When Novartis acquired Avidity Biosciences last year, the goal was to expand its neuroscience pipeline and gain access to Avidity’s RNA-targeting delivery platform. But Avidity had more to offer in its pipeline.
Just six months later, Atrium Therapeutics emerged from the aftermath of that deal, with Avidity spinning out the new independent, publicly traded company to focus on precision RNA therapeutics for rare, genetic cardiomyopathies. Atrium also has a licensing agreement with Novartis for the RNA-targeting delivery platform.
“We are launching with our own programs and with a focus in the precision cardio space, but using all of that validated platform technology,” said Kathleen Gallagher, Atrium Therapeutics’ president and CEO.
Designed to deliver small interfering RNA directly to the heart muscle, Atrium’s platform is aimed at a historically difficult feat.
“RNA therapeutics have, for years, really made an impact on people, but they have been limited to diseases that stemmed from the liver,” Gallagher said.
This platform’s siRNA therapeutics are linked to a monoclonal antibody that targets the transferrin receptor.
“We’re utilizing the body’s own mechanism of transmitting things around it to deliver our medicine,” Gallagher said.
Atrium’s launch comes with $270 million worth of funding, a Novartis-validated platform, and pipeline candidates with the potential to treat the underlying cause of rare cardiomyopathies with no approved therapies. Both of its lead assets are in the early stages of development, with the company aiming to file an IND for one in the second half of 2026 and for the other in 2027. The company’s two preclinical programs are targeting other undisclosed types of cardiomyopathies, Gallagher said.
Atrium’s leadership is also stacked with folks who made the leap from Avidity, including Gallagher, who was most recently Avidity’s chief program officer and oversaw the neuromuscular and precision cardiology programs while guiding its portfolio strategy and corporate affairs. Prior to that, she was the global program head for Avidity’s myotonic dystrophy type 1 program.
“I was able to help lead the team in developing the phase 3 trial that is currently ongoing and move that program forward into late-stage development,” she said.
That technology was designed at Avidity, which was the first company to successfully deliver RNA therapeutics to muscle tissue in humans. Now Atrium is applying it to the heart.
While Atrium’s first two candidates target rare genetic cardiomyopathies, the overall market is relatively large, with about 2 million people in the U.S. living with cardiomyopathy. Leveraging genetics could also broaden Atrium’s reach, since researchers believe about 50% of cardiomyopathies have an underlying genetic driver, Gallagher said.
“Each one of these therapies may have an ability to treat a broader patient group within the genetic cardiopathy patient population,” Gallagher said of Atrium’s first two candidates.
She envisions the company starting in those rare diseases to establish a proof of concept “then take those learnings and go into these broader-based populations.”
In the meantime, their lead assets will target the underlying cause of severe, life-threatening conditions. ATR 1072 is in development to treat protein kinase AMP-activated non-catalytic subunit Gamma 2 syndrome while ATR 1086 was designed to treat phospholamban cardiomyopathy.
“These patients today are basically getting symptomatic treatment. In some cases that could mean they’re getting a pacemaker or it could mean they’re on a generic heart failure medication,” Gallagher said. “But they aren’t getting anything that actually targets the root cause of their disease. They’re getting symptomatic management, but they’re not actually stopping the progression of the disease.”
A ‘burgeoning field’
Gallagher said it’s an exciting time to be working in this “burgeoning field,” not only because of the unmet medical need but also the growing interest.
“More patients are getting genetically tested when they have some of these symptoms. And we're also seeing more companies realize that this is a space where there is a non-bet medical need,” she said.
Among those companies is Alnylam Pharmaceuticals, which last year scored an FDA nod for its RNAi therapeutic, Amvuttra, to treat cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis. Alnylam also recently inked a research collaboration with Tenaya Therapeutics to identify and validate cardiovascular disease genetic targets.
“We're kind of at the tip of the spear here and able to pave the path for this type of medicine in the cardiology space,” Gallagher said.
