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Isis gains milestone from GSK for rare disease trial and Aegerion takes them on in HoFH

Authored by Karl Simpson

Isis Pharmaceuticals is developing a drug for the treatment of the rare diseases transthyretin amyloidosis. The disease is a severe and rare genetic disorder characterized by dysfunction of the peripheral nerve and/or heart tissues. The company have now announced that they earned a further milestone on the drug, ISIS-TTRRX, an antisense drug.

Isis Pharmaceuticals is developing the drug with partner GSK, a large pharmaceutical company with a commitment to rare diseases. The milestone related to the advancement of the ongoing phase 2/3 study in patients with familial amyloid polyneuropathy (FAP). This milestone of $2m, is added to $20m already paid to the company and is the first milestone of a potential $50m the company will be eligible for as the study progresses. GSK also has an option to extend the agreement with Isis which would involve a licensing arrangement, triggering a number of other payments linked to the product license, development and commercialisation milestones or royalties.

Isis Pharmaceuticals is a company looking to drive its antisense technology towards the development of various therapeutic assets for multiple disease indications, including cancer and rare diseases. The company has already enjoyed some success with its pipeline, which currently houses 28 drugs, when it gained FDA regulatory approval with partner Genzyme for the product KYNAMRO™ for the treatment of HoFH (homozygous familial hypercholesterolemia), a product which rivals Aegerion’s Juxtapid.

Aegerion are showing that they are competing incredibly well in this commercialisation head-to-head after the company announced results yesterday, pointing to very impressive early numbers for Juxtapid sales. Aegerion has already garnered $7.7m in Juxtapid sales since its launch earlier this year. Aegerion expects to exceed its prior guidance of 250-300 patients on Juxtapid therapy by the end of 2013, leading to raised guidance on sales and a 26% leap in yesterday’s share price.

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