Regenxbio Stock Climbs on Positive 3-Year Data for Diabetic Retinopathy Gene Therapy

Regenxbio Inc. (NASDAQ: RGNX) shares gained 3.5% in pre-market trading after the company announced positive three-year follow-up data for its gene therapy candidate, surabgene lomparvovec, in patients with non-proliferative diabetic retinopathy (NPDR).

Key Clinical Findings

The results, presented on September 24 at the Retina Society 59th Annual Scientific Meeting, came from the Phase II ALTITUDE study. The data showed sustained efficacy and a favorable safety profile over a three-year period.

Key takeaways from the pivotal Dose Level 3 cohort include:

• 60% of participants who reached the three-year mark achieved a greater than two-step improvement on the Diabetic Retinopathy Severity Scale (DRSS), a clinically meaningful endpoint.
• Patients in this group experienced no vision-threatening events.
• No additional treatments for diabetic retinopathy were required for these participants over the three years.

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In a statement, Chief Medical Officer Steve Pakola called the results "highly encouraging," adding that they "reinforce the potential of sura-vec to change the treatment landscape for the millions of patients with non-proliferative diabetic retinopathy."

Market Impact and Outlook

The positive data provided a clear catalyst for the stock, which had been trading near its 52-week low of $5.46. The durability of the treatment effect is a critical factor for gene therapies targeting chronic conditions, and these results offer investors increased confidence in the drug's long-term potential.

Following the announcement, analysts at Stifel reiterated a Buy rating on Regenxbio with a $42 price target. The data is particularly relevant as the same dose level is being used in the company's ongoing Phase IIb/III NAAVIGATE pivotal trial, directly supporting the rationale for the registrational program.