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NEW YORK - Neurogene Inc. (Nasdaq: NGNE), a clinical-stage biotech company valued at $276 million, disclosed an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. The company’s stock has shown remarkable momentum, gaining over 31% in the past week, according to InvestingPro data. The presentation detailed a monitoring and treatment algorithm for the rare condition hemophagocytic lymphohistiocytosis (HLH), which can develop following high-dose adeno-associated virus (AAV) gene therapy. The algorithm is designed to detect and treat HLH early, which has been effective in reversing the syndrome’s progression.
The algorithm includes daily monitoring for elevated ferritin levels, fever, and falling blood counts, known as the ’three Fs,’ in the first week post-dosing. This approach is based on the HLH-2004 study, which found these signs in over 90% of HLH cases. The treatment protocol incorporates high-dose corticosteroids as the first line of defense, with anakinra, an IL-1 receptor antagonist, as the second-line treatment.
Rachel McMinn, Ph.D., Founder and CEO of Neurogene, expressed her appreciation for the chance to share their findings with the gene therapy community. She emphasized the importance of early monitoring and treatment for HLH and the potential benefits of sharing clinical trial outcomes for broader learning. InvestingPro analysis reveals the company maintains a strong liquidity position with a current ratio of 21.03, though it’s currently burning through cash while advancing its clinical programs.
In its ongoing Phase 1/2 trial of NGN-401 gene therapy for Rett syndrome, Neurogene is using a lower AAV dose level, within the E13 vg/kg range, which has not been associated with HLH. The company aims to balance efficacy and safety by maximizing potency and purity in its gene therapy products and has developed a novel EXACT™ transgene regulation platform technology to achieve therapeutic levels while minimizing transgene toxicity.
Neurogene, committed to treating neurological diseases, has established a gene therapy manufacturing facility in Houston, Texas, where CGMP production of NGN-401 took place, supporting pivotal clinical development activities.
This news is based on a press release statement and includes forward-looking statements that involve risks, uncertainties, and assumptions. Neurogene’s statements about the NGN-401 trial and HLH treatment protocol are predictions based on current expectations, and actual results could differ materially. Investors should note that Neurogene is scheduled to report earnings in 3 days, on May 19, 2025. For deeper insights into Neurogene’s financial health and growth prospects, InvestingPro subscribers can access exclusive analysis, including 8 additional ProTips and a comprehensive Pro Research Report, part of the platform’s coverage of over 1,400 US stocks. The company has identified risks in its filings with the Securities and Exchange Commission, including its Annual Report for the year ended December 31, 2024, and its Quarterly Report for the quarter ended March 31, 2025.
In other recent news, Neurogene Inc. has been at the center of several notable developments. Baird analyst Joel Beatty downgraded Neurogene’s stock from Outperform to Neutral, adjusting the price target from $38.00 to $24.00 due to perceived regulatory uncertainties. Meanwhile, BMO Capital Markets also revised its price target for Neurogene, lowering it from $45.00 to $16.00, although it maintained an Outperform rating. The adjustment followed the unfortunate death of a patient administered with a high dose of NGN-401 and the departure of Peter Marks from the company.
Neurogene has also amended an agreement with Baker Bros. Advisors LP, increasing the ownership threshold for board nominations and issuing warrants as part of a strategic financial management move. Additionally, the company announced progress in its EXACT™ transgene regulation technology, which could significantly impact its NGN-401 gene therapy candidate for Rett syndrome. This technology has shown promise in preclinical studies, supporting the launch of a Phase 1/2 clinical trial.
Neurogene has received several designations from the FDA, which may expedite the development of NGN-401. These developments come amidst broader industry concerns following the resignation of Peter Marks from the FDA, which has affected investor confidence across the biotech sector.
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