Eton Pharmaceuticals, Inc. (“Eton” or the “Company”) (Nasdaq:
ETON), an innovative pharmaceutical company focused on developing
and commercializing treatments for rare diseases announced the full
readout and compelling results from the clinical trial evaluating
PKU GOLIKE as a protein substitute for the treatment of
phenylketonuria (PKU) in patients during prolonged fasting periods.
The study demonstrated that PKU GOLIKE, administered as the last
daily dose and compared to standard amino acid protein substitutes,
improved metabolic control by reducing harmful phenylalanine (Phe)
levels and increasing beneficial tyrosine (Tyr) levels, both
essential for brain function and metabolic health.
PKU patients often experience significant
fluctuations in blood Phe levels during prolonged fasting periods,
particularly at night, when protein breakdown causes Phe
concentrations to peak in the early morning. These fluctuations are
associated with cognitive difficulties and overall health impacts,
making nighttime metabolic control an important focus in PKU
management.
The study was sponsored by Relief Therapeutics
Holding SA, and was a randomized, crossover, controlled clinical
study conducted by the Inherited Metabolic Disorders Unit at
Birmingham Children’s Hospital, UK, on pediatric patients with
classical PKU, the condition’s most severe form. The trial compared
PKU GOLIKE to standard amino acid protein substitutes in managing
metabolic parameters during overnight fasting, the longest fasting
period within 24 hours.
At the end of the one-week treatment period,
patients receiving PKU GOLIKE as the last daily protein substitute
dose showed a statistically significant reduction in blood Phe
levels compared to those receiving standard amino acid substitutes
(P=0.0002) and a statistically significant increase in blood Tyr
levels (P=0.0113). Compared to baseline levels measured prior to
the start of treatment, the PKU GOLIKE group achieved an average
17.8% reduction in blood Phe levels (P=0.0484) and an average 33.8%
increase in blood Tyr levels (P=0.0008) upon awakening after the
overnight fasting period. In comparison, when treated with standard
amino acid protein substitutes, the same patients experienced an
average 27.6% increase in blood Phe levels (P=0.0063) and no
significant improvement in blood Tyr levels. Blood sample analysis
at three early morning time points across the two groups revealed
no significant differences in peak Phe levels upon reawakening in
either group.
Highlighting the clinical significance of the
findings, Prof. Anita MacDonald, principal investigator and leading
dietitian in inherited metabolic disorders at Birmingham Children’s
Hospital, stated: “Giving one dose of PKU GOLIKE as the final daily
dose of protein substitute resulted in consistently better
metabolic control in our cohort of patients with PKU. They all had
classical PKU and were a particularly challenging group to
control.”
These results confirm that PKU GOLIKE’s
prolonged-release profile provides clinically and statistically
significant improvements in metabolic control during extended
fasting periods compared to standard amino acid protein
substitutes. Eton expects these findings to support the adoption of
PKU GOLIKE among healthcare providers and within the PKU
community.
The study findings will be presented in a poster
titled A Prolonged-Release Formula Has a Positive Impact on Morning
Phenylalanine and Tyrosine Fluctuations in Patients with Classical
Phenylketonuria at the 2025 American College of Medical Genetics
and Genomics (ACMG) Annual Clinical Genetics Meeting, March 18-22,
2025, in Los Angeles, California.
Eton promotes PKU GOLIKE with its existing
metabolic sales force, which also promotes Eton’s Carglumic Acid,
Betaine, and Nitisinone products. PKU patients’ care is typically
overseen by metabolic geneticists and their support staff of nurse
practitioners and registered dieticians. Medical formulas for PKU
are frequently covered by insurance and are regulated by the FDA as
medical food products. Patients and healthcare professionals
seeking additional information or requesting a product sample can
visit pkugolike.com.
For more information on this study
(NCT05487378), please visit clinicaltrials.gov.
About Eton PharmaceuticalsEton
is an innovative pharmaceutical company focused on developing and
commercializing treatments for rare diseases. The Company currently
has five commercial rare disease products: ALKINDI SPRINKLE®, PKU
GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The
Company has three additional product candidates in late-stage
development: ET-400, ET-600, and ZENEO® hydrocortisone
autoinjector. For more information, please visit our website at
etonpharma.com.
About Phenylketonuria
(PKU)Phenylketonuria (PKU) is caused by a defect of the
enzyme needed to break down phenylalanine (Phe), leading to a toxic
buildup of Phe from the consumption of foods containing protein or
aspartame. Untreated PKU can result in global developmental delay
or severe irreversible intellectual disability, as well as growth
failure, hypopigmentation, motor deficits, ataxia and seizures.
Living with PKU requires a limited diet and very careful
management. If left unmanaged, PKU can lead to devastating
consequences, such as brain damage. People living with PKU do not
have the ability to metabolize Phe, which is found in many foods,
and they require supplementation of amino acid-based
phenylalanine-free medical formulas as part of an effort to prevent
protein deficiency and optimize metabolic control. Medical formulas
used in PKU are challenged to provide a range of amino acids slowly
and without a medicinal aftertaste.
About PKU GOLIKE®PKU GOLIKE®
products are foods for special medical purposes (FSMPs) for the
dietary management of PKU in both children and adults for use under
medical supervision. Developed with Relief’s proprietary,
patent-protected Physiomimic Technology™ drug delivery platform,
PKU GOLIKE® products are the first prolonged-release amino acid
FSMPs, characterized by a special coating that ensures
physiological absorption of the amino acids mirroring that of
natural proteins. The special coating also masks the unpleasant
taste, odor, and aftertaste of the amino acids. PKU GOLIKE PLUS®
granules are flavorless and can be mixed with many foods. PKU
GOLIKE® products contain all 19 amino acids that people with PKU
need to maintain neurological and muscular health and PKU GOLIKE
PLUS® granules are fortified with 27 essential vitamins and
minerals, including ones normally found in protein-rich foods like
iron, calcium and vitamin B12. The PKU GOLIKE® line of products are
available in convenient packets (PKU GOLIKE PLUS® 3-16 and 16+) and
medical formula bars (PKU GOLIKE BAR®). PKU GOLIKE® products have
been commercially available in the U.S. since October 2022. For
more information, visit pkugolike.com. (Please note this site is
intended for U.S. audiences only).
Forward-Looking
StatementsStatements contained in this press release
regarding matters that are not historical facts are
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements
associated with the expected ability of Eton to undertake certain
activities and accomplish certain goals and objectives. These
statements include but are not limited to statements regarding
Eton’s business strategy, Eton’s plans to develop and commercialize
its product candidates, the safety and efficacy of Eton’s product
candidates, Eton’s plans and expected timing with respect to
regulatory filings and approvals, and the size and growth potential
of the markets for Eton’s product candidates. Because such
statements are subject to risks and uncertainties, actual results
may differ materially from those expressed or implied by such
forward-looking statements. Words such as “believes,”
“anticipates,” “plans,” “expects,” “intends,” “will,” “goal,”
“potential” and similar expressions are intended to identify
forward-looking statements. These forward-looking statements are
based upon Eton’s current expectations and involve assumptions that
may never materialize or may prove to be incorrect. Actual results
and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of
various risks and uncertainties, which include, without limitation,
risks associated with the process of discovering, developing and
commercializing drugs that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around
such drugs. These and other risks concerning Eton’s development
programs and financial position are described in additional detail
in Eton’s filings with the Securities and Exchange Commission. All
forward-looking statements contained in this press release speak
only as of the date on which they were made. Eton undertakes no
obligation to update such statements to reflect events that occur
or circumstances that exist after the date on which they were
made.
Investor Relations:Lisa M.
Wilson, In-Site Communications, Inc.T: 212-452-2793E:
lwilson@insitecony.com
Source: Eton Pharmaceuticals, Inc.
Eton Pharmaceuticals (NASDAQ:ETON)
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Eton Pharmaceuticals (NASDAQ:ETON)
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