Mizuho names its top SMID biotech pick for November

Mizuho Securities has designated Regenxbio Inc (NASDAQ: RGNX) as its top small- and midcap biotechnology pick for November. The firm has maintained an Outperform rating on the stock, with a price target of $18.00, up from the previous $7.71. Regenxbio is currently advancing two gene therapy candidates that analysts believe have franchise potential worth billions of dollars.

The company reported third-quarter results that surpassed revenue expectations while demonstrating lower operating expenses than anticipated. These positive financials support Mizuho's bullish outlook on Regenxbio. The company has also reaffirmed key milestones in its late-stage pipeline, which bolster its investment thesis.

Regenxbio's lead treatment, sura-vec, is a gene therapy for wet age-related macular degeneration. The company has received top-line Phase 3 data, with a target of at least a 50% reduction in treatment burden expected by the fourth quarter of 2026. Management aims to make a compelling case for accelerated approval of sura-vec, bolstered by a statistically significant correlation between microdystrophin and NSAA, a substantial safety database of over 60 patients, and a unique construct incorporating a C-terminal domain.

In the field of Duchenne muscular dystrophy, Regenxbio is preparing for a pre-Bulletin of New Drugs (BLA) meeting with the FDA in Q4 2026, followed by a BLA submission in Q1 2027. The company has already trained around 500 surgeons on the administration of sura-vec, with a focus on high-volume practices to ensure commercial readiness.

Regenxbio has partnered with AbbVie on a global Phase 3b study involving approximately 560 patients, primarily for reimbursement purposes.