Ax­some shares rock­et up on a sur­prise win for late-stage Alzheimer’s ag­i­ta­tion study — but this is no easy layup

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.

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.