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Agreements

Date: 2016-05-03

Type of information: Product acquisition

Compound: Kynamro® (mipomersen sodium)

Company: Ionis Pharmaceuticals (USA - CA) Kastle Therapeutics (USA - IL)

Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases

Type agreement:

product acquisition

Action mechanism:

antisense oligonucleotide. Mipomersen is a first-in-class apo-B synthesis inhibitor and acts by blocking the production of apolipoprotein B (apoB), the protein that provides the structural core for these atherogenic particles, including LDL and lipoprotein-a (Lp(a)).

Disease: homozygous familial hypercholesterolemia (HoFH)

Details:

* On May 3, 2016, Ionis Pharmaceuticals and Kastle Therapeutics announced that Kastle has acquired global rights to develop and commercialize Kynamro® (mipomersen sodium) injection. Kynamro®) is approved in the United States for use in patients with homozygous familial hypercholesterolemia (HoFH) to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apoB), total cholesterol (TC) and non-high density lipoprotein-cholesterol (non-HDL-C) as an adjunct to lipid lowering medications and diet.

Kastle was formed in 2015 to focus on acquiring, developing and commercializing pharmaceuticals targeted toward diseases with high unmet medical needs. Kynamro® is Kastle's first acquisition.

Financial terms:

Under the terms of the agreement, Ionis is eligible to receive up to $95 million, which includes a $15 million up-front payment, a $10 million payment three years from today and up to $70 million in sales milestones. Beginning in 2017, Ionis will earn royalties on global sales of Kynamro® in the mid to low teens. In addition, Ionis will receive a 10 percent common equity position in Kastle's parent company. Sanofi Genzyme will earn a 3 percent royalty on sales of Kynamro® and 3 percent of the cash payments Ionis receives from Kastle.

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