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Pfizer pays $250m upfront to Akcea for phase 2 antisense drug

Has potential in cardiovascular and metabolic diseases


Pfizer has bolted on an antisense drug with potential in cardiovascular and metabolic diseases to its pipeline via a licensing deal with Ionis’ Akcea affiliate.

The big pharma – which has traditionally relied on acquisitions and licensing deals to keep its pipeline stocked – is paying $250m upfront for rights to AKCEA-ANGPTL3-LRx, an antisense oligonucleotide designed to inhibit the production of angiopoietin-like 3 (ANGPTL3) protein in the liver.

AKCEA-ANGPTL3-LRx is already in a phase 2a study involving patients with type 2 diabetes, elevated blood triglycerides and non-alcoholic fatty liver disease (NAFLD), which Pfizer said are all indications with “unmet medical need” and “broad market potential”.

Pfizer’s $250m fee will be split between Akcea and Ionis, and the two companies will also share milestone payments of up to $1.3bn, plus double-digit royalties on sales if the antisense drug reaches the market.

Responsibility for developing AKCEA-ANGPTL3-LRx goes to Pfizer from phase 2 onwards and it has global rights to the drug, although Akcea has some opt-in commercial rights in the US and certain other unidentified markets.

It’s the second sizeable deal for Akcea this year after Novartis paid $150m upfront for rights to its lipoprotein(a) antisense drug AKCEA-APO(a)-LRx in February, with another $1.6bn in milestones on offer. The Swiss pharma group took an option on two of Akcea’s lipoprotein-targeting drug candidates in 2017.

Both deals reflect something of a resurgence in cardiovascular drug development, fuelled by newly-emerged targets, after many years of stagnation which resulted in the market becoming dominated by older generic drugs.

New therapies like Novartis’ heart failure therapy Entresto (sacubitril/valsartan) and cholesterol-lowering PCSK9 inhibitors from Amgen and Sanofi/Regeneron have re-injected growth into the market, although they have faced payer resistance.

ANGPTL3 is a protein that regulates triglyceride and phospholipid levels in the blood, both of which are a risk factor for atherosclerotic coronary heart disease when elevated, and also affects glucose and energy metabolism.

“AKCEA-ANGPTL3-LRx is a novel therapy that will complement our clinical mid-stage internal medicine pipeline,” said Mikael Dolsten, chief scientific officer and president of worldwide R&D at Pfizer.

He added: “we believe that our deep expertise in cardiovascular and metabolic diseases will help allow this program to reach its maximum potential for patients.”

Founded in 2015, Akcea spun out of Ionis two years later armed with four drug candidates for treating diseases caused by lipid disorders.

It’s had some disruption of late with the departure of three top executives including chief executive Paula Soteropoulos, which came as a big surprise given the company is in the roll-out stage for two new products, Tegsedi (inotersen) for hereditary transthyretin-mediated amyloidosis and Waylivra (volanesorsen) for familial chylomicronemia syndrome (FCS).

Article by
Phil Taylor

8th October 2019

From: Sales



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