Novartis has agreed to pay $150 million to Ionis Pharmaceuticals and Boston-based Akcea Therapeutics for the rights to a clinical-stage drug meant to treat patients with abnormally high levels of a protein that is considered a risk factor for cardiovascular disease.
The experimental drug, known as AKCEA-APO(a)-LRx by Akcea and TQJ230 by the Swiss pharma giant, was discovered by Ionis, which is based near San Diego in Carlsbad, CA, and developed jointly by Ionis and Akcea. It is designed to lower elevated levels of lipoprotein(a), or Lp(a), in patients with cardiovascular disease by inhibiting production of a protein that promotes the formation of plaques in the arteries and blood clots.
Unlike some cases of high cholesterol, elevated Lp(a) is generally not manageable through diet or exercise, according to the Lipoprotein(a) Foundation. High Lp(a) levels are viewed as a genetic cause of coronary artery disease, heart attack, stroke, and peripheral arterial disease, according to Akcea. There are eight million to 10 million “treatable” patients with cardiovascular disease and elevated levels of Lp(a), according to Akcea. No FDA approved treatment exists to treat the condition.
Akcea (NASDAQ: AKCA) spun off from Ionis (NASDAQ: IONS) in 2017 with a $125 million initial public offering. Ionis founded the company as its lipid-drug focused unit in 2014 and Novartis (NYSE: NVS) began collaborating with Akcea a little over two years ago, when it paid $75 million upfront for the right to license two of the company’s compounds, including this one. The drug is part of a class of RNA-based therapeutics called antisense drugs, which are designed to block the production of harmful proteins by synthesizing a strand of nucleic acid that binds to a gene’s messenger RNA and stops it from making the disease-causing protein.
Akcea’s stock price rose to $31.91 per share on the news Monday, up 17 percent from the closing price Friday. Shares of Ionis’s stock rose 5 percent to $61.67 apiece. Novartis stock remained flat at about $91 per share.
Boston-based Akcea presented Phase 2 results for the drug candidate in November at an American Heart Association conference in Chicago. Results from the dose-ranging study were promising: Nearly all 48 patients in the cohort who received a 20 mg dose weekly had Lp(a) levels reduced below a threshold the company used to indicates whether a patient is at risk for heart attack or other cardiovascular-related event. About 81 percent of patients who received a 60 mg dose every four weeks also had levels fall below that threshold, according to Akcea.
Most side effects were mild, according to the company. The most common side effect was a reaction at the injection site, which was reported by about 26 percent of the 286 patients evaluated in the study. One patient ended treatment due to the side effect.
Akcea will split the license fee from Novartis with Ionis, which will receive its $75 million cut in the form of Akcea common stock.