Isis Pharmaceuticals said today it won a $2 million milestone payment from GlaxoSmithKline (GSK) related to advancement of an ongoing Phase II/III study of ISIS-TTRRx, an antisense drug being developed with the pharma giant for the rare genetic disease transthyretin (TTR) amyloidosis.

The payment represents the second milestone payment attained this year, and the first portion of up to $50 million in milestone payments for which Isis is eligible based on the progress of the Phase II/III study. ISIS-TTRRx, which inhibits the production of the carrier protein TTR, is being assessed for patients with familial amyloid polyneuropathy (FAP), the indication for which FDA has granted Isis fast-track designation and orphan drug status.

Isis has already received $20 million in up-front and milestone payments before the dosing of the first patient in the Phase II/III study. That includes a $7.5 million payment Isis earned in February upon initiation of the study.

If GSK elects to exercise its option to exclusively license the ISIS-TTRRx program, Isis would also be eligible to receive a license fee, additional regulatory and sales milestone payments, and double-digit royalties on sales of ISIS-TTRRx.

The Phase II/III study of ISIS-TTRRx is a 15-month randomized, double-blind, placebo-controlled, international trial designed to measure the effects of ISIS-TTRRx given to patients for 65 weeks on neurological dysfunction and quality of life. The study (NCT01737398) was recruiting patients as of July 19, according to

Isis’ pipeline includes 28 drugs designed for a variety of diseases, with emphasis on cardiovascular, metabolic, severe and rare diseases, and cancer. The company’s lead product, the once-weekly injection anti-LDL cholesterol drug Kynamro, is being commercialized by collaboration partner Genzyme for patients with the rare genetic condition homozygous familial hypercholesterolemia (HoFH).

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