Ionis, Astrazeneca win FDA approval of competitor to Alnylam drug

Dive Brief:

• The Food and Drug Administration on Thursday approved a new medicine for a form of the rare disease transthyretin amyloidosis, clearing AstraZeneca and Ionis Pharmaceuticals’ Wainua for adults with the condition. 
• The drug, formerly known as eplontersen, has been approved for use in transthyretin amyloidosis polyneuropathy, the variety of the disease that affects nerves. Its price hasn’t yet been disclosed, though RBC Capital Markets’ Luca Issi predicted something “generally on par” with a rival Alnylam Pharmaceuticals’ drug after discussions with Ionis executives.
• Wainua’s clearance begins the first of what could be several near-term drug launches for Ionis, which has historically favored licensing out its products but has changed its strategy under CEO Brett Monia. The company splits rights to Wainua with AstraZeneca, but has multiple wholly owned drugs in advanced testing. 

Dive Insight:

The approval of Wainua opens up a new front in a long-running battle between Ionis and Alnylam. 

The two companies are pioneers in different forms of RNA drug making, and each of them has used their respective approaches to bring several rare disease drugs to market. 

Yet their ambitions have overlapped in transthyretin amyloidosis, an inherited or acquired condition in which a misfolded protein accumulates in the body, damaging the nerves, heart or both.

For years, Alnylam has had the upper hand. Its first drug for people with transthyretin amyloidosis polyneuropathy, Onpattro, had a dominant market position compared to Ionis’ rival Tegsedi. Alnylam also beat Ionis to market with a newer, more convenient version now sold as Amvuttra. Those two drugs generated about $650 million for Alnylam in 2022 and currently comprise a roughly $900 million franchise, wrote RBC’s Issi. Tegsedi, meanwhile, is now largely sold by the Swedish firm Sobi following a series of strategic and restructuring moves by Ionis. 

Wall Street analysts believe Ionis may have a better chance to compete with Wainua. It was developed with a newer type of technology called LICA, and appears to be both more potent and safer than Tegsedi. Unlike Tegsedi, for instance, Wainua showed the potential in testing to halt disease progression. The FDA also hasn’t required patients to be monitored for low platelet counts or potential kidney problems. 

In dual press releases, Ionis and AstraZeneca touted Wainua as the first medicine for the rare disease that can be self-administered with an auto-injector. By comparison, a healthcare professional must administer Amvuttra by subcutaneous injection either at home or in a doctor’s office. 

Amvuttra requires less frequent dosing than Wainua — four injections a year, versus once-monthly — and its effects kick in quicker, noted RBC’s Issi. Still, “we think eplontersen can narrow the gap” and eventually capture about a third of the market, Stifel analyst Paul Matteis wrote to clients on Friday.

A bigger battle lies ahead. Amvuttra and Wainua are being tested in late-stage studies in a more common form of transthyretin amyloidosis that affects the heart. Alnylam is expecting results in early 2024, while Ionis and AstraZeneca could follow a year later. The financial implications are significant, as the only available medication for that form of the disease, Pfizer’s Vyndamax, is a blockbuster medicine. 

Drugs like Amvuttra and Wainua are thought to be more potent because of how they work. 

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