Regenxbio (RGNX) launched a pivotal study of its eye disease-targeting gene therapy, prompting RGNX stock to pop on Wednesday.
Late Tuesday, the biotech company also provided a roadmap for 2021. It’s planning to test a gene therapy in a form of muscular dystrophy and hopes to have Phase 2 data from another key test in the third quarter.
Chardan analyst Gbola Amusa sees RGNX stock as a top pick for 2021. Its eye-disease treatment is being tested in people with wet age-related macular degeneration, or wet AMD. Leaders in this market include Roche (RHHBY) and Regeneron Pharmaceuticals (REGN).
“Credible pivotal-stage AMD companies trade in the $2 billion to $7 billion market-cap range,” Amusa said in a report to clients. He notes competitors include Apellis Pharmaceuticals (APLS), Kodiak Sciences (KOD) and Iveric Bio (ISEE).
In morning trading on today’s stock market, RGNX stock rose 4.1% near 46.80. Earlier, shares popped as high as 10.1%.
RGNX Stock Jumps On Gene Therapy Update
Regenxbio will need to complete two Phase 3 studies before asking for approval of its wet AMD gene therapy in 2024.
The first will enroll roughly 300 patients and test Regenxbio’s treatment against ranibizumab, the generic name for Roche’s Lucentis. The second will pit Regenxbio’s gene therapy against the generic form of Regeneron’s Eylea.
Modest success in this market would make Regenxbio’s gene therapy a blockbuster, Chardan’s Amusa said.
Regenxbio’s late-stage effort tests an injection under the retina. The company is also studying the same gene therapy in wet AMD and diabetic retinopathy delivered via an injection in the suprachoroidal space. This is the space between the sclera and choroid of the eye.
The company expects to report interim results of that test in the third quarter. Chardan’s Amusa notes the suprachoroidal injection has been well tolerated with no inflammation. He has a buy rating and 100 price target on RGNX stock.
Muscular Dystrophy Test Planned
Regenxbio also announced plans to test a gene therapy in Duchenne muscular dystrophy. It will ask the Food and Drug Administration for permission to begin human testing in mid-2021.
This form of muscular dystrophy is caused by mutations in the gene that codes for dystrophin, a key protein for muscle strength and function.
SVB Leerink analyst Mani Foroohar also raised his price target on RGNX stock to 37 from 34, but kept his market perform rating following the update.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
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