Results, RNAi

Prize fight in Paris: Alnylam and Ionis slug it out over PhIII data in preparation for a blockbuster showdown

This morning Alnylam $ALNY rolled out a battery of stellar Phase III data on their blockbuster contender patisiran, which promises to go on to become the company’s first approved and marketed drug after 15 long years of RNAi discovery and development work. And as of now, it looks like they’ll be going head-to-head with the antisense experts at Ionis $IONS, the underdogs in what promises to be a long running fight for a small group of patients suffering from rare cases of hereditary ATTR (hATTR) amyloidosis.

John Maraganore

For Alnylam, the key points in the APOLLO data unveiled today in Paris focus on an impressive 34-point mean difference in the modified neuropathy impairment score (mNIS+7) at 18 months, with a 6-point improvement for patients which underscores that they got somewhat better over that period. There was also a 21.1-point mean improvement in the quality of life score, with a 6.7-point improvement to show that the patients also felt better.

Just hours earlier, it was Ionis that took center stage, adding to its Phase III data rollout with a 19.73-point mean change in disease scale at 15 months, and an 8.69-point benefit at 8 months. Their quality of life scores registered an 11.68-point mean improvement, with a 6.4-point benefit that showed their patients also responded better with treatment.

“We beat them on both,” Alnylam CEO John Maraganore tells me flatly, “with the caveat that these aren’t comparative (head-to-head) studies. We clearly have a more substantial treatment effect.”

“This is a big win,” adds the CEO. “It’s a nice way to debut the advent of this technology in medicine.”

The market agreed with Maraganore. Alnylam shares shot up 13%, with Ionis down 8% after the matchup.

Stanley Crooke. Image: OTS

Maraganore — who’s stuck with RNAi through the good years and the bad as Big Pharma dropped out or came into the emerging field — hasn’t made his own peak sales projections here, but he also isn’t shy of noting analysts’ projections ranging up to $2 billion worth of yearly revenue. And he’s already been ramping up commercial activities in the US and Europe, while their big partner Sanofi $SNY takes on the rest of the world.

Ionis CEO Stan Crooke has heard the analysts cheering Alnylam. And he’s not about to roll over and play dead now.

“We think we’ll win in the marketplace,” Crooke told me in the lead-up to today’s data reveal, adding that “there are people who think otherwise.”

So what does Ionis have that Alnylam doesn’t? Crooke — who hadn’t seen the Alnylam results when we talked ahead of Thursday’s sessions — says there’s lots on the table.

First, he can point to 8-month and 15-month results which demonstrate significant success ahead the 18-month mark at Alnylam. He’s not talking price yet — no one does at this stage — but “the cost of therapy will be substantially better.” And he’s heard Alnylam hinting about Soliris style prices, which could land it on the list of the top 10 most expensive therapies.

“We’ll win because we think inotersen is easy to use,” he adds, adding pointedly that this isn’t their first time out on the market. Where the Alnylam therapy has to be infused, exposing patients to reactions that can leave them unable to work, he’ll be selling a treatment that can be self-administered at home.

Ionis, though, also had to deal with the death of a patient from thrombocytopenia during its study, throwing in an added safety factor that could play to Alnylam’s advantage — as many analysts would be willing to tell you. Since that case occurred, though, the biotech added a more careful screening process to prevent any new incidents.

“I think both drugs will be very successful,” Crooke sums up. But anyone who counts Ionis out now, he adds, is in for a big surprise.

The sales numbers will provide the score on who’s winning.


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