Axsome Therapeutics’ AXS-05 is currently in Phase III for major depressive disorder (MDD) and was granted a breakthrough therapy designation by the US Food and Drug Administration (FDA) in March 2019.

In a late-stage study, the drug was shown to regulate mood by improving the communication between brain cells and increasing levels of serotonin, noradrenaline and dopamine. The success of this study helps the company enter the global $13bn market for depression therapies.

If approved, Axsome Therapeutics’ AXS-05 will experience competition from other brands already established in the market. However, the market for depression is known for its high level of clinical need, as many patients do not respond to first-line anti-depressants and experience side effects. Patients who were given AXS-05 in the GEMINI Phase III trial demonstrated a significant improvement compared to placebo following one week of treatment, and AXS-05 was found to be well tolerated. As such, GlobalData forecasts that AXS-05 will generate global sales of $538m by 2025.

AXS-05 has the potential to be successful, as current antidepressants on the market have struggled to provide adequate treatment responses in patients and can take longer than one month to achieve clinically meaningful responses. The promising results of the GEMINI study are significant because they help to address a public health need with a novel, orally-administered, fast-acting, and effective antidepressant that can be safely taken at home.

AXS-05 is a fixed-dose combination of bupropion and dextromethorphan that is administered orally. The former is an older depression drug and the latter is an over-the-counter cough suppressant. AXS-05 acts by targeting the N-methyl-D-aspartate (NMDA) receptor, sigma 1 receptor, CHRNA3, CHRNB4, SLC6A3, SLC6A2, and SLC6A4, as well as by inhibiting the reuptake of serotonin, norepinephrine, and dopamine.

The trial data suggest that AXS-05, as an oral NMDA receptor antagonist with multimodal effects, may become a successful new treatment for MDD. Now that the drug’s efficacy has been demonstrated in both the GEMINI trial and the previously completed ASCEND study, Axsome is aiming to file a New Drug Application (NDA) for AXS-05 in H2 2020.