03/03/2021 | Press release | Distributed by Public on 03/03/2021 06:05
- On track to initiate three Phase 1/2 clinical programs in the first half of 2021: PBGM01 for GM1 gangliosidosis (GM1), PBFT02 for frontotemporal dementia with granulin mutations, and PBKR03 for Krabbe disease -
- Opened first U.S. site for PBGM01's Imagine-1 global clinical program and actively recruiting patients; other site activations in progress -
- Opening new CMC research and development site to support analytics, assay development, and product testing in 2Q 2021 -
- Strengthened financial position with recent public offering that raised $166M in net proceeds -
- Management to host conference call today at 8:30 a.m. ET -
PHILADELPHIA, March 03, 2021 (GLOBE NEWSWIRE) -- 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 fourth quarter and full year ended December 31, 2020 and provided recent business highlights.
'At Passage Bio, we are on a mission to provide life-transforming therapies to patients with devastating CNS diseases, and we are pleased with the foundational work we accomplished in 2020 as we transition to a clinical-stage company in 2021,' said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. 'In the year ahead, our priorities are progressing three clinical development programs, all set to begin in the first half of 2021, and continuing to expand our pipeline and internal operations. We are confident that with the expertise of our talented internal team, our partnership with University of Pennsylvania's Gene Therapy Program, and our relationships with patient and physician groups that we are well positioned for strong execution across all our upcoming milestones.'
Recent Corporate Highlights
Anticipated Upcoming Milestones
Fourth Quarter and Full Year 2020 Financial Results
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 6758094. 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 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:
Investors:
Stuart Henderson
Passage Bio
[email protected]
Media:
Gwen Fisher
Passage Bio
215.407.1548
[email protected]
Passage Bio, Inc.
Balance Sheets
(unaudited)
December 31, | ||||||||
(in thousands, except share data) | 2020 | 2019 | ||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 135,002 | $ | 158,874 | ||||
Marketable securities | 169,815 | - | ||||||
Prepaid expenses | 1,405 | 156 | ||||||
Prepaid research and development | 10,961 | 6,745 | ||||||
Total current assets | 317,183 | 165,775 | ||||||
Other assets | 8,029 | 11,751 | ||||||
Total assets | $ | 328,007 | $ | 178,613 | ||||
Liabilities, convertible preferred stock and stockholders' equity (deficit) | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 5,265 | $ | 629 | ||||
Accrued expenses and other current liabilities | 15,910 | 3,052 | ||||||
Total current liabilities | 21,175 | 3,681 | ||||||
Deferred rent | 2,077 | 504 | ||||||
Other liabilities | 41 | 76 | ||||||
Total liabilities | 23,293 | 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; 45,917,084 shares issued and 45,614,807 shares outstanding at December 31, 2020 and 5,194,518 shares issued and 4,293,039 shares outstanding at December 31, 2019 | 4 | - | ||||||
Additional paid‑in capital | 475,617 | 2,410 | ||||||
Accumulated other comprehensive loss | (12 | ) | - | |||||
Accumulated deficit | (170,895 | ) | (58,663 | ) | ||||
Total stockholders' equity (deficit) | 304,714 | (56,253 | ) | |||||
Total liabilities, convertible preferred stock and stockholders' equity (deficit) | $ | 328,007 | $ | 178,613 |
Statements of Operations and Comprehensive Loss
(unaudited)
Three Months Ended December 31, | Year Ended December 31, | |||||||||||||||
(in thousands, except share and per share data) | 2020 | 2019 | 2020 | 2019 | ||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 27,932 | $ | 9,972 | $ | 81,788 | $ | 29,738 | ||||||||
Acquired in‑process research and development | 1,000 | - | 1,000 | 500 | ||||||||||||
General and administrative | 10,124 | 3,620 | 30,114 | 6,951 | ||||||||||||
Loss from operations | (39,056 | ) | (13,592 | ) | (112,902 | ) | (37,189 | ) | ||||||||
Change in fair value of future tranche right liability | - | - | - | (9,141 | ) | |||||||||||
Interest income, net | 112 | 441 | 670 | 696 | ||||||||||||
Net loss | $ | (38,944 | ) | $ | (13,151 | ) | $ | (112,232 | ) | $ | (45,634 | ) | ||||
Per share information: | ||||||||||||||||
Net loss per share of common stock, basic and diluted | $ | (0.85 | ) | $ | (3.07 | ) | $ | (2.91 | ) | $ | (10.77 | ) | ||||
Weighted average common shares outstanding, basic and diluted | 45,571,132 | 4,290,598 | 38,615,967 | 4,236,061 | ||||||||||||
Comprehensive loss: | ||||||||||||||||
Net loss | $ | (38,944 | ) | $ | (13,151 | ) | $ | (112,232 | ) | $ | (45,634 | ) | ||||
Unrealized loss on available-for-sale securities | 28 | - | (12 | ) | - | |||||||||||
Comprehensive loss | $ | (38,916 | ) | $ | (13,151 | ) | $ | (112,244 | ) | $ | (45,634 | ) |