Rare Daily Staff

Pfizer is paying Akcea Therapeutics and its parent Ionis Pharmaceuticals $250 million upfront for an exclusive global license for AKCEA-ANGPTL3-LRx, an experimental antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases.

The deal is valued at up to $1.55 billion. Akcea will split the fees equally with Ionis and provide Ionis with $125 million in Akcea common stock for its share of the upfront fee.

AKCEA-ANGPTL3-LRx is an experimental antisense therapy designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver, a key regulator of triglycerides, cholesterol, glucose and energy metabolism.

The potential therapeutic benefits of ANGPTL3 reduction are supported by the discovery that people with a genetic deficiency in ANGPTL3 have reduced levels of low-density lipoprotein cholesterol and triglycerides, and a decreased risk of diabetes and cardiovascular disease. In a phase 1/2 study, patients treated with AKCEA-ANGPTL3-LRx achieved robust, dose-dependent reductions of ANGPTL3, triglycerides, LDL-cholesterol and total cholesterol with a positive safety and tolerability profile.

AKCEA-ANGPTL3-LRx is currently being evaluated in a phase 2 study in patients with type 2 diabetes, hypertriglyceridemia, and non-alcoholic fatty liver disease. AKCEA-ANGPTL3-LRx was discovered by Ionis and has been co-developed by Akcea and Ionis.

Under the terms of their agreement, Akcea and Ionis are also eligible to receive development, regulatory and sales milestone payments of up to $1.3 billion and tiered, double-digit royalties on annual worldwide net sales following marketing approval of AKCEA-ANGPTL3-LRx. Future milestone payments and royalties will be split equally between Akcea and Ionis.

Pfizer is responsible for all development and regulatory activities and costs beyond those associated with the ongoing phase 2 study. Prior to regulatory filing for marketing approval, Akcea retains the option, prior to regulatory filing for marketing approval, to participate in certain commercialization activities with Pfizer in the United States and certain additional markets on pre-defined terms and based on meeting pre-defined criteria.

“AKCEA-ANGPTL3-LRx has the potential to treat people suffering from certain cardiovascular and metabolic diseases. Given the unmet medical need for this patient population and the broad market potential, we believe Pfizer’s expertise and breadth of experience in cardiovascular and metabolic diseases makes it well suited to accelerate clinical development of AKCEA-ANGPTL3-LRx, and to deliver it to patients in need of additional therapies for these life threatening diseases,” said Damien McDevitt,  interim chief executive officer at Akcea.

Photo: Damien McDevitt, interim CEO at Akcea.

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