Two years after mid-stage results first sent their stock flying, Axsome announced the FDA has accepted their application for a new depression drug and promised to give it a hastened look.
The agency has given priority review to Axsome’s lead drug, AXS-05, for the treatment of major depressive disorder. The agency said they will give a decision on what would be Axsome’s first approved drug by Aug. 22, the company announced.
AXS-05 first made headlines for the New York City biotech in 2019, when Axsome announced that it successfully improved symptoms of depression in an 80-person, 6-week study — a major, albeit preliminary, win in a disease where many a drug has gone to die. Axsome’s stock soared from less than $3 to more than $7, and it kept soaring as Axsome validated the results with a Phase III trial and added a surprise win Alzheimer’s-related agitation.
Their stock $AXSM creeped up again on the regulatory news, rising 14% from $52.89 to $60.25.
An approval would mark the first of what Axsome hopes are a series of launches, as they look to finish pivotal trials for AXS-05 in Alzheimer’s and file another drug, AXS-07, for approval in migraine. A molecule for narcolepsy and fibromyalgia are also in mid-to-late stage studies.
Axsome’s Phase III success was a rare victory in a field that has swallowed up an array of promising candidates over the years, in part because patients often have a strong placebo effect. Their readout came just 11 days after Sage revealed their lead drug failed a pivotal trial for major depression, dealing the much-hyped Cambridge biotech a significant setback and cutting their stock in half.
In Axsome’s Phase III study, 327 patients with major depression were randomized to receive either an AXS-05 tablet or placebo tablet for 6 weeks. Patients who received AXS-05 saw their scores on a standard depression scale fall by a median of 16.2 points, compared with 11.9 points for patients on placebo — a p value of .002. The drug also hit on all five secondary endpoints.
AXS-05 is a combination of the decades-old depression drug bupropion and the molecule dextromethorphan, commonly known as cough syrup. Dextromethorphan plugs several receptors in the brain, including the commonly targeted NMDA receptors. Bupropion is used to lengthen how long the dextromethorphan molecule remains in the body.
Axsome received breakthrough designations for both major depression and has also Alzheimer’s agitation. A Phase III trial for the latter indication is now underway. Success, though, has not been universal: Last year, AXS-05 failed a Phase III trial for treatment-resistant depression, with the compound failing to show a statistically significant improvement over bupropion alone.
Analysts expect Alzheimer’s may ultimately provide a larger market than depression. But they’ve said there’s potential for significant revenue.
Opinion leaders “don’t view the drug as a ‘game changer,’” SVB Leerink’s Marc Goodman wrote in a note in February, “but it would likely be a welcome new alternative and probably get used after augmentation therapy.”