What happened

Shares of Solid Biosciences (SLDB 5.26%) are up 24% as of 11:15 a.m. EDT as the company prepares to present long-term data for three patients in a phase 1/2 study of its treatment for Duchenne muscular dystrophy. The presentation will take place at the World Muscle Society Virtual Congress.  

A family outside of their front door.

Image source: Getty Images.

So what

Solid's drug candidate -- SGT-001 -- is a gene transfer therapy designed to address the absence of the dystrophin protein in those with the disease. Early data suggests SGT-001 could slow or stop the progression of Duchenne muscular dystrophy regardless of how far the disease has progressed.

The market for treatments is estimated to be slightly less than $1 billion, but it is growing more than 40% per year. To date, only one drug has been approved by the Food and Drug Administration (FDA) to treat Duchenne. That's a weekly hourlong infusion created by a subsidiary of a Japanese pharmaceutical company. Notably, that treatment from is a piece of synthetic messenger RNA. That's the same method both Moderna and BioNTech used to create their COVID-19 vaccines.

Now what

Shareholders will eagerly await the data on the three high-dose patients. In 2018, the drug was put on clinical hold by the FDA. Another hold was placed in 2019 when the agency determined a trial participant had suffered a kidney injury associated with SGT-001. In May, Solid shared yet another safety event during its first-quarter earnings call. 

Despite the concerns, Ultragenyx Pharmaceutical (RARE 1.23%) established a partnership with Solid last October. The deal gave Ultragenyx an exclusive license to certain products in exchange for a $40 million investment. It could be worth $255 million based on milestone payments. If the data today is positive, it should give the company negotiating leverage for any potential buyout. It might also give it a leg up in the race for a treatment. Sarepta Therapeutics (SRPT 1.30%) and Pfizer (PFE 0.76%) have Duchenne programs of their own.