Neurogene’s NGN-401 gene therapy scores well in Rett syndrome

New York, USA-based rare neurological disease drug developer Neurogene (Nasdaq: NGNE) has announced positive interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase I/II open-label trial designed to evaluate NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome. Low-dose NGN-401 has demonstrated a favorable safety profile.

Investors, however, were not impressed, sending the firm’s share plunging 44% to $40.00 by close of trading, as Neurogene also disclosed that it became aware yesterday that the third high-dose participant, who was recently dosed, experienced a treatment-related serious adverse event (AE) described as "consistent with known risks of AAV gene therapy."

“Today marks an important day for Neurogene and the Rett syndrome community as we share positive interim data for NGN-401 from our low-dose cohort that shows the first four participants demonstrated meaningful gains of skills and developmental milestones in core clinical domains of Rett syndrome, which are not expected to occur when compared to and contextualized against the natural history of Rett syndrome. Data were also concordant across multiple scales and show consistency of effect across patients, despite their unique clinical presentations at baseline,” said Rachel McMinn, founder and chief executive of Neurogene.