UP­DAT­ED: Ab­b­Vie adds to the Botox war chest with a 12th FDA ap­proval — but com­pe­ti­tion is fast ap­proach­ing

Com­pe­ti­tion is clos­ing in on Ab­b­Vie’s block­buster Botox fran­chise — de­spite the drug­mak­er’s best ef­forts to avoid it. In the mean­time, the Chica­go area drug gi­ant can take some so­lace in a new in­di­ca­tion that will add to the block­buster drug’s war chest.

The FDA on Wednes­day gave Botox the green light to treat de­tru­sor (blad­der mus­cle) over­ac­tiv­i­ty as­so­ci­at­ed with neu­ro­log­i­cal con­di­tions in chil­dren 5 and old­er who were in­tol­er­ant to or didn’t im­prove on an­ti­cholin­er­gic meds, Ab­b­Vie said in a state­ment. Botox is now the first neu­ro­tox­in ap­proved for such use, the drug­mak­er said.

“While al­ways sat­is­fy­ing to bring forth new in­di­ca­tions, it is par­tic­u­lar­ly re­ward­ing when we can help ad­vance care for pe­di­atric pa­tients,” Mitchell Brin, Ab­b­Vie’s se­nior VP and CSO of Botox and neu­ro­tox­ins, said in a state­ment.

Blad­der mus­cle over­ac­tiv­i­ty oc­curs when the spinal cord and blad­der can’t com­mu­ni­cate ef­fec­tive­ly, caus­ing the blad­der mus­cle to in­vol­un­tar­i­ly con­tract. This in­creas­es pres­sure in the blad­der and re­duces blad­der ca­pac­i­ty, which can cause pa­tients to leak urine and suf­fer blad­der and kid­ney dam­age over time. In chil­dren, the most com­mon neu­ro­log­i­cal cause is spina bi­fi­da, though it can al­so be spurred by con­di­tions like trans­verse myelitis and spinal cord in­jury.

“When car­ing for pe­di­atric pa­tients with neu­ro­genic de­tru­sor over­ac­tiv­i­ty, we strive to re­duce blad­der pres­sure and in­crease the blad­der’s ca­pac­i­ty. Pre­vi­ous­ly, treat­ment op­tions were lim­it­ed pri­mar­i­ly to an­ti­cholin­er­gic med­i­cines, where long-term use needs to be con­sid­ered care­ful­ly, in ad­di­tion to surgery,” said Paul Austin, chief of pe­di­atric urol­o­gy at Texas Chil­dren’s Hos­pi­tal and pro­fes­sor of urol­o­gy at Bay­lor Col­lege of Med­i­cine.

Ab­b­Vie sub­mit­ted a sup­ple­men­tal BLA for the con­di­tion back in June, and said it ex­pect­ed a de­ci­sion by Q1 2021. Right on time, the OK was based on a Phase III study with more than 100 chil­dren, which showed that Botox re­duced day­time uri­nary in­con­ti­nence episodes, low­ered max­i­mum blad­der pres­sure and in­creased blad­der ca­pac­i­ty af­ter six weeks.

The most com­mon ad­verse re­ac­tions were bac­teri­uria (20%), uri­nary tract in­fec­tion (7%), leuko­cy­turia (7%), and hema­turia (3%), ac­cord­ing to Ab­b­Vie.

Al­ler­gan snagged its first ap­proval for Botox back in 1989 for two rare eye mus­cle dis­or­ders — ble­pharospasm and stra­bis­mus — then stacked on a va­ri­ety of oth­er in­di­ca­tions, in­clud­ing chron­ic mi­graine, over­ac­tive blad­der, cer­vi­cal dys­to­nia, spas­tic­i­ty and se­vere un­der­arm sweat­ing.

The fran­chise be­came Ab­b­Vie’s in 2019 when it sealed a $63 bil­lion deal to buy out Al­ler­gan. Ab­b­Vie im­me­di­ate­ly carved off Botox’s cos­met­ic use and Al­ler­gans’ oth­er cos­met­ic port­fo­lio in­to an Al­ler­gan Aes­thet­ics sub­sidiary and kept the drug’s ther­a­peu­tic in­di­ca­tions un­der the larg­er com­pa­ny’s di­rect um­brel­la.

Al­ler­gan has put up a tough fight against Botox com­peti­tors, in­clud­ing Evo­lus, which Al­ler­gan took to court on al­le­ga­tions that its Jeu­veau was de­vel­oped us­ing a stolen trade se­cret. In De­cem­ber, Evo­lus was is­sued a 21-month ban on the im­por­ta­tion or sale of the wrin­kle treat­ment — which was re­duced from an ini­tial 10-year ban pro­posed by a judge, Bloomberg re­port­ed. 

Still, oth­er ri­vals are cir­cling in on Botox, in­clud­ing Re­vance, which read out pos­i­tive Phase III re­sults for its Dax­i­bot­u­linum­tox­i­nA in Oc­to­ber. The FDA de­ferred its de­ci­sion on Re­vance’s BLA in No­vem­ber due to Covid-re­lat­ed trav­el re­stric­tions that kept the agency from per­form­ing an in­spec­tion of the com­pa­ny’s man­u­fac­tur­ing fa­cil­i­ty. And In June, Re­vance and My­lan an­nounced plans to move for­ward with a sep­a­rate pro­gram for a Botox biosim­i­lar.

Botox raked in $991.3 mil­lion in net sales in 2019, a 9.3% in­crease from 2018.

This sto­ry has been up­dat­ed to add that the new in­di­ca­tion in­cludes chil­dren who are in­tol­er­ant to an­ti­cholin­er­gic meds. 

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, 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.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slapdown, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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Paul Hudson, Getty Images

How does Paul Hud­son's $13.5M comp pack­age stack up against oth­er CEOs? He's in the 'first quar­tile'

Paul Hudson arrived at Sanofi like a hurricane, chopping off duds in the pipeline, shaking up the C-suite, striking big M&A deals and jumping into the Covid-19 vaccine race — all in an attempt to reboot a pharma giant notorious for its setbacks.

Now, we’re getting a look at what the CEO brought home in his first year on the job.

When all is said and done, Hudson will have made about $6.7 million in 2020, about $2.5 million of which has already been paid. The bigger figure includes a $2.3 million bonus that’s subject to approval at an April meeting, and another $1.8 million in variable compensation that has yet to be paid.

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CMO Levi Garraway (Genentech)

Fo­cus­ing on the bright side, FDA OKs Roche's Actem­ra for rare lung dis­ease de­spite PhI­II flop

Actemra’s failure to hit the primary endpoint in a Phase III study didn’t stop the FDA from granting Roche priority review. And it’s certainly not standing in the way of a sixth approval for Roche’s IL-6 drug.

Regulators have cleared Actemra, or tocilizumab, for systemic sclerosis-associated interstitial lung disease in adult patients. Roche’s big Genentech subsidiary notes that it is the first biologic approved for this rare disease.