On Monday, H.C. Wainwright reaffirmed its Buy rating and $55.00 stock price target for Neurogene (NASDAQ:NGNE), following the biotech company's recent update on its Rett syndrome treatment, NGN-401.
The analyst from the firm highlighted the promising low-dose efficacy data from the ongoing trials. Despite a severe adverse event (SAE) occurring in a high-dose patient, the firm remains confident in the drug's potential, particularly at lower doses.
Neurogene reported that a patient treated with a high dose of NGN-401 on November 5 experienced a severe adverse event, which was disclosed to the company's management on November 11. The patient, unfortunately, passed away due to systemic hyperinflammatory syndrome, a severe immune reaction linked to high doses of adeno-associated virus (AAV) vectors.
The condition can trigger abnormal cytokine release and potentially fatal complications such as hemophagocytic lymphohistiocytosis (HLH) and multi-system inflammatory syndrome.
Despite this incident, the safety profile of NGN-401 at lower doses has been deemed highly favorable by H.C. Wainwright, with only mild adverse events observed. The firm believes that the therapeutic potential of the low-dose treatment remains significant and could offer a viable regulatory path forward.
The U.S. Food and Drug Administration (FDA) has reviewed the safety data for NGN-401 and has allowed Neurogene to continue with the trials at the 1x1015 low-dose level. Looking ahead, Neurogene anticipates providing updates on the pivotal trial design in the first half of 2025 and expects to release more interim Phase 1/2 data in the second half of 2025.
In summary, H.C. Wainwright has reiterated its positive stance on Neurogene, maintaining the 12-month price target of $55 for the company's stock. The firm's outlook is based on the promising low-dose efficacy data and the FDA's decision to allow trials to proceed, despite the recent adverse event in a high-dose patient.
In other recent news, Neurogene Inc. has experienced significant developments. The biopharmaceutical company reported a net loss of $18.5 million in Q2 2024, with R&D expenses reaching $15.7 million.
However, the firm secured approximately $200 million in oversubscribed PIPE financing, expected to support the completion of enrollment for the study of NGN-401, their investigational therapy for Rett syndrome, into the second half of 2027.
Recent developments include a patient death in the Phase 1/2 clinical trial for Rett syndrome due to a severe hyperinflammatory syndrome related to high doses of adeno-associated virus (AAV). Despite this, the FDA has permitted the trial to continue with a lower dose.
Analysts from Stifel, BMO Capital Markets, and Baird have adjusted their price targets for Neurogene, reflecting their positive outlook on the preliminary Phase 1/2 data for NGN-401.
The company also terminated its Batten's disease program recently. The details of the registrational study are expected to be released in the first half of 2025. These are among the latest developments in Neurogene's journey with its investigational therapy, NGN-401.
InvestingPro Insights
Recent InvestingPro data provides additional context to Neurogene's (NASDAQ:NGNE) current financial situation and market performance. Despite H.C. Wainwright's optimistic outlook, the company faces some financial challenges. InvestingPro Tips reveal that Neurogene is quickly burning through cash and is not expected to be profitable this year. This aligns with the company's focus on clinical trials and drug development, which often require significant investment before generating revenue.
The stock's recent performance has been volatile, with InvestingPro data showing a 58.03% decline in the past month and a 41.9% drop over the last three months. This volatility may reflect investor reactions to the recent severe adverse event in the high-dose trial and the subsequent FDA review.
However, it's worth noting that Neurogene holds more cash than debt on its balance sheet, which could provide some financial flexibility as it continues its clinical trials. The company's market cap stands at $301.02 million, with a price-to-book ratio of 2.19, indicating that investors are still valuing the company above its book value despite recent setbacks.
For investors seeking a more comprehensive analysis, InvestingPro offers 11 additional tips for Neurogene, providing a deeper understanding of the company's financial health and market position.
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