NEW YORK - Neurogene Inc. (NASDAQ:NGNE) has announced positive interim clinical data from its ongoing Phase 1/2 trial of NGN-401, a gene therapy aimed at treating Rett syndrome in pediatric patients. The treatment showed favorable safety and meaningful improvement in participants, suggesting potential gains in skills and developmental milestones.
The low-dose cohort of the trial, comprising four female pediatric patients, demonstrated consistent improvements across various assessments of Rett syndrome. Notably, all participants achieved a score indicating significant clinical improvement on the Clinical Global Impression-Improvement scale. Additionally, improvements in the Rett Syndrome Behavior Questionnaire ranged from 28 to 52 percent from baseline, and participants acquired skills in core clinical domains of the disease.
No serious adverse events related to the treatment have been reported, and most adverse events were responsive to steroids. However, a recent serious adverse event consistent with known risks of adeno-associated virus gene therapy was observed in a high-dose participant.
Neurogene has also initiated an adolescent/adult cohort in the trial and has aligned with the FDA on manufacturing requirements for a future registrational trial and potential product launch.
The company plans to complete enrollment for the low-dose pediatric cohort in the fourth quarter of 2024 and will provide an update on the registrational trial design in the first half of 2025. Additional interim data is expected in the second half of 2025.
In contrast, Neurogene disclosed that it does not plan to advance its NGN-101 gene therapy program for CLN5 Batten disease, as the FDA denied its Regenerative Medicine Advance Therapy application.
Today, Neurogene will host an investor/analyst webcast to discuss the interim data from the NGN-401 clinical trial.
NGN-401 is an investigational therapy that delivers the MECP2 gene, which is implicated in Rett syndrome, using Neurogene's proprietary EXACT transgene regulation technology to avoid toxicity from gene overexpression.
This article is based on a press release statement from Neurogene Inc.
In other recent news, Neurogene Inc. has secured approximately $200 million in an oversubscribed PIPE financing. This financing, involving prominent healthcare investment firms such as RTW Investments and Casdin Capital, is expected to extend Neurogene's financial runway into the second half of 2027. These funds are intended to support the completion of enrollment for a study of their gene therapy, NGN-401, for Rett syndrome, among other corporate purposes.
The company reported a net loss of $18.5 million for the second quarter of 2024, with research and development expenses amounting to $15.7 million. Neurogene's gene therapy candidate, NGN-401, has received the Regenerative Medicine Advanced Therapy designation from the FDA, which is anticipated to expedite the approval process.
In terms of analyst ratings, BMO Capital and William Blair both maintained an Outperform rating on Neurogene shares, while H.C. Wainwright adjusted the share price target to $49 from $51, maintaining a Buy rating. Investors are closely watching the upcoming Phase 1/2 clinical data from the low-dose cohort of their NGN-401 study, and an update in the first quarter of 2025, which will include interim Phase 1/2 clinical data and a regulatory update for NGN-101 in the treatment of CLN5 Batten disease. These are recent developments that could potentially impact the company's trajectory.
InvestingPro Insights
As Neurogene Inc. (NASDAQ:NGNE) reports positive interim clinical data for its Rett syndrome gene therapy, investors may be interested in the company's financial position and market performance. According to InvestingPro data, Neurogene's market capitalization stands at $934.76 million, reflecting the market's current valuation of the company's potential.
The stock has shown remarkable performance, with a 351.72% price total return over the past year and a 251.96% return year-to-date. This strong momentum aligns with the positive clinical trial results and could indicate growing investor confidence in Neurogene's gene therapy pipeline.
However, it's important to note that Neurogene is currently trading at a high earnings multiple, with a P/E ratio of 264.67. This suggests that investors are pricing in significant future growth expectations, which may be influenced by the promising NGN-401 trial results.
InvestingPro Tips highlight that Neurogene holds more cash than debt on its balance sheet, which could provide financial flexibility as the company advances its clinical programs. Additionally, liquid assets exceed short-term obligations, potentially offering a buffer against operational costs associated with ongoing trials and potential product launches.
It's worth noting that analysts do not anticipate the company will be profitable this year, which is not uncommon for biotech firms in the clinical stage of drug development. The company's focus on advancing its lead candidate, NGN-401, and the decision not to pursue the NGN-101 program for CLN5 Batten disease, may impact its near-term financial outlook.
For investors seeking a more comprehensive analysis, InvestingPro offers 17 additional tips for Neurogene, providing a deeper understanding of the company's financial health and market position.
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