Regenxbio a new Outperform at Mizuho on potential of two pipeline drugs

Mizuho initiated coverage on Regenxbio (NASDAQ:RGNX) with an outperform rating and $22.00 price target, citing its gene therapy pipeline. The stock is down 48% YTD. FDA placed a clinical hold on RGX-121, but the company reported positive Phase 2 data for diabetic retinopathy. Insider buying was also noted.

Regenxbio (RGNX) stock rose 3.5% in pre-market trading after presenting positive three-year data from its Phase II ALTITUDE study. 60% of participants showed clinically meaningful improvement, with no adverse events. Stifel maintained a Buy rating with a $42 price target. The broader market was modestly positive, but Regenxbio's pipeline developments drove the move.

Regenxbio Inc (RGNX) shares rose 4.8% premarket after presenting positive 3-year data for its gene therapy, surabgene lomparvovec, in diabetic retinopathy. The study showed sustained improvement without additional treatment. The company also settled patent litigation with Sarepta Therapeutics for $39.0 million.

Nasdaq and S&P 500 futures rose despite surging Treasury yields. Akamai (AKAM) surged 20% after a $11.6B deal with Anthropic. Tesla (TSLA) trended upward following a next-gen Roadster unveil and Semi truck production news. SpaceX (SPCX) gained on AI model predictions. Bloom Energy (BE) rose 1% after reaffirming Oracle's (ORCL) fuel-cell contract. Comcast (CMCSA) fell on analyst downgrades. Regenxbio (RGNX) rose 2% on positive gene therapy data.

REGENXBIO (Nasdaq: RGNX) presented positive three-year data from its Phase II ALTITUDE study of surabgene lomparvovec for diabetic retinopathy at the Retina Society Annual Meeting. The data showed durable efficacy with 60% of participants achieving a >2-step improvement on the DRSS. No new safety signals were observed. The company is advancing the treatment in a Phase IIb/III trial.

The FDA is scrutinizing REGENXBIO's RGX-202 and Solid Biosciences' SGT-003, both Duchenne muscular dystrophy treatments, for accelerated approval. The FDA requires evidence that microdystrophin expression predicts clinical benefit, citing Pfizer's failed CIFFREO trial as a cautionary example. REGENXBIO reported 93% of patients reached 10% microdystrophin expression, but functional data is limited. The FDA seeks robust evidence before approval, given the permanent nature of gene therapy.