
we presented PK modeling data from OLPRUVA in both adult and
pediatric virtual patients, showing an increase in drug exposure
under fasting conditions.”
Presentation Details
The data for arimoclomol presented at SSIEM is
summarized below:
Poster Number: 21260 |
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Title: |
Efficacy Results from a 12-month Double-blind Randomized Trial of
Arimoclomol for Treatment of Niemann Pick Disease Type C –
presenting an improved 4-Domain NPC Clinical Severity Scale |
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Authors: |
Marc Patterson, Sven Guenther and Christine i Dali |
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Summary: |
The treatment effect of arimoclomol was evaluated in a 12-month,
double-blind, placebo-controlled clinical trial (NCT02612129) using
the original 5-domain Niemann pick type C clinical severity scale
(5DNPCCSS) and the modified 4-domain Niemann Pick type C clinical
severity scale (4DNPCCSS). A statistically significant treatment
effect was shown using the modified 4DNPCCSS and the prespecified
5DNPCCSS primary endpoint in the 12-month clinical trial,
representing a clinically meaningful reduction in disease
progression with arimoclomol treatment compared to placebo. |
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Poster Number: 21271 |
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Title: |
Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment
in Patients with Niemann Pick Type C – Data from 48 Months Open
Label Trial |
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Authors: |
Marc Patterson, Eugene Mengel, Sven Guenther and Christine i
Dali |
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Summary: |
The long-term safety and efficacy of arimoclomol in the 12-month
double-blind, and 48-month open-label extension (OLE) portion of
the clinical trial (NCT02612129) were presented using the 4-Domain
Niemann Pick Type C Clinical Severity scale (4DNPCCSS) which
evaluates ambulation, speech, swallowing and fine motor skills. For
those patients transitioning from placebo to arimoclomol at the
start of the open-label extension period, the mean annual rate of
disease progression reduced from an annual rate of change of 1.9
points during the double-blind phase, to a rate of 0.3 in the first
12 months of treatment, remained numerically smaller for the rest
of the trial, and was comparable between the double-blind phase of
the trial and the open-label extension phase of the trial.
Additionally, arimoclomol was well tolerated with no new safety
signals observed. |
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Poster Number: PO-212 |
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Title: |
Arimoclomol for the Treatment of NPC in a Real-World Setting:
Long-term Outcomes from an Expanded Access Program in the USA |
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Authors: |
Walla Al-Hertani, Elizabeth M. Berry-Kravis, Raymond Wang, Marc
Patterson, Can Ficicioglu, Loren Pena, Kristina Julich, Damara
Ortiz, Paula Schleifer, Caroline Hastings, Paul Hillman, Ronan
O'Reilly, Christine Dali and Daniel Gallo |
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Summary: |
The long-term safety and efficacy of arimoclomol in a real-world
setting were evaluated in a total of 56 adult and pediatric
patients in the U.S. arimoclomol expanded access program (EAP)
trial (NCT04316637). Data presented included over 3 years of U.S.
EAP clinical outcomes and demonstrated that adult and pediatric
patients treated with arimoclomol, including those with and without
miglustat as a component of their routine clinical care,
experienced relatively stable disease as measured by the 5DNPCCSS
and 4DNPCCSS and show that arimoclomol was well tolerated. |
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Poster Number: 20950 |
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Title: |
Arimoclomol safety profile in the treatment of NPC in a Real-World
setting: Long-term data from an Expanded Access program in the
USA |
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Authors: |
Can Ficicioglu, Elizabeth M. Berry-Kravis, Walla Al-Hertani,
Raymond Wang, Marc Patterson, Loren Pena, Kristina Julich, Damara
Ortiz, Paula Schleifer, Paul Hillman, Caroline Hastings, Ronan
O'Reilly, Christine Dali and Daniel Gallo |
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Summary: |
Safety data from 94 adult and pediatric participants in the
arimoclomol US EAP were presented. Safety outcomes reported from
exposure periods spanning as much as 46 months of treatment,
demonstrated a safety profile consistent with the published
clinical trial experience of arimoclomol in NPC. |
The data for OLPRUVA presented at SSIEM is
summarized below:
Poster Number: PO-609 |
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Title: |
Modeling the Pharmacokinetics of Phenylbutyrate in Fed and Fasted
States |
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Authors: |
Steiner, Rebecca Baillie, Tongli Zhang, Christina Friedrich,
Meredith Hart, Mike Reed |
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Summary: |
Pharmacokinetic modeling evaluating whether sodium phenylbutyrate
(NaPBA) could be safely and effectively administered while fasting
showed greater absorption and bioavailability with increased drug
exposure in fasted administration of NaPBA compared to fed
administration of NaPBA and glycerol phenylbutyrate (GPB) in both
adult and child virtual patients, which is predicted to increase
efficacy in proportion to increased drug exposure, theoretically
allowing for a 30% dose decrease compared to fed conditions. |
About the SSIEM 2024 Annual
Symposium
The Society for the Study of Inborn Errors of
Metabolism (SSIEM) 2024 Annual Symposium took place September 3-6,
2024, in Porto, Portugal. The annual symposium is intended to
foster the study of inherited metabolic disorders and promote the
exchange of ideas between professionals in different disciplines
who are researching inborn errors of metabolism (IEM).
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,
investigational drug 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 action date (PDUFA date) of September 21,
2024.
About Urea Cycle Disorders
UCDs are a group of rare, genetic disorders that
can cause harmful ammonia to build up in the blood, potentially
resulting in brain damage and neurocognitive impairments if ammonia
levels are not controlled. Any increase in ammonia over time is
serious. Therefore, it is important to adhere to any dietary
protein restrictions and have alternative medication options to
help control ammonia levels.
About OLPRUVA®
OLPRUVA (sodium phenylbutyrate) was approved for
the treatment of certain UCDs in December 2022 and has recently
been marketed under the brand name, OLPRUVA®. OLPRUVA (sodium
phenylbutyrate) for oral suspension is a prescription medicine used
along with certain therapies, including changes in diet, for the
long-term management of adults and children weighing 44 pounds (20
kg) or greater and with a body surface area (BSA) of 1.2 m2 or
greater, with UCDs, involving deficiencies of carbamylphosphate
synthetase (CPS), ornithine transcarbamylase (OTC), or
argininosuccinic acid synthetase (AS). OLPRUVA is not used to treat
rapid increase of ammonia in the blood (acute hyperammonemia),
which can be life-threatening and requires emergency medical
treatment. For more information, please visit www.OLPRUVA.com.
Important Safety
Information
Certain medicines may increase the level of
ammonia in your blood or cause serious side effects when taken
during treatment with OLPRUVA. Tell your doctor about all the
medicines you or your child take, especially if you or your child
take corticosteroids, valproic acid, haloperidol, and/or
probenecid.
OLPRUVA can cause serious side effects,
including: 1) nervous system problems (neurotoxicity). Symptoms
include sleepiness, tiredness, lightheadedness, vomiting, nausea,
headache, confusion, 2) low potassium levels in your blood
(hypokalemia) and 3) conditions related to swelling (edema).
OLPRUVA contains salt (sodium), which can cause swelling from salt
and water retention. Tell your doctor right away if you or your
child get any of these symptoms. Your doctor may do certain blood
tests to check for side effects during treatment with OLPRUVA. If
you have certain medical conditions such as heart, liver or kidney
problems, are pregnant/planning to get pregnant or breast-feeding,
your doctor will decide if OLPRUVA is right for you.
The most common side effects of OLPRUVA include
absent or irregular menstrual periods, decreased appetite, body
odor, bad taste or avoiding foods you ate prior to getting sick
(taste aversion). These are not all of the possible side effects of
OLPRUVA. Call your doctor for medical advice about side effects.
You may report side effects to FDA at 1-800-FDA-1088.
About Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc. 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 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
promise and zpotential 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 benefits of any of our products or product
candidates for any specific disease or at any dosage; our strategic
and product development objectives, including with respect to
becoming a leading, commercially focused rare disease company;
potential revenues from our arimoclomol expanded access program;
the potential for royalty and milestone contributions, the
presentation of data at conferences; and the timing of any of the
foregoing. 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, Zevra’s Quarterly Report on Form 10-Q for the three months
ended June 30, 2024, 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
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