Neurogene Releases Q2 2024 Financial Results and Updates

Neurogene Inc., a clinical-stage company specializing in genetic medicines for rare neurological disorders, reported its financial results for the second quarter of 2024 and shared significant corporate milestones.

Dr. Rachel McMinn, the CEO and Founder of Neurogene, noted the importance of the company's NGN-401 gene therapy for Rett syndrome receiving the Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA. This designation, along with selection for the FDA’s START Pilot Program, promises enhanced collaboration with the FDA, potentially accelerating NGN-401’s development.

Key advancements in the NGN-401 program were highlighted, including the first high-dose patient dosing in May and an early favorable safety profile. By mid-June, the low-dose NGN-401 remained well-tolerated among the initial three patients in Cohort 1. Looking ahead, Neurogene anticipates interim efficacy data from the low-dose cohort by the end of the year, with further data on both low- and high-dose treatments expected in the latter half of 2025.

Significant milestones for the second quarter of 2024 include:

NGN-401 Gene Therapy for Rett Syndrome:
- NGN-401 received RMAT designation and was chosen for the START Pilot Program, enabling frequent FDA consultations to discuss product-specific development.
- High-dose NGN-401 was administered to the first patient in May, showing a favorable early safety profile.
- At the International Rett Syndrome Foundation ASCEND Summit in June, data from low-dose Cohort 1 indicated no new treatment-related adverse events, with all adverse events being mild, transient, or resolving.
- No signs of MeCP2 overexpression toxicity were reported, even in patients with mild genetic variants.
- Neurogene expects to report interim clinical efficacy data from Cohort 1 in Q4 2024 and further interim data from Cohort 2 in the second half of 2025.
- The company aims to complete enrollment for Cohort 1 by the second half of 2024.

NGN-101 Gene Therapy for CLN5 Batten Disease:
- Enrollment has been completed, and interim clinical data along with a regulatory update is anticipated in Q1 2025.
- Due to the rarity of CLN5 Batten disease, streamlined regulatory pathways are crucial for ongoing investment in the program.

Additional Corporate Updates:
- Neurogene plans to introduce another product candidate using transgene regulation technology into clinical trials in 2025.

Upcoming Events:
- Presentations and meetings are scheduled at the H.C. Wainwright 26th Annual Global Investment Conference on September 9, the Cantor Global Healthcare Conference on September 18, and the Cell & Gene Meeting on the Mesa from October 7-9.

Financial Overview for Q2 2024:
- Cash Position: Neurogene reported $153.9 million in cash, cash equivalents, and investments as of June 30, 2024, expected to fund operations into the second half of 2026.
- R&D Expenses: Increased to $15.7 million from $10.3 million year-over-year, driven by clinical trial costs, preclinical program expenses, and higher compensation due to increased R&D headcount.
- G&A Expenses: Rose to $5.3 million from $2.3 million in the previous year, primarily due to increased employee-related costs, professional fees, rent, and corporate expenses.
- Net Loss: The net loss for Q2 2024 was $18.5 million, compared to $11.9 million in Q2 2023.

Neurogene's mission revolves around creating treatments for severe neurological diseases to improve the lives of affected patients and families. The company is at the forefront of developing innovative approaches to gene therapy, focusing on maximizing distribution to target tissues and ensuring product potency and safety. The construction of a state-of-the-art gene therapy manufacturing facility in Houston further underlines Neurogene's commitment to advancing clinical development activities.

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