Passage Bio Receives FDA Clearance of IND Application for PBFT02 Gene Therapy Candidate for Treatment of Patients with Fronto...
January 28 2021 - 7:00AM
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 the
U.S. Food and Drug Administration (FDA) has cleared an
investigational new drug (IND) application for PBFT02, an
adeno-associated virus (AAV)-delivery gene therapy that is being
studied for the treatment of patients with Frontotemporal Dementia
(FTD) with granulin (GRN) mutations. FTD is a debilitating form of
early onset dementia that currently has no approved
disease-modifying therapies.
“We are pleased to be advancing our second therapy into clinical
development in our quest to bring transformative medicines to
patients who need them,” said Bruce Goldsmith, Ph.D., chief
executive officer of Passage Bio. “FTD can have a devastating
impact on a person’s quality of life and create a substantial
caregiving and economic burden for families. We are excited to
investigate the potential of PBFT02 as a treatment for FTD-GRN as
we initiate our clinical development program in the coming
months.”
FTD is one of the more common causes of early-onset (midlife)
dementia, causing impairment in behavior, language and executive
function, and occurs at similar frequency to Alzheimer’s disease in
patients younger than 65 years. In approximately 5 to 10 percent of
individuals with FTD – 3,000 to 6,000 in the United States – the
disease occurs because of mutations in the GRN gene, causing a
deficiency of progranulin (PGRN). PGRN is a complex and highly
conserved protein. The mechanism by which PGRN deficiency results
in FTD is uncertain, but increasing evidence points to PGRN’s role
in lysosomal function. The rapid progression of FTD results in an
average survival of eight years after onset of symptoms.
Passage Bio is developing PBFT02 to treat FTD-GRN as a single
dose delivered via intra-cisterna magna (ICM) injection. The gene
therapy utilizes an AAV1 viral vector to deliver a modified DNA
encoding the GRN gene to a patient's cells. The goal of this vector
and delivery approach is to provide higher than normal levels of
PGRN to the central nervous system to overcome the progranulin
deficiency in GRN mutation carriers, who have been observed to have
reduced cerebrospinal fluid PGRN levels ranging from 30% to 50% of
the PGRN levels observed in normal, mutation non-carriers.
Clinical Development of PBFT02 Supported by University
of Pennsylvania’s Gene Therapy Program (GTP) Pre-Clinical
Data
Passage Bio is advancing PBFT02 into the clinic supported by
preclinical data generated by its collaborator, University of
Pennsylvania’s Gene Therapy Program (GTP). The data, published in
the peer-reviewed scientific journal Annals of Clinical and
Translational Neurology, showed that a single administration of an
optimized AAV containing the GRN gene resulted in elevated levels
of PGRN in the brain and cerebral spinal fluid (CSF), reduced
lysosomal storage lesions, normalized lysosomal enzyme expression
and corrected microgliosis in a mouse model of progranulin
deficiency. A single administration of PBFT02 via the optimized
AAV1-GRN vector demonstrated transduction broadly across the brain,
including a very high transduction of ependymal cells that line the
ventricles of the brain and are involved with CSF production,
resulting in CSF progranulin levels of more than 50-fold
normal.
The FDA has granted an Orphan Drug designation for PBFT02 for
the treatment of FTD-GRN.
Phase 1/2 Study Initiation Anticipated for
1H21
Passage Bio expects to initiate a Phase1/2 clinical trial for
PBFT02 in the first half of 2021. The trial is designed as a
dose-escalation study of a single ICM dose of PBFT02 in subjects
with FTD and heterozygous mutations in the GRN gene. The primary
endpoint of the Phase 1/2 study is safety and tolerability;
secondary endpoints include CSF progranulin levels, disease
biomarkers, and clinical outcome measure. Initial data from the
trial is anticipated to potentially readout in late 2021 or early
2022, depending on the timing of when the first patient is treated
in the study.
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.
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.com Zofia.mita@sternir.com
Passage Bio Media:
Gwen FisherPassage Bio215-407-1548gfisher@passagebio.com
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