Passage Bio Reports Third Quarter 2020 Financial Results and Recent Business Highlights
November 10 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 reported financial results
for the third quarter ended September 30, 2020 and provided recent
business highlights.
“Over the past quarter, we have made important strides in
strengthening our operations to support the initiation of three
Phase 1/2 clinical trials for our lead programs in infantile GM1,
FTD-GRN, and Krabbe disease in the first half of 2021,” said Bruce
Goldsmith, Ph.D., president and chief executive officer of Passage
Bio. “We are particularly pleased with our progress in terms of
patient identification, clinical trial site preparedness and
manufacturing readiness. We also remain confident that we will
receive FDA clearance for our investigational new drug application
for PBGM01 for the treatment of infantile GM1. This has all been
made possible because of the highly experienced, talented team that
we have assembled over the past year to be ready to execute
seamlessly in 2021 on our path to providing life-transforming gene
therapies for patients with rare, monogenic CNS disorders.”
Recent Corporate
Highlights
- Work progresses in anticipation of
U.S. Food and Drug Administration (FDA) clearance for
investigational new drug (IND) application for PBGM01: In
August 2020, Passage Bio received its official letter from FDA
confirming that the clinical hold of its IND for PBGM01 for the
treatment of infantile GM1 gangliosidosis (GM1) was due solely to
questions concerning the biocompatibility of the proposed intra
cisterna magna (ICM) delivery device. Passage Bio is working
diligently to resolve the clinical hold and, based on feedback from
FDA, is conducting biocompatibility risk assessments and testing of
the ICM device. The company remains confident that the IND will be
cleared. As a result of these efforts to obtain FDA clearance of
the device, Passage Bio now plans to initiate its Phase 1/2 trial
in the first quarter of 2021 and to report initial 30-day safety
and biomarker data mid-year 2021.
- Dedicated GMP manufacturing suite is
complete: Construction and qualification of
Passage Bio’s dedicated suite at Catalent are complete. With this
suite, the company will now be able to provide clinical supplies
for its lead pipeline products through early commercialization and
expects to initiate manufacturing activities in the coming months.
Clinical supply for the GM1 Phase 1/2 trial is already in place
through Passage Bio’s ongoing partnership with Catalent.
- Orphan Drug Designation (ODD) granted by
European Commission (EC) for PBGM01 – In October
2020, the EC granted ODD to PBGM01 for the treatment of GM1, a rare
and often life-threatening monogenic recessive lysosomal storage
disease caused by mutations in the GLB1 gene, resulting in rapidly
progressing neurodegeneration. PBGM01 has previously been granted
ODD and RPDD by FDA for the treatment of GM1.
- Rare Pediatric Disease Designation (RPDD) and
ODD granted by FDA for PBKR03 – In October 2020,
FDA granted RPDD and ODD to PBKR03 for the treatment of Krabbe
disease, a rare and often life-threatening lysosomal storage
disease caused by mutations in the GALC gene resulting in
progressive damage to both the brain and peripheral nervous
system.
- Collaboration with Invitae in place to
facilitate genetic testing and support diagnosis of patients with
GM1 – Passage Bio recently announced it has
entered into a collaboration with Invitae, a leading medical
genetic testing company, to offer free genetic testing and
counselling for GM1 through Invitae’s Detect Lysosomal Storage
Disorders program to encourage early diagnosis. As part of the
collaboration, Invitae will also provide educational information to
patients and clinicians regarding clinical trials.
- Recently published preclinical data in
peer-reviewed journals support advancement into the clinic of
PBGM01 and PBFT02 – Passage Bio earlier announced
the publications of preclinical data from the University of
Pennsylvania’s Gene Therapy Program supporting the potential of
PBGM01 and PBFT02 to correct the underlying genetic defect
associated with GM1 and frontotemporal dementia (FTD) with a
granulin (GRN) mutation, respectively. Data from a mouse model of
GM1 demonstrated that a single intracerebroventricular injection of
an optimized adeno-associated virus (AAV) into the cerebral spinal
fluid (CSF) resulted in significant expression of β-gal in the
brain and peripheral tissues, and demonstrated dose-related
reductions in neuronal lysosomal storage lesions, reduced
neurological impairment and improvement in survival. Data from the
FTD mouse model showed a single administration of an AAV containing
the GRN gene resulted in elevated levels of progranulin in the
brain and CSF, reduced lysosomal storage lesions, normalized
lysosomal enzyme expression and corrected microgliosis. These GM1
data were published in Human Gene Therapy, and FTD-GRN data were
published in Annals of Clinical and Translational Neurology.
Anticipated Upcoming
Milestones
- Initiate Phase 1/2 trial for the Company’s lead program,
PBGM01, for the treatment of patients with infantile GM1 in the
first quarter of 2021. Report initial 30-day safety and biomarker
data mid-year 2021.
- Continue to advance lead programs PBFT02 for the treatment of
FTD-GRN and PBKR03 for the treatment of Krabbe disease toward Phase
1/2 clinical trial initiations in the first half of 2021.
- Continue to advance pre-clinical programs for PBML04
(Metachromatic leukodystrophy), PBLA05 (Amyotrophic lateral
sclerosis) and PBCM06 (Charcot-Marie-Tooth Disease Type
2A).
Third Quarter 2020 Financial
Results
- Cash
Position: Cash,
cash equivalents and marketable securities were $335.7 million as
of September 30, 2020 as compared to $158.9 million as of
December 31, 2019.
- Research and Development (R&D)
Expenses: R&D
expenses were $20.8 million for the quarter ended September 30,
2020, compared to $10.4 million for the same quarter in
2019.
- General and Administrative (G&A)
Expenses: G&A expenses were $7.8 million
for the quarter ended September 30, 2020, compared to $1.2 million
for the same quarter in 2019.
- Net Loss: Net loss was
$28.5 million, or a net loss of $0.63 per basic and diluted share,
for the quarter ended September 30, 2020, compared to $11.4
million, or a net loss of $2.68 per basic and diluted share, for
the same quarter in 2019.
Conference Call Details
Passage 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 5679946. A live audio webcast of the
event will be available on the Investors & Media 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 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.
Passage Bio,
Inc.Balance
Sheets(unaudited)
(in thousands, except share
data) |
|
September 30, 2020 |
|
December 31, 2019 |
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
200,570 |
|
|
$ |
158,874 |
|
Marketable securities |
|
|
135,131 |
|
|
|
— |
|
Prepaid expenses |
|
|
1,748 |
|
|
|
156 |
|
Prepaid research and development |
|
|
12,058 |
|
|
|
6,745 |
|
Total current assets |
|
|
349,507 |
|
|
|
165,775 |
|
Property and equipment,
net |
|
|
915 |
|
|
|
1,087 |
|
Other assets |
|
|
8,269 |
|
|
|
11,751 |
|
Total assets |
|
$ |
358,691 |
|
|
$ |
178,613 |
|
Liabilities,
convertible preferred stock and stockholders’ equity
(deficit) |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
3,441 |
|
|
$ |
629 |
|
Accrued expenses and other current liabilities |
|
|
15,662 |
|
|
|
3,052 |
|
Total current liabilities |
|
|
19,103 |
|
|
|
3,681 |
|
Deferred rent |
|
|
643 |
|
|
|
504 |
|
Other liabilities |
|
|
42 |
|
|
|
76 |
|
Total liabilities |
|
|
19,788 |
|
|
|
4,261 |
|
Convertible preferred stock,
$0.0001 par value: |
|
|
|
|
|
|
Series A‑1 convertible preferred stock: 63,023,258 shares
authorized, issued and outstanding at
December 31, 2019 |
|
|
— |
|
|
|
74,397 |
|
Series A‑2 convertible preferred stock: 22,209,301 shares
authorized; issued and outstanding at
December 31, 2019 |
|
|
— |
|
|
|
46,311 |
|
Series B convertible preferred stock: 33,592,907 shares
authorized, issued and outstanding at
December 31, 2019 |
|
|
— |
|
|
|
109,897 |
|
Total convertible preferred stock |
|
|
— |
|
|
|
230,605 |
|
Commitments and Contingencies
(note 7) |
|
|
|
|
|
|
Stockholders’ equity
(deficit): |
|
|
|
|
|
|
Common stock, $0.0001 par value: 300,000,000 shares authorized;
45,885,052 shares issued and 45,543,682 shares outstanding at
September 30, 2020 and 5,194,518 shares issued and
4,293,039 shares outstanding at December 31, 2019 |
|
|
4 |
|
|
|
— |
|
Additional paid‑in capital |
|
|
470,890 |
|
|
|
2,410 |
|
Accumulated other comprehensive loss |
|
|
(40 |
) |
|
|
— |
|
Accumulated deficit |
|
|
(131,951 |
) |
|
|
(58,663 |
) |
Total stockholders’ equity (deficit) |
|
|
338,903 |
|
|
|
(56,253 |
) |
Total liabilities, convertible preferred stock and stockholders’
equity (deficit) |
|
$ |
358,691 |
|
|
$ |
178,613 |
|
|
|
|
|
|
|
|
|
|
Statements of
Operations(unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
|
Nine Months Ended
September 30, |
(in thousands, except share and per share
data) |
|
2020 |
|
|
2019 |
|
|
|
2020 |
|
|
2019 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
20,837 |
|
|
$ |
10,434 |
|
|
|
$ |
53,856 |
|
|
$ |
19,766 |
|
Acquired in‑process research and development |
|
|
— |
|
|
|
— |
|
|
|
|
— |
|
|
|
500 |
|
General and administrative |
|
|
7,793 |
|
|
|
1,209 |
|
|
|
|
19,990 |
|
|
|
3,331 |
|
Loss from operations |
|
|
(28,630 |
) |
|
|
(11,643 |
) |
|
|
|
(73,846 |
) |
|
|
(23,597 |
) |
Change in fair value of future
tranche right liability |
|
|
— |
|
|
|
— |
|
|
|
|
— |
|
|
|
(9,141 |
) |
Interest income, net |
|
|
99 |
|
|
|
255 |
|
|
|
|
558 |
|
|
|
255 |
|
Net loss |
|
$ |
(28,531 |
) |
|
$ |
(11,388 |
) |
|
|
$ |
(73,288 |
) |
|
$ |
(32,483 |
) |
Per share information: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share of common
stock, basic and diluted |
|
$ |
(0.63 |
) |
|
$ |
(2.68 |
) |
|
|
$ |
(2.02 |
) |
|
$ |
(7.70 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
|
45,503,794 |
|
|
|
4,248,835 |
|
|
|
|
36,273,495 |
|
|
|
4,218,907 |
|
Comprehensive loss: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
$ |
(28,531 |
) |
|
$ |
(11,388 |
) |
|
|
$ |
(73,288 |
) |
|
$ |
(32,483 |
) |
Unrealized loss on available-for-sale investments |
|
|
(40 |
) |
|
|
— |
|
|
|
|
(40 |
) |
|
|
— |
|
Comprehensive loss |
|
$ |
(28,571 |
) |
|
$ |
(11,388 |
) |
|
|
$ |
(73,328 |
) |
|
$ |
(32,483 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
For further information, please
contact:
Investors:Sarah McCabe and Zofia MitaStern Investor Relations,
Inc.sarah.mccabe@sternir.comzofia.mita@sternir.com
Media:Gwen FisherPassage
Bio215.407.1548gfisher@passagebio.com
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