StemCell Therapy

Pfizer has signed a deal with Ionis affiliate Akcea Therapeutics, to develop a potential blockbuster antisense therapy to treat patients with certain cardiovascular and metabolic diseases including type 2 diabetes.

The terms of the deal give a clue as to Pfizers assessment of the drugs potential: Akcea and Ionis will receive a fairly modest $250 million up front, split between the two companies, and Akcea will settle its $125 million obligation to Ionis in common stock.

But the companies are also eligible to receive development, regulatory and sales milestone payments of up to$1.3 billionand tiered, double-digit royalties on annual worldwide net sales following marketing approval of the drug codenamed AKCEA-ANGPTL3-LRx.

AKCEA-ANGPTL3-LRx is designed to reduce the production of angiopoietin-like protein 3 (ANGPTL3) and is a regulator of triglycerides, cholesterol, glucose, and energy metabolism.

It is currently in a phase 2 study in patients with type 2 diabetes, hypertriglyceridemia, and non-alcoholic steatohepatitis (NASH) indications that could generate substantial sales following approval from regulators.

Akceas antisense drugs work by blocking the action of messenger RNA, which carries instructions to the cell from the DNA to code for a particular protein.

Antisense drugs are made from components such as synthetic strands of DNA, which target a specific RNA sequence and stop it from producing the protein.

Future milestone payments and royalties will be split equally between Akcea and Ionis, and Pfizer will pay for all development and regulatory costs beyond the ongoing phase 2 study.

Before a filing for marketing approval, Akcea has an option to take part in some marketing activities with Pfizer in the US, and some other markets if certain conditions are met.

Akcea already has two antisense therapies approved Tegsedi (inotersen) for the rare disease hereditary transthyretin-related amyloidosis (hATTR) and Waylivra (volanesorsen) for familial chylomicronemia syndrome.

Damien McDevitt, interim chief executive officer at Akcea, said: AKCEA-ANGPTL3-LRxhas 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 Pfizers 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.

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Pfizer inks deal with Ionis and Akcea for potential blockbuster diabetes and NASH drug - - pharmaphorum

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