REGENXBIO

Shares of REGENXBIO declined 37% on a mixed data readout and other updates from the company’s first quarter earnings call Thursday.

Comprehending the spate of recent rejections in the cell and gene therapy space may require looking no further than early-stage clinical trials of candidates from REGENXBIO, Excision BioTherapeutics and Intellia Therapeutics.

Avlayah is the first Hunter syndrome therapy approved to address the condition’s neurologic complications, according to Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research.

The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.

In a complete response letter published by the FDA on Monday, the agency said a resubmission for REGENXBIO’s Hunter syndrome gene therapy should provide evidence of normalized or improved biomarker levels or neurodevelopmental outcomes.

A lower court had previously ruled for Sarepta in the companies’ long-running dispute, finding that REGENXBIO’s AAV patent was invalid because its elements were naturally occurring. The appeals court on Friday said that this original decision adopts a “narrow” view of the invention.

The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.

With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.