Enrolled first FTD-GRN patient in Cohort 2 in upliFT-D trial
Plan to present updated safety and biomarker data from Cohort 1 FTD-GRN patients treated with PBFT02 at 14th International Conference on Frontotemporal Dementias (ISFTD2024) in September 2024
Achieved alignment with Food and Drug Administration (FDA) on the company's proposed clinical development plans to expand the upliFT-D trial to assess PBFT02 in FTD patients with C9orf72 gene mutations
Completed out-licensing of pediatric lysosomal storage disease programs to GEMMA Biotherapeutics
Strong balance sheet to support continued execution, with cash runway to fund operations extended to the end of Q2 2026
PHILADELPHIA, Aug. 08, 2024 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported financial results for the second quarter ended June 30, 2024 and provided recent business highlights.
"This has been a pivotal quarter for our company, marked by significant momentum in our upliFT-D trial, increased clarity around our strategy to expand PBFT02 into additional adult neurodegenerative indications, and out-licensing of our pediatric lysosomal storage disorder programs," said Will Chou, M.D., president and chief executive officer of Passage Bio. "The positive recommendation of the IDMC to proceed to Cohort 2 dosing in our upliFT-D trial for FTD-GRN reinforces the well-tolerated safety profile of PBFT02 when combined with our revised immunosuppression regimen, and we are excited to present updated safety and biomarker data from Cohort 1 at the ISFTD2024 conference in Amsterdam this September. We are also pleased the FDA is aligned with our proposed expansion of our upliFT-D trial to include FTD-C9orf72 patients and expect to initiate dosing of these patients in the first half of 2025. The company remains on track to gain regulatory feedback on the pathway to treating ALS patients with PBFT02 in the second half of 2024."
Dr. Chou continued, "Furthermore, we completed the out-licensing of our pediatric lysosomal storage disease programs to GEMMA Biotherapeutics. Dr. Wilson and the entire GEMMABio team are committed to developing these promising therapies for pediatric patients with life-threatening conditions. We look forward to our continued research and collaboration efforts with their team, driving advancements and hope for those affected by neurodegenerative diseases."
Recent Highlights
Anticipated Upcoming Milestones:
FTD-GRN
FTD-C9orf72 and ALS
Second Quarter 2024 Financial Results
About Passage Bio
Passage Bio (NASDAQ:PASG) is a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases. Our primary focus is the development and advancement of cutting-edge, one-time therapies designed to target the underlying pathology of these conditions. Passage Bio's lead product candidate, PBFT02, seeks to treat neurodegenerative conditions, including frontotemporal dementia, by elevating progranulin levels to restore lysosomal function and slow disease progression.
To learn more about Passage Bio and our steadfast commitment to protecting patients and families against loss in neurodegenerative conditions, please visit: 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 the initiation of dosing of FTD-C9orf72 patients, feedback from regulatory authorities, the progress of clinical studies and the availability of clinical data from such trials; our expectations about our collaborators' and partners' ability to execute key initiatives; our ability to receive milestone payments from our partners; our expectations about cash runway; and the ability of our product candidates to treat their respective target 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; 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 and per share data) | June 30, 2024 | December 31, 2023 | ||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 24,770 | $ | 21,709 | ||||
| Marketable securities | 67,003 | 92,585 | ||||||
| Prepaid expenses and other current assets | 1,397 | 923 | ||||||
| Prepaid research and development | 1,830 | 2,742 | ||||||
| Total current assets | 95,000 | 117,959 | ||||||
| Property and equipment, net | 13,054 | 15,295 | ||||||
| Right of use assets - operating leases | 16,822 | 16,858 | ||||||
| Other assets | 516 | 433 | ||||||
| Total assets | $ | 125,392 | $ | 150,545 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 621 | $ | 1,298 | ||||
| Accrued expenses and other current liabilities | 8,063 | 11,670 | ||||||
| Operating lease liabilities | 3,701 | 3,373 | ||||||
| Total current liabilities | 12,385 | 16,341 | ||||||
| Operating lease liabilities - noncurrent | 22,450 | 22,921 | ||||||
| Total liabilities | 34,835 | 39,262 | ||||||
| Stockholders' equity: | ||||||||
| Preferred stock, $0.0001 par value: 10,000,000 shares authorized; no shares issued and outstanding at both June 30, 2024 and December 31, 2023 | — | — | ||||||
| Common stock, $0.0001 par value: 300,000,000 shares authorized; 61,754,786 shares issued and outstanding at June 30, 2024 and 54,944,130 shares issued and outstanding at December 31, 2023 | 6 | 5 | ||||||
| Additional paid‑in capital | 717,788 | 705,789 | ||||||
| Accumulated other comprehensive income (loss) | (67 | ) | (43 | ) | ||||
| Accumulated deficit | (627,170 | ) | (594,468 | ) | ||||
| Total stockholders' equity | 90,557 | 111,283 | ||||||
| Total liabilities and stockholders' equity | $ | 125,392 | $ | 150,545 | ||||
| Passage Bio, Inc. Statements of Operations and Comprehensive Loss (Unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| (in thousands, except share and per share data) | 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 10,430 | $ | 17,324 | $ | 21,965 | $ | 34,160 | ||||||||
| General and administrative | 6,510 | 8,064 | 13,025 | 27,111 | ||||||||||||
| Impairment of long-lived assets | 438 | — | 438 | — | ||||||||||||
| Loss from operations | (17,378 | ) | (25,388 | ) | (35,428 | ) | (61,271 | ) | ||||||||
| Other income (expense), net | 1,387 | 1,532 | 2,726 | 3,077 | ||||||||||||
| Net loss | $ | (15,991 | ) | $ | (23,856 | ) | $ | (32,702 | ) | $ | (58,194 | ) | ||||
| Per share information: | ||||||||||||||||
| Net loss per share of common stock, basic and diluted | $ | (0.26 | ) | $ | (0.44 | ) | $ | (0.55 | ) | $ | (1.06 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 61,682,475 | 54,683,817 | 58,989,007 | 54,651,488 | ||||||||||||
| Comprehensive loss: | ||||||||||||||||
| Net loss | $ | (15,991 | ) | $ | (23,856 | ) | $ | (32,702 | ) | $ | (58,194 | ) | ||||
| Unrealized gain (loss) on marketable securities | 2 | 87 | (24 | ) | 626 | |||||||||||
| Comprehensive loss | $ | (15,989 | ) | $ | (23,769 | ) | $ | (32,726 | ) | $ | (57,568 | ) | ||||
For further information, please contact:
Investors:
Stuart Henderson
Passage Bio
267.866.0114
shenderson@passagebio.com
Media:
Mike Beyer
Sam Brown Inc. Healthcare Communications
312.961.2502
MikeBeyer@sambrown.com
