Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company
focused on developing transformative therapies for rare, monogenic
central nervous system disorders, today announced that the U.S.
Food and Drug Administration (FDA) has granted Orphan Drug and Rare
Pediatric Disease (RPD) designations to PBKR03 for the treatment of
Krabbe disease (Globoid Cell Leukodystrophy). Passage Bio expects
to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021.
Krabbe disease is a rare and often life-threatening lysosomal
storage disease that presents early in the patient’s life,
resulting in progressive damage to both the brain and peripheral
nervous system.
“Receiving both Orphan Drug and Rare Pediatric Disease
designations for PBKR03 underscore the urgent unmet medical need
for children with Krabbe disease, for which there are no approved
treatments,” said Bruce Goldsmith, Ph.D., president and chief
executive officer of Passage Bio. “We are encouraged about the
potential of PBKR03 as a life-altering therapy for this underserved
patient population, and we look forward to working with the FDA as
we solidify our plans to advance PBKR03 into clinical testing in
2021.”
The FDA grants Orphan Drug designation to drugs and biologics
intended for the treatment, diagnosis or prevention of rare
diseases or conditions affecting fewer than 200,000 people in the
United States. Orphan Drug designation affords Passage Bio the
potential for certain benefits, including up to seven years of
market exclusivity, assistance in the drug development process, tax
credits for clinical development, and exemptions from certain FDA
fees.
RPD designation is granted by the FDA to encourage treatments
for serious or life-threatening diseases primarily affecting
children 18 years of age and younger and fewer than 200,000 people
in the United States. Under the RPD program, and subject to FDA
approval of PBKR03 for the treatment of Krabbe disease, Passage Bio
would be eligible to receive one priority review voucher, which
could then be redeemed to receive priority review for any
subsequent marketing application, or sold or transferred to other
companies for their programs.
About Krabbe Disease
Krabbe disease is a rare and often life-threatening lysosomal
storage disease 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. The early infantile form of the
disease is the most severe and common, typically manifesting before
six months of age and accounting for 60% to 70% of diagnoses. In
these patients, the disease course is highly predictable and
rapidly progresses to include loss of acquired milestones, staring
episodes, apnea, peripheral neuropathy, severe weakness,
unresponsiveness to stimuli, seizures, blindness, deafness and
eventual death by two years of age. Late infantile patients,
defined by onset between seven to twelve months of age, present
similar symptoms and have a median survival of approximately five
years from onset of symptoms. There are currently no
disease-modifying therapies for Krabbe disease, and we believe
incidence may be 2.6 in 100,000 births, which is higher than
reported due to lack of adequate screening at birth.
About PBKR03
PBKR03 is an AAV-delivered gene therapy encoding GALC currently
in late preclinical development for the treatment of infantile
Krabbe disease, in which patients have mutations in the gene that
codes for galactosylceramidase (GAL-C). Low GAL-C activity results
in accumulation of psychosine which is toxic to the myelin
producing oligodendrocytes of the CNS and Schwann cells in the
periphery, resulting in damage to both the central and peripheral
nervous systems. PBKR03 utilizes a next-generation proprietary
AAVhu68 capsid to deliver, through intra-cisterna magna
administration, a functional GALC gene. In preclinical models,
PBKR03 has shown meaningful transduction of both the central and
peripheral nervous system, with restoration of myelination in the
brain and peripheral nerves. PBKR03 has the potential to treat both
the central nervous system and peripheral nerve manifestations
observed in Krabbe disease patients.
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 unparalleled 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:
Investors:Sarah McCabe and Zofia MitaStern
Investor Relations, Inc.sarah.mccabe@sternir.com
zofia.mita@sternir.com
Media:Gwen FisherPassage
Bio215.407.1548gfisher@passagebio.com
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