Bio­haven touts a pair of PhI­II wins for mi­graine pill, but shares ric­o­chet off of weak da­ta

Bio­haven has re­leased a set of pos­i­tive Phase III da­ta for their fast-act­ing mi­graine drug, which close­ly par­al­lel a ri­val at Al­ler­gan and trail well be­hind Eli Lil­ly’s las­mid­i­tan. And the biotech will now join the scram­ble un­der­way for an ap­proval in a field that’s about to be se­vere­ly dis­rupt­ed.

In­vestors didn’t like the num­bers ear­ly Mon­day morn­ing, send­ing shares $BHVN down 40%. That clear­ly wasn’t the way Bio­haven was bet­ting. The biotech paid Bris­tol-My­ers Squibb $50 mil­lion just a cou­ple of weeks ago to re­struc­ture their li­cens­ing deal, cut­ting the roy­al­ty stream to the sin­gle dig­its. But in the hours up to the close, the stock — backed by some heavy­weight mar­ket play­ers — went on a roller coast­er ride in the red, end­ing the day down 7%.

Al­ler­gan — which will have an­oth­er Phase III tri­al to re­port on — saw its shares rise a bit, while Lil­ly stock jumped close to 2%.

The co-pri­ma­ry end­points for Bio­haven’s oral rimegepant (BHV-3000) was pain free­dom and free­dom from most both­er­some symp­tom 2 hours af­ter tak­ing the pill in two piv­otal stud­ies.

Their pain free­dom rate was 19.2% and 19.6% for the drug, ver­sus 14.2% and 12% for the place­bo arm. Free­dom from MBS was 37.6% and 36.6% ver­sus 25.2% and 27.7% in the sug­ar pill group.

That ev­i­dent­ly may have been sta­tis­ti­cal­ly sig­nif­i­cant, but it wasn’t a big enough spread to con­vince skep­tics won­der­ing about the drug’s clin­i­cal sig­nif­i­cance in a com­pet­i­tive field.

Com­pare rimegepant to Al­ler­gan’s oral CGRP ubro­gepant, where the first Phase III re­port­ed out free­dom for pain at 2 hours for 19.2% and 21.2% for two dif­fer­ent dos­es and 11.8% in the place­bo arm; 38.6% and 37.7% on MBS and 27.8% in the place­bo arm.

Eli Lil­ly’s las­mid­i­tan — which works by hit­ting 5-HT1F re­cep­tors — did even bet­ter, de­spite be­ing dogged by high place­bo rates. Lil­ly $LLY, which paid $960 mil­lion to buy CoLu­cid and las­mid­i­tan, al­so has a CGRP drug that has per­formed in line with the rest of the in­jecta­bles.

Two hours af­ter tak­ing las­mid­i­tan or a place­bo, here were the the lat­est Phase III re­spons­es on free­dom from pain by dosage: 28.6% for 50 mg (p=0.003); 31.4% for 100 mg (p<0.001); 38.8% for 200 mg (p<0.001) and 21.3% for place­bo. In­ves­ti­ga­tors al­so nailed an end­point on free­ing pa­tients of their most both­er­some symp­tom, whether that was nau­sea, sen­si­tiv­i­ty to sound or sen­si­tiv­i­ty to light. The da­ta: 40% for 50 mg (p=0.009); 44.2% for 100 mg (p<0.001); 48.7% for 200 mg (p<0.001) and 33.5% for place­bo.

Back-to-back Phase II­Is, how­ev­er, won’t trans­late in­to a quick FDA fil­ing. Bio­haven not­ed in a re­lease that it won’t be ready to file un­til 2019, keep­ing up with Al­ler­gan on that score.

Vlad Coric

The ri­vals here are bet­ting that they can take mar­ket share for treat­ing mi­graines as heavy­weights com­pete over in­jectable CGRP drugs head­ed to reg­u­la­tors for the pre­ven­tion of acute mi­graine. Am­gen and No­var­tis lead the way on that front, with a drug un­der re­view. Te­va, and Alder are an­gling for a fol­lowup po­si­tion.

The one com­mon char­ac­ter­is­tic of these drugs is that they all per­form in re­mark­ably sim­i­lar ways, leav­ing oral de­liv­ery and price as two fea­tures that these two new ther­a­pies can com­pete on. Al­ler­gan has al­so had to ex­plain some cas­es of liv­er tox in its first Phase III — a con­cern for the class — while Bio­haven says it saw noth­ing to alarm in­ves­ti­ga­tors in its Phase III stud­ies.

Bio­haven CEO Vlad Coric had this to say:

By com­bin­ing pos­i­tive ef­fi­ca­cy re­sults and a fa­vor­able safe­ty pro­file with ease of oral dos­ing, we be­lieve that rimegepant will rep­re­sent a sig­nif­i­cant im­prove­ment over ex­ist­ing treat­ment op­tions.

Bio­haven shares al­so took a beat­ing last fall when their Phase II/III study of tri­grilu­zole demon­strat­ed not on­ly that it didn’t work bet­ter than a place­bo in treat­ing spin­ocere­bel­lar atax­ia, but pa­tients in the study al­so re­port­ed in larg­er num­bers that the place­bo helped them more than the drug.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.