H.C. Wainwright Reiterates Buy Rating on Regenxbio Inc. (RGNX)

H.C. Wainwright analyst Yi Chen reiterated a Buy rating and $39.00 price target on Regenxbio Inc. (NASDAQ: RGNX).

The analyst commented: "REGENXBIO announced new long-term data on September 3 from the Phase 1/2/3 CAMPSIITE trial of RGX-121 at the pivotal dose level that demonstrated an 85% median reduction of cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, which approached normal levels and sustained for up to two years. Of note, HS D2S6 is considered a surrogate endpoint that is reasonably likely to predict clinical benefit in MPS II (or Hunter syndrome), a rare disease caused by a deficiency in the lysosomal enzyme I2S. RGX-121 expressed protein is structurally identical to normal I2S, thus providing a permanent source of secreted I2S and improvement of brain function and systemic symptoms. As previously reported, the pivotal phase of the CAMPSIITE trial met its primary endpoint—RGX-121 treatment achieved decreased CSF levels of D2S6 below maximum attenuated disease levels at 16 weeks (p=0.00016). At the pivotal dose level, 80% of patients were enzyme replacement therapy (ERT) free at last time point, up to more than 18 months post-dosing. RGX-121 was well tolerated across 25 patients in the CAMPSIITE trial. The most common TEAEs were vomiting in 11 (73.3%) participants, followed by pyrexia, cough, and gastroenteritis in 9 (60.0%) participants. The majority of TEAEs were mild-moderate in severity. No TEAEs led to study discontinuation. Management noted that the submission of a rolling BLA in 3Q24 is on track. We note that a confirmatory trial is expected to begin enrollment in 2H25 prior to potential FDA approval under the accelerated approval pathway. Potential approval of RGX-121 could allow the company to receive a Priority Review Voucher. We reiterate our Buy rating and $39 PT."

For an analyst ratings summary and ratings history on Regenxbio Inc. click here. For more ratings news on Regenxbio Inc. click here.

Shares of Regenxbio Inc. closed at $11.28 yesterday.

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