In the hunt for a $100M IPO, Bio­haven pol­ish­es up dis­cards from Bris­tol-My­ers, As­traZeneca

Bio­haven CEO Vlad Coric

When Bio­haven Phar­ma­ceu­ti­cal spun out of Yale a few years ago, com­pa­ny ex­ec­u­tives talked a lot about their fo­cus on de­pres­sion and anx­i­ety — tough tar­gets in R&D by any ac­count. But on Fri­day the stealthy Bio­haven jumped out with a $100 mil­lion IPO fil­ing, af­ter un­der­go­ing a rad­i­cal, low-key re­vamp of its pipeline fo­cus, clear­ly aimed at of­fer­ing in­vestors a chance at a late-stage biotech eye­ing near-term FDA ap­pli­ca­tions.

Now, ac­cord­ing to the S-1, the New Haven-based biotech’s lead drug is a CGRP ther­a­py for mi­graine, a crowd­ed field in the bio­phar­ma in­dus­try with big and lit­tle com­peti­tors — Eli Lil­ly, Am­gen/No­var­tis, Te­va and Alder — lin­ing up NDAs. A num­bers-redact­ed li­cens­ing agree­ment filed just days ago re­veals Bio­haven grabbed its drug from Bris­tol-My­ers Squibb. The S-1 spells out a pact that in­cludes up to about $350 mil­lion in de­vel­op­ment and sales mile­stones, but on­ly $9 mil­lion due in 90 days of the fil­ing — $5 mil­lion of that up front.

There’s noth­ing un­usu­al about a biotech com­pa­ny grab­bing drugs off a phar­ma com­pa­ny’s shelf for mis­fit drugs. Vivek Ra­maswamy did the same with GSK when he set up Ax­o­vant $AX­ON and quick­ly turned it in­to a record-set­ting biotech IPO, af­ter tak­ing a failed Alzheimer’s drug for $5 mil­lion up­front. This is a tried and true ap­proach, which al­ways rais­es ques­tions about why these drugs, with hu­man da­ta, are so cheap to come by.

The S-1 for Bio­haven in­cludes the boast that their CGRP drug is the on­ly one to have pos­i­tive mid-stage da­ta on four key end­points rel­a­tive to place­bo. What it ap­par­ent­ly left out was that Bris­tol-My­ers al­so in­clud­ed a cheap gener­ic trip­tan — suma­trip­tan — in the mid-stage study, which beat out all three dos­es of their drug. Bris­tol-My­ers sub­se­quent­ly shelved the drug, un­til Bio­haven came along with a new plan to re­vive it in the clin­ic.

That could take some ex­plain­ing, though the S-1 notes that trip­tans are known for headache re­cur­rence and oth­er side ef­fects. Pa­tients that want to knock out a mi­graine may pre­fer a more ef­fec­tive drug, though pay­ers aren’t like­ly to be wild about cov­er­ing it as a front­line ther­a­py. Bio­haven plans to put the drug through two Phase III stud­ies in the sec­ond half of this year and then take the piv­otal da­ta to the FDA in H1 2018.

In fair­ness, the ri­vals in this field have all been com­pared to place­bos for their ef­fi­ca­cy da­ta. And Bio­haven’s Phase III specif­i­cal­ly in­cludes trip­tan-re­sis­tant pa­tients or oth­ers who would not be good can­di­dates for that class of ther­a­pies.

The in­ves­ti­ga­tors in­volved in Bio­haven al­so had fo­cused on ke­t­a­mine, a NM­DA re­cep­tor an­tag­o­nist that’s in­spired a long line­up of aca­d­e­m­ic stud­ies. The horse tran­quil­iz­er is bet­ter known in par­ty cir­cles as Spe­cial K for its psy­che­del­ic qual­i­ties.


In this case, Bio­haven in-li­censed an NM­DA an­tag­o­nist from As­traZeneca in a $210 mil­lion deal, well af­ter the phar­ma gi­ant re­treat­ed from the field. Like Bris­tol-My­ers, Bio­haven nev­er be­fore dis­closed that it did a deal with As­traZeneca, re­fer­ring on­ly to ties with blue-chip phar­mas. Co­in­ci­den­tal­ly, I cov­ered the drug — lan­icem­ine (AZD6765)  — back in 2014 when As­traZeneca un­cer­e­mo­ni­ous­ly swept it out the back door fol­low­ing a failed mid-stage study for de­pres­sion.

But in­stead of stay­ing fo­cused on de­pres­sion, the com­pa­ny took a cue from some pre­clin­i­cal mouse tests and re­pur­posed it for symp­toms of Rett syn­drome. The FDA oblig­ing­ly gave the drug, one of many NM­DA drugs, an or­phan des­ig­na­tion which comes with a set of spe­cial in­cen­tives.

Bio­haven’s mid­dle drug is from its glu­ta­mate mod­u­la­tion plat­form. The drug is called tri­grilu­zole, a re­for­mu­la­tion of rilu­zole cur­rent­ly in a Phase II/III bioe­quiv­a­lence study for the treat­ment of atax­ia with an ini­tial fo­cus on spin­ocere­bel­lar atax­ia, or SCA. Da­ta is due out in ear­ly 2018. Bio­haven ac­quired that drug from ALS Bio­phar­ma.

Cur­rent­ly based in the British Vir­gin Is­lands, Bio­haven is set­ting up a sub­sidiary in the low-tax haven of Ire­land, where the com­pa­ny will tech­ni­cal­ly move its domi­cile. It al­so has a few strings to con­sid­er with Bris­tol-My­ers and As­traZeneca, which have some claw-back pro­vi­sions in their con­tracts and a right of first ne­go­ti­a­tion if they ever come back up on the mar­ket.

Pe­ter Kolchin­sky

The com­pa­ny has burned through more than $75 mil­lion, but raised more than $100 mil­lion from a group that in­clud­ed crossover in­vestor RA Cap­i­tal, which de­lights in com­ing in­to a deal about a year ahead of what the MBA crowd likes to call a val­ue in­flec­tion point. RA’s Pe­ter Kolchin­sky was named to the board as an ob­serv­er a few weeks ago.

CEO and Yale pro­fes­sor Vlad Coric, a Bris­tol-My­ers vet with con­sid­er­able ex­pe­ri­ence in drug de­vel­op­ment, scored $784,428 in to­tal com­pen­sa­tion last year and owns 7% of the com­pa­ny. Ex­ec­u­tive chair­man De­clan Doogan runs Portage Biotech, the biggest share­hold­er and seed provider with 28.3% of the stock. Vi­vo Cap­i­tal has a 6.7% chunk and RA is in for 5.1%.

Not long ago Doogan al­so arranged to have Bio­haven in­vest in an­oth­er Yale spin­out, Kleo Phar­ma­ceu­ti­cals, which is de­vel­op­ing new ther­a­pies that re­cruit T-cells in an im­mune sys­tem at­tack on can­cer cells and oth­er tar­gets.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

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Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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In a boost to Rit­ux­an fran­chise, Roche nabs quick ap­proval for po­latuzum­ab ve­dotin

Roche’s lat­est an­ti­body-drug con­ju­gate has crossed the FDA fin­ish line, gain­ing an ac­cel­er­at­ed ap­proval a full two months ahead of sched­ule.

Po­livy, or po­latuzum­ab ve­dotin, is a first-in-class drug tar­get­ing CD79b — a pro­tein promi­nent in B-cell non-Hodgkin lym­phoma. It will now be mar­ket­ed for dif­fuse large B-cell lym­phoma as part of a reg­i­men that al­so in­cludes the chemother­a­py ben­damus­tine and a ver­sion of rit­ux­imab (Rit­ux­an).

Ab­b­Vie touts new da­ta for Hu­mi­ra suc­ces­sor; Gilead inks dis­cov­ery deal

→ Ab­b­Vie is tout­ing new pos­i­tive da­ta com­par­ing their ag­ing block­buster Hu­mi­ra with their hoped-for block­buster upadac­i­tinib. Over 48 weeks a larg­er pro­por­tion of pa­tients tak­ing the ex­per­i­men­tal drug ex­pe­ri­enced clin­i­cal re­mis­sion than in the con­trol arm with Hu­mi­ra. Their drug brought in $20 bil­lion last year, top­ping the scales in the num­ber 1 slot.

→ Gilead has turned to Van­cou­ver-based Ab­Cellera for its lat­est dis­cov­ery deal. Ab­Cellera will use its know-how in “sin­gle-cell screen­ing of nat­ur­al im­mune sources” to find an­ti­body can­di­dates for Gilead to pur­sue in the in­fec­tious dis­ease field. The deal in­cludes an up­front and mile­stones.