NEW YORK – Neurogene on Monday said after the market closed that it will stop advancing NGN-101 as a treatment for CLN5 Batten disease, a very rare genetic disorder.
The New York-based company said it applied for regenerative medicine advanced therapy designation with the US Food and Drug Administration, but the regulator did not grant it. Sponsors developing drugs with this designation can have more interaction with the agency during the development process, and any resulting regulatory application may be eligible for accelerated approval. Neurogene said it is currently evaluating other options for the NGN-101 gene therapy program, since continued investment was predicated on establishing a streamlined registrational pathway with the FDA.
Also on Monday, Neurogene reported interim clinical data from the first four female, pediatric Rett syndrome patients treated with a low-dose of its NGN-401 gene therapy in an ongoing open-label Phase I/II trial. NGN-401 uses an adeno-associated virus serotype 9 vector to deliver a full-length functional MECP2 gene using the firm's EXACT transgene regulation technology.
NGN-401 is under evaluation in the FDA's Support for clinical Trials Advancing Rare disease Therapeutics, or START, pilot program, and has received regenerative medicine advanced therapy, orphan drug, fast-track, and rare pediatric disease designations from the FDA.
All patients achieved a rating of "much improved" from baseline on the Clinical Global Impression-Improvement scale, a seven-point measure that clinicians use to rate patients' outcomes. Patients also improved between 28 percent and 52 percent from baseline on the Rett Syndrome Behavior Questionnaire, which is completed by caregivers.
After receiving NGN-401, patients acquired new skills and hit developmental milestones related to hand function or fine motor functions, language or communication capabilities, or ambulation or gross motor skills that were unexpected in this population based on natural history data from a US National Institutes of Health study, according to the company.
"Data were also concordant across multiple scales and show consistency of effect across patients, despite their unique clinical presentations at baseline," Neurogene Founder and CEO Rachel McMinn said in a statement.
The firm said low-dose NGN-401 has demonstrated a favorable safety profile in five patients, as well as in two patients treated with high-dose NGN-401. However, a third patient recently treated with a high-dose of the gene therapy experienced an emerging treatment-related serious adverse event that's consistent with known risks of AAV gene therapy, Neurogene said.
Neurogene's stock price was $70.04 per share when the Nasdaq opened Monday morning, but had dropped 31 percent to $48.28 per share by the time the market opened Tuesday.
Neurogene has initiated an adolescent and adult cohort in the NGN-401 clinical trial, in which it will enroll three patients at least 16 years of age. The company also said it plans to provide an update in the first half of 2025 on the design of a planned registrational trial to continue to assess NGN-401 and will report additional interim Phase I/II trial data in the second half of 2025.