NEW YORK - Neurogene Inc. (NASDAQ: NGNE), a clinical-stage biotech company, has announced initial safety results from a Phase 1/2 trial of its gene therapy, NGN-401, for patients with Rett syndrome. The treatment was reported to be well-tolerated in the first three patients, with follow-up periods ranging from three to nine months.
The ongoing trial aims to evaluate the safety, tolerability, and preliminary efficacy of NGN-401, which incorporates the company's proprietary EXACT transgene regulation technology. This approach is designed to provide therapeutic levels of protein expression to crucial areas of the brain and nervous system affected by Rett syndrome.
According to the company, the trial has not observed any signs or symptoms of overexpression toxicity in patients, including one with a mild variant of the disease.
The three patients, aged 4 to 7 years, received the treatment via intracerebroventricular infusion. All adverse events related to NGN-401 have been classified as mild and are either transient or resolving. To date, no serious adverse events related to the treatment or infusion procedure have been reported.
Dr. Rachel McMinn, CEO of Neurogene, stated that the interim safety data presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting demonstrate a favorable tolerability profile for NGN-401. The company remains on track to provide interim efficacy data from the first cohort of the trial in the fourth quarter of 2024.
The trial is still enrolling female patients between the ages of 4 and 10 with classic Rett Syndrome, with the goal of assessing two dose levels of NGN-401. Dr. Bernhard Suter, the principal investigator of the trial, highlighted the high unmet need for new treatments for Rett syndrome and the potential of gene therapy to address the underlying cause with a one-time treatment.
Neurogene's mission is to develop genetic medicines for rare neurological diseases, with a focus on optimizing efficacy and safety profiles through novel approaches. The company has established a gene therapy manufacturing facility in Houston, Texas, which produced NGN-401 for the trial.
While the company has expressed optimism about the therapeutic potential of NGN-401, it also cautions that statements about the future plans and prospects are forward-looking and subject to risks and uncertainties. Further interim data, including from the high-dose second cohort, is expected in the second half of 2025.
This article is based on a press release statement from Neurogene Inc.
InvestingPro Insights
As Neurogene Inc. (NASDAQ: NGNE) continues to make strides in the development of its gene therapy NGN-401, investors are closely monitoring the company's financial health and stock performance. According to InvestingPro data, Neurogene holds a market capitalization of $422.12 million, with a strikingly low P/E ratio of 1.23, suggesting that the stock may be undervalued relative to its near-term earnings growth potential.
Despite the company's promising clinical developments, it's important to note that Neurogene has experienced weak gross profit margins, with a gross profit of -$44.39 million over the last twelve months as of Q1 2023. This could be indicative of the high costs associated with the research and development phase typical of clinical-stage biotech companies. Additionally, the stock has seen significant volatility, with a 1-month price total return of -20.58%, yet it has also demonstrated a strong 6-month price total return of 117.28%.
InvestingPro Tips highlight that while Neurogene is trading at a high earnings multiple with a P/E ratio (adjusted for the last twelve months as of Q4 2023) of 22.28, it also has liquid assets that exceed its short-term obligations, providing some financial stability. Moreover, the company does not pay a dividend, which is common for growth-focused biotech firms that reinvest earnings back into research and development.
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