Shares of Avidity Biosciences surged to a record high Wednesday after the company showed its hand in the cardiovascular space — a first for the company's RNA platform.
This has the potential to be "game-changing," Chief Executive Sarah Boyce told Investor's Business Daily in a recent interview. Avidity plans to test out treatments for two rare diseases, PLN cardiomyopathy and PRKAG2 syndrome.
Both diseases are driven by genetic causes. In laboratory testing, Avidity's drugs lowered the amount of disease-causing protein in the heart by about 80%.
Generally, cardiologists don't study the genetics of their patients.
"We are proud to be the first company to successfully deliver siRNA (small interfering RNA) directly to skeletal muscle and now, we have demonstrated that we can deliver siRNA against targets in the heart in preclinical studies," Boyce said in a written statement.
Avidity Biosciences stock jumped 12.4% to 52.50. Shares trounced their previous record high at 50.76.
Avidity Biosciences Enters Cardio Space
There are no approved treatments for PLN cardiomyopathy or PRKAG2 syndrome. They are caused by mutations in the PLN and PRKAG2 genes, respectively. The former causes dilation of the heart chambers, sudden cardiac death and heart failure. The latter leads to thickening of the heart muscle, irregular heartbeats and other problems.
So far, Avidity Biosciences has tested its treatments in nonhuman primates.
"However, it is important to note the small sample of NHP data presented, and, as yet, no data presented from a disease model," Chardan analyst Keay Nakae said in a report. "A major feature of both diseases is the physiological changes that occur in the heart, and it is yet to be determined if either of these therapies will lead to a functional reversal of those changes."
Still, the move is unique for the RNA space. These drugs try to silence the genes responsible for making disease-causing proteins. Biotech companies first focused on delivering RNA-targeting drugs to the liver. Now, they're expanding into other tissue types.
Separately, Avidity Biosciences ultimately plans to test 10 cardiovascular targets with partner Bristol Myers Squibb.
"We're looking to lead the space," Boyce told IBD.
Top 1% Biotech Stock
Avidity Biosciences stock had soared more than 416% this year, as of Tuesday's close.
Shares have a perfect IBD Digital Relative Strength Rating of 99. This puts them in the top 1% of all stocks in terms of 12-month performance.
This story has been corrected to reflect Avidity is developing the PLN cardiomyopathy and PRKAG2 syndrome treatments alone.
Follow Allison Gatlin on X, the platform formerly known as Twitter, at @IBD_AGatlin.
