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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials.

Vas Narasimhan (AP Images)

Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Gene Wang, Immetas co-founder and CEO (file photo)

Im­metas Ther­a­peu­tics nabs $11M Se­ries A to nar­row their bis­pe­cif­ic work tar­get­ing in­flam­ma­tion in age-re­lat­ed dis­eases

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.