Shares of Ax­some Ther­a­peu­tics $AXSM shot up Mon­day morn­ing on the sur­prise news that its lead drug scored sta­tis­ti­cal­ly sig­nif­i­cant re­sults for Alzheimer’s ag­i­ta­tion, a rel­a­tive­ly green field in drug R&D, in what’s billed as a piv­otal Phase II/III tri­al. But the com­pa­ny will have plen­ty of high-risk work to do be­fore it can ex­pect an FDA ap­proval.

Com­ing off the ropes of a late-stage fail for AXS-05 in treat­ment-re­sis­tant de­pres­sion, the biotech re­ports that their drug scored well against a place­bo based on a care­giv­er-rat­ed scale of Alzheimer’s pa­tients’ be­hav­ior, which in­cludes track­ing episodes of scream­ing or hit­ting. At week 5 re­searchers tracked mean re­duc­tions from base­line of 15.4 points for AXS-05 and 11.5 points for place­bo, giv­ing them a p val­ue of 0.010.

The drug, a com­bi­na­tion of 2 ther­a­pies — dex­tromethor­phan and bupro­pi­on — al­so beat out bupro­pi­on alone. And the drug achieved su­pe­ri­or­i­ty at week 3, ac­cord­ing to the com­pa­ny.

An­a­lysts, mean­while, have been pri­mar­i­ly track­ing this drug’s po­ten­tial in ma­jor de­pres­sive dis­or­der, where the com­pa­ny is lin­ing up a near-term ap­pli­ca­tion with the FDA af­ter re­port­ing up­beat da­ta in one of the tough­est fields in R&D. That left ex­pec­ta­tions low as the biotech ac­cel­er­at­ed the study read­out due to Covid-19 in­to Q2 from Q3.

Marc Good­man at SVB Leerink re­cent­ly not­ed:

In fact, we don’t be­lieve in­vestors in­clude any­thing for this in­di­ca­tion, and with good da­ta, we would as­sume the stock could be up $18 if in­vestors as­sume $650M of added peak sales at 50% POS, which we think is very rea­son­able.

Ac­tu­al­ly, the ini­tial re­sponse was much bet­ter than that, as Ax­some stock gy­rat­ed up more than 40% on the news. This is an un­cer­tain are­na, though, with re­searchers of­ten forced to stick with dif­fi­cult end­points and tough place­bo re­spons­es as care­givers track re­spons­es.

Good­man and oth­ers like to point out that no oth­er drug has been specif­i­cal­ly ap­proved for Alzheimer’s ag­i­ta­tion, leav­ing this a big op­por­tu­ni­ty. But oth­ers have tried and failed to make the da­ta stick, and this is an area where reg­u­la­tors will like­ly de­mand plen­ty of pos­i­tive da­ta.

Ot­su­ka’s Avanir un­der­scored just how dif­fi­cult this could be with their pro­gram for AVP-786 — deudex­tromethor­phan hy­dro­bro­mide [d6-DM]/quini­dine sul­fate) — which first scored a win in the first Phase III and then flopped in the sec­ond last fall.

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.