Neurogene gearing up to apply for approval of Rett gene therapy
Company hopes to use results of ongoing Phase 3 trial to form basis for request
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With a Phase 3 trial currently in progress, Neurogene is getting ready to file an application seeking approval of NGN-401, the company’s experimental gene therapy for Rett syndrome.
Neurogene recently completed dosing in the Phase 3 Embolden clinical trial (NCT05898620), which is testing NGN-401 in 25 female Rett patients ages 3 and older. Assuming that findings are positive, Neurogene hopes to use the results as a basis to file a biologics license application (BLA), a formal request seeking approval of the gene therapy in the U.S.
Neurogene is already preparing for the BLA submission. Most recently, the company began a process performance qualification (PPQ) campaign, which is a procedure that aims to ensure the gene therapy can be reliably and consistently manufactured at a quality that meets all regulatory requirements.
“With initiation of our PPQ campaign, ongoing preparations for a potential BLA submission and a strengthened balance sheet supporting commercial-readiness activities, we believe we are well positioned to bring NGN-401 to patients and families as quickly as possible, if approved,” Rachel McMinn, PhD, founder and CEO of Neurogene, said in a company press release.
Long-term data suggest gene therapy led to functional improvement
Rett is chiefly caused by mutations in the MECP2 gene. NGN-401 is designed to provide the body’s cells with a working version of this gene, alongside molecular machinery that helps keep the gene’s activity within the normal range. The therapy is administered directly into fluid-filled spaces within the brain.
In a previous Phase 1/2 trial, NGN-401 treatment was administered to 10 people with Rett syndrome. Long-term data have suggested that the gene therapy led to functional improvements. Most recently, data announced in June showed all 10 participants experienced improvements in the Clinical Global Impression-Improvement (CGI-I) scale, which is a clinician-rated measurement of overall health status.
All of the patients also achieved at least one new developmental milestone. Seven of the 10 achieved more than one new milestone, with an average of 4.7 new milestones across all the patients. According to Neurogene, milestone gains have generally progressed in the order that occurs during early development, and the gains have shown no sign of plateauing or fading. The company said these trends suggest that the therapy may help restart development post-treatment.
The clinically meaningful benefit observed across all participants [in the Phase 1/2 trial], including 100% of treated participants improving on CGI-I and gaining an average of 4.7 developmental milestones, strengthens our confidence in the program as we advance toward topline Embolden registrational data.
As of mid-August, safety data from patients in the Phase 1/2 study and the Phase 3 Embolden trial have shown that NGN-401 has been “generally well-tolerated,” Neurogene said. The company didn’t give specifics on safety findings.
The goal of the Phase 3 Embolden study is to see how many patients gain developmental milestones and skills that Rett patients rarely achieve without treatment, while also being rated as improved by their doctors on the CGI-I. Top-line results are expected in the second half of 2027.
“The clinically meaningful benefit observed across all participants [in the Phase 1/2 trial], including 100% of treated participants improving on CGI-I and gaining an average of 4.7 developmental milestones, strengthens our confidence in the program as we advance toward topline Embolden registrational data,” McMinn said.
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