Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company
focused on developing transformative therapies for rare monogenic
central nervous system (CNS) disorders, today announced that U.S.
Food and Drug Administration (FDA) has cleared an investigational
new drug (IND) application for the company's lead product
candidate, PBGM01, an adeno-associated virus (AAV)-delivery gene
therapy that is being studied for the treatment of infantile GM1
gangliosidosis (GM1). GM1 is a rare and often life-threatening CNS
disorder with no approved disease-modifying therapies available.
The company expects to dose the first patient for the global PBGM01
clinical trial program in the first quarter of 2021.
“FDA clearance of our IND represents a significant milestone
that supports the transition of Passage Bio to a clinical-stage
company,” said Bruce Goldsmith, Ph.D., president and chief
executive officer of Passage Bio. “It is an achievement that
reflects the potential of our bold science, the deep value of our
partnership with the University of Pennsylvania’s Gene Therapy
Program, and the dedication of our entire team to our shared
mission to deliver one-time gene therapies that improve the lives
of patients with rare CNS disorders.”
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 in neurons throughout the
brain, causing rapidly progressive neurodegeneration. GM1 manifests
with hypotonia (reduced muscle tone), progressive CNS dysfunction,
and rapid developmental regression. Life expectancy for infants
with GM1 is two to four years, and infantile GM1 represents
approximately 60 percent of the global GM1 incidence of 0.5 to 1 in
100,000 live births.
“I am pleased that Passage Bio has FDA clearance to proceed with
this study,” said James Wilson, M.D., Ph.D., director of the Gene
Therapy Program at the University of Pennsylvania (Penn) and chief
scientific advisor of Passage Bio. “We look forward to Passage
Bio’s clinical advancement of this therapy that aims to slow or
potentially halt the rapidly progressive neurodegeneration seen in
infants affected by this condition.”
Imagine-1 study to commence in 1Q 2021
“Our team is actively preparing for the start of our global
Phase 1/2 trial, and is committed to ensuring eligible patients
have a pathway to clinical sites in the United States as well as in
other countries when they begin to open later in the first quarter,
so that we can explore the potential of PBGM01 as a
disease-modifying treatment for infantile GM1 that is so
desperately needed,” said Gary Romano, M.D., Ph.D., chief medical
officer of Passage Bio.
Imagine-1 is a 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 phase
1 / 2 clinical program will enroll a total of four cohorts of two
patients each, with separate dose-escalation cohorts for late onset
infantile GM1 and early onset infantile GM1. Passage Bio plans to
report initial 30-day safety and biomarker data mid-year 2021 for
Imagine-1.
The clearance of Passage Bio’s IND application from FDA follows
receipt of Clinical Trial Authorization for PBGM01 from the United
Kingdom’s (UK) Medicines Healthcare Products Regulatory Agency,
which was announced on December 10, 2020.
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 β-gal
enzyme activity and subsequent neurodegeneration. PBGM01 utilizes a
next-generation AAVhu68 capsid administered through the
intra-cisterna magna to deliver a functional GLB1 gene encoding
β-gal to the brain and peripheral tissues. By reducing the
accumulation of GM1 gangliosides, PBGM01 has the potential to
reverse neuronal toxicity, thereby restoring developmental
potential. In preclinical models, PBGM01 has demonstrated broad
brain distribution and high levels of expression of the β-gal
enzyme in both the CNS and critical peripheral organs, suggesting
potential treatment for both the CNS and peripheral manifestations
of GM1. PBGM01 has been granted Orphan Drug Designations by FDA and
the European Commission as well as a Rare Pediatric Disease
Designation by FDA.
About Passage Bio
At Passage Bio (Nasdaq: PASG), we are on a mission to provide
life-transforming gene therapies for patients with rare, monogenic
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 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.
Penn Financial Disclosure
Dr. Wilson is a Penn faculty member as well as a scientific
collaborator, consultant and co-founder of Passage Bio. As such, he
holds an equity stake in the company, receives sponsored research
funding from Passage Bio, and as an inventor of certain Penn
intellectual property that is licensed to Passage Bio, may receive
additional financial benefits in the future. The University of
Pennsylvania also receives sponsored research funding from Passage
Bio and has licensed intellectual property to the company that may
result in future financial returns to Penn.
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 our planned IND submissions,
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 the underlying causes of 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:Sarah McCabe and Zofia MitaStern Investor
Relations,
Inc.212-362-1200sarah.mccabe@sternir.comZofia.mita@sternir.com
Passage Bio Media:Gwen FisherPassage
Bio215-407-1548gfisher@passagebio.com
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