Avidity Biosciences unveiled "compelling" results from a muscular dystrophy study Wednesday, leading RNA stock to skyrocket more than 50% to a three-month high.
The biotech company tested its treatment in patients with myotonic dystrophy 1. This form of muscular dystrophy is caused by a change in a gene known as DMPK, which makes a protein involved in muscle function. After a single dose, Avidity's drug led to an average 45% reduction in the amount of protein created by that gene. Every patient in the study responded.
SVB Securities analyst Joseph Schwartz called the results "compelling." Avidity tested two doses of its drug, dubbed AOC 1001, in a study of 20 patients. Fifteen patients received the treatment.
"We think the initial data (are) promising for 1001's clinical profile as we would expect the treatment response to deepen over time," he said in a note to clients.
On the stock market today, RNA stock launched itself 55.3% to 17.05.
RNA Stock: Silencing Genes
Avidity is part of a group of companies attempting to silence genes responsible for troublesome proteins. In a first-ever move, Avidity injected its treatment into patients' muscles. The results suggest knocking down the DMPK protein could benefit these patients.
SVB's Schwartz says this could allow Avidity to enter a big market. He currently projects roughly $4.2 billion in gross global sales for AOC 1001. He kept his outperform rating on RNA stock.
Importantly, the drug appears safe so far. Earlier this year, the Food and Drug Administration blocked Avidity from enrolling new patients in its study after one patient who received AOC 1001 experienced a serious side effect.
"It seems as though Avidity expects to submit their response to the partial clinical hold in the first quarter, and receive a response by the FDA within 30 days or so, which all sounds encouraging for the potential to remove the hold in the relatively near term," he said.
The news Wednesday helped RNA stock retake its key moving averages, according to MarketSmith.com.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
