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.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

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KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.

Ear­ly-stage can­cer biotech nails $85M C round; Flem­ming Orn­skov's Gal­der­ma scores 'break­through' sta­tus

→ Zentalis Pharmaceuticals just nabbed an $85 million round from a syndicate that includes Matrix Capital, Viking Global Investors, Redmile Group, Farallon Capital, Perceptive Advisors, Surveyor Capital and Eventide Asset Management. Their lead drug is ZN-c5, which is currently in Phase I/II trials. The biotech describes that drug as a “potential best-in-class oral Selective Estrogen Receptor Degrader for estrogen receptor-positive, HER2-negative (ER+/ HER2-) breast cancer.”

UP­DAT­ED: Ob­sE­va makes case for best-in-class hor­mone sup­pres­sive ther­a­py in pos­i­tive uter­ine fi­broid study

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).