Mizuho names its top SMID biotech pick for November

Mizuho Securities has selected its top pick in the small- and midcap biotechnology sector for November, maintaining an Outperform rating.

The gene therapy company reported third-quarter results that beat revenue expectations and showed lower operating expenses than estimated. The company reaffirmed key late-stage pipeline milestones that underpin Mizuho's bullish thesis on the stock.

1. Regenxbio Inc (NASDAQ:RGNX) - Mizuho maintained an Outperform rating with a price target of $18.00, up from $7.71.

Regenxbio is advancing two gene therapy candidates that Mizuho believes have multi-billion-dollar franchise potential.

The company confirmed critical upcoming milestones for sura-vec, its treatment for wet age-related macular degeneration, with top-line Phase 3 data expected in the fourth quarter of 2026. Management reiterated the efficacy target of at least 50% reduction in treatment burden with a meaningful share of patients becoming injection-free.

For RGX-202 in Duchenne muscular dystrophy, the company is preparing for a fourth-quarter 2026 pre-BLA meeting with the FDA, followed by a BLA submission in the first quarter of 2027. The company has positive Phase 3 data in hand and aims to make the case for accelerated approval.

Management highlighted that a statistically significant microdystrophin-to-NSAA correlation, a safety database of over 60 patients, and a unique construct including a C-terminal domain should collectively support accelerated approval.

Third-quarter revenue of $9.2 million exceeded Mizuho's estimate of $5.9 million. The company has trained approximately 500 surgeons on sura-vec administration, focusing on high-volume practices for potential commercial readiness. Partner AbbVie is conducting a global Phase 3b study with approximately 560 patients, primarily for reimbursement purposes.