Neurogene Q1 2024 Financial Results and Recent Updates
Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company committed to developing genetic medicines for rare neurological diseases, has released its financial results for the first quarter of 2024 and provided updates on its corporate progress.
Founder and CEO Rachel McMinn, Ph.D., highlighted significant advancements in their NGN-401 Rett syndrome gene therapy program. The company has dosed its third patient, expanded the trial to include more patients and a high-dose cohort, and received approval to conduct the trial in Australia. Favorable safety data from the trial, presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, indicated that NGN-401 has been generally well-tolerated. Neurogene plans to release interim efficacy data from the low-dose cohort in the fourth quarter of 2024.
Neurogene's strategy includes expanding into other disease areas that could benefit from gene therapy with transgene regulation. They aim to advance another product candidate into clinical trials in 2025. Financially, the company remains strong, with sufficient cash to fund operations into the second half of 2026.
First Quarter 2024 Highlights and Anticipated Milestones:
NGN-401 Gene Therapy for Rett Syndrome:
- Presented favorable safety data from the first three pediatric patients in the low-dose Cohort 1 at the ASGCT Annual Meeting. The therapy has been well-tolerated with only mild adverse events, all of which are known risks of AAV administration and asymptomatic.
- No signs of MeCP2 overexpression toxicity were reported.
- Received approval from the Australian Human Research Ethics Committee (HREC) to conduct the trial in Australia, making it the third region where the trial is cleared.
- Interim clinical data, including efficacy from Cohort 1, is expected in Q4 2024. Data from Cohort 2 is anticipated in the second half of 2025.
- The trial has been expanded to include a high-dose Cohort 2 and additional patients in low-dose Cohort 1. This expansion aims to create a comprehensive data package to inform future NGN-401 studies.
- Plans to complete enrollment in Cohort 1 in the second half of 2024 and begin enrollment for Cohort 2 in Q2 2024.
NGN-101 Gene Therapy for CLN5 Batten Disease:
- Enrollment in high-dose Cohort 3 continues, with interim clinical data and regulatory updates expected in the second half of 2024. Due to the rarity of the disease, alignment with the U.S. Food and Drug Administration on a streamlined registrational pathway is crucial for continued investment.
Additional Corporate Updates:
- Advancing an early-stage portfolio with plans for another product candidate using transgene regulation technology to enter clinical trials in 2025.
Upcoming Events:
- Management will present at the 5th Annual Goldman Sachs Global Healthcare Conference on June 12.
- Presentation of safety data from the Phase 1/2 NGN-401 gene therapy trial for Rett syndrome at the 2024 IRSF Rett Syndrome Scientific Meeting on June 18-19.
First Quarter 2024 Financial Results:
- Cash, cash equivalents, and investments as of March 31, 2024, were $169.5 million. The company expects these funds to cover operations into the second half of 2026.
- Research and Development (R&D) expenses increased to $13.5 million from $10.3 million in the prior year, driven by higher NGN-401 clinical trial costs and increased early discovery program costs.
- General and Administrative (G&A) expenses rose to $5.2 million from $2.8 million, due to higher compensation and benefits, professional fees, and costs associated with becoming a public company.
- Net loss for the quarter was $16.9 million, compared to $12.3 million in the previous year.
Neurogene remains committed to developing innovative treatments for neurological diseases, leveraging their proprietary EXACT transgene regulation platform to optimize efficacy and safety in gene therapy. The company's state-of-the-art manufacturing facility in Houston, Texas, supports the clinical development of their gene therapy products, including NGN-401.
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