On Tuesday, H.C. Wainwright initiated coverage of Regenxbio Inc. (NASDAQ:RGNX), a clinical-stage biotechnology company, with a Buy rating and a price target of $36.00. The firm highlighted Regenxbio's focus on developing gene therapies for ophthalmic disorders and rare diseases utilizing its proprietary NAV technology platform. This platform includes a portfolio of adeno-associated virus (AAV) vectors, such as AAV8 and AAV9, which have been clinically and commercially validated.
The NAV AAV9 vector has been used in the development of Zolgensma, a treatment for spinal muscular atrophy (SMA) marketed by Novartis (SIX:NOVN), which achieved $1.2 billion in net sales in 2023. Regenxbio's pipeline features several clinical programs, including ABBV-RGX-314 for retinal diseases like wet AMD (NASDAQ:AMD) and diabetic retinopathy, RGX-121 for the neurodegenerative disease MPS II (Hunter syndrome), and RGX-202 for the neuromuscular disease DMD (Duchenne muscular dystrophy).
H.C. Wainwright pointed out that there are several near- to mid-term catalysts for Regenxbio, including topline results from the pivotal trials of ABBV-RGX-314 in wet AMD expected in 2025, the submission of a Biologics License Application for RGX-121 in MPS II in the second half of 2024, and the initiation of a pivotal trial for RGX-202 in DMD also in the second half of 2024.
The firm noted the significance of the Rare Pediatric Disease designations granted to both RGX-121 and RGX-202, which could lead to the company receiving a Priority Review Voucher worth approximately $100 million upon potential regulatory approval.
The analyst's optimistic outlook is based on the potential market value of the Priority Review Voucher and the company's robust pipeline of gene therapy candidates. The $36.00 price target reflects a 12-month projection for the company's stock value.
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