Full Press Release Details
Passage Bio Announces Plan to Deliver
on Multiple Meaningful Catalysts in 2021
- Investigational New Drug Applications (IND) submitted for PBFT02 for Frontotemporal Dementia with
Granulin Mutation (FTD-GRN) and PBKR03 for Krabbe Disease -
- Three clinical programs to begin in 1H21 -
- Metachromatic leukodystrophy program, PBML04, advanced to IND-enabling phase -
- Additional option exercised from Penn's world-class Gene Therapy Program for undisclosed program focused on an adult CNS disorder -
- New manufacturing laboratory to support analytical capabilities, clinical product testing and assay validation slated to open in 2Q21 -
- PBFT02 for FTD-GRN becomes latest of company's three most advanced programs to receive FDA orphan drug designation -
PHILADELPHIA, January 11, 2021 - Passage Bio, Inc.
(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous
system (CNS) disorders, today provided a corporate outlook detailing the expansion of the company's pipeline, new regulatory
filings, projected program milestones, and research and development progress. Bruce Goldsmith, Ph.D., president and chief executive
officer of Passage Bio, will highlight the company's recent progress and plans for the future in his presentation today at
the 39th Annual J.P. Morgan Healthcare Conference.
In his remarks, Dr. Goldsmith will highlight several components
of the Company's progress and timelines, including:
"It is widely reported that there are approximately 7,000
rare diseases. Less discussed is that out of these 7,000 rare diseases, there are more than 790 rare monogenic CNS diseases, the
area where we are focused," said Dr. Goldsmith. "The diseases we are targeting are particularly devastating and have
no approved disease-modifying treatment options, which means there is tremendous opportunity to make a difference for patients.
At Passage Bio, we believe we are well positioned with our collaboration with Penn's Gene Therapy Program, our corporate
model, robust pipeline, manufacturing capability, strong cash position, and experienced team to succeed in our mission to deliver
on the promise of gene therapies for patients with rare monogenic CNS disorders."
Passage Bio's collaboration with Penn's world-class
Gene Therapy Program (GTP) provides the company with access to cutting-edge research, expertise and next-generation technologies.
GTP is recognized worldwide for its innovative gene therapy capabilities, AAV vector technology manufacturing, and IND expertise.
Through the Penn GTP collaboration, Passage Bio has licensing options for a total of 17 gene therapy research programs focused
on rare monogenic disorders of the CNS through 2025. The company has exercised seven options to date, including four programs for
pediatric and three programs for adult rare monogenic CNS disorders.
"Through our relationship with GTP coupled with our business
model, we have a well-defined path for clinical success," explained Dr. Goldsmith. "We believe our pipeline has a higher
probability of technical and regulatory success, in part because we are able to optimize delivery approaches, select candidates
based on empirical research findings and integrate next-generation innovations from GTP as appropriate. Additionally, all our current
therapies are designed for direct delivery to the CNS, which we believe offers a number of benefits, including avoiding crossing
the blood brain barrier, better CNS biodistribution and lower dosing compared to systemic delivery."
Dr. Goldsmith added: "We spent 2020 establishing a solid
foundation for our company to execute seamlessly against our ambitious future plans. Our progress has laid the groundwork for clinical
trial preparedness and manufacturing readiness, which will be critical to successfully deliver on several meaningful catalysts
The company's three most advanced programs are: PBGM01
for infantile GM1 and PBKR03 for Krabbe disease, which are pediatric programs; and PBFT02 for FTD-GRN, which is an adult program.
Passage Bio is also continuing to progress preclinical pediatric programs PBCM06 for Charcot-Marie-Tooth neuropathy type 2A (CMT2A)
and PBML04 for MLD, which was recently advanced into IND-enabling studies. The company's additional adult preclinical programs
are PBAL05 for amyotrophic lateral sclerosis (ALS) and a seventh program, for which the company recently exercised an option and
has not yet disclosed the therapeutic target.
Significant Pipeline and Corporate Advancement
The company's lead program PBGM01 is entering the clinic
to study its safety and efficacy in addressing infantile GM1, a rare and often life-threatening CNS disorder with no approved disease-modifying
therapies. The global Phase 1/2 study, Imagine-1, is expected to enroll the first patient in the first quarter of 2021 and is expected
to report initial 30-day safety and biomarker data mid-year 2021. PBGM01 has received regulatory agency clearance from FDA and
MHRA. The investigational therapy has also received orphan drug and rare pediatric disease designations from FDA, as well as orphan
drug designation from the European Medicines Agency for the treatment of GM1.
The company has submitted investigational new drug (IND) applications
to FDA for Phase 1/2 clinical studies of PBFT02 in FTD-GRN, a devastating form of early onset dementia; and PBKR03 in Krabbe disease,
a pediatric disease with rapid progression, typically resulting in death by 2 years old. The company recently received orphan drug
designation from FDA for PBFT02 for FTD-GRN. PBKRO3 has orphan drug and rare pediatric disease designations from FDA for treatment
of Krabbe disease. Passage Bio anticipates the start of both clinical programs in the first half of 2021. Initial data from these
trials are anticipated to potentially readout in late 2021 or early 2022, depending on the timing of first patient treated in each
In parallel with pipeline advancement, the company has focused
on establishing manufacturing and global distribution from clinical development through initial commercialization. Clinical supply
for PBGM01 for our phase 1 / 2 global trial, manufactured through our partnership with Catalent, is already in place. We have also
manufactured clinical supplies to initiate clinical trials for our next two most advanced programs - PBKRO3 for Krabbe disease
and PBFT02 for FTD-GRN. In December, Passage Bio announced that it had completed construction and started production at its dedicated
Current Good Manufacturing Practices (CGMP) manufacturing suite at Catalent Cell & Gene Therapy's facility in Maryland.
The company also announced the planned opening in the second quarter of 2021 of a gene therapy manufacturing research and development
site at the Princeton West Innovation Campus in Hopewell, New Jersey, for Chemistry, Manufacturing and Controls (CMC) laboratory
operations to support analytics, assay development, and product testing for the company's gene therapy programs.
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