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On Wednesday, Stifel analysts maintained a Buy rating for Neurogene (NASDAQ:NGNE) shares, with a steady price target of $46.00. According to InvestingPro data, analyst targets range from $38 to $72, suggesting significant upside potential despite the stock’s challenging year, during which it has declined over 71%. The stock currently trades near its 52-week low of $13.47, with an RSI indicating oversold conditions. The affirmation comes after Neurogene’s fourth quarter 2024 update, which indicated that the company’s development plans are progressing as expected. Stifel highlighted that the anticipated regulatory update for Neurogene’s NGN-401 therapy is scheduled for the first half of 2025, with additional interim clinical data from the phase 1/2 study in Rett syndrome expected in the second half of the year.
The analysts recently engaged with Neurogene’s management during Stifel’s CNS Forum, where the company expressed confidence in the clinical efficacy of the low-dose NGN-401 treatment. Initial data showed consistent improvement across multiple clinical scales, such as the Clinical Global Impressions of Improvement (CGII), Rett Syndrome Behaviour Questionnaire (RSBQ), and developmental milestones. These improvements were significant compared to the natural history of Rett syndrome, where patients typically do not exhibit spontaneous progress.
Furthermore, the safety profile of NGN-401 has been well-tolerated, particularly at a dose level that has historically been below the threshold where other adeno-associated virus gene therapy (AAV-GTx) programs have observed rare but severe immune reactions. In response, Neurogene is taking additional risk mitigation measures to further ensure patient safety. InvestingPro analysis reveals the company’s overall financial health score is currently weak at 1.67, though it maintains a strong liquidity position with a current ratio of 20.84, indicating robust short-term financial stability.
Looking ahead, the next significant event for Neurogene will be the regulatory update in the first half of 2025. Due to the severity of Rett syndrome and the ineffectiveness of gene therapy sham-controls, Neurogene anticipates that a single-arm pivotal study will be conducted, potentially utilizing either a delayed-start or a natural history comparator.
Lastly, the company’s financial position was noted to be strong, with current cash reserves expected to fund operations well into the second half of 2027. This financial stability supports Neurogene’s ongoing research and development efforts. However, InvestingPro data shows the company is quickly burning through cash, with negative free cash flow of $70.9 million in the last twelve months. For deeper insights into Neurogene’s financial health and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers, along with 14 additional ProTips for informed decision-making.
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