Regenxbio Prioritizes Pipeline, Cuts 15 Percent of Workforce

Rare Daily Staff

Regenxbio announced a strategic pipeline prioritization and corporate restructuring intended to significantly reduce operating expenses while continuing to support value generation from the company’s pipeline of AAV therapeutics.

“Today, we are following the data and making necessary decisions to focus our capabilities and resources on these differentiated product candidates, which represent opportunities to bring ground-breaking AAV Therapeutics to millions of patients,” said Kenneth Mills, president and CEO of Regenxbio. He noted that the company reported positive clinical data over the past two months from investigational treatments for diabetic retinopathy, and Duchenne, as well as its efforts to expedite the BLA for its treatment for mucopolysaccharidosis type II.

Under the new plan, the highest priority programs are ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, which is being developed in collaboration with AbbVie; and RGX-202 for the treatment of Duchenne; and RGX-121 for the treatment mucopolysaccharidosis type II.

Regenxbio said it will pursue strategic alternatives, including potential partnering, for its other rare neurodegenerative disease clinical stage programs: RGX-111 for the treatment of severe mucopolysaccharidosis type I, RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, and RGX-381 for the treatment of the ocular manifestations of CLN2 disease.

The restructuring plan includes a 15 percent reduction in workforce, primarily in rare neurodegenerative disease development, early research, and other general and administrative areas. The company expects to incur approximately $4 million in one-time restructuring costs in the fourth quarter of 2023.

As a result of the portfolio prioritization and corporate restructuring, Regenxbio anticipates total savings of at least $100 million over the next two years, which along with $365 million in cash, cash equivalents and marketable securities as of September 30, 2023, are now expected to fund operations into the second half of 2025.