Regenxbio stock gains on positive gene therapy trial data

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.

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.

Six Montgomery County companies raised over $566 million in Q3. Forbright (Nasdaq) completed an IPO, raising $142.2 million. Xometry (Nasdaq) announced a $225 million secondary offering. RegenxBio (Nasdaq) raised $90.04 million in a public offering. Georgiamune secured $92.69 million in venture funding. Arculus Solutions raised $1.95 million in Seed-1 funding. Jesse & Ben raised $15 million in Series A funding.

Regenxbio Inc. (RGNX) faced an FDA clinical hold for RGX-121, its gene therapy for Hunter syndrome, due to spinal abnormalities in five patients. Shares fell over 24% premarket. The abnormalities were deemed non-serious and likely benign, with patients asymptomatic. The FDA's full clinical-hold letter and additional data are awaited to determine next steps.