Akcea Therapeutics Inc., a majority owned affiliate of Ionis Pharmaceuticals Inc., and Pfizer Inc. signed an exclusive, worldwide licensing deal for AKCEA-ANGPTL-3-LRx, which is being developed to treat cardiovascular and metabolic diseases. The deal follows a period during which Akcea's stock (NASDAQ:AKCA) has sagged prior to rising Monday and big changes in the company's C-suite.

Akcea and Ionis will equally split a $250 million up-front license fee. Both could also receive development, regulatory and sales milestone payment of up to $1.3 billion. Should AKCEA-ANGPTL-3-LRx become FDA approved, both companies could also receive tiered, double-digit royalties on annual worldwide net sales. Akcea will pay its $125 million obligation to Ionis in Akcea common stock. Any future milestone payments and royalties will be split equally between the two companies.

Pfizer will handle all development and regulatory activities costs incurred beyond the drug's current phase II study. Akcea will have the right to participate in some commercialization activities in the U.S. and additional markets with Pfizer.

Boston-based Akcea's stock finished Monday up 31.3% , while shares of Ionis (NASDAQ:IONS) gained about 2.5%.

Some management shuffling pushed the stock downward in the past several weeks, as CEO Paula Soteropoulos, President Sarah Boyce and Chief Operating Officer Jeff Goldberg left the company on Sept. 23. A few days before, on Sept. 20, the stock sold for $22.46, but shares dropped to $18.04 on Sept. 23 and drifted downward to $14.70 by Oct. 1. Shares had bounced back to $19.87 by the time the market closed Monday.

Damien McDevitt, a member of the board, is the newly appointed interim CEO. He comes from Ionis, where he was chief business officer, leading the charge for corporate communications, investor relations, business development, competitive intelligence and alliance management. He also spent more than 20 years at Glaxosmithkline plc.

AKCEA-ANGPTL3-LRx, an antisense therapy, 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 to treat multiple lipid disorders or rare hyperlipidemias. Preclinical data showed it inhibited liver fat accumulation and lowered blood levels of low-density lipoprotein cholesterol (LDL-C) and very low density lipoprotein cholesterol.

Preclinical studies indicated an ANGPTL3 genetic deficiency in adults leads to lower levels of LDL-C and triglycerides, ultimately leading to lower chances of developing diabetes and cardiovascular disease.

AKCEA-ANGPTL3-LRx continues the phase II study begun in December 2017 and set for completion in February 2020. The randomized, double-blind, placebo-controlled, dose-finding study follows 144 adult participants who are administered AKCEA-ANGPTL3-LRx subcutaneously to patients with hypertriglyceridemia, type 2 diabetes mellitus and nonalcoholic fatty liver disease. The three cohorts in the study are linked to a placebo comparator, which is randomly assigned to one of the dosing cohorts.

The primary outcome measure of the phase II is to determine the percent change in fasting triacylglycerol level from baseline, with a six-month time frame. Secondary outcome measures include determining the drug's safety over a six-month period.

Last month, Akcea released phase I clinical data for AKCEA-TTR-LRx, which is designed to inhibit the production of transthyretin (TTR) and is being studied in patients with both the hereditary and the wild type forms of TTR amyloidosis (ATTR). In the phase I, randomized, double-blind, placebo-controlled, dose-escalation study, healthy volunteers were administered AKCEA-TTR-LRx or placebo via a single subcutaneous injection or once every four weeks for 13 weeks followed by an additional 13-week period where patients did not receive treatment. Results showed patients who received injections of 45 mg monthly achieved a mean reduction in TTR levels of 86% at week 13 and patients who received injections of 90 mg monthly achieved a mean reduction in TTR levels of 94% at week 13. There were no severe adverse events in patients treated with AKCEA-TTR-LRx.

Akcea and Ionis also plan to initiate the phase III program for AKCEA-TTR-LRx later this year.

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