Dive Brief:
- Regenxbio revealed new data from an early trial of a gene therapy for Duchenne muscular dystrophy that suggests it may work for older patients with the muscle-wasting disease.
- The findings are potentially significant because the only approved gene therapy for Duchenne is currently limited to patients who are 4 or 5 years old, able to walk and don’t have a genetic mutation that can negatively affect treatment. Sarepta Therapeutics is seeking an expanded label for that product, sold as Elevidys, despite the failure of a confirmatory trial late last year.
- Regenxbio has data from five patients who have received its RGX-202 therapy without any serious side effects. On Tuesday, the company reported promising results for a 12-year-old patient and said new recordings of patients given the treatment show evidence of strength and functional improvement. The company’s shares surged 15% on the news.
Dive Insight:
Though very early, the data could offer a new path forward for Regenxbio, which to date has largely been known for its adeno-associated virus vectors used to deliver gene therapies, and an experimental eye disease treatment in development with AbbVie.
Tuesday’s stock increase suggests a switch in investor focus that may provide a “tailwind” to Regenxbio for the next year, Leerink analyst Mani Foroohar wrote in a note to clients Wednesday. Leerink raised its rating on the stock to “outperform” from “market perform” based on the new data.
Duchenne is an inherited disease that primarily affects boys, eroding their muscles until many end up in a wheelchair at an early age. Given the severity of the disease, the Food and Drug Administration has appeared to take a more flexible approach in approving new therapies — a stance that may provide Regenxbio a favorable regulatory path forward, Foroohar wrote.
The opposite may be true of the company’s more advanced program in wet age-related macular degeneration. Experimental gene therapies for eye conditions have suffered failures and would be competing with approved drugs such as Eylea and Lucentis that have proven safe and effective, leaving the regulatory bar high for genetic medicines.
Regenxbio has a long way to go with its Duchenne therapy. The company plans to begin a pivotal trial in the second half of this year. If successful, the therapy would still have to carve out its path in the market and may face new competition from a treatment under development at Pfizer.