Bioblast Pharma Announces Sale of its Trehalose Clinical Development Programs to Seelos Therapeutics
February 19 2019 - 7:45AM
Bioblast Pharma Ltd. ( Nasdaq: ORPN), a clinical-stage,
orphan disease-focused biotechnology company, today announced the
sale of its Trehalose clinical development programs (including its
advanced phase 2 clinical program of Trehalose to treat
Oculoharyngeal Muscular Dystrophy (OPMD) to Seelos Therapeutics,
Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company.
Under the terms of the agreement, Seelos will pay Bioblast a
payment of $3.5 million in two instalments; $1.5 million payable
upon closing of the sale and the other $2.0 million will be paid
upon the first anniversary of the closing of the sale.
Potential additional milestone payments of up to $17.0 million will
be made upon completion of the clinical study and approval of a New
Drug Application (NDA) by the Food and Drug Administration (FDA),
as well as royalties.
Bioblast announced on February 14, 2019, that it has signed a
collaboration agreement with Team Sanfilippo (TSF) for the
development of Trehalose for Sanfilippo Syndrome. Under the terms
of the collaboration, TSF plans to conduct an open label study in
up to 20 Sanfilippo Syndrome patients, which will be followed for
safety and efficacy. If successful, based on the results of such
study, Seelos, as successor to Bioblast, may determine to continue
the development of Trehalose towards a pivotal study for this extra
rare disease while maintaining full commercial rights. TSF recently
received FDA approval for this study.
All Trehalose programs are currently expected to be further
developed by Seelos following Bioblast’s previously announced
business combination with Enlivex Therapeutics Ltd., which is
expected to close in the first quarter of 2019. The current
shareholders of Bioblast will have the opportunity to benefit from
the potential success of these programs, as well as potentially
other programs for the Trehalose technology after the transaction
with Enlivex closes and pursuant to the previously announced
Contingent Value Rights (CVR) agreement with Enlivex.
“We have great faith in the efficacy of Trehalose for the
treatment of several rare diseases” Dalia Megiddo MD, Interim CEO
of Bioblast commented: “We are confident that Seelos will continue
to develop the clinical programs skilfully for the benefit of the
patients as well as for Bioblast’s and Seelos’ shareholders.”
“We view Trehalose as a promising therapy for many
patients suffering from rare diseases caused by accumulation of
proteins and other large molecules in their cells. ,"
commented Raj Mehra. Chief Executive Officer of Seelos “We
look forward to further developing this therapy for the benefit
of patients suffering from rare diseases.”
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 Ltd. 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 Seelos Therapeutics, Inc.
Seelos Therapeutics, Inc. ( Nasdaq: SEEL) is a clinical-stage
biopharmaceutical company focused on the development and
advancement of novel therapeutics to address unmet medical needs
for the benefit of patients with central nervous system (CNS)
disorders and in rare diseases. Seelos’s robust portfolio includes
several late-stage clinical assets targeting psychiatric and
movement disorders, including orphan diseases. Seelos is based in
New York, New York. For more information, please visit Seelos
website: www.seelostherapeutics.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, 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 and its successful sale to Seelos, as well as
receipt of unconditional and contingent consideration. 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.
Contact:
Bioblast Pharma Dalia Megiddo MD, CEO
dalia@bioblast-pharma.com
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