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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.

The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.

Pfizer announces the first data from its Metsera-acquired pipeline just ahead of its earnings call, where analysts pressed execs for more details; Merck and Roche also released Q4 and full year earnings, with Eli Lilly, Novo Nordisk and others reporting Wednesday; REGENXBIO hits a regulatory snag ahead of its upcoming PDUFA; more.

In what is shaping up to be a back-loaded month, the FDA is set to release a slew of regulatory decisions in February, including two that would expand the labels of blockbuster drugs.

The FDA has postponed its decision date for Regenxbio’s Hunter syndrome gene therapy to review additional longer-term clinical data for the asset.

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.

In exchange for its investigational gene therapies, Regenxbio will receive $110 million upfront and up to $700 million in milestones. After hitting an all-time low of $6.95 at close of business yesterday, the stock surged on the news by nearly 20% before markets opened Tuesday.

REGENXBIO Inc. announced that Curran Simpson, Chief Operating Officer, has been appointed as President, Chief Executive Officer and a member of the Board of Directors, effective July 1, 2024.

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.

REGENXBIO Inc. announced it will participate in the following upcoming investor conferences.