After leadership shuffle, Akcea hands heart drug to Pfizer for $250M

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Before Pfizer files the antisense therapy treatment for approval, Akcea Therapeutics may opt into “certain commercialization activities” with Pfizer in the U.S. and “certain additional markets.” (rawpixel/Pixabay)

After playing an unexpected game of executive musical chairs, Akcea Therapeutics has inked a deal with a potential rival. For $250 million upfront, Akcea is licensing an antisense therapy to Pfizer to be developed for “certain cardiovascular and metabolic diseases.” 

Akcea is already testing the treatment, AKCEA-ANGPTL3-LRx, in a phase 2 study involving patients with Type 2 diabetes, hypertriglyceridemia and nonalcoholic fatty liver disease. Under the deal, Akcea and Ionis Therapeutics—the company that spun four of its programs out into Akcea in 2015—will split the license fee as well as milestone payments that could total up to $1.3 billion. Pfizer will take over all development and regulatory work beyond the ongoing phase 2 study. 

Before Pfizer files the treatment for approval, Akcea may opt into “certain commercialization activities” with Pfizer in the U.S. and “certain additional markets,” the companies said in a statement. 

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The antisense treatment is designed the cut the production of a protein called angiopoietin-like 3 (ANGPTL3) in the liver. The protein plays a key role in regulating triglycerides, cholesterol, glucose and energy metabolism. 

RELATED: Ionis CBO takes over Akcea as CEO, CBO and COO head out the door 

“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 Akcea’s interim CEO Damien McDevitt, Ph.D., in the statement. McDevitt, the former chief business officer of Ionis, took over Akcea after the departures of its previous CEO Paula Soteropoulos, CBO Sarah Boyce and COO Jeff Goldberg. 

“AKCEA-ANGPTL3-LRx 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,” said Mikael Dolsten, chief scientific officer and president of worldwide R&D at Pfizer. 

The deal comes five months after Pfizer’s tafamidis scored FDA nods to treat both the wild-type and hereditary forms of a rare and fatal heart disease called ATTR cardiomyopathy. Though Pfizer’s treatment, dubbed Vyndaqel, trailed Alnylam’s Onpattro and Ionis/Akcea’s Tegsedi to market, they won’t compete head to head just yet. The latter two meds are approved for a different variant of ATTR: hereditary ATTR polyneuropathy, a form of the disease that Vyndaqel isn’t yet approved to treat. 

RELATED: Pfizer's tafamidis wins blockbuster nod—and gets a lower price—to rival Alnylam, Ionis 

However, Alnylam is working toward an approval in ATTR cardiomyopathy and Pfizer hopes to move Vyndaqel into the other variant. If and when that happens, Vyndaqel might have a leg up over its competitors as it is a pill rather than an injection and has a relatively low price—$225,000 per year compared to $450,000 per year for both Tegsedi and Onpattro.

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