Bioblast’s Special Medical Advisor to Make a Presentation Today On Trehalose as a Potential Therapeutic Agent for OPMD
September 26 2016 - 8:00AM
Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan
disease-focused biotechnology company, today announced that Zohar
Argov, MD, Special Medical Advisor to the Executive Chairman of
Bioblast Pharma, will present an invited lecture on “Trehalose in
Oculopharyngeal Muscular Dystrophy (OPMD)” at the annual scientific
meeting of the Muscle Study Group Society (MSGS) to be held today
in Snowbird, Utah. The MSGS is an interdisciplinary US-based group
devoted to the development of treatments for neuromuscular
disorders.
The lecture will summarize the general approach that Bioblast is
taking in the use of trehalose as a potential therapeutic agent for
OPMD. The topics to be discussed include:
- OPMD: with emphasis on its pathogenesis involving accumulation
of an abnormal PABN1 protein. The main features of the disease will
be presented, including the progressive swallowing difficulties
(dysphagia) and pattern of muscle weakness.
- Trehalose: its newly understood mechanism of action that
includes activation (enhancing) of autophagy and lysosomal
activity. The basic common mechanism is presumed to be through
cellular ‘glucose starvation’ which leads to the activation of
these cellular pathways.
- Results of the phase 2 trial of trehalose in OPMD including an
extension study: The main results include improvement in swallowing
functions as measured by drinking times of fluids as well as by
swallowing quality of life measurements. Trends of positive
response to trehalose in various muscle functions will be presented
as well.
- Pharmacokinetics: trehalose in normal animals and humans as
well as in OPMD patients. Bioblast’s preclinical and clinical work
emphasizes that the mode of administration of trehalose is critical
to achieving therapeutic serum concentrations. Due to the active
metabolism in the gut, trehalose must be administered intravenously
(IV). In rats, IV administration after a single dose results in
high plasma levels of trehalose for 8 hours, while muscle tissue
levels remain high for more than 48 hours. In normal human
subjects, trehalose has linear kinetics with exposure increasing
proportionally with increasing dose. In OPMD patients in a Phase 2
trial, trehalose levels after a single 27 gram IV dose reach
equivalent plasma levels to those in the animal model, with a
half-life of 1.8 hours.
- Safety: in an OPMD phase 2 clinical trial in which patients
received once weekly IV dosing of 27 grams for up to 52 weeks,
trehalose was generally safe and well tolerated. There were no
drug-related serious adverse events and no changes in safety labs
including ECG. Trehalose is a disaccharide that is metabolized to
glucose; however, there was no clinically meaningful change in
plasma glucose levels during or following the infusion of
trehalose.
- Preclinical: the animal basis for the action of trehalose in a
mouse model of OPMD.
About Professor Argov
Professor Zohar Argov, MD, is world renowned for his studies and
discoveries in myology and neurological diseases. He is Professor
(Emeritus) of Neurology & Josephine Frank Kanrich Chair of
Neuromuscular Diseases at Hebrew University Hadassah Medical
School, Jerusalem, Israel. Professor Argov is also an Adjunct
Professor at the Department of Neurology/Neurosurgery, Montreal
Neurological Institute, McGill University, Montreal, Canada. He was
President of the European Neurological Society and Chairman of the
European Neurological Society Muscle & Neuromuscular Disorders
Subcommittee. Prof. Argov currently serves on the executive
committee of the World Muscle Society.
Professor Argov, a board-certified neurologist, earned his
medical degree from Hebrew University Hadassah Medical School in
Jerusalem, Israel.
About Trehalose 90mg/mL IV Solution
Trehalose 90mg/mL IV solution is a chemical chaperone that
protects against pathological processes in cells. It has been
shown to reduce pathological aggregation of proteins within cells
in several diseases associated with abnormal cellular-protein
aggregation as well as acting as an autophagy enhancer.
Trehalose 90mg/mL IV solution has been documented as demonstrating
significant promise in preclinical animal models of OPMD and other
PolyA/PolyQ diseases.
In OPMD, trehalose 90mg/mL IV solution is being developed to
prevent the aggregation of the pathological protein (PABPN1) in
muscle cells, the hallmark of the disease, by stabilizing the
protein, reducing the formation of protein aggregations, and
promoting their clearance from cells through autophagy, thus
preventing muscle cell death.
About Oculopharyngeal Muscular Dystrophy
(OPMD)
OPMD is an inherited myopathy characterized by dysphagia
(difficulty in swallowing), eyelid drooping (ptosis), the loss of
muscle strength, and weakness in multiple muscles of the
limbs. Symptoms generally appear in mid-life and get worse
over time. As the dysphagia becomes more severe, patients
become malnourished, lose significant weight, and may suffer from
repeated incidents of aspiration pneumonia. Aspiration pneumonia
and severe emaciation may result in death. The disease is caused by
a genetic mutation responsible for the creation of a mutant protein
(PABPN1) with an expanded polyalanine domain that aggregates within
patient muscle cells. There is currently no approved
pharmacologic treatment for OPMD.
About the Muscle Study Group Society
The Muscle Study Group Society (MSGS), is a consortium of
scientific investigators from academic and research centers who are
committed to the cooperative planning, implementation, analysis and
reporting of controlled clinical trials and of other research for
muscle and other neuromuscular diseases. The MSGS aims to advance
knowledge about the cause(s), pathogenesis, epidemiology, and
clinical manifestations of muscle disease and related neuromuscular
disorders and to develop and implement strategies to examine
promising therapeutic interventions.
About Bioblast Pharma Ltd.
Bioblast Pharma is a clinical-stage biotechnology company
committed to developing clinically meaningful therapies for
patients with rare and ultra-rare genetic diseases, with a lead
drug candidate -- trehalose 90mg/mL solution -- in Phase 2
development. Bioblast was founded in 2012 and is traded on
the NASDAQ under the symbol “ORPN”. For more information, please
visit our website, www.bioblastpharma.com, the content of which is
not incorporated herein by reference.
Forward Looking Statements
This press release contains forward-looking statements within
the meaning of the "safe harbor" provisions of the Private
Securities Litigation Reform Act of 1995 and other Federal
securities laws. Historic results of scientific research and
clinical and preclinical trials do not guarantee that the
conclusions of future research or trials would not suggest
different conclusions or those historic results referred to in this
press release would not be interpreted differently in light of
additional research and clinical and preclinical trial results.
Because such statements deal with future events and are based on
Bioblast Pharma Ltd.'s current expectations, they are subject to
various risks and uncertainties and actual results, performance or
achievements of Bioblast Pharma could differ materially from those
described in or implied by the statements in this press release,
including those discussed under the heading "Risk Factors" in
Bioblast Pharma's Annual Report on Form 20-F filed with the
Securities and Exchange Commission ("SEC") on March 29, 2016, and
in any subsequent filings with the SEC. Except as otherwise
required by law, Bioblast Pharma disclaims any intention or
obligation to update or revise any forward-looking statements,
which speak only as of the date hereof, whether as a result of new
information, future events or circumstances or otherwise.
INVESTOR CONTACT:
Matthew P. Duffy
Managing Director
LifeSci Advisors, LLC
Telephone: 212-915-0685
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