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.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

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For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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