Passage Bio Receives FDA Clearance of IND Application for PBML04 for Treatment of Metachromatic Leukodystrophy
June 08 2022 - 7:30AM
Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic
medicines company focused on developing transformative therapies
for central nervous system disorders, today announced that the U.S.
Food and Drug Administration (FDA) has cleared its Investigational
New Drug (IND) application for PBML04, an adeno-associated virus
(AAV)-delivery gene therapy that is being studied for the treatment
of Metachromatic Leukodystrophy (MLD). MLD is a rare, fatal,
pediatric, lysosomal storage disease that currently has limited
available treatment options.
“We are thrilled to have received IND clearance for our program
in metachromatic leukodystrophy, marking our fourth IND clearance
as a company and our third pediatric lysosomal storage disorder
program to reach clinical development,” said Edgar (Chip) Cale,
interim chief executive officer of Passage Bio. “Clearance of this
IND is a testament to the quality preclinical data supporting
PBML04 through our partnership with the University of
Pennsylvania’s Gene Therapy Program and the strong CMC and
analytics capabilities we have developed internally. MLD, GM1
gangliosidosis and Krabbe disease are similar diseases each caused
by a single gene mutation leading to deficiency of a critical
enzyme. Our approach and clinical development plans across these
three programs also share similarities, including use of our
next-generation, proprietary AAVhu68 capsid and ICM delivery. We
are excited by the promise of PBML04 to offer a potentially
transformative treatment for this devastating disease.”
MLD is a fatal inherited disease that is caused by mutations in
the arylsulfatase-A (ARSA) gene which reduces enzyme activity,
leading to progressive build-up of toxic sulfatides in the central
and peripheral nervous system. We are targeting infantile-onset
MLD, which is characterized by progressive muscle weakness,
rigidity, gait disorder, developmental delays and is typically
fatal by five years of age. The estimated worldwide incidence of
MLD is approximately 1 in 100,000 live births.
The Phase 1 clinical trial will utilize intra-cisterna magna
(ICM) administration to deliver an AAVhu68 capsid to express ARSA
and potentially address both central nervous system and peripheral
manifestations of this devastating disease.
About Passage BioPassage Bio (Nasdaq: PASG) is
a clinical-stage genetic medicines company on a mission to provide
life-transforming therapies for patients with CNS diseases with
limited or no approved treatment options. Our portfolio spans
pediatric and adult CNS indications, and we are currently advancing
three clinical programs in GM1 gangliosidosis, Krabbe disease and
frontotemporal dementia with several additional programs in
preclinical development. Based in Philadelphia, PA, our company has
established a strategic collaboration and licensing agreement with
the renowned University of Pennsylvania’s Gene Therapy Program to
conduct our discovery and IND-enabling preclinical work. Through
this collaboration, we have enhanced access to a broad portfolio of
gene therapy candidates and future gene therapy innovations that we
then pair with our deep clinical, regulatory, manufacturing and
commercial expertise to rapidly advance our robust pipeline of
optimized gene therapies. As we work with speed and tenacity, we
are always mindful of patients who may be able to benefit from our
therapies. More information is available at www.passagebio.com.
Forward-Looking StatementsThis press release
contains “forward-looking statements” within the meaning of, and
made pursuant to the safe harbor provisions of, the Private
Securities Litigation Reform Act of 1995, including, but not
limited to: our expectations about timing and execution of
anticipated milestones, including initiation of clinical trials and
the availability of clinical data from such trials; our
expectations about our collaborators’ and partners’ ability to
execute key initiatives; our expectations about manufacturing plans
and strategies; our expectations about cash runway; and the ability
of our lead product candidates to treat their respective target
monogenic CNS disorders. These forward-looking statements may be
accompanied by such words as “aim,” “anticipate,” “believe,”
“could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,”
“might,” “plan,” “potential,” “possible,” “will,” “would,” and
other words and terms of similar meaning. These statements involve
risks and uncertainties that could cause actual results to differ
materially from those reflected in such statements, including: our
ability to develop and obtain regulatory approval for our product
candidates; the timing and results of preclinical studies and
clinical trials; risks associated with clinical trials, including
our ability to adequately manage clinical activities, unexpected
concerns that may arise from additional data or analysis obtained
during clinical trials, regulatory authorities may require
additional information or further studies, or may fail to approve
or may delay approval of our drug candidates; the occurrence of
adverse safety events; the risk that positive results in a
preclinical study or clinical trial may not be replicated in
subsequent trials or success in early stage clinical trials may not
be predictive of results in later stage clinical trials; failure to
protect and enforce our intellectual property, and other
proprietary rights; our dependence on collaborators and other third
parties for the development and manufacture of product candidates
and other aspects of our business, which are outside of our full
control; risks associated with current and potential delays, work
stoppages, or supply chain disruptions caused by the coronavirus
pandemic; and the other risks and uncertainties that are described
in the Risk Factors section in documents the company files from
time to time with the Securities and Exchange Commission (SEC), and
other reports as filed with the SEC. Passage Bio undertakes no
obligation to publicly update any forward-looking statement,
whether written or oral, that may be made from time to time,
whether as a result of new information, future developments or
otherwise.
For further information, please contact:
Passage Bio Investors:Stuart HendersonPassage
Bio267.866.0114shenderson@passagebio.com
Passage Bio Media:Mike BeyerSam Brown Inc.
Healthcare Communications312-961-2502MikeBeyer@sambrown.com
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