Bioblast Pharma Reports Results of a Clinical Trial of Trehalose in Oculopharyngeal Muscular Dystrophy (OPMD) Patients
September 12 2016 - 5:00AM
Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan
disease-focused biotechnology company, today announced results of
the Extension study for patients who completed HOPEMD, the
Company’s initial clinical trial for patients with Oculopharyngeal
Muscular Dystrophy (OPMD). This Extension study was a multi-center,
randomized, open-label study with a non-treatment concurrent
control arm that enrolled 22 patients, 16 of whom continued to
receive trehalose 90mg/mL IV solution while 6 were in the
non-treatment group.
OPMD is a rare progressive muscle-wasting
disease characterized by severe swallowing difficulties (dysphagia)
leading to malnutrition, aspiration of food into the lungs and
recurrent episodes of aspiration pneumonia, as well as more
generalized, progressive muscle weakness.
Efficacy: Change in Drinking Time from
Baseline
Patients enrolled in the Extension study were
evaluated by means of a timed cold water drinking test, a commonly
used dysphagia measurement used for decades to help diagnose and
assess the severity of OPMD. At the end of the 12 month period:
- For the patients who continued on trehalose treatment (the blue
line in the following chart), the cold water drinking test time
remained relatively stable across the treatment period
- For the patients who were not on treatment (the red line in the
following chart), the cold water drinking test time worsened
A chart accompanying this release is available
at http://www.globenewswire.com/NewsRoom/AttachmentNg/0ef29d0a-abb3-440b-8909-140f629422a0
Twenty-two patients were enrolled and 21
contributed data through the first seven months of the Extension
trial, 16 on trehalose and 5 not on drug treatment; at the end of
the study, 10 patients remained on trehalose while 3 were not on
drug treatment. The average time for those in the treatment arm was
46.9 weeks on trehalose.
Bernard Brais, MD, PhD, Professor of Neurology,
McGill University, co-director of the neuromuscular group of the
Montreal Neurological Institute and Hospital and world-renowned
expert in OPMD who was a principal investigator on both the HOPEMD
and Extension studies stated, “This data provides preliminary
evidence that when trehalose is stopped, patients return to a level
of symptoms they had prior to taking the drug. This suggests that
trehalose influences at least the major symptom of dysphagia in
patients, further supporting that it may influence the evolution of
this late onset muscular dystrophy.”
Warren Wasiewski, MD, Bioblast’s Chief Medical
Officer said, “We recognize that these results are in a small
number of patients and that while there were treatment and
non-treatment arms, this was not a double-blind, placebo-controlled
study. Nevertheless, we are encouraged by the fact that the
improvement found first in the HOPEMD study is sustainable over an
extended period of time and that the difference between patients
who continued on treatment and those who were randomized to be
withdrawn from treatment in this extension study has provided us
with valuable information for our upcoming Phase 2b double-blind,
placebo-controlled trial, which is planned to enroll more than 70
patients with OPMD.”
Based on the results in the Extension study (as
well as from the earlier HOPEMD study), the primary efficacy
measures in the Phase 2b trial will be the cold water drinking test
and dysphagia related quality of life questionnaires (SWAL-QOL and
Sydney Swallowing Questionnaire). Other tests, including those
related to muscle function and power and weight, will be evaluated
as secondary endpoints.
More information from the Extension
trial
There were three main objectives of this
study:
- To determine the long-term effect of trehalose on disease
progression as assessed by the changes in the disease markers as
well as in the patients’ swallowing quality of life.
- To assess which efficacy measures were appropriate for
consideration as endpoints in the upcoming Phase 2b trial. The
efficacy measures included change from baseline in cold water
drinking test time, video fluoroscopy, the total score and total
symptom score on the patient reported swallowing quality of life
questionnaire (SWAL-QOL), functional muscle testing, muscle
strength testing by handheld dynamometer, histologic changes on
percutaneous muscle biopsy, and weight.
- To assess safety parameters in the upcoming Phase 2b trial,
including the frequency, severity, and duration of adverse events
(AEs), as well as clinically significant laboratory abnormalities
after administration of trehalose. The results from the Extension
study indicated that:
- Trehalose was generally safe and well tolerated.
- There were no clinically significant changes in safety
labs.
- There was one serious adverse event (SAE) unrelated to drug
treatment.
- There were no infusion reactions or adverse events leading to
discontinuation.
Treatment emergent adverse events (TEAE)
occurred in 15 of the 16 treatment arm patients (93.8%). There was
one SAE, a kidney stone, that was considered unrelated to
trehalose. There were two possibly related TEAEs; urinary tract
infection and sclerodactylia. Most AEs were mild to moderate in
intensity. There were no clinically significant changes in any
safety lab parameters, which included assessments of chemistry,
hematology, coagulation, liver enzymes, and renal function tests.
There was no change in physical examination or electrocardiogram.
There were no infusion reactions and no discontinuations due to
adverse events.
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. For example, we are using forward looking
statements when we discuss our plans to conduct a Phase 2b clinical
study, and the design and timing of such study. In addition,
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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