Neurogene Inc (NASDAQ: NGNE) surged by over 30 per cent Friday after the FDA approved continuation of a clinical study that took somebody’s life. The biotech company is now required to use a lower dose.
The tragic incident occurred this month during a Phase I/II trial on the rare disease treatment developer’s Rett syndrome drug. The patient had a rare and ultimately fatal immune response to the therapeutic, “NGN-401.”
“We are deeply saddened for the family,” Neurogene said in a press release on Nov. 18 before the girl passed away days later. The biotech operator’s stock had plummeted after the adverse event was initially revealed in a previous statement on Nov. 11.
Rett syndrome is a rare genetic neurological condition that primarily impacts young girls. It causes female children, and boys in rare cases, to lose control of motor skills, have speech and breathing problems, behavioural issues and seizures.
One in 10,000 girls are impacted by the condition. There is no cure, only management options.
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Children are Neurogene’s main concern
The company’s other primary focus is CLN5 Batten disease, a condition linked to a specific gene mutation. It causes kids to experience neurological decline in their early years, usually between 4 and 7. Blindness and epilepsy can also result from the ailment.
“What makes Batten disease so complex is that it is caused by a mutation in any one of 13 different genes called ceroid lipofuscinosis, neuronal [CLN] genes,” the Canadian Institutes of Health Research says. “Each CLN gene is associated with a different subtype of the disease [CLN1, CLN2, etc.]”
Like Rett syndrome, there is no known cure, only management options. Neurogene has been involved with a clinical study on its treatment for the disease, NGN-101, since 2022. The extensive trial is expected to conclude in 2028.
“Efficacy assessments in this study will evaluate motor, language, visual and cognitive function,” the biotech operator explained.
Neurogene is actively engaged in a research partnership with the University of Edinburgh in Scotland.
On Aug 6., the treatment developer got to ring the opening bell at the Nasdaq Exchange to celebrate its listing. This was made possible through the company’s merger with Neoleukin Therapeutics just under one year ago. The “NGNE” ticker listing followed this development.
Neurogene made it onto the Russell 3000 Index (INDEXRUSSELL: RUA) in July.
rowan@mugglehead.com
