Passage Bio Reports Second Quarter 2023 Financial Results and Provides Recent Business Highlights
August 07 2023 - 7:15AM
Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic
medicines company focused on developing transformative therapies
for central nervous system (CNS) disorders, today reported
financial results for the second quarter ended June 30, 2023 and
provided recent business highlights.
“We continued to make steady progress advancing our lead
clinical programs in the second quarter and further improved our
financial strength, extending our cash runway into the fourth
quarter of 2025. By extending our operating runway, we can continue
to focus on strong execution of our GM1 and FTD programs and
delivering meaningful clinical data from each program over the
coming quarters,” said William Chou, M.D., president and chief
executive officer of Passage Bio. “In GM1, we are encouraged by the
promising data shared from Cohorts 1 through 4, further validating
the potential of PBGM01 as a transformative therapy in both early
and late infantile GM1 gangliosidosis. Additionally, we are
thrilled to have already dosed the first patient at Dose 3,
demonstrating effective execution across the program. In FTD, we
are excited by emerging data from initial treated patients, which
indicates translation from preclinical models into the clinic. We
are experiencing strong momentum in the FTD program with six
clinical trial sites in Brazil, Canada, and the United States now
initiated, and several GRN positive patients undergoing evaluation
for study eligibility. We remain dedicated to progressing our two
lead clinical programs in our mission to bring transformative
therapies to those affected by devastating CNS disorders.”
Recent Highlights
- Reported promising interim safety, biomarker and
survival data from Cohorts 1-4 of Imagine-1 study: Interim
data from eight patients treated in Cohorts 1-4 showed that PBGM01
continued to be well-tolerated and exhibited a favorable safety and
immunological profile at both doses tested in early and late
infantile GM1. Administration of PBGM01 led to durable and
dose-dependent increases in CSF β-Gal activity, with Dose 2
increasing enzyme activity in three of four patients treated,
exceeding average levels seen in healthy controls. Dose 2 PBGM01
also demonstrated the ability to achieve normal levels of CSF GM1
gangliosides at twelve months after treatment (based on a healthy
adult reference sample). Additionally, Imagine-1 study participants
showed initial evidence of improved survival relative to natural
history data.
- Treated first patient at highest dose level (Dose 3) in
Imagine-1 study in July: The company is actively
recruiting additional patients in Imagine-1 study to be treated at
the highest dose, Dose 3, of PBGM01 in Cohorts 5 (late infantile)
and 6 (early infantile). The amended study protocol has been
approved at several clinical trial sites in multiple countries,
including Brazil, Turkey and the United States. Dosing of patients
in Cohorts 5 and 6 may occur concurrently, and the company expects
to report initial biomarker and safety data from Dose 3 by
mid-2024.
- Experiencing strong momentum in global Phase 1/2
upliFT-D trial of PBFT02 for the treatment of patients with
frontotemporal dementia (FTD) with granulin mutations
(GRN): The company
continues to experience strong momentum across the FTD program,
with six clinical trial sites now initiated across Brazil, Canada,
and the United States. Patient enrollment is advancing globally
with multiple GRN+ patients identified and being evaluated for
eligibility. Initial safety and biomarker data from Cohort 1
patients is expected in Q4 2023.
- Cash runway extended into Q4 2025: Following
an organizational restructuring in July and implementation of
prudent cash management strategies, the company significantly
extended its cash runway into the fourth quarter of 2025. This
provides the company with ample resources to deliver on meaningful
clinical data from its two ongoing clinical trials.
- Appointed Dolan Sondhi,
Ph.D. to the company’s board of directors: In July 2023,
the company announced the appointment of Dolan Sondhi, Ph.D.,
Professor of Research in the Department of Genetic Medicine at
Weill Cornell Medicine, to its board of directors and her
appointment to the Nominating and Corporate Governance Committee.
Dr. Sondhi is a highly accomplished research scientist with
expertise in the development and translation of adeno-associated
virus (AAV) gene therapies for genetic disorders, with a particular
emphasis on the CNS, including lysosomal storage diseases and
neurodegenerative conditions.
Anticipated Upcoming Milestones
- Present initial safety and biomarker data for Cohort 1 in
upliFT-D clinical trial for FTD in Q4 2023.
- Present initial safety and biomarker data for Dose 3 in
Imagine-1 clinical trial for GM1 gangliosidosis by mid-2024.
Second Quarter 2023 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities were $151.5 million as of June 30, 2023, as
compared to $239.3 million as of June 30, 2022. The Company expects
current cash, cash equivalents and marketable securities to fund
operations into the fourth quarter of 2025.
- Research and Development (R&D)
Expenses: R&D expenses were $17.3 million for the
quarter ended June 30, 2023, as compared to $26.8 million as of
June 30, 2022.
- General and Administrative (G&A)
Expenses: G&A expenses were $8.1 million for the
quarter ended June 30, 2023, as compared to $13.0 million as of
June 30, 2022.
- Net Loss: Net loss was $23.9 million, or
a net loss of $0.44 per basic and diluted share, for the quarter
ended June 30, 2023, compared to $39.5 million, or a net loss of
$0.73 per basic and diluted share, for the same quarter in
2022.
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 beta-galactosidase enzyme activity and
subsequent neurodegeneration. PBGM01 utilizes a next-generation
AAVhu68 capsid administered through the cisterna magna to deliver a
functional GLB1 gene encoding beta-galactosidase to the brain and
peripheral tissues. By increasing beta-galactosidase activity,
PBGM01 has the potential to reduce accumulation of toxic GM1
gangliosides and reverse neuronal toxicity, thereby restoring
developmental potential. In preclinical models, PBGM01 has
demonstrated broad brain distribution and high levels of expression
of the beta-galactosidase enzyme in both the CNS and critical
peripheral organs, suggesting potential treatment for both the CNS
and peripheral manifestations of GM1.
About GM1 GM1, a rare monogenic lysosomal
storage disease, is caused by mutations in the GLB1 gene, which
encodes the lysosomal enzyme beta-galactosidase (β-gal). Reduced
β-gal activity results in the accumulation of toxic levels of GM1
gangliosides in neurons throughout the brain, causing rapidly
progressive neurodegeneration. GM1 accumulation also results in
progressive damage to other tissues including the heart, liver, and
bones and manifests with hypotonia (reduced muscle tone),
progressive CNS dysfunction, seizures, and rapid developmental
regression. Life expectancy for infants with GM1 ranges from 2-10
years, and infantile GM1 represents approximately 60 percent of the
global GM1 incidence of 0.5 to 1 in 100,000 live births.
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
clinical programs in GM1 gangliosidosis and frontotemporal dementia
and our preclinical pipeline, including programs in amyotrophic
lateral sclerosis and Huntington’s disease. 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 timing and execution of
anticipated milestones, including the progress 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 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.
|
Passage Bio, Inc.Balance
Sheets |
|
|
|
|
|
|
|
|
|
(Unaudited) |
|
|
|
(in thousands, except share and per share
data) |
|
June 30, 2023 |
|
December 31, 2022 |
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
30,291 |
|
|
$ |
34,601 |
|
Marketable securities |
|
|
121,189 |
|
|
|
155,009 |
|
Prepaid expenses and other current assets |
|
|
1,230 |
|
|
|
926 |
|
Prepaid research and development |
|
|
915 |
|
|
|
6,508 |
|
Total current assets |
|
|
153,625 |
|
|
|
197,044 |
|
Property and equipment,
net |
|
|
20,225 |
|
|
|
22,515 |
|
Right of use assets -
operating leases |
|
|
19,413 |
|
|
|
19,723 |
|
Other assets |
|
|
433 |
|
|
|
4,267 |
|
Total assets |
|
$ |
193,696 |
|
|
$ |
243,549 |
|
Liabilities and
stockholders’ equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
3,383 |
|
|
$ |
4,065 |
|
Accrued expenses and other current liabilities |
|
|
13,161 |
|
|
|
11,011 |
|
Operating lease liabilities |
|
|
3,326 |
|
|
|
3,275 |
|
Total current liabilities |
|
|
19,870 |
|
|
|
18,351 |
|
Operating lease liabilities -
noncurrent |
|
|
23,412 |
|
|
|
23,832 |
|
Total liabilities |
|
|
43,282 |
|
|
|
42,183 |
|
|
|
|
|
|
|
|
Stockholders’ equity: |
|
|
|
|
|
|
Common stock, $0.0001 par value: 300,000,000 shares authorized;
54,745,680 shares issued and outstanding at June 30, 2023
and 54,614,690 shares issued and outstanding at
December 31, 2022 |
|
|
5 |
|
|
|
5 |
|
Additional paid-in capital |
|
|
701,349 |
|
|
|
694,733 |
|
Accumulated other comprehensive income (loss) |
|
|
(340 |
) |
|
|
(966 |
) |
Accumulated deficit |
|
|
(550,600 |
) |
|
|
(492,406 |
) |
Total stockholders’ equity |
|
|
150,414 |
|
|
|
201,366 |
|
Total liabilities and stockholders’ equity |
|
$ |
193,696 |
|
|
$ |
243,549 |
|
|
Passage Bio, Inc.Statements of
Operations and Comprehensive
Loss(Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
(in thousands, except share and per share
data) |
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
17,324 |
|
|
$ |
26,821 |
|
|
$ |
34,160 |
|
|
$ |
53,034 |
|
Acquired in-process research and development |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
1,500 |
|
General and administrative |
|
|
8,064 |
|
|
|
12,991 |
|
|
|
27,111 |
|
|
|
28,090 |
|
Loss from operations |
|
|
(25,388 |
) |
|
|
(39,812 |
) |
|
|
(61,271 |
) |
|
|
(82,624 |
) |
Other income (expense),
net |
|
|
1,532 |
|
|
|
270 |
|
|
|
3,077 |
|
|
|
271 |
|
Net loss |
|
$ |
(23,856 |
) |
|
$ |
(39,542 |
) |
|
$ |
(58,194 |
) |
|
$ |
(82,353 |
) |
Per share information: |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share of common
stock, basic and diluted |
|
$ |
(0.44 |
) |
|
$ |
(0.73 |
) |
|
$ |
(1.06 |
) |
|
$ |
(1.52 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
|
54,683,817 |
|
|
|
54,386,318 |
|
|
|
54,651,488 |
|
|
|
54,331,340 |
|
Comprehensive loss: |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
$ |
(23,856 |
) |
|
$ |
(39,542 |
) |
|
$ |
(58,194 |
) |
|
$ |
(82,353 |
) |
Unrealized gain (loss) on marketable securities |
|
|
87 |
|
|
|
(244 |
) |
|
|
626 |
|
|
|
(1,165 |
) |
Comprehensive loss |
|
$ |
(23,769 |
) |
|
$ |
(39,786 |
) |
|
$ |
(57,568 |
) |
|
$ |
(83,518 |
) |
For further information, please contact:
Investors:Stuart HendersonPassage
Bio267.866.0114shenderson@passagebio.com
Media:Mike BeyerSam Brown Inc. Healthcare
Communications312.961.2502MikeBeyer@sambrown.com
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