Aquinox(NLTX) - 2024 Q1 - Quarterly Results

CEO Statement and Corporate Strategy CEO highlights NGN-401 program progress, trial expansion, strong financial position with cash into H2 2026, and new clinical candidate plans for 2025 - Significant progress in NGN-401 Rett syndrome program, with trial expanding to Australia and a high-dose cohort[2] - The company plans to advance an additional product candidate into clinical trials in 2025, leveraging its transgene regulation platform[2] - Neurogene confirms a strong financial position, with sufficient cash runway to fund operations into the second half of 2026[2] Clinical Program Updates Neurogene provides updates on key clinical trials, including NGN-401 expansion, NGN-101 enrollment, and plans for a new candidate in 2025 NGN-401 for Rett Syndrome (Phase 1/2 Trial) NGN-401 trial shows favorable safety, no MeCP2 overexpression toxicity, expands to Australia, with interim efficacy data expected in Q4 2024 - Favorable safety data showed NGN-401 was generally well-tolerated in the first three patients, with all treatment-related adverse events being mild (Grade 1)[3] - No signs of MeCP2 overexpression toxicity were reported, addressing a key safety concern[3] - The trial has been cleared to proceed in Australia, the third region for the study[3] - Key upcoming milestones include interim efficacy data from Cohort 1 in Q4 2024 and additional data from Cohort 2 in H2 2025[3] NGN-101 for CLN5 Batten Disease (Phase 1/2 Trial) NGN-101 trial enrollment continues for Cohort 3, with an H2 2024 update, contingent on FDA alignment for a streamlined registrational pathway - Enrollment continues for the high-dose Cohort 3, with an interim clinical and regulatory update planned for H2 2024[4] - Future investment in the NGN-101 program is contingent upon FDA agreement on a streamlined registrational pathway[4] Early-Stage Portfolio Development Neurogene actively advances its early-stage portfolio, planning to move an additional product candidate into clinical trials in 2025 - The company plans to advance an additional product candidate into the clinic in 2025[5] First Quarter 2024 Financial Results Neurogene reported a Q1 2024 net loss of $16.9 million, with $169.5 million cash extending runway into H2 2026, driven by increased operating expenses Summary Financial Metrics | Financial Metric | Q1 2024 | Q1 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | R&D Expenses | $13.5M | $10.3M | +$3.2M | | G&A Expenses | $5.2M | $2.8M | +$2.4M | | Net Loss | $16.9M | $12.3M | +$4.6M | - Cash, cash equivalents, and investments totaled $169.5 million as of March 31, 2024[8] - The current cash position is expected to fund operations into the second half of 2026[8] - The increase in R&D expenses was driven by NGN-401 clinical trial costs and preclinical discovery programs, while G&A expenses rose due to higher headcount and public company costs[8] Condensed Consolidated Balance Sheets As of March 31, 2024, total assets were $195.4 million, liabilities $24.6 million, and stockholders' equity $170.8 million, compared to year-end 2023 figures Balance Sheet Summary | (In Thousands of U.S. dollars) | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $150,140 | $148,210 | | Total assets | $195,350 | $222,573 | | Liabilities & Equity | | | | Total liabilities | $24,573 | $36,549 | | Stockholders' equity | $170,777 | $186,024 | | Total liabilities and stockholders' equity | $195,350 | $222,573 | Condensed Consolidated Statements of Operations Q1 2024 net loss increased to $16.9 million from $12.3 million in Q1 2023, driven by higher total operating expenses of $18.8 million Statements of Operations Summary | (In thousands of U.S. dollars) | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Research and development | $13,541 | $10,283 | | General and administrative | $5,238 | $2,752 | | Total operating expenses | $18,779 | $13,035 | | Loss from operations | ($18,779) | ($13,035) | | Net loss | ($16,921) | ($12,263) | | Net loss per share, basic and diluted | ($1.00) | ($28.28) |