• Novartis said Monday it will exercise a licensing option on an experimental heart drug, paying $75 million to both Ionis Pharmaceuticals and Akcea Therapeutics as it takes over development and plans a Phase 3 cardiovascular outcomes trial.
• The experimental therapy is focused on lowering elevated levels of lipoprotein(a), or Lp(a), an independent genetic factor of cardiovascular disease. A Phase 2 study of about 270 patients showed the drug’s effectiveness at bringing down Lp(a) levels below 50 mg/dL, a threshold for Lp(a)-driven cardiac events such as a heart attack or a stroke.
• The $150 million payment is evenly split between Ionis and its majority-owned affiliate, Akcea, with shares in both respectively increasing by roughly 5% and 15% in value Monday.

Novartis is showing an appetite for staying in the cardiovascular space despite some high-level misses in the past few years. 

In 2017, an experimental treatment called serelaxin failed a Phase 3 heart study, crimping hopes for a drug the Swiss pharma had previously pegged as a potential blockbuster. And last October the Food and Drug Administration rejected canakinumab for a label expansion as a cardiovascular risk reduction therapy, despite a positive six-year-long Phase 3 study that enrolled more than 10,000 patients.

But Novartis feels confident enough in this investigational Lp(a)-lowering drug to commit once again in cardio. In addition to the $150 million payout, Novartis is on the hook for the costs of running the Phase 3 study, which will focus on Lp(a) patients.

The drugmaker first partnered with Ionis and Akcea in January 2017 over two cardiovascular drugs, including this experimental therapy which Novartis has now renamed TQJ230 from AKCEA-APO(a)-LRx.

Efficacy in lowering Lp(a) could set the experimental therapy apart from other cholesterol-lowering drugs, particularly statins, which do not affect Lp(a), Brett Monia, Ionis’ chief operating officer said in an interview with BioPharma Dive last month.

In a Feb. 25 statement, Novartis’ chief medical officer John Tsai echoed the market opportunity for TQJ230. “No treatments are currently available to substantially lower Lp(a),” Tsai said. “People with this inherited risk factor are facing cardiovascular risks that cannot be addressed effectively with lifestyle changes.”

Phase 3 study design details have yet to be shared by Novartis, although the Phase 2 study tested a wide array of dosing and frequency levels for the therapy that could serve as a guide.

The mid-stage clinical results were presented last November at the American Heart Association Scientific Sessions. 

While approval is a long ways out and will hinge upon successful Phase 3 results, a commercialized lipoprotein candidate could bolster Novartis’ cardiovascular offerings, which are now heavily dependent on Entresto (sacubitril/valsartan), which posted $1 billion in net sales in 2018.