Passage Bio Reports Second Quarter 2020 Financial Results and Recent Business Highlights
August 13 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 second quarter ended June 30, 2020 and provided recent
business highlights.
“We have made substantial progress over the past
quarter, including the submission of our first IND application to
the FDA for a Phase 1/2 clinical trial in infantile GM1 patients
with PBGM01, demonstrating our team’s ability to work
collaboratively with our partners at the Gene Therapy Program led
by Dr. James Wilson,” said Gary Romano, M.D., Ph.D, chief medical
officer of Passage Bio. “We are confident that we can efficiently
and successfully address the FDA clinical hold questions related to
biocompatibility of our proposed ICM delivery device so that we can
begin to dose patients before the end of this year or early next
year. Importantly, we continue to believe that the initial clinical
safety and biomarker data from this trial will be available late in
the first half of 2021.”
Bruce Goldsmith, Ph.D., president and chief
executive officer of Passage Bio, said: “I am proud of the
world-class team we are building at Passage Bio, including
substantial expansion of the clinical, manufacturing, and corporate
operation teams. We have also been effective at navigating the
unpredictable environment caused by COVID-19 both internally and
with our key external partners to maintain our pipeline
advancement. This has enabled us to make significant progress
toward delivering on the promise of PBGM01 for patients suffering
from GM1. We look forward to treating our first patient as well as
demonstrating the potential of all our investigational product
candidates as safe and effective treatment options for devastating
rare diseases like GM1. With our operational progress and the
continued strength of our balance sheet, together with our robust
pipeline, we are well positioned to achieve these goals.”
Recent Business Highlights
- Received feedback from U.S. Food and Drug
Administration (FDA) on Investigational New Drug (IND) application
for PBGM01 – In June 2020, Passage Bio submitted its first
ever IND for PBGM01 for the treatment of GM1 gangliosidosis to the
FDA in collaboration with the University of Pennsylvania’s Gene
Therapy Program. Following this submission, the Company was
notified that the IND was placed on clinical hold pending
additional risk assessments of the biocompatibility of the proposed
ICM delivery device. The ICM route of administration delivers
PBGM01 directly to the brain into the cisterna magna, a space
within the lower portion of the brain, with techniques and delivery
devices commonly used both in current medical practice and other
clinical trials, including those for gene therapy. The Company is
evaluating options for conducting additional risk assessments while
it awaits official written feedback from the FDA. During the IND
review, the Company addressed specific clinical and protocol
questions raised by the FDA, and the agency confirmed that there
are no further clinical information requests. As a result of the
clinical hold, the Company now expects to initiate dosing of its
Phase 1/2 trial late in the fourth quarter of 2020 or early in the
first quarter of 2021 and remains on track to report initial 30 day
safety and biomarker data late in the first half of 2021.
- Added to Russell 2000® Index – In June 2020,
Passage Bio was added to the Russell 2000® Index, a subset of the
Russell 3000® Index, which measures the performance of the
small-cap segment of the U.S. equity market.
- Expanded gene therapy collaboration with the University
of Pennsylvania (UPenn) – In May 2020, Passage Bio
expanded its research and development collaboration and licensing
agreement with the University of Pennsylvania. The amendment
increased the number of remaining available licensing options for
programs to treat rare monogenic CNS disorders from six to eleven
and extended the window for the exercise of options by three years.
Accordingly, the window to exercise all eleven remaining options
extends to May 2025. The Company also received exclusive rights and
licenses, subject to limitations, to certain technologies resulting
from discovery research at Gene Therapy Program (GTP) for Passage
Bio products developed with GTP, such as novel capsids, toxicity
reduction technologies and delivery and formulation
improvements.
- Announced presentation of preclinical data models of
Krabbe Disease at American Society of Gene & Cell Therapy
(ASGCT) 23rd Annual Meeting – In May 2020, the University
of Pennsylvania’s Gene Therapy Program, Passage Bio’s preclinical
development partner, presented encouraging preclinical data from a
Krabbe dog and Twitcher mouse model at the ASGCT Annual Meeting
demonstrating the potential of an AAVhu68 capsid carrying a
functional GALC gene to normalize GALC enzyme activity and nerve
conduction, leading to dose-dependent phenotypic correction and
increased survival. Passage Bio is currently developing an AAVhu68
GALC therapeutic, PBKR03, for the treatment of infantile Krabbe
disease and expects to initiate a Phase 1/2 trial in the first half
of 2021.
- Granted Orphan Drug and Rare Pediatric Disease
designation by FDA for PBGM01 – In April and May 2020,
respectively, the FDA granted Orphan Drug and Rare Pediatric
Disease designation to PBGM01 for the treatment of infantile GM1.
Orphan Drug designation is intended to promote the development of
safe and effective treatments for rare diseases through certain
financial and market exclusivity incentives, and Rare Pediatric
Disease designation is intended to encourage treatments for
life-threatening disease affecting children of 18 years of age or
younger by qualifying the sponsor for a priority review voucher
upon approval that may be sold or transferred.
Anticipated Upcoming Milestones
- Initiate a Phase 1/2 trial for the lead program, PBGM01, for
the treatment of patients with infantile GM1 late in the fourth
quarter of 2020 or early in the first quarter of 2021. Report
initial 30-day safety and biomarker data late in the first half of
2021.
- Continue to advance lead programs PBFT02 for the treatment of
frontotemporal dementia (FTD) and PBKR03 for the treatment of
Krabbe disease toward clinical trial initiations in the first half
of 2021.
- Continue to advance PBML04, PBLA05 and PBCM06 toward
IND-enabling studies.
Second Quarter 2020 Financial Results
- Cash Position: Cash and
cash equivalents were $353.4 million as of June 30, 2020 as
compared to $158.9 million as of December 31, 2019.
- Research and Development (R&D)
Expenses: R&D expenses were
$19.9 million for the quarter ended June 30, 2020, compared to $6.3
million for the same quarter in 2019. The increase was
primarily due to an increase of $4.5 million in costs incurred with
the University of Pennsylvania in preparation for several IND
filings, an increase of $4.0 million in clinical manufacturing
costs, a $1.4 million increase in clinical development costs and a
$0.5 million increase in consulting expense as we prepare for our
clinical trials to begin in the second half of 2020 and early 2021.
The Company also had a $3.1 million increase in
personnel‑related costs and a $0.1 million increase in facility and
other costs due to increases in employee headcount in the R&D
function.
- General and Administrative (G&A)
Expenses: G&A expenses were $7.4 million for the
quarter ended June 30, 2020, compared to $1.0 million for the same
quarter in 2019. The increase was primarily due to a $4.7
million increase in personnel-related and share‑based compensation
expense due to increases in employee headcount. The Company’s
professional fees and facility costs also increased by
$0.7 million and $1.0 million, respectively, as Passage
Bio expanded its operations to support its R&D efforts.
- Net Loss: Net loss was $27.2 million, or
a net loss of $0.60 per basic and diluted share, for the quarter
ended June 30, 2020, compared to $13.4 million, or a net loss of
$3.19 per basic and diluted share, for the quarter ended June 30,
2019.
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
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 Passage Bio is a genetic
medicines company focused on developing transformative therapies
for rare, monogenic central nervous system disorders with limited
or no approved treatment options. The company is based in
Philadelphia, PA and has a research, collaboration and license
agreement with the University of Pennsylvania and its Gene Therapy
Program (GTP). The GTP conducts discovery and IND-enabling
preclinical work and Passage Bio conducts all clinical development,
regulatory strategy and commercialization activities under the
agreement. The company has a development portfolio of six product
candidates, with the option to license eleven more, with lead
programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe
disease.
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 our planned IND submissions,
resolution of the clinical hold on PBGM01, initiation of clinical
trials and the availability of clinical data from such trials; our
cash forecasts, our expectations about our collaborators’ and
partners’ ability to execute key initiatives; 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, obtain regulatory
approval for and commercialize our product candidates; the timing
and results of preclinical studies and clinical trials; 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; 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; failure to protect and enforce our
intellectual property, and other proprietary rights; failure to
successfully execute or realize the anticipated benefits of our
strategic and growth initiatives; risks relating to technology
failures or breaches; our dependence on collaborators and other
third parties for the development 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 COVID-19 pandemic; risks
associated with current and potential future healthcare reforms;
risks relating to attracting and retaining key personnel; failure
to comply with legal and regulatory requirements; risks relating to
access to capital and credit markets; 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) |
|
|
|
|
|
|
June
30, |
|
December
31, |
(in thousands, except share data) |
|
2020 |
|
2019 |
Assets |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
353,423 |
|
|
$ |
158,874 |
|
Prepaid expenses |
|
|
2,176 |
|
|
|
156 |
|
Prepaid research and development |
|
|
12,631 |
|
|
|
6,745 |
|
Total current assets |
|
|
368,230 |
|
|
|
165,775 |
|
Property and equipment, net |
|
|
1,096 |
|
|
|
1,087 |
|
Other assets |
|
|
8,771 |
|
|
|
11,751 |
|
Total assets |
|
$ |
378,097 |
|
|
$ |
178,613 |
|
Liabilities, convertible preferred stock and stockholders’
equity (deficit) |
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
|
$ |
7,214 |
|
|
$ |
629 |
|
Accrued expenses and other current liabilities |
|
|
6,920 |
|
|
|
3,052 |
|
Total current liabilities |
|
|
14,134 |
|
|
|
3,681 |
|
Deferred rent |
|
|
524 |
|
|
|
504 |
|
Other liabilities |
|
|
43 |
|
|
|
76 |
|
Total liabilities |
|
|
14,701 |
|
|
|
4,261 |
|
Convertible preferred stock, $0.0001 par value: |
|
|
|
|
Series A‑1 convertible preferred stock: No shares authorized,
issued and outstanding at March 31, 2020; 63,023,258 shares
authorized, issued and outstanding at December 31, 2019 |
|
|
- |
|
|
|
74,397 |
|
Series A‑2 convertible preferred stock: No shares authorized,
issued and outstanding at March 31, 2020; 22,209,301 shares
authorized; issued and outstanding at December 31, 2019 |
|
|
- |
|
|
|
46,311 |
|
Series B convertible preferred stock: No shares authorized, issued
and outstanding at March 31, 2020; 33,592,907 shares authorized,
issued and outstanding at December 31, 2019 |
|
|
- |
|
|
|
109,897 |
|
Total convertible preferred stock |
|
|
- |
|
|
|
230,605 |
|
Stockholders’ equity (deficit) : |
|
|
|
|
Common stock, $0.0001 par value: 100,000,000 shares authorized;
45,797,195 shares issued and 45,350,687 shares outstanding at March
31, 2020 and 5,194,518 shares issued and 4,293,039 shares
outstanding at December 31, 2019 |
|
|
4 |
|
|
|
- |
|
Additional paid-in capital |
|
|
466,812 |
|
|
|
2,410 |
|
Accumulated deficit |
|
|
(103,420 |
) |
|
|
(58,663 |
) |
Total stockholders' equity (deficit) |
|
|
363,396 |
|
|
|
(56,253 |
) |
Total liabilities, convertible preferred stock and
stockholders' equity (deficit) |
|
|
|
$ |
378,097 |
|
|
$ |
178,613 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Statements of Operations |
|
|
|
|
|
|
|
|
|
(unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended June 30, |
|
Six Months
Ended June 30, |
(in thousands, except share and per share
data) |
|
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
$ |
19,902 |
|
|
$ |
6,299 |
|
|
$ |
33,019 |
|
|
$ |
9,332 |
|
Acquired in-process research and development |
|
|
|
- |
|
|
|
500 |
|
|
|
- |
|
|
|
500 |
|
General and administrative |
|
|
|
7,402 |
|
|
|
968 |
|
|
|
12,197 |
|
|
|
2,122 |
|
Loss from operations |
|
|
|
(27,304 |
) |
|
|
(7,767 |
) |
|
|
(45,216 |
) |
|
|
(11,954 |
) |
Change in fair value of future tranche right liability |
|
|
|
- |
|
|
|
(5,659 |
) |
|
|
- |
|
|
|
(9,141 |
) |
Interest income |
|
|
|
132 |
|
|
|
- |
|
|
|
459 |
|
|
|
- |
|
Net loss |
|
|
|
$ |
(27,172 |
) |
|
$ |
(13,426 |
) |
|
$ |
(44,757 |
) |
|
$ |
(21,095 |
) |
Per share information: |
|
|
|
|
|
|
|
|
|
Net loss per share of common stock, basic and diluted |
|
|
$ |
(0.60 |
) |
|
$ |
(3.19 |
) |
|
$ |
(1.42 |
) |
|
$ |
(5.02 |
) |
Weighted average common shares outstanding, basic and diluted |
|
|
|
45,386,308 |
|
|
|
4,209,716 |
|
|
|
31,581,851 |
|
|
|
4,203,694 |
|
|
|
|
|
|
|
|
|
|
|
|
|
For further information, please contact:
Investors:Sarah McCabe and Zofia MitaStern Investor Relations,
Inc.sarah.mccabe@sternir.com zofia.mita@sternir.com
Media:Gwen FisherPassage
Bio215.407.1548gfisher@passagebio.com
Passage Bio (NASDAQ:PASG)
Historical Stock Chart
From Sep 2024 to Oct 2024
Passage Bio (NASDAQ:PASG)
Historical Stock Chart
From Oct 2023 to Oct 2024