Bioblast Pharma Announces Collaboration with Team Sanfilippo to Evaluate Trehalose for the Treatment of Sanfilippo Syndrome...
February 14 2019 - 7:05PM
Bioblast Pharma Ltd. (NasdaqCM: ORPN) (“Bioblast”), a
clinical-stage, orphan disease-focused biotechnology company, and
Team Sanfilippo (“TSF”), a nonprofit medical research foundation
founded by parents of children with Sanfilippo Syndrome today
announced a clinical collaboration to evaluate the safety and
efficacy of Bioblast’s proprietary Trehalose solution for the
treatment of patients with Sanfilippo syndrome.
Under the terms of the collaboration, TSF plans
to conduct an open label study in up to 20 Sanfilippo patients,
which will be followed for safety and efficacy. If successful,
based on the results of such study, Bioblast may determine to
continue the development of Trehalose towards a pivotal study for
this extra rare disease while maintaining full commercial rights.
TSF received FDA approval for this study was last week.
"We are excited to be working with TSF to
explore the potential of Trehalose in this rare and devastating
disease,” said Dalia Megiddo, M.D., interim Chief Executive
Officer of Bioblast. "Based on the known mechanism of action of
Trehalose on diseased lysosomes, several preclinical studies and
case reports, we have high expectations from this proof of concept
study. We have great respect and admiration for the brave parents
of Sanfilippo children on their fighting spirit. This program is
currently expected to remain in place following Bioblast’s
previously announced business combination with Enlivex Therapeutics
Ltd., which is expected to close in the first quarter of this year.
The current shareholders of Bioblast will have the opportunity to
benefit from the potential success of this program, as well as
potentially other programs for the Trehalose technology after the
transaction with Enlivex closes and pursuant to the previously
announced CVR agreement with Enlivex.
“We view Trehalose as a great promise to our
children, families and community," commented Kathleen Buckley,
President of the New York-based Team Sanfilippo. “We are eager to
start the study and plan to do so in the very near future. We have
been following the Trehalose potential and programs closely for
several years and feel that it could indeed make a difference in
the life of our loved ones.”
About Trehalose Trehalose is a
protein stabilizer that also activates autophagy and crosses the
blood-brain-barrier. Trehalose is a low molecular weight
disaccharide (.342 kDa) that protects against pathological
processes in cells. It has been shown to penetrate muscle and cross
the blood brain barrier. In animal models of several diseases
associated with abnormal cellular-protein aggregation, it has been
shown to reduce pathological aggregation of misfolded proteins as
well as to activate autophagy pathways through the activation of
Transcription Factor EB (“TFEB”), a key factor in lysosomal and
autophagy gene expression. Activation of TFEB is an emerging
therapeutic target for a number of diseases with pathologic
accumulation of storage material. Trehalose has been studied in
prior phase 2 clinical studies in over 70 patients with two rare
diseases and was found both safe and effective.
About Bioblast Pharma Bioblast
Pharma is a clinical-stage biotechnology company committed to
developing clinically meaningful therapies for patients with rare
and ultra-rare genetic diseases. Bioblast 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.
About Team Sanfilippo
TSF is a nonprofit medical research foundation
founded in 2008 by parents of children with Sanfilippo Syndrome.
TSF’s mission is to fund potential therapies that can be in
clinical trials in the near future. Team Sanfilippo is dedicated to
provide assistance to families to gain access to clinical trials,
treatments and compassionate use. Team Sanfilippo remains dedicated
to getting children of all ages access to clinical trials and
treatments and assistance to families enrolled in clinical
trials.
About Sanfilippo Syndrome:
Sanfilippo Syndrome, or mucopolysaccharidosis
III (MPS-III), is a rare autosomal recessive lysosomal storage
disease. It is caused by a deficiency in one of the enzymes needed
to break down the glycosaminoglycan (GAG) heparan sulfate (which is
found in the extra-cellular matrix and on cell surface
glycoproteins).
Affected children generally do not show any
signs or symptoms at birth. In early childhood, they begin to
develop developmental disability and loss of previously learned
skills. In later stages of the disorder, they may develop seizures
and movement disorders. Patients with Sanfilippo Syndrome usually
live into adolescence or early adulthood.
According to [source], the number of new
instances of Sanfilippo Syndrome is estimated at 1 in 70,000 live
births and overall prevalence estimates range from 1 to 9 in
1,000,000 people. The number of children suffering from this
syndrome is estimated at very few thousands world wide.
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. Forward-looking statements can be
identified by words such as “will,” “would,” “anticipate,”
“expect,” “believe,” “plan,” “hope,” “estimates,” “suggests,” “has
the potential to,” “projects”, “assumes”, “goal”, “targets”,
“likely”, “should” or “intend,” and other words and phrases of
similar meanings, the negative of these terms, and similar
references to anticipated or expected events, activities, trends,
future periods or results. Forward-looking statements are based on
management’s current expectations and are subject to risks and
uncertainties that are difficult to predict and, accordingly, our
actual results may differ materially from the results discussed or
implied in our forward-looking statements. For example,
Bioblast is using forward-looking statements when it discusses the
plan to initiate a clinical study to treat Sanfilippo Syndrome,
potential success thereof and next steps assuming such success, the
expected closing of the merger with Enlivex, the potential of the
CVR agreement with Enlivex and potential for benefit to the
Bioblast shareholders from the Trehalose program. Because such
statements deal with future events and are based on Bioblast’s
current expectations, they are subject to various risks and
uncertainties and actual results, performance or achievements of
Bioblast 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’s Annual Report on
Form 20-F filed with the Securities and Exchange Commission (“SEC”)
on April 24, 2018 and Exhibit 99.2 to Bioblast’s Report on Form 6-K
furnished to the SEC on December 6, 2018, and in any subsequent
filings with the SEC. Except as otherwise required by law, Bioblast
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.
Contacts:
Bioblast Pharma, Ltd.
Dr. Dalia Megiddo, CEO
dalia@bioblast-pharma.com
TSF:
Kathleen Buckley , President
kbuckley12866@gmail.com
Bioblast Pharma Ltd. (NASDAQ:ORPN)
Historical Stock Chart
From Jul 2024 to Aug 2024
Bioblast Pharma Ltd. (NASDAQ:ORPN)
Historical Stock Chart
From Aug 2023 to Aug 2024