FDA, New Products

Ionis and Akcea get their shot at competing with Alnylam for a rare disease group — but it’s not expected to be pretty

Ionis $IONS and Akcea $AKCA have been given a green light to go head-to-head into a marketing fight with Alnylam for treating extremely rare cases of TTR amyloidosis — with one arm tied behind their back on efficacy and both commercial feet hobbled by a worrisome safety issue.

Akcea — a spinout from Ionis, which still controls a majority of the company — put out the word of the delayed OK for Tegsedi (inotersen) on Friday night, which comes after a regulatory delay that put them one step behind the competition. 

It’s not the delay that presents the biggest problem, though. That honor goes to the black box warning about the drug’s side effects, which includes the risk of thrombocytopenia — low platelet counts that carry the risk of serious bleeding. Patients will need to be carefully monitored for the side effects, while Alnylam’s drug comes with a much cleaner safety profile that will be clearly preferred by the patient population and their doctors.

Steve Miller

The approval did come with an upbeat comment from Express Scripts CMO Steve Miller, who spotlighted the weekly injection regimen — a slight advantage in Akcea’s favor — and the chance for some head-to-head price discounts.

“Tegsedi is a weekly self-administered injection, that should reduce administration costs and drug wastage,” noted an approving Miller. “By introducing competition into the marketplace this should drive down the total cost of care.”

The companies, though, start out at exactly the same price of $450,000 a year wholesale, which Alnylam set. Any discounting on price will now be worked out with payers, and Alnylam is vowing to get aggressive to stay on top, hoping to prove that its shift to marketing can be accomplished with expertise. Alnylam has estimated the net price at $345,000 with individual deals being worked out on a pay-for-performance basis.

Not to be discounted in all this is Pfizer $PFE, which is hustling along tafamidis after scoring positive data on transthyretin amyloid cardiomyopathy. Pfizer is already rolling out an expanded access program that will sign up patients with the disease as they shoot for a quick OK of their own.

This disease is triggered by a misfolding protein which then builds up in organs, threatening patients with an early death. 

The Akcea/Ionis team have had plenty of setbacks to deal with this year. Most notably the FDA recently rejected volanesorsen, their drug for regulating plasma triglyceride for patients with rare cases of familial chylomicronemia syndrome, even after an expert panel review gave the companies a thumbs up on marketing. The rejection forced the company to restructure, laying off 10% of its workforce as they plan a comeback.

Topping it all off, Ionis CEO Stanley Crooke opted to jump off the Akcea board Monday, departing a company he created. He’ll be replaced by Ionis BD chief Damien McDevitt.

Akcea’s stock dropped 7% Monday morning. Ionis dropped 4%. Alnylam was down too, just under 2%.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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