REGENXBIO stated that the FDA has placed a clinical hold on the company’s experimental gene therapy program for the treatment of rare childhood diseases.
- The company said that the FDA hold has impacted two experimental gene therapies, RGX-111 and RGX-121.
- REGENXBIO added that the tumor was identified during a routine brain MRI of an asymptomatic five-year-old participant in its RGX-111 trial.
- The company said that the participant had received intracisternal RGX-111 four years prior.
REGENXBIO Inc.’s (RGNX) shares tumbled more than 29% in Wednesday’s opening trade after the company announced an update from the U.S. Food and Drug Administration (FDA).
REGENXBIO stated that the FDA has placed a clinical hold on the company’s experimental gene therapy program for the treatment of rare childhood diseases.
The company said that the FDA hold has impacted two experimental gene therapies, RGX-111 and RGX-121.
Why Did FDA Place A Clinical Hold?
REGENXBIO stated that the FDA’s clinical hold was placed after a brain tumor was found in one patient in the trial.
The company added that the tumor was identified during a routine brain magnetic resonance imaging (MRI) of an asymptomatic five-year-old participant in its RGX-111 trial. REGENXBIO said that the participant had received intracisternal RGX-111 four years prior.
The company said that an investigation into whether this serious adverse event (SAE) is drug-related is ongoing, and that causality has not been established yet.
While the SAE involved a patient in the RGX-111 trial, the FDA has also placed a hold on the RGX-121 gene therapy, citing the similarities in products, study populations, and shared risk between the clinical studies, the company said.
“These are separate therapies, and the positive safety profile of RGX-121 in more than 30 patients treated, including those dosed nearly seven years ago, remains unchanged,” said REGENXBIO CEO Curran Simpson.
What Did REGENXBIO’s Experimental Therapies Treat?
The RGX-111 gene therapy is aimed at treating MPS I, also known as Hurler syndrome. This is a genetic disorder that results in the buildup of large sugar molecules in lysosomes, causing damage to several organ systems.
The RGX-121 gene therapy is aimed at treating MPS II, also known as Hunter syndrome, which results in the buildup of large sugar molecules in body tissues. This causes abnormalities in body organs, including the heart, skeleton, and respiratory system.
How Did Stocktwits Users React?
Retail sentiment on Stocktwits around REGENXBIO was in the ‘bearish’ territory, with message volumes at ‘extremely high’ levels at the time of writing.
One user thinks that the stock decline is an “overreaction.”
RGNX stock is down 34% year-to-date, but up 25% over the past 12 months.
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