Financial Performance - The net loss for the three months ended September 30, 2023, was $19,340,000, compared to a net loss of $27,110,000 for the same period last year, representing a 28.5% improvement[12]. - The net loss per share for the three months ended September 30, 2023, was $(0.31), compared to $(0.49) for the same period last year[12]. - Comprehensive loss for the three months ended September 30, 2023, was $19,241,000, compared to $26,962,000 for the same period last year, indicating a 28.5% reduction[12]. - For the nine months ended September 30, 2024, the net loss was $52,042 thousand, a decrease from a net loss of $85,304 thousand for the same period in 2023, representing a 39% improvement[19]. - For the three months ended September 30, 2024, the net loss was $19.3 million, compared to a net loss of $27.1 million for the same period in 2023, representing a 29% improvement[155]. - For the nine months ended September 30, 2024, the net loss was $52.0 million, down from $85.3 million in the same period in 2023, indicating a 39% reduction in losses[155]. - As of September 30, 2024, the accumulated deficit stood at $646.5 million[155]. Research and Development - Research and development expenses for the three months ended September 30, 2023, were $8,656,000, while for the nine months ended, they totaled $30,621,000[12]. - Research and development expenses decreased by $6.4 million to $8.7 million for the three months ended September 30, 2024, from $15.1 million for the same period in 2023[186]. - Research and development expenses decreased by $18.6 million to $30.6 million for the nine months ended September 30, 2024, from $49.3 million for the same period in 2023[195]. - The company has paused development of certain research programs to reduce operating expenses, specifically a program for TLE[154]. - Research and development expenses are anticipated to remain consistent in the near future, with potential increases as product candidates progress into later-stage clinical trials[180]. Cash Flow and Liquidity - The total accumulated deficit as of September 30, 2023, was $(646,510,000)[15]. - Cash and cash equivalents at the end of the period were $32,292 thousand, slightly down from $33,579 thousand at the end of the previous period[19]. - The company had cash, cash equivalents, and marketable securities totaling $84.8 million as of September 30, 2024, expected to fund operations until the end of Q2 2026[158]. - The net cash used in operating activities for the nine months ended September 30, 2024, was $39,512 thousand, compared to $58,748 thousand for the same period in 2023, indicating a 33% reduction in cash outflow[19]. - The company expects existing cash and marketable securities will fund operating expenses and capital expenditures until the end of the second quarter of 2026[203]. Impairment and Expenses - The company reported an impairment of long-lived assets amounting to $4,795,000 for the three months ended September 30, 2023[12]. - The Company recognized impairment expenses for property and equipment of $2.3 million and $2.7 million for the three and nine months ended September 30, 2024, respectively, compared to $3.2 million for the same periods in 2023[39]. - The Company recognized an impairment expense of $4.8 million related to certain asset groups, including $2.5 million for ROU assets and $2.3 million for property and equipment, during the three and nine months ended September 30, 2024[88]. - Impairment expense of long-lived assets recorded was $4.8 million during the three months ended September 30, 2024, compared to $5.4 million for the same period in 2023[192][193]. - Impairment expense of long-lived assets recorded was $5.2 million during the nine months ended September 30, 2024, compared to $5.4 million for the same period in 2023[199][200]. Shareholder Equity and Stock Options - The weighted average common shares outstanding for the three months ended September 30, 2023, were 61,763,346, an increase from 54,789,410 shares in the prior year[12]. - The Company had 12,012,051 stock options outstanding as of September 30, 2024, with a weighted average exercise price of $3.89[118]. - The total unrecognized compensation expense related to unvested stock option awards was $6.9 million, expected to be recognized over 2.3 years[118]. - The Company has 1,379,590 shares available for future grants under the 2020 Employee Stock Purchase Plan as of September 30, 2024[122]. Collaborations and Agreements - The company entered into a new research collaboration and license agreement with GEMMA Biotherapeutics and amended its collaboration with the University of Pennsylvania[21]. - The Company entered into sublicense agreements with Gemma, receiving an initial payment of $5,000,000 for licenses and clinical product supply, with an additional $5,000,000 due in December 2024[73]. - The Gemma Collaboration Agreement includes payments of up to $16.5 million per product candidate for Huntington's disease and future CNS indications, with additional sales milestone payments of up to $55.0 million based on annual worldwide net sales[163]. - The Company is obligated to make payments of up to $16.5 million per product candidate upon achieving specific development milestones under the Penn License Agreement[96]. - The Company is required to pay tiered royalties in the mid-single digits percentage on annual worldwide net sales of licensed products upon successful commercialization[97]. Legal and Regulatory Matters - The Company recorded a $1.0 million loss contingency for a legal proceeding as of September 30, 2024[111]. - The Company is involved in litigation regarding a claim of breach of contract, with the plaintiff seeking a mid-single digit million dollar amount, and a jury awarded $1.0 million to the plaintiff on the breach of contract claim[125][126]. - The FDA has granted Orphan Drug Designation and Fast Track Designation for PBFT02 for the treatment of FTD and FTD-GRN[145]. Clinical Development - The lead clinical product candidate, PBFT02, aims to elevate progranulin levels to enhance lysosomal function and slow disease progression in neurodegenerative diseases, specifically targeting frontotemporal dementia caused by progranulin deficiency[130]. - PBFT02 has shown promising biomarker data, with cerebrospinal fluid (CSF) progranulin levels increasing from 10.7 to 27.3 ng/mL at 6 months post-treatment, significantly higher than the healthy control range of 3.3 to 8.2 ng/mL[142]. - The Company plans to initiate dosing for PBFT02 in treating FTD-C9orf72 patients in the first half of 2025 and expects regulatory feedback on the clinical pathway for ALS patients in the second half of 2024[149]. - The Company has completed dosing of Cohort 1 in the upliFT-D trial and is continuing to study Dose 1 in Cohort 2 based on robust PGRN expression observed[143]. - The Company anticipates reporting 12-month follow-up data from Cohort 1 and interim data from Cohort 2 in the first half of 2025[144].
Passage BIO(PASG) - 2024 Q3 - Quarterly Report