Banking on Positive Initial Data for DMD Gene Therapy, RegenxBio Raises $140 Million

Rare Daily Staff

Two days after reporting positive initial efficacy data from its Duchenne muscular dystrophy trial that sent its stock up 15 percent, RegenxBio tapped the public markets to raise $140 million to advance its pipeline of AAV gene therapies.

The company priced an underwritten upsized public offering of 44.6 million shares of its common stock at $23.00 per share and 1.5 million pre-funded warrants at $22.9999 per warrant, before underwriting discounts and commissions. In addition, RegenxBio has granted the underwriters a 30-day option to purchase an additional 913,050 shares of common stock at the public offering price, less underwriting discounts and commissions.

Duchenne muscular dystrophy (DMD) is a severe, progressive, degenerative muscle disease, affecting 1 in 3,500 to 5,000 boys born each year worldwide. Duchenne is caused by mutations in the Duchenne gene which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy, and premature death.

RegenxBio’ data suggests that its gene therapy RGX-202 may work for older patients who are not currently eligible for the only approved gene therapy Elevidys, which is limited to patients 4 to 5 years of age.

“RGX-202 at dose level 2 is demonstrating significantly increased microdystrophin expression in a 12-year-old patient,” said Kenneth Mills, president and CEO of RegenexBio in the statement reporting the trial data. “We know there is an insufficient level of data available to the community for boys older than 7 years, and we are committed to being transparent with our data for a Duchenne community in need of new treatment options that can meaningfully impact disease.”

Photo: Kenneth Mills, president and CEO of RegenexBio