Diversifies Seelos’ pipeline in rare diseases
Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage
biopharmaceutical company, announced today a definitive agreement
with Bioblast Pharma Ltd., (Nasdaq: ORPN), whereby Seelos acquired
all development and commercial rights to Bioblast’s proprietary
trehalose 90 mg/mL IV (Trehalose) solution as well as the existing
inventory of the drug which should be sufficient to fulfill its
current research needs. Trehalose, which is currently an
investigational molecule, has been studied in prior phase 2
clinical studies in over 70 patients with two rare diseases and
demonstrated a favorable safety profile and promising efficacy
signal.
Under the terms of the acquisition, Seelos assumed a
collaborative agreement with Team Sanfilippo Foundation (TSF), a
nonprofit medical research foundation founded by parents of
children with Sanfilippo syndrome. TSF, upon approval by the FDA,
plans to begin an open label, Phase 2(b) clinical trial in up to 20
patients with Sanfilippo syndrome and Seelos will provide the
clinical supply of Trehalose. The terms of the agreement entitle
Seelos access to all clinical data from this trial. Based on the
pre-clinical and in-vitro studies, there is a sound scientific
rationale for developing Trehalose for the treatment of Sanfilippo
syndrome.
“The acquisition of Trehalose, along with our collaboration with
TSF, are each important to understand in terms of their
significance,” said Raj Mehra, PhD, Chairman, Founder, and Chief
Executive Officer, Seelos Therapeutics. “Taken together, these are
validating achievements for Seelos’ collaborative approach and
speaks to the clinical and moral imperative to address the needs of
patients with Sanfilippo syndrome, the need to rapidly advance
novel therapeutics like Trehalose, and the need to continue to
evaluate Trehalose in a range of such rare and devastating diseases
including continuing the prior work in oculopharyngeal muscular
dystrophy and spinocerebellar ataxia type 3.”
“Our hope through this study is to evaluate the safety and
efficacy of Trehalose in all forms of Sanfilippo, which is a
devastating terminal disease that took the lives of 16 children
last year,” said Kathleen Buckley, President of the New York-based
TSF. “Our team has tirelessly worked to raise proceeds to fund this
study. If successful, we will work with the regulatory agencies in
the US and EU to get this drug approved and into the hands of our
families.”
Under the terms of the agreement, Seelos made an upfront payment
to Bioblast and will make certain additional milestone payments
upon the achievement of clinical and regulatory milestones, plus a
royalty upon clinical success, and approval for commercialization.
Full terms of this agreement are disclosed in the Current Report on
Form 8-K, filed by Seelos with the Securities and Exchange
Commission today.
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 90 mg/mL IV solution has demonstrated promising
clinical potential in prior phase 2 clinical development for
oculopharyngeal muscular dystrophy (OPMD) and spinocerebellar
ataxia type 3 (SCA3, also called Machado Joseph disease), with
encouraging safety and efficacy results thus far. These
pathological proteins aggregate within cells, eventually leading to
cell death. Prior preclinical studies indicate that this platform
has the potential to prevent mutant protein aggregation in other
devastating PolyA/PolyQ diseases.
Two U.S. patents for parental administration of trehalose exist
for patients with OPMD and SCA3; both are expected to expire in
2033. In addition, Orphan Drug Designation for OPMD and SCA3 has
been secured in the U.S. and in the EU.
About Seelos Therapeutics, Inc.
Seelos Therapeutics, Inc. 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. The Company’s portfolio includes several late-stage
clinical assets targeting psychiatric and movement disorders,
including orphan diseases. Seelos is based in New York, New York.
Seelos’ common stock is traded on The Nasdaq Capital Market under
the symbol "SEEL". For more information, please visit our website:
www.seelostherapeutics.com, the content of which is not
incorporated herein by reference.
About Bioblast Pharma Ltd.
Bioblast Pharma Ltd. (Nasdaq: ORPN) is a clinical-stage
biotechnology company committed to developing clinically meaningful
therapies for patients with rare and ultra-rare genetic diseases.
For more information, please visit: www.bioblastpharma.com, the
content of which is not incorporated herein by reference.
About Team Sanfilippo Foundation
Team Sanfilippo Foundation (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 providing 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. For more information, please visit teamsanfilippo.org.
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 Seelos’ future operations and its
ability to successfully initiate and complete clinical trials and
achieve regulatory milestones and related timing; the nature,
strategy and focus of Seelos’ business; the development and
commercial potential and potential benefits of any of Seelos’
product candidates including Trehalose; and that Seelos’ product
candidates have the potential to address critical unmet needs of
patients with serious diseases and conditions. Seelos may not
actually achieve the plans, carry out the intentions or meet the
expectations or projections disclosed in the forward-looking
statements and you should not place undue reliance on these
forward-looking statements. Because such statements deal with
future events and are based on Seelos’ current expectations, they
are subject to various risks and uncertainties and actual results,
performance or achievements of Seelos could differ materially from
those described in or implied by the statements in this press
release, including: the uncertainties associated with the clinical
development and regulatory approval of Seelos’ product candidates,
including potential delays in the commencement, enrollment and
completion of clinical trials; the potential that earlier clinical
trials and studies of Seelos’ product candidates may not be
predictive of future results; and the requirement for additional
capital to continue to advance these product candidates, which may
not be available on favorable terms or at all. The foregoing review
of important factors that could cause actual events to differ from
expectations should not be construed as exhaustive and should be
read in conjunction with statements that are included herein and
elsewhere, including the those risks discussed in Seelos’ filings
with the Securities and Exchange Commission. Except as otherwise
required by law, Seelos 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 information:
Anthony Marciano Head of Corporate Communications Seelos
Therapeutics, Inc. anthony.marciano@seelostx.com
www.seelostherapeutics.com
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