Neurogene Stock Tanks After Adverse Event In Early-Stage Rett Syndrome Gene Therapy Trial

On Monday, Neurogene Inc. NGNE released interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase 1/2 open-label trial of NGN-401 gene therapy for female pediatric patients with Rett syndrome, a rare genetic neurological and developmental disorder.

Low-dose (1E15 vg) and high-dose (3E15 vg) NGN-401 have been well-tolerated with a favorable safety profile in the first seven pediatric participants (N=5 low-dose; N=2 high-dose):

• No treatment-related serious adverse events (SAEs).
• No signs or symptoms indicative of MeCP2 overexpression toxicity.
• Most treatment-related adverse events (AEs) are known potential risks of adeno-associated virus (AAV), have been responsive to steroids, and are resolved or are resolving.
• No intracerebroventricular (ICV)-related AEs.
• No seizures for any participants following NGN-401 treatment.

The company said it became aware of an emerging treatment-related SAE consistent with known risks of AAV gene therapy in the third high-dose participant who was recently dosed.

Low-dose interim efficacy data (N=4) showed that all participants achieved a rating of “much improved,” or a score of 2, on the clinician-rated Clinical Global Impression Scale of Improvement (CGI-I) from baseline; a score of < 3 is considered clinically meaningful.

All participants acquired skills and/or developmental milestones in one or more core clinical domains of Rett syndrome – hand function/fine motor, language/communication and ambulation/gross motor.

Neurogene initiated an adolescent/adult Cohort 3 to gain initial data on the potential of NGN-401 to treat a broader patient population. This cohort will enroll three participants ages 16 and above at the high dose.

The company also said it does not expect to move forward with the NGN-101 CLN5 Batten disease gene therapy program.

Price Action: NGNE stock is down 44% at $40.05 at last check Tuesday.

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