Passage Bio’s PBGM01 Receives Orphan Drug Designation from EMA for Treatment of GM1 Gangliosidosis
October 26 2020 - 7:00AM
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 European
Commission (EC) has granted orphan drug designation for the
company’s lead gene therapy candidate PBGM01 for the treatment of
GM1 gangliosidosis (GM1). PBGM01, an adeno-associated virus
(AAV)-delivery gene therapy, has previously been granted Orphan
Drug Designation and Rare Pediatric Disease Designation by the U.S.
Food and Drug Administration (FDA) for the treatment of GM1. GM1 is
a rare and often life-threatening central nervous system disorder
with no approved disease-modifying therapies available.
“GM1, most common and severe in infants, results in rapid
neurodegeneration and is simply devastating for patients and their
families,” said Bruce Goldsmith, Ph.D., president and chief
executive officer of Passage Bio. “PBGM01 is a potentially
transformative gene therapy that may lead to the preservation of
neurological function and an improvement in developmental potential
and survival in patients with GM1. Receiving orphan drug
designation for PBGM01 in the European Union is an important
milestone that will propel our efforts to bring this much-needed
therapy to waiting patients.”
The EC grants orphan drug designation based on a positive
opinion issued by the EMA Committee for Orphan Medicinal Products
(COMP). To qualify for orphan designation, an investigational
medicinal product must be intended to treat a chronically
debilitating or life-threatening condition that affects fewer than
five in 10,000 people across the European Union and there is no
effective treatment approved in EU. With EMA orphan drug
designation, Passage Bio will receive certain benefits and
incentives including 10-year market exclusivity for the approved
therapeutic indication once PBGM01 receives marketing authorization
as well as clinical protocol assistance and reduced regulatory
fees.
Passage Bio expects to initiate dosing of PBGM01 in a Phase 1/2
trial late in the fourth quarter of 2020 or early in the first
quarter of 2021, with initial 30-day safety and biomarker data
expected in the late first half of 2021. The trial will be an
open-label, dose escalation study of PBGM01 administered by a
single injection into the intra cisterna magna, or ICM, in
pediatric subjects with infantile GM1.
About GM1
GM1 gangliosidosis (GM1) is a rare and often life-threatening
monogenic recessive lysosomal storage disease caused by mutations
in the GLB1 gene, which encodes lysosomal acid beta-galactosidase
(β-gal). Reduced β-gal activity results in the accumulation of
toxic levels of GM1 ganglioside in neurons throughout the brain,
causing rapidly progressing neurodegeneration. GM1 manifests as a
continuum of disease and is most severe in the Infantile form,
which is characterized by onset in the first 6 months of life 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 62.5% of the incidence of 0.5 to 1 in 100,000 live
births.
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 intra-cisterna
magna (ICM) 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 wide
uptake of the β-gal enzyme in both the central nervous system (CNS)
and critical peripheral organs, suggesting potential treatment for
both the CNS and peripheral manifestations of GM1.
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.212-362-1200sarah.mccabe@sternir.comzofia.mita@sternir.com
Media:Gwen FisherPassage
Bio215-407-1548gfisher@passagebio.com
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