Neurogene Inc. (NASDAQ:NGNE), a biopharmaceutical company, reported the death of a patient enrolled in its Phase 1/2 clinical trial for Rett syndrome, according to a recent SEC filing. The patient, who was dosed on November 5 with 3E15 vg of NGN-401, developed a rare and severe hyperinflammatory syndrome related to high doses of adeno-associated virus (AAV) and subsequently passed away.
The U.S. Food and Drug Administration (FDA) has permitted the trial to continue, but with a lower dose of 1E15 vg for both pediatric and adolescent/adult cohorts. Neurogene plans to employ this lower dose in its upcoming registrational trial design.
This news follows a critical condition report of the same patient, indicating the severity of the adverse reaction. The information disclosed in the SEC filing emphasizes that the data is furnished and not filed, and therefore is not subject to the liabilities of Section 18 of the Securities Exchange Act of 1934, nor is it incorporated by reference into any filing under the Exchange Act or the Securities Act of 1933, unless specifically referenced.
Neurogene, previously known as Neoleukin Therapeutics (NASDAQ:NGNE), Inc., and before that as Aquinox Pharmaceuticals, Inc., is headquartered in New York and specializes in pharmaceutical preparations. The company's focus on Rett syndrome, a rare genetic neurological disorder, underscores its commitment to addressing unmet medical needs in neurology.
In other recent news, Neurogene has experienced a series of significant developments. The biotech firm reported a net loss of $18.5 million in Q2 2024, with R&D expenses reaching $15.7 million. However, the company 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.
Analysts from Stifel, BMO Capital Markets, Baird, and H.C. Wainwright have adjusted their price targets for Neurogene, reflecting their positive outlook on the preliminary Phase 1/2 data for NGN-401. However, a serious adverse event was identified in a patient receiving a high dose of the treatment. Despite this, the company has been given the green light by the FDA to continue with the low-dose arm of the study, which has shown a clean safety profile.
Neurogene's management is actively planning the registrational study, with details expected to be released in the first half of 2025. The company also terminated its Batten's disease program recently. These are among the latest developments in Neurogene's journey with its investigational therapy, NGN-401.
InvestingPro Insights
Recent data from InvestingPro sheds light on Neurogene Inc.'s financial position and market performance, providing context to the clinical trial developments. The company's market capitalization stands at $201.73 million, reflecting its current valuation in light of recent events.
InvestingPro Tips highlight that Neurogene holds more cash than debt on its balance sheet, which could be crucial for funding ongoing research and navigating challenges in clinical trials. However, the company is quickly burning through cash, a common characteristic of biopharmaceutical firms in the development stage.
The stock's recent performance aligns with the clinical trial setback, with InvestingPro data showing a significant 60.89% decline in the past week and a 70.89% drop over the last month. This volatility is consistent with the InvestingPro Tip indicating that the stock generally trades with high price volatility.
Financially, Neurogene faces challenges with weak gross profit margins and is not profitable over the last twelve months. Analysts do not anticipate the company will be profitable this year, which is typical for early-stage biopharmaceutical companies focused on research and development.
For investors seeking a more comprehensive analysis, InvestingPro offers 14 additional tips for Neurogene, providing a deeper understanding of the company's financial health and market position.
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