Small AI biotech BioX­cel surges on pos­i­tive PhI­II re­sults, set­ting up de­men­tia and Alzheimer’s test

A small biotech added a few hun­dred mil­lion dol­lars in mar­ket val­ue Mon­day morn­ing af­ter an­nounc­ing pos­i­tive Phase III re­sults from their lead neu­rol­o­gy drug.

The drug, known as BX­CL501, comes from BioX­cel, one of a slate of biotechs that has raised mil­lions in re­cent years on the promise of us­ing ar­ti­fi­cial in­tel­li­gence and oth­er ad­vanced com­pu­ta­tion­al tech­niques to speed up the slug­gish pace of drug de­vel­op­ment. Sim­i­lar to the more promi­nent Re­cur­sion, the com­pa­ny aimed to use its tech to find com­pounds that oth­er com­pa­nies or re­searchers had val­i­dat­ed to some de­gree and re­pur­pose them, par­tic­u­lar­ly in the ar­eas of neu­rode­gen­er­a­tion, men­tal health and im­muno-on­col­o­gy.

Now, in a pair of near­ly 400-per­son Phase III tri­als for bipo­lar dis­or­der and schiz­o­phre­nia, the lead drug ef­fec­tive­ly calmed pa­tients’ ag­i­ta­tion, as mea­sured by a stan­dard scor­ing sys­tem. It al­so hit sec­ondary end­points, show­ing the ef­fect be­gan as ear­ly as 20 min­utes af­ter dos­ing.

The com­pa­ny said it plans to sub­mit for ap­proval in the first quar­ter of 2021. Their stock soared 31%, from $52.99 to $69.40 pre-mar­ket on the news.

“In our view, these da­ta are very com­pelling,” Guggen­heim Part­ners’ Yatin Sune­ja wrote in a note to in­vestors, “and we ex­pect ap­proval in both in­di­ca­tions.”

Sune­ja said the re­sults looked “in-line or bet­ter” than Ada­suve, the Alexza drug used as the stan­dard of care for ag­i­ta­tion. She pre­dict­ed $500 mil­lion in peak sales.

When BioX­cel filed for its IPO in 2017, though, the com­pa­ny spent as much time talk­ing about its lead drug’s po­ten­tial in Alzheimer’s and de­men­tia as it did about schiz­o­phre­nia. Al­though fur­ther be­hind, that in­di­ca­tion — so sought out by biotech in re­cent years — re­mains the com­pa­ny’s largest mar­ket po­ten­tial. A Phase I/II tri­al is now un­der­way.

“The larg­er fo­cus for in­vestors is de­men­tia, larg­er mar­ket than schiz­o­phre­nia/bipo­lar,” Sun­Trust’s Robyn Kar­nauskas wrote in a note to in­vestors.

She said the Phase III num­bers sup­port­ed $800 mil­lion in peak sales, while point­ing to even greater po­ten­tial in neu­rode­gen­er­a­tion. “We be­lieve da­ta helps val­i­date tar­get­ing a cen­tral mech­a­nism re­lat­ed to acute ag­i­ta­tion and as such, should help ex­pand BX­CL501 in­to ad­di­tion­al in­di­ca­tions,” she said.

That mech­a­nism in­volves a re­for­mu­la­tion of Pifz­er’s 21-year-old se­da­tion drug Pre­cedex. BioX­cel cre­at­ed a sub­lin­gual ver­sion of the drug that can be giv­en eas­i­ly and pro­duce a calm­ing ef­fect with­out knock­ing a pa­tient out. Specif­i­cal­ly, it ac­ti­vat­ed the al­pha-2 re­cep­tor, the path­way through which nor­ep­i­neph­rine, among oth­er neu­ro-chem­i­cals, trav­els.

In the Phase III stud­ies, the drug re­duced pa­tients’ “ex­cite­ment” score on the Pos­i­tive and Neg­a­tive Syn­drome Scale for schiz­o­phre­nia and bipo­lar dis­or­der. Across 381 schiz­o­phre­nia pa­tients, those on the high dose had 10.3 point re­duc­tion af­ter 2 hours, com­pared with 8.5 on the low dose and 4.8 on place­bo. Across 378 bipo­lar pa­tients, the high dose arm and 10.4 point re­duc­tion, com­pared with 9.1 on the low dose and 5 on place­bo.

Pa­tients al­so showed sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments on the Ag­i­ta­tion and Calm­ness Eval­u­a­tion Scale (ACES), and clin­i­cal glob­al im­pres­sion.

So­cial im­age: Vi­mal Mehta, BioX­cel CEO

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Aduhelm OK 'bit­ter­sweet' for ALS ad­vo­cates; Con­trast­ing Covid-19 vac­cine read­outs; GSK joins TIG­IT bat­tle; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With the busiest days of June now behind us, we’re starting to think seriously about the second half of the year. In August, we have scheduled a special report where Endpoints will compile a list of the 20 most influential R&D executives in biopharma. Know a luminary who should definitely be included? Nominate them now.

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Who are the lu­mi­nar­ies dri­ving the biggest ad­vances in bio­phar­ma R&D? End­points News is ask­ing for your nom­i­na­tions for a spe­cial re­port

In biopharma, driving a drug to market is the ultimate goal — but none of that happens without a strong research and development program. At the most successful companies, those R&D efforts are spearheaded by true innovators in the field who are always looking for that next novel mechanism of action or breakthrough safety profile.

Now, Endpoints News is asking you to tell us who those guiding lights are.

Leen Kawas, Athira CEO

Biotech founder placed on leave as $400M Alzheimer's start­up idea comes un­der scruti­ny

Athira Pharma, the Alzheimer’s biotech that emerged out of obscurity last year and raised nearly $400 million for a dark-horse approach to treating neurodegeneration, has found itself in sudden turmoil.

On Tuesday evening, the company released a terse statement announcing that CEO and founder Leen Kawas had been placed on administrative leave while an independent review board investigated “actions stemming” from her doctoral research at Washington State University. Mark Litton, who joined the company as COO two years ago, will take over day-to-day operations, they said.

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Bris­tol My­ers breaks the bank on Ei­sai's fo­late re­cep­tor ADC drug, lay­ing out more than $3B+ for rights

For years, innovation in oncology has been a crapshoot with Big Pharma — the whales at the table — dropping the big bucks for the key to the next generation of tumor fighters. Bristol Myers Squibb hasn’t exactly made a name for being an innovator in the space, but that doesn’t mean it won’t splash in when it sees a potential winner.

Now, with a massive check in hand, the drugmaker is willing to put its intuition to the test.

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Michael Chambers (L) and John Ballantyne

Dana­her strikes deal to buy boom­ing next-gen man­u­fac­tur­er Alde­vron for $9.6B

Life sciences conglomerate Danaher Corp. $DHR has struck a deal to buy the fast-growing Aldevron, one of the world’s top manufacturers of hotly sought-after plasmid DNA, mRNA and recombinant proteins for the burgeoning world of vaccine and drugmakers pushing some game-changing technologies.

Buyout talks set the stage for Danaher to settle on a $9.6 billion cash pact to acquire the private Fargo, ND-based company — a key supplier for a disruptive new Covid vaccine as well as a host of gene and cell therapy and CRISPR gene editing players — founded by Michael Chambers and CSO John Ballantyne as a crew of 2 back in 1998.

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Jeff Albers, Blueprint CEO

Blue­print Med­i­cines nabs 4th ap­proval in bid to­ward prof­itabil­i­ty

Blueprint Medicines’ push to profitability continues.

On Wednesday, the Cambridge biotech announced the FDA approved its longtime lead drug, Ayvakit, for advanced systemic mastocytosis, a group of debilitating rare diseases where one type of immune cell — mast cells — builds up uncontrollably in a particular organ. The decision came on the heels of Phase III trials showing that more than half of late-stage patients who received the drug responded to it and did so for just over three years.

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Franz-Werner Haas, CureVac CEO (Christoph Schmidt/picture-alliance/dpa/AP Images)

UP­DAT­ED: Cure­Vac blames vari­ants as a close­ly-watched Covid vac­cine goes down in flames, fail­ing piv­otal study with woe­ful da­ta

CureVac was widely expected to come in with a late but likely late-stage winner in the race to develop new vaccines for the Covid-19 pandemic. Instead, late Wednesday, the German biotech said their mRNA candidate CVnCoV flat failed a pivotal trial — quashing any hopes for a quick entry in the blockbuster field and gutting their share price.

CVnCoV demonstrated an interim vaccine efficacy of 47% against COVID-19 disease of any severity and did not meet prespecified statistical success criteria. Initial analyses suggest age and strain dependent efficacy.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Days af­ter con­tro­ver­sy greet­ed Bio­gen's block­buster Alzheimer's OK, the big biotech con­cedes a set­back on the tau front

Just days after triggering a maelstrom of controversy with their decision to launch an unproven Alzheimer’s drug with a $56,000 price, Biogen $BIIB is back with the latest data on its mid-stage tau drug.

And it’s not good.

The big biotech says that gosuranemab — targeted at tau, the second leading drug target in Alzheimer’s — flat failed its Phase II and will now be taken out and dumped in the mass grave for all but one other Alzheimer’s drug in the past generation.

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