Neurogene(NGNE) - 2024 Q3 - Quarterly Results

Q3 2024 Financial Results and Corporate Updates Neurogene's Q3 2024 report details financial performance, clinical pipeline advancements, and strategic corporate developments, including a significant capital raise Financial Performance Neurogene's Q3 2024 net loss increased to $20.2 million, driven by higher R&D and G&A expenses reflecting increased clinical trial activities and headcount Financial Metric Comparison | Financial Metric | Q3 2024 (in millions) | Q3 2023 (in millions) | Change | | :--- | :--- | :--- | :--- | | R&D Expenses | $16.3 | $11.6 | +$4.7M | | G&A Expenses | $5.9 | $3.6 | +$2.3M | | Net Loss | $20.2 | $14.6 | +$5.6M | - The increase in R&D expenses was primarily due to higher costs for the NGN-401 clinical trial, preclinical discovery programs, and increased compensation expenses from a larger R&D team[10] - The rise in G&A expenses was mainly driven by increased employee-related costs, professional fees, rent, and other corporate expenses[10] Clinical Pipeline Updates Neurogene advances NGN-401 for Rett syndrome at the 1E15 vg dose after positive data, while pausing the 3E15 vg dose and discontinuing the NGN-101 program for CLN5 Batten disease following FDA's RMAT denial NGN-401 for Rett Syndrome NGN-401 for Rett syndrome shows positive interim data and a favorable safety profile at the 1E15 vg dose, with the 3E15 vg dose paused due to a serious adverse event - Positive interim data from the 1E15 vg dose showed consistent improvements, with all four participants rated as "much improved" on the CGI-I scale and showing a 28% to 52% improvement on the RSBQ[2][5] - The 1E15 vg dose was well-tolerated, with all treatment-related adverse events being mild (Grade 1)[5] - The company paused the 3E15 vg dose following a treatment-related serious adverse event (SAE) and does not plan to enroll more participants at this dose[3] - The FDA has reviewed safety data and allowed the trial to proceed with the 1E15 vg dose; an update on the registrational trial design is expected in the first half of 2025[3][5] NGN-101 for CLN5 Batten Disease Neurogene will not proceed with the NGN-101 program for CLN5 Batten disease after the FDA denied its RMAT application - Neurogene announced it does not expect to move forward with the NGN-101 gene therapy program for CLN5 Batten disease at this time[6] - The decision was made after the FDA denied the company's Regenerative Medicine Advance Therapy (RMAT) application, which was considered necessary for a streamlined registrational pathway due to the rarity of the disease[6] Corporate and Strategic Developments Neurogene significantly strengthened its financial position by executing an oversubscribed private placement of approximately $200 million, extending its cash runway into the second half of 2027 and supporting future clinical advancements - Executed an oversubscribed private placement, raising approximately $200 million in gross proceeds[9] - The company's cash, cash equivalents, and investments totaled $139.0 million as of September 30, 2024; combined with private placement proceeds, the cash runway is expected to last into the second half of 2027[1][8][10] - Neurogene plans to advance an additional product candidate into clinical trials in 2025[9] Financial Statements The condensed consolidated financial statements reflect a decrease in total assets to $164.1 million as of September 30, 2024, and a net loss of $20.2 million for Q3 2024 Condensed Consolidated Balance Sheets The balance sheets detail assets, liabilities, and stockholders' equity, showing a decrease in total assets from year-end 2023 to Q3 2024 | (In thousands of U.S. dollars) | September 30, 2024 | December 31, 2023 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $66,633 | $148,210 | | Total assets | $164,120 | $222,573 | | Liabilities and Stockholders' Equity | | | | Total liabilities | $26,706 | $36,549 | | Stockholders' equity | $137,414 | $186,024 | | Total liabilities and stockholders' equity | $164,120 | $222,573 | Condensed Consolidated Statements of Operations The statements of operations present the company's operating expenses, loss from operations, and net loss for the three and nine months ended September 30, 2024 | (In thousands of U.S. dollars) | Three Months Ended Sep 30, 2024 | Three Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2024 | Nine Months Ended Sep 30, 2023 | | :--- | :--- | :--- | :--- | :--- | | Total operating expenses | $22,180 | $15,219 | $62,018 | $40,850 | | Loss from operations | $(22,180) | $(15,219) | $(61,093) | $(40,850) | | Net loss | $(20,217) | $(14,577) | $(55,630) | $(38,700) | | Net loss per share, basic and diluted | $(1.19) | $(32.67) | $(3.29) | $(87.66) |