🔹The company said it is planning to submit a new drug application (NDA) to the US Food and Drug Administration (FDA) for the orally-administered candidate in this indication later this year.
🔹AD is the most common form of dementia, affecting approximately seven million people in the US. The neurodegenerative disease slowly destroys memory and thinking skills and, eventually, the ability to carry out simple tasks.
🔹Up to 70% of AD patients report agitation, which is characterised by emotional distress as well as verbal and physical aggressiveness, and has been associated with accelerated cognitive decline.
🔹In its latest update of AXS-05, Axsome reported that the 295-patient phase 3 ACCORD-2 trial met its primary endpoint, with the drug demonstrating a statistically significantly delay in the time to relapse of agitation, as assessed by the Cohen-Mansfield Agitation Inventory (CMAI) total score, compared to placebo.
AXS-05 also achieved ACCORD-2’s key secondary endpoint of relapse prevention, and was shown to reduce worsening for overall AD severity versus placebo.
🔹The company noted that the late-stage ADVANCE-2 trial of 408 AD patients did not demonstrate statistical significance for the primary endpoint, change in the CMAI total score from baseline to week five, but said the results for the primary and nearly all secondary endpoints “numerically favoured” AXS-05 over placebo.
🔹The candidate was shown to be safe and well tolerated, and was not associated with increased risk of falls, cognitive decline or sedation in either controlled study, or in the long-term safety trial of the drug in patients who were treated for six and 12 months.
🔹Axsome’s chief executive officer, Herriot Tabuteau, said: “With the strong results of the ACCORD-2 trial, AXS-05 has now demonstrated substantial and statistically significant improvements in AD agitation across three pivotal, phase 3, placebo-controlled trials [ADVANCE-1, ACCORD-1 and ACCORD-2], underscoring its potential to provide meaningful benefit to patients living with this condition and their families.
The improvements in the AXS-05 arm relative to placebo in ADVANCE-2 did not reach statistical significance. However, we are pleased with the very positive controlled safety data from this trial which will be an essential part of our planned NDA submission of AXS-05 in AD agitation, which is targeted for the second half of 2025.”