Passage Bio Reports First Quarter 2022 Financial Results and Provides Recent Business Highlights
May 16 2022 - 7:00AM
Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic
medicines company focused on developing transformative therapies
for central nervous system disorders, today reported financial
results for the first quarter ended March 31, 2022 and provided
recent business highlights.
“We remain focused on execution across our ongoing clinical
programs and are excited to have dosed additional patients in our
Imagine-1 trial for GM1 gangliosidosis as well as the first patient
in our GALax-C trial for infantile Krabbe disease,” said Bruce
Goldsmith, Ph.D., president and chief executive officer of Passage
Bio. “We look forward to reporting data as these programs advance
throughout 2022, including additional clinical and biomarker data
from Cohort 1 in Imagine-1 at the ASGCT conference this week. We
continue to open additional clinical sites for our FTD program and
expect to dose the first patient in mid-2022.”
“We are thrilled to have recently submitted an IND for PBML04 in
metachromatic leukodystrophy, our third rare, pediatric, lysosomal
storage disorder program,” Dr. Goldsmith added. “We also recently
completed prioritization of our additional pipeline programs as
part of our strategic initiatives to extend our cash runway into
the second quarter of 2024 and look forward to continuing to
advance our programs in amyotrophic lateral sclerosis and
Huntington’s Disease and our ongoing exploratory research programs
in Alzheimer’s Disease and temporal lobe epilepsy. Rights to our
programs for Canavan disease, Charcot-Marie-Tooth Type 2A and
Parkinson’s disease have been returned to the University of
Pennsylvania allowing for the future development of these programs.
We remain deeply committed to our mission of developing
transformative therapies for people with devastating CNS
disorders.”
Recent Highlights
- Dosed first patient in Cohort 2 (late infantile, high
dose) and completed dosing of Cohort 3 (early infantile, low dose)
of the Imagine-1 clinical trial: Initial safety and
biomarker data are expected from both cohorts in the second half of
2022. The company plans to share additional clinical data from
Cohort 1 during an oral presentation at the ASGCT conference on
Wednesday, May 18, 2022.
- Dosed first patient in Cohort 1 of the GALax-C clinical
trial in March: Initial safety and biomarker data are
expected by year-end 2022.
- Preclinical data supporting ongoing clinical study of
PBKR03 in Krabbe disease published in Human Gene
Therapy: The company announced the
publication of the robust pre-clinical studies that supported
clinical study initiation for PBKR03. In the paper, Juliette
Hordeaux, D.V.M., Ph.D., D.E.C.V.P., and colleagues from University
of Pennsylvania’s Gene Therapy Program (GTP), report marked
improvements in both disease progression and key biomarkers in
large and small animal models of Krabbe disease following a single
administration of PBKR03, with no observed dose-limiting
toxicities.
- Submitted an Investigational New Drug (IND) application
to the U.S. Food and Drug Administration for a Phase 1/2 clinical
program for PBML04: An IND has been submitted for PBML04
in metachromatic leukodystrophy (MLD), a rare, pediatric, lysosomal
storage disorder. PBML04 utilizes the same next-generation
proprietary capsid as PBGM01 and PBKR03 to deliver, through
intra-cisterna magna (ICM) administration, a functional ARSA gene
into the CSF. Preclinical data supporting the planned clinical
development of PBML04 will be presented by GTP colleagues at the
ASGCT conference on Wednesday, May 18, 2022.
- Completed strategic prioritization to reduce operating
expenses and extend cash runway into the second quarter of
2024: In March, the company announced a 13 percent
reduction in workforce and plans to prioritize research and
development programs to reduce operating expenses. The company has
completed its prioritization and will continue to advance its
ongoing three clinical programs as well as its preclinical programs
in metachromatic leukodystrophy, amyotrophic lateral sclerosis and
Huntington’s disease and its exploratory research programs in
Alzheimer’s disease and temporal lobe epilepsy. The company has
returned its rights to programs in Canavan disease,
Charcot-Marie-Tooth Type 2A and Parkinson’s disease to the
University of Pennsylvania allowing for the future development of
these programs. The company continues to hold 8 additional license
options.
Anticipated Upcoming Milestones
- Multiple presentations and posters at the ASGCT 25th Annual
Meeting taking place May 16-19, 2022, including additional clinical
and biomarker data from Cohort 1 of Imagine-1 and preclinical data
supporting PBML04.
- Present interim safety and biomarker data for Cohorts 2 (late
infantile, high dose) and 3 (early infantile, low dose) for
Imagine-1 clinical trial for GM1 in the second half of 2022.
- Dose first patient in Phase 1/2 study with PBFT02 for FTD-GRN
in mid-2022. Additional clinical data milestone timing to be
provided following dosing of first patient.
- Present interim safety and biomarker data for Cohort 1 for
GALax-C Phase 1/2 clinical trial for Krabbe disease by year-end
2022.
First Quarter 2022 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities were $267.1 million as of March 31, 2022, as
compared to $315.8 million as of December 31, 2021. The
Company expects current cash and cash equivalents to fund
operations into the second quarter of 2024.
- Research and Development (R&D)
Expenses: R&D expenses were $26.2 million for the
first quarter ended March 31, 2022, compared to $25.0 million for
the same quarter in 2021. Acquired in-process R&D expenses
were $1.5 million for the first quarter ended March 31, 2022,
compared to $1.5 million in the same quarter of 2021.
- General and Administrative (G&A)
Expenses: G&A expenses were $15.1 million for the
first quarter ended March 31, 2022, compared to $12.5 million for
the same quarter in 2021.
- Net Loss: Net loss was $42.8 million, or
a net loss of $0.79 per basic and diluted share, for the first
quarter ended March 31, 2022, compared to $38.9 million, or a net
loss of $0.76 per basic and diluted share, for the same quarter in
2021.
Conference Call DetailsPassage Bio will host a
conference call and webcast today at 8:30 a.m. ET. To access
the live conference call, please dial 833-528-0605 (domestic) or
830-221-9711 (international) and reference conference ID number
6960234. A live audio webcast of the event will be available on the
Investors & News section of Passage Bio’s website at
investors.passagebio.com. The archived webcast will be available on
Passage Bio's website approximately two hours after the completion
of the event and for 30 days following the call.
About Passage BioPassage 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 timing and execution of
anticipated 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; 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.
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
|
Passage Bio, Inc.Balance
Sheets |
|
|
|
|
|
|
|
|
|
(Unaudited) |
|
|
|
(in thousands, except share data) |
|
March 31, 2022 |
|
December 31, 2021 |
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
101,614 |
|
|
$ |
128,965 |
|
Marketable securities |
|
|
165,476 |
|
|
|
186,808 |
|
Prepaid expenses and other current assets |
|
|
2,835 |
|
|
|
1,726 |
|
Prepaid research and development |
|
|
13,076 |
|
|
|
7,567 |
|
Total current assets |
|
|
283,001 |
|
|
|
325,066 |
|
Property and equipment,
net |
|
|
24,292 |
|
|
|
23,806 |
|
Right of use assets -
operating leases |
|
|
20,212 |
|
|
|
- |
|
Other assets |
|
|
5,719 |
|
|
|
6,204 |
|
Total assets |
|
$ |
333,224 |
|
|
$ |
355,076 |
|
Liabilities and
stockholders’ equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
9,135 |
|
|
$ |
9,448 |
|
Accrued expenses and other current liabilities |
|
|
15,227 |
|
|
|
20,050 |
|
Operating lease liabilities |
|
|
3,119 |
|
|
|
- |
|
Total current liabilities |
|
|
27,481 |
|
|
|
29,498 |
|
Operating lease liabilities -
noncurrent |
|
|
24,432 |
|
|
|
- |
|
Deferred rent |
|
|
- |
|
|
|
6,921 |
|
Total liabilities |
|
|
51,913 |
|
|
|
36,419 |
|
|
|
|
|
|
|
|
Commitments and
Contingencies |
|
|
|
|
|
|
|
|
|
|
|
|
|
Stockholders’ equity: |
|
|
|
|
|
|
Common stock, $0.0001 par value: 300,000,000 shares authorized;
54,307,691 shares issued and outstanding at March 31, 2022 and
54,244,996 shares issued and outstanding at December 31, 2021 |
|
|
5 |
|
|
|
5 |
|
Additional paid-in capital |
|
|
681,732 |
|
|
|
675,346 |
|
Accumulated other comprehensive income (loss) |
|
|
(1,334 |
) |
|
|
(413 |
) |
Accumulated deficit |
|
|
(399,092 |
) |
|
|
(356,281 |
) |
Total stockholders’ equity |
|
|
281,311 |
|
|
|
318,657 |
|
Total liabilities and stockholders’ equity |
|
$ |
333,224 |
|
|
$ |
355,076 |
|
Passage Bio, Inc.Statements of
Operations and Comprehensive
Loss(Unaudited) |
|
|
|
Three Months Ended March 31, |
(in thousands, except share and per share
data) |
|
2022 |
|
2021 |
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
$ |
26,213 |
|
|
$ |
24,970 |
|
Acquired in-process research and development |
|
|
1,500 |
|
|
|
1,500 |
|
General and administrative |
|
|
15,099 |
|
|
|
12,464 |
|
Loss from operations |
|
|
(42,812 |
) |
|
|
(38,934 |
) |
Interest income, net |
|
|
1 |
|
|
|
52 |
|
Net loss |
|
$ |
(42,811 |
) |
|
$ |
(38,882 |
) |
Per share information: |
|
|
|
|
|
|
Net loss per share of common
stock, basic and diluted |
|
$ |
(0.79 |
) |
|
$ |
(0.76 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
|
54,275,751 |
|
|
|
51,331,449 |
|
Comprehensive loss: |
|
|
|
|
|
|
Net loss |
|
$ |
(42,811 |
) |
|
$ |
(38,882 |
) |
Unrealized gain (loss) on marketable securities |
|
|
(921 |
) |
|
|
5 |
|
Comprehensive loss |
|
$ |
(43,732 |
) |
|
$ |
(38,877 |
) |
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