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NEW YORK - Neurogene Inc. (NASDAQ:NGNE), a clinical-stage biotechnology company currently trading at $14.95 per share, has received written agreement from the U.S. Food and Drug Administration on key aspects of its registrational trial design for NGN-401, a gene therapy targeting Rett syndrome in females aged 3 years and older. According to InvestingPro analysis, the company appears undervalued based on its Fair Value assessment.
The trial, named Embolden, will be a single-arm, open-label, baseline-controlled study evaluating a composite primary endpoint that assesses both clinical improvement and developmental milestone achievement. The company has proposed enrolling 18 participants, pending final FDA feedback.
The primary endpoint will measure response at 12 months post-treatment, with participants considered responders if they achieve a Clinical Global Impression-Improvement score of 3 or better and gain at least one developmental milestone from a predefined list of 28 skills. These milestones span areas including hand function, communication, and mobility.
Neurogene reported that the Phase 1/2 portion of the trial is fully enrolled, with a total of 10 participants having received the 1E15 vector genomes dose. Five participants were dosed in the first half of 2025, with no evidence of hemophagocytic lymphohistiocytosis or hyperinflammatory syndrome observed at this dose level.
Previously disclosed data from the first four participants showed each achieved a "much improved" rating on the Clinical Global Impression-Improvement scale, with participants collectively gaining 23 developmental skills across multiple domains. While the clinical progress is promising, InvestingPro data reveals the company’s financial health score is rated as ’FAIR’, with analysts not expecting profitability this year.
"I have been impressed with the improvements observed in participants after NGN-401 administration, which have included global improvement in signs and symptoms of Rett syndrome and gains of multi-domain developmental milestones," said Dr. Elizabeth Berry-Kravis, Professor at Rush University and principal investigator in the trial, according to the company’s press release.
Neurogene has begun registrational trial initiation activities and plans to share updated clinical efficacy and safety data in the second half of 2025. The company also announced it has extended its cash runway into early 2028, with $292.6 million in cash and investments as of March 31, 2025. With a current ratio of 21.03, the company’s liquid assets significantly exceed its short-term obligations, though InvestingPro analysis indicates rapid cash burn remains a concern. Discover more insights about Neurogene’s financial health and growth potential in the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.
In other recent news, Neurogene has been in the spotlight for several developments concerning its NGN-401 gene therapy. Craig-Hallum initiated coverage with a buy rating, highlighting the potential of NGN-401 for treating Rett syndrome, and set a price target of $50. BMO Capital raised its price target for Neurogene from $22 to $26, citing effective management of ferritin levels in patients under the updated HLH monitoring protocol, which enhanced their confidence in the treatment. Stifel maintained its Buy rating with a target of $46, noting the competitive landscape in the Rett Gene Therapy sector and the significance of forthcoming data from Neurogene.
Additionally, Neurogene presented at the American Society of Gene & Cell Therapy (ASGCT) meeting, detailing a treatment strategy for the rare condition hemophagocytic lymphohistiocytosis (HLH) that can occur post-gene therapy. The company shared an algorithm for early detection and treatment of HLH, emphasizing the importance of early intervention. Neurogene’s ongoing Phase 1/2 trial for NGN-401 is using a lower AAV dose level, which has not been associated with HLH, aiming to balance efficacy and safety. These developments reflect Neurogene’s commitment to advancing its gene therapy programs and addressing challenges in the treatment of neurological diseases.
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