Racing against Alnylam, Ionis posts positive PhIII ATTR efficacy numbers, but questions linger

One of the big rivalries in biotech right now is the race between Ionis $IONS and Alnylam $ALNY to field a new RNA therapy for hereditary TTR amyloidosis (hATTR) with polyneuropathy. And after Alnylam drew cheers for its top-line win, Ionis is rolling out a big peek at the bottom line.

Researchers posted a mean 19.73-point benefit for the neuropathy impairment score, its mNIS+7 co-primary endpoint after 15 months of treatment, saying that they could start tracking a statistically significant benefit at 8 months.

Stanley Crooke, Ionis CEO

We don’t know yet what Alnylam’s numbers are, though plenty of analysts are willing to give them the edge in the race — and even though Ionis can rely on a much more easily used therapy, with a subcutaneous injection compared to an infusion process.

Delivery, though, is a distant second compared to safety, and we still have a lot more to learn on that score from Ionis. Its therapy was linked with several cases of thrombocytopenia, low platelet levels, with one patient dead from intracranial hemorrhage as well as two cases of renal toxicity.

All Ionis says in the statement — repeating earlier assurances to analysts — is that the safety events are “monitorable and manageable.”

That won’t be enough to satisfy the doubters, though, who are tracking just how fast these drugs take effect and just what impact safety will have in terms of adoption. Everyone is waiting now for the 1st European ATTR Amyloidosis Meeting for Patients and Doctors, on November 2, where full data from both studies will be put on display.

Look for applications to be filed later this year as both companies jockey for position.

“The pivotal inotersen data presented today represent new hope for patients suffering with hATTR. A 20-point benefit in mNIS+7 is unprecedented and could mean the difference between the ability to walk and being confined to a wheelchair for patients suffering from this debilitating and fatal disease,” said Annabel Wang, associate professor of neurology at the University of California, Irvine, School of Medicine. “Based on benefit observed in neurological and quality of life endpoints, inotersen treatment has shown the potential to effectively change the relentless progression of this disease and offers a convenient, at-home administration method, providing patients further liberation from the burden of hATTR.”

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