Neurocrine’s Ingrezza, fresh off its first FDA approval and aiming for another, just hit a major roadblock.
Ingrezza flopped a phase 2 study in pediatric Tourette Syndrome patients, the San Diego-based biotech announced late Tuesday, throwing a wrench into the company's big expansion plans. The med missed the study’s primary endpoint, failing outdo placebo in registering a change on the Yale Global Tic Severity Scale (YGTSS).
It was Ingrezza's second Tourette shortfall this year; in January, a phase 2 trial in adults failed to deliver the goods. And the Tourette use is key to Neurocrine's growth plans for the new med.
The good news for Neurocrine, though? “The program is not dead, in our view,” Leerink Partners analyst Paul Matteis wrote in a note to clients. Neurocrine executives, putting a positive spin on the shortfall, said it had underestimated the dose needed to deliver benefits in kids—but now that it knows, it can start up a new-and-improved phase 2b study.
Matteis, for one, bought that explanation for the misfire, noting that it’s “supported by an association between drug exposure and tic reduction and a very low discontinuation rate,” both seen in the failed trial.
The bad news? Matteis thinks the setback could delay an approval in Tourette by two years, and he’s not predicting any Ingrezza revenue in that department until 2022. Jefferies’ Biren Amin was only slightly more optimistic, pushing back the expected launch year from 2020 to 2021.
The biotech’s pediatric work had been seen as “the main focus for Neurocrine,” after a phase 2 trial in adults hit a snag in January, Barclays analyst Geoff Meacham wrote to investors at the time. That study also missed its primary endpoint in change-from-baseline improvement on the YGTSS, though it did show Ingrezza could significantly improve overall symptoms. The trial also offered some insights the company could use in setting up its phase 3.
With both less-than-stellar trials in mind, though, “we continue to assume a 30% odds of success” in securing a Tourette nod, “which we think is fair,” Matteis wrote. And the way he sees it, company management’s “strong track record of drug development in CNS” is pushing those odds up in Neurocrine’s favor.
Either way, it’ll likely be at least a few years before Ingrezza has another indication on its label, meaning Neurocrine’s commercial focus will revolve around the tardive dyskinesia (TD) approval it picked up last month. Neurocrine is looking to milk its head start as much as it can before Teva’s Austedo, a Huntington’s med angling for a TD nod, enters the picture.
To do so, the first-to-market drugmaker will have to up TD awareness, a task the company has long been working on. It’s fielded a group of 12 medical science liaisons for over a year, and they’ve spent that time engaging with national and regional opinion leaders, CEO Kevin Gorman said in an April interview.