Regenxbio Inc. (RGNX) announced promising outcomes from the Phase I/II/III CAMPSIITE trial of RGX-121, aimed at treating Mucopolysaccharidosis Type II (Hunter syndrome), a rare genetic disorder.
The study indicated that RGX-121 facilitated a significant and sustained reduction in cerebrospinal fluid (CSF) levels of HS D2S6, a critical biomarker of brain disease in Hunter syndrome. This suggests that RGX-121 could potentially become the first gene therapy and a one-time treatment for the condition.
Additionally, long-term data from the trial revealed that patients administered RGX-121 at the pivotal dose level experienced a median reduction of 85% in heparan sulfate D2S6 levels in their CSF, approaching normal levels and maintaining this reduction for up to two years.
Regenxbio plans to commence a rolling submission of a Biologics License Application, utilizing the accelerated approval pathway in the third quarter of 2024.
At present, Regenxbio's shares are trading at $12.15 on the Nasdaq, reflecting a 0.16 percent dip.
