Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic
medicines company focused on developing transformative therapies
for central nervous system (CNS) disorders, today announced the
presentation of longer-term clinical and biomarker data from Cohort
1 in Imagine-1, a Phase 1/2 study of PBGM01, a gene therapy for GM1
gangliosidosis (GM1). David Weinstein, M.D. M.M.Sc., senior vice
president, Clinical Development, Passage Bio, will present the data
at 8:30 a.m. ET during a late-breaker oral presentation at the
American Society of Gene and Cell Therapy (ASGCT) 25th Annual
Meeting, being held in Washington, D.C. and virtually.
“We continue to be encouraged by the interim data from the first
cohort in our Imagine-1 trial and the potential promise it offers
for patients. These data show that the low dose of PBGM01 is
well-tolerated, has a favorable safety profile and exerts a
biological effect that appears to translate to improvement in
developmental milestones for these children,” said Mark Forman,
M.D., Ph.D., chief medical officer, Passage Bio. “We look forward
to continuing to advance the Imagine-1 study through additional
cohorts and are excited to gather more data to see the impact of
earlier intervention, which we believe may be important based on
the initial clinical findings presented to date. The life
expectancy for children with GM1 ranges from two to 10 years, so we
are eager to pursue the opportunity to potentially offer the first
disease-modifying treatment for this disease as quickly as
possible.”
Key Highlights from Late-Breaker Presentation at ASGCT
Include:
- Low dose PBGM01 was well-tolerated and had a favorable
safety profile with patient 1 data through 13 months and patient 2
data through seven months: No serious adverse events,
complications related to ICM administration or evidence of dorsal
root ganglion (DRG) toxicity were observed in either patient.
- Both patients showed continued improvement across
developmental areas on the Vineland-II (caregiver-assed) and
Bayley-III (investigator-assed) scales: Through the
12-month assessments following PBGM01 administration, improvement
from baseline was observed and documented across all developmental
areas for patient 1, with notable progression in motor and language
domains. Improvement across multiple developmental domains was also
observed and documented for patient 2, despite this patient having
severe developmental delay at baseline and a prior history of
regression.
- Volumetric MRI data showed meaningful growth in brain
volume for patient 1, who was 15 months of age at gene
transfer: Volumetric MRI showed increases in brain volume
at 12 months compared to baseline for patient 1 in multiple brain
regions. Patient 2, who had severe developmental delay at baseline
and was 31 months of age at gene transfer, showed a slight decline
in brain volume from baseline at six months.
- Longer-term biomarker data
for Beta-galactosidase enzyme activity in CSF and serum showed
functional transgene expression: CSF beta-galactosidase
activity for both patients increased above levels measured in
natural history study (NHS) samples following dosing and were
sustained at the upper limit of NHS values. Serum
beta-galactosidase activity also increased following administration
of PBGM01 for both patients, with patient 1 levels above NHS values
at 12-months post-treatment and patient 2 levels normalizing to NHS
values at 6-months post-treatment.
The Imagine-1 study continues to advance. The first patient in
Cohort 2, high dose, late infantile, and both patients in Cohort 3,
low dose, early infantile, have been dosed. Interim safety and
biomarker data from both cohorts are expected in the second half of
2022.
About Imagine-1 Imagine-1 is a Phase 1/2,
global, open-label, dose-escalation study of PBGM01 administered by
a single injection into the cisterna magna in pediatric subjects
with early and late infantile GM1. The primary goal of the study is
to first assess safety and tolerability and then efficacy of PBGM01
in patients. The U.S. Food and Drug Administration (FDA) has
granted PBGM01 Fast Track, Orphan Drug, and Rare Pediatric Disease
designations. PBGM01 has also received an Orphan designation from
the European Commission.
To learn more about the clinical trial program, please visit
ClinicalTrials.gov: NCT04713475.
About PBGM01 PBGM01 is an AAV-delivery gene
therapy currently being developed for the treatment of infantile
GM1, in which patients have mutations in the GLB1 gene causing
little or no residual beta-galactosidase enzyme activity and
subsequent neurodegeneration. PBGM01 utilizes a next-generation
AAVhu68 capsid administered through the cisterna magna to deliver a
functional GLB1 gene encoding beta-galactosidase to the brain and
peripheral tissues. By increasing beta-galactosidase activity,
PBGM01 has the potential to reduce accumulation of toxic GM1
gangliosides and reverse neuronal toxicity, thereby restoring
developmental potential. In preclinical models, PBGM01 has
demonstrated broad brain distribution and high levels of expression
of the beta-galactosidase enzyme in both the CNS and critical
peripheral organs, suggesting potential treatment for both the CNS
and peripheral manifestations of GM1.
About GM1 GM1, a rare monogenic lysosomal
storage disease, is caused by mutations in the GLB1 gene, which
encodes the lysosomal enzyme beta-galactosidase (β-gal).
Reduced β-gal activity results in the accumulation of toxic
levels of GM1 gangliosides in neurons throughout the brain, causing
rapidly progressive neurodegeneration. GM1 accumulation also
results in progressive damage to other tissues including the heart,
liver, and bones and manifests with hypotonia (reduced muscle
tone), progressive CNS dysfunction, seizures, and rapid
developmental regression. Life expectancy for infants with GM1
ranges from 2-10 years, and infantile GM1 represents approximately
60 percent of the global GM1 incidence of 0.5 to 1 in 100,000 live
births.
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 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
Imagine-1 trial; 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; and the
ability of our lead product candidates to treat their respective
target 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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