Bio­haven is bet­ting hun­dreds of mil­lions it can hus­tle ahead of ri­vals in a block­buster race. A quick M&A deal could pay off back­ers big time

Bio­haven $BHVN may not have any­thing like the mar­ket caps of its ri­vals at Al­ler­gan $AGN and Eli Lil­ly $LLY, but they’re not let­ting the David vs. Go­liath sta­tus of their com­pe­ti­tion stop them from mak­ing a big bet on a fast win for a new oral drug to treat acute mi­graine. And they’re backed by a group that has an eye on a po­ten­tial buy­out.

The biotech has ac­quired a pri­or­i­ty re­view vouch­er from GW Re­search for $105 mil­lion, look­ing to shave 4 months off their mar­ket­ing ap­pli­ca­tion for rimegepant — a year af­ter they man­aged to dis­en­chant its in­vestors with their pos­i­tive Phase III da­ta, which failed to dra­mat­i­cal­ly break away from the place­bo group.

Bio­haven says it plans to sub­mit their ap­pli­ca­tion some time in the next few months, which would pro­vide a de­ci­sion from the FDA be­fore year’s end. That would put them right along­side their di­rect ri­vals at Al­ler­gan, which sub­mit­ted their ap­pli­ca­tion for ubro­gepant, which now faces a Q4 PDU­FA dead­line.

Both drugs are oral CGRP re­cep­tor an­tag­o­nists, both in-li­censed from phar­ma gi­ants. Al­ler­gan, which re­cent­ly ex­pe­ri­enced a big set­back on a late-stage drug, got its ther­a­py from Mer­ck. And Bio­haven bagged a li­cens­ing deal with Bris­tol-My­ers Squibb.

Bio­haven ne­go­ti­at­ed a $200 mil­lion fi­nanc­ing deal with Roy­al­ty Phar­ma to pay for the PRV as well as tee up $75 mil­lion if the FDA pro­vides a stamp of ap­proval. Div­ing in­to the SEC fil­ing, the biotech made clear that Roy­al­ty is set to buy $200 mil­lion in shares in 2 batch­es. And they’re not look­ing for in­ter­est. If a change of con­trol — or a buy­out — comes along any­time in the next few years, RP gets a pay­back of 1.5 to 2x on the shares. If no ap­proval comes with no buy­out, the pay­back is 2x, or $400 mil­lion.

Bio­haven and its ri­vals are fol­low­ing a wave of new­ly ap­proved in­jectable CGRP drugs from Eli Lil­ly, Te­va and Am­gen/No­var­tis, which took the lead in that are­na. Lil­ly is al­so an­gling ahead with las­mid­i­tan, an oral se­lec­tive sero­tonin 5-HT1F ag­o­nist which it sub­mit­ted last No­vem­ber.

These oral drugs are be­ing po­si­tioned to take away mar­ket share from gener­ic trip­tans, which typ­i­cal­ly have a tran­sient ef­fect. The mi­graine mar­ket has long been con­sid­ered wide open to drug de­vel­op­ers ad­vanc­ing bet­ter so­lu­tions for mil­lions of pa­tients.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Jay Flatley, new Zymergen CEO

Fol­low­ing Au­gust melt­down, Zymer­gen hints at sal­vage plans — cut­ting jobs and rene­go­ti­at­ing loans

Two months after a spectacular implosion that saw its founding CEO leave his post amid customer reports its only product didn’t work, Zymergen provided the first peek behind the curtain for its plans moving forward.

In an SEC filing Wednesday, Zymergen told regulators it would slash about 100 jobs and had renegotiated a $100 million loan from Perceptive that loomed like a storm cloud over the company, moving up the maturity date 18 months to June 30, 2022. Jed Dean, one of Zymergen’s three co-founders and VP of operations, will also step down at the end of the month.

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