FDA gives Axsome speedy review for depression drug, while other indications wait in the wings – Endpoints News

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.”