Full Press Release Details
Passage Bio Reports Third Quarter 2020 Financial Results and Recent Business Highlights
- Dosing for first patient in PBGM01 Phase 1/2 trial anticipated in 1Q2021; initial safety and biomarker data expected mid-year 2021 -
- Phase 1/2 clinical trials for FTD-GRN and Krabbe expected to initiate in 1H21 -
- Completion of dedicated manufacturing suite achieved to ensure control of supply of company's lead products through early commercialization -
- Management to host conference call today at 8:30 a.m. ET -
Philadelphia, PA - November 10, 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 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
Anticipated Upcoming Milestones
Third Quarter 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 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.
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.
| (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 45,534,682 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 and Comprehensive Loss
| 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 securities | (40) | - | (40) | - | |||||||||
| Comprehensive loss | $ | (28,571) | $ | (11,388) | $ | (73,328) | $ | (32,483) |
For further information, please contact:
Sarah McCabe and Zofia Mita
Stern Investor Relations, Inc.