Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic
medicines company focused on developing transformative therapies
for central nervous system (CNS) disorders, announced that the
first patient with early infantile Krabbe disease has received
PBKR03, an adeno-associated virus (AAV)-delivery gene therapy, in
its global Phase 1/2 clinical trial, GALax-C.
“It is gratifying to dose the first patient in our GALax-C
trial,” said Bruce Goldsmith, Ph.D., president and chief executive
officer of Passage Bio. “We now have two global clinical trials
with patients dosed under way. The advancement of these two
clinical trials represents significant progress for our company in
our mission to develop transformative medicines for people with
devastating CNS disorders. We look forward to reporting initial
safety and biomarker data from GALax-C by end of 2022.”
Krabbe disease is a rare pediatric lysosomal storage disorder
caused by mutations in the GALC gene, which encodes
galactosylceramidase, an enzyme that breaks down galactosylceramide
and psychosine. Without adequate levels of galactosylceramidase,
psychosine accumulates, causing widespread death of
myelin-producing cells and progressive damage to nerves in both the
brain and peripheral tissues of affected children. This is
characterized in children by loss of acquired milestones, staring
episodes, apnea, peripheral neuropathy, severe weakness,
unresponsiveness to stimuli, seizures, blindness, and deafness.
Life expectancy for early infantile Krabbe disease, the most severe
form, is two years.
Eliseo Salinas, M.D., MSc., chief research and development
officer at Passage Bio, noted: “For GALax-C, we are using a
proprietary AAVhu68 capsid, the same vector that we are using in
our GM1 gangliosidosis clinical trial. We were pleased to recently
report interim clinical data from the first two patients in the GM1
trial, showing meaningful clinical improvements and a positive
safety profile for the therapy. We are hopeful about the benefits
PBKR03 may have for patients with Krabbe disease. We also are
grateful to the children, families, and clinical trial
investigators who have chosen to participate in our studies.”
Roberto Giugliani, M.D., Ph.D., Department of Genetics UFRGS and
Medical Genetics Service HCPA, Porto Alegre, Brazil, who is an
expert in lysosomal storage disorders and a clinical trial
investigator for Passage Bio’s Imagine-1 and GALax-C clinical
studies, said: “It is encouraging to see this clinical trial
program advancing, especially considering the urgent medical need
for this patient population. Currently there are no approved
disease-modifying treatment options for children with Krabbe
disease, which has devastating impact on patients as well as their
families.”
The U.S. Food and Drug Administration (FDA) has granted PBKR03
Fast Track, Orphan Drug, and Rare Pediatric Disease designations.
PBKR03 has also received an Orphan designation from the European
Commission.
About GALax-C (NCT04771416)
GALax-C is a Phase 1/2 global, open-label, dose-escalation study
of PBKR03 administered by single injection into the cisterna magna
in pediatric subjects with early infantile Krabbe disease who are
>1 to 9 months of age at enrollment.
The GALax-C study will investigate low and high dose PBKR03 in
two age groups, beginning with a low dose cohort of children
aged 4 to 9 months. Enrollment is currently ongoing for Cohort 1 at
sites globally. The primary endpoint of the study is to evaluate
the safety and tolerability of PBKR03. Secondary endpoints include
disease biomarkers, and clinical outcome measures. GALax-C is a
two-year study with a three-year safety extension.
Passage Bio is undertaking a number of initiatives to identify
eligible patients, such as screening efforts to support early and
accurate identification of children with Krabbe disease, as well as
no-charge genetic testing for patients suspected of having Krabbe
disease through collaboration with Invitae.
More information about GALax-C can be found
at https://clinicaltrials.gov/ct2/show/NCT04771416
About PBKR03
PBKR03 utilizes a next-generation proprietary AAV capsid to
deliver, through intra-cisterna magna (ICM) administration, a
functional GALC gene to patients with mutations in the gene that
codes for galactosylceramidase (GAL-C). The gene therapy has the
potential to treat both the central nervous system and peripheral
nerve manifestations observed in patients with Krabbe disease.
PBKR03 is supported by extensive preclinical studies conducted
by Passage Bio’s collaborator, the University of Pennsylvania’s
Gene Therapy Program. The studies showed meaningful transduction of
both the central and peripheral nervous system in animal models,
with restoration of myelination in the brain and peripheral nerves.
In a naturally occurring Krabbe animal model, a single ICM
injection of an AAVhu68 capsid containing the normal canine GALC
gene showed normalization of GALC activity, reduction of cerebral
spinal fluid psychosine levels, normalization of peripheral nerve
conduction velocity, improvement in brain myelination, reduction in
brain inflammation and increased survival.
About Passage Bio
At Passage Bio (Nasdaq: PASG), we are on a mission to provide
life-transforming genetic medicines for patients with CNS diseases
that replace their suffering with boundless possibility, all while
building lasting relationships with the communities we serve. 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. This provides our team with 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 into
clinical testing. 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 Statements
This 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 subsequent events in our GALax-C
study; 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:Gwen FisherPassage
Bio215-407-1548gfisher@passagebio.com
Passage Bio (NASDAQ:PASG)
Historical Stock Chart
From Jul 2024 to Aug 2024
Passage Bio (NASDAQ:PASG)
Historical Stock Chart
From Aug 2023 to Aug 2024