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.

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.