PHILADELPHIA, PA — Passage Bio, Inc. (Nasdaq: PASG) this week reported financial results for the fourth quarter and full-year ended December 31, 2022, and provided recent business highlights.
“We recently presented encouraging interim safety, biomarker and efficacy data on PBGM01 in the first 3 cohorts in our Imagine-1 study for GM1 gangliosidosis, giving us further confidence that PBGM01 continues to exert a biological effect across infantile GM1 patients. We look forward to continuing to generate data with a planned Cohort 4 data readout expected mid-year, and based on the promising data we’ve seen so far, expanding the study to treat patients at an even higher dose,” said William Chou, M.D., chief executive officer of Passage Bio. “We are also pleased to report that enrollment efforts are well underway in our upliFT-D trial in FTD, and we expect to report initial safety and biomarker data from Cohort 1 in the second half of this year. 2023 is an exciting year for Passage as we focus on operational execution and generation of clinical data across our two lead programs. We are supported by a strong balance sheet with cash runway into the first half of 2025 and remain committed to generating value for both patients and stakeholders as we work to develop life transforming therapeutics for devastating CNS disorders.”
- Presented interim data at 19th Annual WORLDSymposium™ showing continued biological effect of PBGM01 in all 6 patients in Cohorts 1-3 in Imagine-1 study: Interim efficacy data showed administration of PBGM01 resulted in stabilization of MRI severity scores in all treated patients through 6 to twelve months of follow-up. Interim biomarker data showed PBGM01 administration led to decreases in β-Gal substrate Dp5 levels in urine, an exploratory biomarker for peripheral β-Gal activity. These data build upon the interim data reported by the company in December 2022, which showed PBGM01 administration was well tolerated and had a favorable safety profile and led to dose dependent increases in CSF β-Gal activity and dose dependent decreases in CSF GM1 ganglioside levels.
- Plan to expand dose escalation portion of Imagine-1 study to treat patients at a higher dose of PBGM01 to inform registrational dose selection: Based on the favorable safety profile of PBGM01 observed in the Imagine-1 study to date, the observed dose-response in key biomarkers, and supporting preclinical safety data, the company intends to treat additional patients at a higher dose than has currently been evaluated. Additionally, the company is revising the study inclusion criteria to maximize the benefit-risk profile of PBGM01. Treating patients at the higher dose will provide important clinical data to determine dose selection and inform discussions with regulatory authorities on the confirmatory study design. Following regulatory review, the company expects to dose the first patient at the higher dose in the second half of 2023.
- Expect to report initial safety and biomarker data from Cohort 1 patients in upliFT-D trial of PBFT02 for the treatment of patients with frontotemporal dementia (FTD) with granulin mutations (GRN) in the second half of 2023: upliFT-D is a global Phase 1/2, multi-center, open-label, dose-escalation study of PBFT02 administered by single injection into the cisterna magna in patients aged 35 to 75 years with early symptomatic FTD-GRN. Enrollment is ongoing at three sites globally.
Anticipated Upcoming Milestones
- Present initial safety and biomarker data for Cohort 4 (early infantile, high dose) in Imagine-1 clinical trial for GM1 in mid-2023.
- Dose first patient at higher dose in Imagine-1 clinical trial for GM1 in the second half of 2023.
- Present initial safety and biomarker data for Cohort 1 in upliFT-D trial for FTD in the second half of 2023.
Fourth Quarter and Full-Year 2022 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were $189.6 million as of December 31, 2022, as compared to $315.8 million as of December 31, 2021. The Company expects current cash, cash equivalents and marketable securities to fund operations into the first half of 2025.
- Research and Development (R&D) Expenses: R&D expenses were $17.7 million for the quarter ended December 31, 2022, and $86.1 million for the year ended December 31, 2022, compared to $33.0 million and $117.7 million for the same quarter and year in 2021.
- General and Administrative (G&A) Expenses: G&A expenses were $10.6 million for the quarter ended December 31, 2022, and $49.3 million for the year ended December 31, 2022, compared to $17.2 million and $60.1 million for the same quarter and year in 2021.
- Net Loss: Net loss was $27.1 million, or a net loss of $0.50 per basic and diluted share, for the quarter and $136.1 million, or a net loss of $2.50 per basic and diluted share, for the year ended December 31, 2022, compared to $51.2 million, or a net loss of $0.95 per basic and diluted share, for the quarter and $185.4 million, or a net loss of $3.48 share, for the year ended December 31, 2021.
Additional details can be found in the Investors & News section of Passage Bio’s website at investors.passagebio.com.
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