Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage
genetic medicines company focused on developing transformative
therapies for central nervous system (CNS) disorders, today
announced that the first patient has been dosed in the global Phase
1/2 upliFT-D clinical trial evaluating PBFT02, an adeno-associated
virus (AAV)-delivery gene therapy for the treatment of patients
with frontotemporal dementia (FTD) with granulin (GRN) mutations.
FTD is a form of early onset dementia with no approved
disease-modifying therapies.
“Dosing the first patient in our upliFT-D trial is an important
milestone for the Passage Bio team and for the PBFT02 program, our
first program in the clinic for adults,” said Edgar (Chip) Cale,
interim chief executive officer of Passage Bio. “We look forward to
continuing our important work to develop PBFT02 as a potential
treatment option for the thousands of people living with FTD-GRN.
We are grateful for the support from the families and clinical
trial investigators who have chosen to participate in our
studies.”
The upliFT-D clinical study evaluates PBFT02 as a single dose
delivered via intra-cisterna magna (ICM) injection. This gene
therapy uses an AAV1 viral vector to deliver a functional copy of
the GRN gene encoding progranulin (PGRN) to a patient's cells. PGRN
is a complex and highly conserved protein thought to have multiple
roles in cell biology, development and inflammation. Emerging
evidence suggests that PGRN deficiency may contribute to lysosomal
dysfunction.
“FTD-GRN is a devastating disease with no approved
disease-modifying therapies, and we are hopeful this trial will
provide evidence that PBFT02 could become a meaningful treatment
option for adults living with FTD-GRN,” said Mark Forman, M.D.,
Ph.D., chief medical officer of Passage Bio. “Our approach, which
employs the AAV1 vector and ICM administration, provides a
potential opportunity to achieve higher than normal levels of PGRN
in the CNS, thereby overcoming the PGRN deficiency in GRN mutation
carriers with a diagnosis of early symptomatic FTD-GRN. We look
forward to building on our preclinical data with this Phase 1/2
trial.”
FTD is one of the more common causes of early-onset dementia. In
approximately 5 to 10 percent of individuals with FTD–3,000 to
6,000 individuals in the United States–the disease occurs because
of mutations in the GRN gene, causing a deficiency of PGRN. FTD
causes impairment in behavior, language and executive function, as
well as changes in personal and social conduct including loss of
inhibition, apathy, and social withdrawal. Progression of FTD
results in an average survival of eight years after the onset of
symptoms.
The U.S. Food and Drug Administration (FDA) has granted PBFT02
Fast Track and Orphan Drug designations. PBFT02 has also received
an Orphan designation from the European Commission.
upliFT-D continues to enroll patients with early symptomatic
FTD-GRN. If you are interested in a referral to a clinical trial
site, please contact Passage Bio here to learn more.
About upliFT-D (NCT04747431)
upliFT-D is a Phase 1/2 global, multi-center, open-label,
dose-escalation study of PBFT02 administered by single injection
into the cisterna magna in patients aged 35 to 75 years with early
symptomatic FTD-GRN.
The upliFT-D study will investigate two dose levels of PBFT02.
The study will sequentially enroll two cohorts, with an optional
third dose level cohort expected to be enrolled based on the
results of the first two cohorts. Enrollment is currently ongoing.
The primary endpoint of the study is to evaluate the safety and
tolerability of PBFT02. Secondary endpoints include disease
biomarkers and clinical outcome measures. upliFT-D is a two-year
study with a three-year safety extension. Passage Bio is pursuing
several initiatives to support clinical trial recruitment and
enrollment, including a collaborative partnership with InformedDNA
to provide no-cost genetic counseling and testing for adults who
have been diagnosed by their physicians with FTD. To date, upliFT-D
has received CTA acceptance in the United States, Canada and
Brazil. More information about upliFT-D can be found here.
About PBFT02
PBFT02 utilizes an AAV1 viral vector to deliver, through ICM
administration, a functional GRN gene to patients with mutations in
the gene that encodes for progranulin (PGRN). This vector and
delivery approach aims to provide higher-than-normal levels of the
PGRN protein to the CNS to overcome the progranulin deficiency in
GRN gene mutation carriers.
PBFT02 is supported by extensive preclinical studies conducted
by Passage Bio’s collaborator, the University of Pennsylvania’s
Gene Therapy Program. The studies showed compelling evidence,
including broad transduction across the brain, including high
transduction of ependymal cells, and demonstrated increases in
cerebrospinal fluid (CSF) PGRN concentrations to >50-fold normal
human CSF PGRN concentrations.
About Passage Bio
Passage 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 future milestones related to our
PBFT02 program; timing and execution of anticipated trial designs
and 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; 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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