BioPharma

Atrium Therapeutics Launches With $270M for RNA Therapies Addressing Rare Cardio Conditions

Atrium spun out of Avidity Biosciences, which Novartis acquired in a $12 billion deal. The Atrium pipeline is led by two RNA therapies with a path to the clinic for rare inherited cardiological disorders that have no FDA-approved drugs.

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When Novartis agreed to buy RNA therapies developer Avidity Biosciences last fall, the companies said the plan was to spin out Avidity’s cardiology assets into a separate, publicly traded company. That biotech has launched bearing the name Atrium Therapeutics.

Atrium’s debut followed Novartis’s Friday close of its $12 billion acquisition of Avidity, which includes two late-clinical programs for rare neuromuscular disorders. The pipeline Atrium inherits consists of two precision cardiology candidates and two undisclosed research targets. Avidity is providing the San Diego-based spinout with about $270 million in cash and cash equivalents.

Avidity’s RNA therapies came from a proprietary platform technology that combines antibodies with RNA to yield a new type of RNA therapy called an antibody oligonucleotide conjugate, or AOC. RNA therapies preferentially target the liver. AOCs leverage the targeting ability of an antibody to deliver RNA to a wide range of tissue types in the body.

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Atrium’s two lead candidates are in preclinical development for rare cardio conditions with no FDA-approved therapies. ATR 1072 is a potential treatment for protein kinase AMP-activated non-catalytic subunit Gamma 2 syndrome (PRKAG2), a rare inherited disorder in which mutations in the PRKAG2 gene lead to early onset cardiomyopathy. Atrium said it expects to file an FDA investigational new drug (IND) application in the second half of this year.

The second cardio program, ATR 1086, is a potential treatment for phospholamban (PLN) cardiomyopathy, a rare inherited cardiac disease caused by mutations in the PLN gene that lead to a significantly higher risk of heart failure and sudden cardiac death. The company plans to start the preclinical research that could support an IND filing for the therapy this year with a goal of an IND submission next year.

Atrium also inherits research collaborations with Bristol Myers Squibb and Eli Lilly. When the Avidity acquisition agreement was announced last October, Novartis CEO Vas Narasimhan said the planned spinoff was not due to antitrust concerns, but rather because it was the most straightforward way to deal with those collaborations.

Per terms of the separation agreement, Avidity shareholders were eligible to receive one share of Atrium stock for every 10 shares of Avidity common stock that they owned. Following the separation, Avidity holds no ownership stake in Atrium. Shares of Atrium began trading on the Nasdaq Friday under a modified version of Avidity’s former “RNA” stock symbol — “RNAM.”

Former Avidity CEO Sarah Boyce is chair of Atrium’s board of directors. Atrium is led by President and CEO Kathleen Gallagher, who was Avidity’s chief program officer.

“The launch of Atrium Therapeutics marks an important milestone for people living with genetic cardiomyopathies,” Gallagher said in a prepared statement. “Patients and families facing these genetically driven rare cardiomyopathies have few if any options that address the underlying cause. Building on Avidity’s pioneering work in targeted RNA delivery, Atrium is positioned to advance precision medicines designed to directly target the biologic drivers of cardiac disease.”

The most advanced Avidity neuromuscular program now under Novartis is delpacibart zotadirsen, or del-zota, an AOC for Duchenne muscular dystrophy. In regulatory filings, Avidity said it reached alignment with the FDA last fall on a path for a regulatory submission, which is planned for 2026. The other Avidity neuromuscular program that now belongs to Novartis is delpacibart etedesiran, or del-desiran, a potential RNA therapy for myotonic dystrophy type 1. Avidity said in regulatory filings that 54-week topline data from a Phase 3 clinical trial are expected in the second half of this year.

Photo: Wong Yu Liang, Getty Images