Axsome says FDA wants more data for Alzheimer’s agitation label expansion, but readout to come sooner

 Axsome Therapeutics said the FDA wants safety information from the biotech’s third Phase III study of its drug candidate for Alzheimer’s disease agitation before submitting a label expansion request.

The regulator wants long-term safety data from patients in that indication, which the company has previously said affects up to 70% of patients with Alzheimer’s. The Phase III completion could come about a year sooner than previously anticipated, though, with the timeline for ADVANCE-2 moving from mid-2025 to the first half of next year, per a quarterly update Monday morning.

FDA indicated it wants data on at least 300 patients treated for six months and 100 patients on the drug for one year, the New York biotech said. The news comes after the agency said no to Eli Lilly’s accelerated pitch for an Alzheimer’s drug last month, citing its request for data on 100 patients for 12 months.

Axsome’s combination drug received regulatory clearance for treating major depressive disorder last August and is marketed as Auvelity. It’s known investigationally as AXS-05 for AD agitation and smoking cessation, for which a Phase II/III will begin in the fourth quarter.

After presenting a win on its second Phase III, ACCORD, in AD agitation last November, Axsome said it would ask regulators about next steps, with Cowen analyst Joseph Thome saying he looked forward to FDA feedback on whether the two late-stage trials “could constitute a regulatory package, with ADVANCE-2 serving as more of a confirmatory trial.”

It appears all three trials will be needed for the regulatory package, which Mizuho Securities analyst Graig Suvannavejh said is “in keeping with the company’s original expectations.”

Axsome CEO Herriot Tabuteau told analysts on last quarter’s earnings call that the ADVANCE-2 trial and its ADVANCE-1 predecessor are “pretty much identical study designs.” The trial is estimated to enroll 350 patients.

Alzheimer’s agitation is also being targeted by Lundbeck and partner Otsuka, who reported their own Phase III success in the indication with their depression and schizophrenia drug Rexulti. Elsewhere, Acadia’s Alzheimer’s-related psychosis drug was given a thumbs-down by FDA’s advisors last year. There are no approved therapies for the indication,

Meanwhile, during its quarterly refresh, Axsome said it plans to resubmit the NDA for its oral migraine candidate, AXS-07 (meloxicam-rizatriptan), in the second half of this year, which Mizuho noted is a “widening of timelines vs. prior guidance of 3Q23.” Manufacturing activities remain ongoing, Axsome said, noting it expects the NDA would go through a six-month review.

https://endpts.com/axsome-says-fda-wants-more-data-for-alzheimers-agitation-label-expansion-but-readout-to-come-sooner/