Drug Development

The stellar antibody shop at Regeneron wins a ‘breakthrough’ tag for its next big PhIII drug

Richard Dunbar

Regeneron $REGN has one of the best track records in the business when it comes to building amazingly effective antibodies. It did it with Dupixent for eczema and Praluent for cholesterol. And now one of its Phase II stars just earned a breakthrough tag from the FDA, which puts it on the inside track at the agency as it goes into pivotal studies for slashing triglyceride.

The drug is evinacumab. The target is once again extraordinarily high levels of LDL cholesterol. But the target is different. This antibody tackles angiopoietin-like protein 3, or ANGPTL3. ANGPTL3 inhibits lipoprotein lipase and endothelial lipase, playing a key role in lipoprotein metabolism.

Researchers are testing the drug in hypercholesterolemia in patients with rare cases of Homozygous Familial Hypercholesterolemia (HoFH). And it could fit comfortably in their portfolio, right next to Praluent.

The FDA cited data from a small Phase II study for the drug in handing out the BTD, which is designed to open doors to developers as they advance significant new drugs with high potential through the clinic. In the Phase II investigators monitored triglyceride levels for at least five months following an injection. Six doses were tested and in the top three dose-groups, triglycerides were cut by 64% to 73%. At the time, Penn’s Richard Dunbar noted two distinct advantages in managing triglycerides: reducing hospitalization for pancreatitis patients and reducing the risk of heart disease.

“Current medications such as fibrates or prescription fish oils effectively lower triglycerides, but leave much to be desired, each only lowering levels by 20 to 50 percent,” Dunbar said at the time. “Validating a drug that lowers triglycerides well beyond that range would undoubtedly take us to the next level, particularly since it could be combined with current oral medications for those patients with extraordinarily high triglycerides who often can’t achieve safe levels with our usual medications. A similar approach has been taken for lowering certain cholesterol with the advent of PCSK9 inhibitors, which utilize a similar monoclonal antibody mechanism.”

Proving that and winning coverage from payers won’t be easy, as Regeneron and its close partner Sanofi are well aware after their Praluent experience. And Sanofi — which shoulders much of the Phase III load on its partnered therapies — already opted out of this antibody. But Regeneron seems ready to push this drug into their wholly-owned late-stage pipeline, tearing off another major Phase III effort for a remarkably effective R&D group.

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