Neurogene(US:NGNE)2025-08-11 20:05Financial Performance - The company reported a net loss of $44.7 million for the six months ended June 30, 2025, compared to a net loss of $35.4 million for the same period in 2024[114]. - The company reported no revenue for the three and six months ended June 30, 2025, compared to $0.9 million for the same periods in 2024[133]. - The net loss for the three months ended June 30, 2025, was $22.0 million, compared to a net loss of $18.5 million in 2024, reflecting a 19% increase in losses[132]. - For the six months ended June 30, 2025, the company used $40.2 million in cash for operating activities, reflecting a net loss of $44.7 million[155]. - For the six months ended June 30, 2024, the company used $37.5 million in cash for operating activities, reflecting a net loss of $35.4 million[156]. Cash and Investments - As of June 30, 2025, the company had an accumulated deficit of $307.0 million and cash, cash equivalents, and short-term investments totaling $274.5 million[114]. - As of June 30, 2025, the company had cash, cash equivalents, and short-term investments totaling $274.5 million[149]. - Net cash flows used in investing activities for the six months ended June 30, 2025, included purchases of investments totaling $149.4 million[158]. - Net cash flows provided by investing activities for the six months ended June 30, 2024, included purchases of investments totaling $42.7 million[159]. - The company anticipates needing additional capital to fund operations, with existing resources expected to last at least 12 months from the filing date[148]. Research and Development - The Phase 1/2 clinical trial of NGN-401 for Rett syndrome has completed dosing with a total of 10 participants receiving the 1E15 vg dose[105]. - Positive interim clinical data showed consistent improvements in developmental milestones across multiple clinical assessments for the first four participants receiving the 1E15 vg dose[102]. - The company received FDA clearance for its Investigational New Drug (IND) application for NGN-401 in January 2023[101]. - The Embolden registrational trial for NGN-401 is designed to evaluate the 1E15 vg dose in females ages three years and older with Rett syndrome[106]. - The company discontinued the 3E15 vg dose level in the NGN-401 trial due to safety concerns related to hyperinflammatory syndromes[107]. - Research and development expenses increased to $19.4 million for the three months ended June 30, 2025, up from $15.7 million in 2024, representing a 23% increase[132]. - Research and development expenses for Q2 2025 were $19.4 million, an increase of 22.9% from $15.7 million in Q2 2024[134]. - For the first half of 2025, research and development expenses totaled $37.1 million, up 26.8% from $29.3 million in the same period of 2024[136]. - The increase in R&D expenses was primarily due to higher costs in the Rett syndrome program, including a $2.4 million rise in clinical trial costs for NGN-401[135]. - The company expects continued increases in R&D and general administrative expenses as it advances clinical development and builds manufacturing capabilities[139][142]. General and Administrative Expenses - General and administrative expenses rose to $6.7 million for the three months ended June 30, 2025, compared to $5.3 million in 2024, a 26% increase[132]. - General and administrative expenses for Q2 2025 were $6.7 million, a 25.5% increase from $5.3 million in Q2 2024[140]. - For the first half of 2025, general and administrative expenses reached $14.9 million, compared to $10.6 million in the same period of 2024, marking an increase of 40.5%[141]. - The company anticipates that general and administrative expenses will increase to support continued research and potential commercialization efforts[126]. Collaborations and Agreements - The collaboration agreement with the University of Edinburgh was extended through December 2026 to support pipeline development and scientific innovation[117]. - The RMAT application for the NGN-101 program was denied, leading the company to evaluate options for the program[111]. - The company is obligated to pay up to $5.3 million in regulatory-related milestones and up to $25.0 million in sales-related milestones under the License Agreement with the University of Edinburgh[171]. Operational and Facility Information - The company established a fully operational cGMP facility in Houston, Texas, to manufacture products for research and clinical studies[112]. - The company has a lease obligation for approximately 6,000 square feet of office space in New York, expiring in June 2026[161]. - The company has a lease for a manufacturing facility in Houston, Texas, expiring in August 2029, with options for two additional five-year terms[162]. - As of June 30, 2025, the total contingent value rights liability was approximately $1.1 million, compared to $1.1 million as of December 31, 2024[168]. Loss Carryforwards - The company has federal and state net operating loss carryforwards of $319.8 million and $39.6 million, respectively, which may offset future taxable income[130]. Interest Income - Interest income increased by $0.9 million in Q2 2025, attributed to a significant rise in cash and short-term investments following a private placement[143]. Other Financial Information - Total operating expenses for the three months ended June 30, 2025, were $26.1 million, an increase of 24% from $21.1 million in 2024[132]. - The company has not generated any commercial revenue from product sales and relies on substantial additional capital to support ongoing operations[116]. - The company has raised aggregate net proceeds of approximately $521.9 million since its inception[114].