Full Press Release Details
Bio Reports Second Quarter 2020 Financial Results and Recent Business Highlights
Submitted IND for PBGM01 Phase 1/2 trial; currently on FDA clinical hold pending additional review of proposed ICM delivery
Anticipate dosing first patient in PBGM01 Phase 1/2 trial in late 4Q2020 or early 1Q2021 -
Initial PBGM01 clinical safety and biomarker data remains on track for 1H2021 read out -
Strong cash balance of $353M expected to fund operations into 2023 -
Management to host conference call today at 8:30 a.m. ET -
Philadelphia, PA - August
13, 2020 - 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
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
Anticipated Upcoming Milestones
Second Quarter 2020 Financial
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.
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 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, 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.
| 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
| 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
Sarah McCabe and Zofia Mita
Stern Investor Relations, Inc.