Akcea & Pfizer Announce $250-Million Licensing Agreement for Investigative Therapy

Akcea Therapeutics, Inc., a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Pfizer Inc. recently announced the companies have entered into a worldwide exclusive licensing agreement for AKCEA-ANGPTL3-L_Rx, an investigational antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases.

AKCEA-ANGPTL3-L_Rx is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver, a key regulator of triglycerides, cholesterol, glucose, and energy metabolism. AKCEA-ANGPTL3-L_Rx is currently being evaluated in a Phase 2 study in patients with Type 2 diabetes, hypertriglyceridemia and non-alcoholic fatty liver disease (NAFLD).

“AKCEA-ANGPTL3-L_Rx 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-L_Rx, and to deliver it to patients in need of additional therapies for these life threatening diseases,” said Damien McDevitt, PhD, Interim Chief Executive Officer at Akcea.

“Pfizer is committed to delivering breakthrough medicines to patients with unmet medical needs,” said Mikael Dolsten, Chief Scientific Officer and President, Worldwide Research & Development and Medical, Pfizer. “AKCEA-ANGPTL3-L_Rx is a novel therapy that will complement our clinical mid-stage internal medicine pipeline, and we believe that our deep expertise in cardiovascular and metabolic diseases will help allow this program to reach its maximum potential for patients.”

Under terms of the agreement, Akcea and Ionis will receive a $250 million upfront license fee, which will be split equally between the two companies. Akcea will settle its $125 million obligation to Ionis in Akcea common stock. The companies 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-L_Rx. 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 has the right, at its option to participate in certain commercialization activities with Pfizer in the US and certain additional markets on pre-defined terms and based on meeting pre-defined criteria.

This transaction is subject to clearance under the Hart-Scott Rodino Antitrust Improvements Act and other customary closing conditions.

AKCEA-ANGPTL3-L_Rx is an investigational antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases. This antisense medicine is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver, a key regulator of triglycerides, cholesterol, glucose and energy metabolism. AKCEA-ANGPTL3-L_Rx was developed using Ionis’ advanced LIgand Conjugated Antisense (LICA) technology platform. 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 (LDL-C) and triglycerides, and a decreased risk of diabetes and cardiovascular disease¹. In a Phase 1/2 study, patients treated with AKCEA-ANGPTL3-L_Rx achieved robust, dose-dependent reductions of ANGPTL3, triglycerides, LDL-cholesterol and total cholesterol with a positive safety and tolerability profile². AKCEA-ANGPTL3-L_Rx is currently being evaluated in a Phase 2 study in patients with Type 2 diabetes, hypertriglyceridemia and non-alcoholic fatty liver disease (NAFLD). AKCEA-ANGPTL3-L_Rx was discovered by Ionis and has been co-developed by Akcea and Ionis.

Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is commercializing TEGSEDI^® (inotersen) and WAYLIVRA^® (volanesorsen) as well as advancing a mature pipeline of novel drugs, including AKCEA-APO(a)-L_Rx, AKCEA-ANGPTL3-L_Rx, AKCEA-APOCIII-L_Rx, and AKCEA-TTR-L_Rx, with the potential to treat multiple diseases. All six drugs were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. TEGSEDI is approved in the U.S., E.U. and Canada. WAYLIVRA is approved in the E.U. and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Boston, MA. For more information, visit www.akceatx.com. and you can follow us on twitter at @akceatx.