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Ionis Options Cardiac Drugs in $1.5 Billion Deal

Heart check

(Gerd Altmann, Pixabay)

6 January 2016. Biotechnology company Ionis Pharmaceuticals is providing drug maker Novartis first access to two experimental drugs for controlling proteins that promote cholesterol and triglycerides, which contribute to heart disease. The option agreement with Novartis could bring Ionis as much as $1.5 billion if Novartis agrees to license and fully commercialize both drugs.

The drugs being optioned are code-named APO(a)-LRx and APOCIII-LRx. APO(a)-LRx reduces a protein known as lipoprotein-a that connects to LDL cholesterol — the “bad” cholesterol —  which collects in blood vessels, and is considered a risk factor for coronary heart disease, atherosclerosis, thrombosis, and stroke. About 1 in 5 people inherit a condition where they produce high quantities of lipoprotein-a, who often cannot control these elevated levels with diet and exercise.

APOCIII-LRx is designed to reduce levels of apolipoprotein CIII, or ApoC-III, a protein produced in the liver that contributes to triglycerides, a fatty substance similar to cholesterol that can also build up in blood vessels and contribute to heart disease. People that produce ApoC-III not only have a higher risk of heart disease, they also often have metabolic disorders such as insulin resistance and metabolic syndrome, as well as a higher risk of type 2 diabetes.

APO(a)-LRx and APOCIII-LRx are the lead products of Akcea Therapeutics, a subsidiary of Ionis that specializes in lipid disorders. In an early- and intermediate-stage clinical trials, APO(a)-LRx was shown to reduce lipoprotein-a in people with elevated levels of the protein an average of 79 percent with a small dose and 92 percent with multiple doses. APOCIII-LRx is currently being tested with healthy volunteers in an early- and intermediate-stage trial.

Both drugs are produced with Ionis’s technology platform known as antisense, which uses short DNA or RNA sequences, called oligonucleotides, designed to complement a specific gene sequence. Ionis says it designs antisense drugs to interact precisely with a specific sequence of RNA, with many of the therapies in its pipeline binding to messenger RNAs, or mRNAs, and blocking the production of disease-causing proteins.

The agreement gives Novartis an option to later license both drugs from Ionis. Under the deal, Novartis is paying Ionis a $225 million option fee and taking a $100 million equity stake in the company. Novartis and Ionis will collaborate on intermediate-stage clinical trials to determine dosages of each drug for subsequent late-stage trials. Following the intermediate-stage studies, Novartis will decide whether to exercise its option to license the drugs.

Should Novartis decide to exercise its option, Ionis, in Carlsbad, California will receive a licensing fee of $150 million for each drug. Ionis will also be eligible for $600 million in development and commercialization milestone payments for APO(a)-LRx and $530 million in milestone payments for APOCIII-LRx. Novartis also agrees to make another $50 million equity investment in either Ionis or Akcea in the next 18 months.

The agreement gives Ionis the option to co-market either drug to lipid disease specialists through Akcea’s sales force. In addition, Ionis will be eligible for royalties on sales of each drug.

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