Mys­tery biotech Em­maus grabs ground­break­ing FDA pan­el back­ing for sick­le cell drug — af­ter qui­et­ly scrap­ping $225M deal

Yu­ta­ka Ni­ihara

A lit­tle-known biotech out of Tor­rance, CA is one big step clos­er to win­ning the first new drug ap­proval for sick­le cell dis­ease in close to 20 years. And it has achieved pre­cious lit­tle at­ten­tion for its years-long clin­i­cal odyssey with the drug — or the $225 mil­lion deal it just qui­et­ly scut­tled days ago.

Em­maus Life Sci­ences won an FDA pan­el vote fa­vor­ing an ap­proval by 10-3, set­ting it on a course for an odds-on ap­proval by the PDU­FA date of Ju­ly 7. The break­through win comes just days af­ter Em­maus filed doc­u­ments with the SEC say­ing that it had called off a move to sell a ma­jor­i­ty in­ter­est in the com­pa­ny to Generex for $10 mil­lion in cash plus $215 mil­lion in stock.

Generex $GNBT has a mar­ket cap of sight­ly more than $5 mil­lion.

Em­maus’ drug is L-glu­t­a­mine, now dubbed En­dari, which CEO and founder Yu­ta­ka Ni­ihara has been work­ing on for years. Healio re­ports the drug won or­phan sta­tus in 2001, with fast track sta­tus com­ing 12 years ago. The Phase III da­ta were re­port­ed in 2014. And Healio’s re­port on the vote re­flect­ed some deep seat­ed reser­va­tions among the pan­el mem­bers tasked with the re­view.

“My ‘yes,’ like many of the [votes], was dif­fi­cult,” not­ed Bri­an I. Ri­ni, act­ing chair­per­son of ODAC. “This could have gone ei­ther way. This is clear­ly a bad dis­ease — worse than can­cer in many ways, most­ly from a stig­ma stand­point — and to com­plete two ran­dom­ized stud­ies is a ma­jor ac­com­plish­ment. Our job, how­ev­er, is to rec­om­mend ap­proval of drugs not based on des­per­ate need, but based on good da­ta. The da­ta were all there and I think it might be help­ful in how we ap­ply this drug clin­i­cal­ly if it is FDA ap­proved.”

One of the pan­el’s pri­ma­ry con­cerns, ac­cord­ing to Bio­Cen­tu­ry, was the high rate of dropouts — 36% com­pared to 24% for the place­bo group. FDA re­view­ers had trou­ble with miss­ing da­ta from the study and ques­tioned the ef­fi­ca­cy, not­ing that a re­duc­tion of one sick­le cell cri­sis over 48 weeks might not pro­vide a clear clin­i­cal ben­e­fit for pa­tients.

Em­maus re­port­ed top-line da­ta back in 2014, say­ing that the drug al­so re­duced hos­pi­tal­iza­tions by 33%.

In­ter­est­ing­ly, Scott Got­tlieb’s role as a part­ner at T.R. Win­ston, a bou­tique in­vest­ment bank, led him to Em­maus Life Sci­ences, which he in­clud­ed on a long list of com­pa­nies he was sev­er­ing con­nec­tions with af­ter tak­ing over as FDA com­mis­sion­er. Got­tlieb com­mit­ted to re­cuse him­self from any agency de­ci­sions on Em­maus, now up for a for­mal ap­proval.

Ni­ihara was sup­posed to be­come ex­ec­u­tive chair­man at Generex $GNBT, an OTC com­pa­ny which ex­e­cut­ed a 1-for-1000 stock split two months ago to re­solve its pen­ny stock sta­tus. Em­maus is an un­list­ed pub­lic com­pa­ny, and in an 8-K on Fri­day the com­pa­ny re­port­ed that it was ter­mi­nat­ing the let­ter of in­tent on the Generex ac­qui­si­tion:

The ter­mi­na­tion of the LOI was based on Generex’s fail­ure to file an amend­ment to its re­stat­ed cer­tifi­cate of in­cor­po­ra­tion ef­fect­ing an in­crease in its au­tho­rized cap­i­tal by May 1, 2017 and the par­ties’ in­abil­i­ty to agree on a res­o­lu­tion of cer­tain key fi­nan­cial ac­count­ing is­sues re­gard­ing the fi­nan­cial con­sol­i­da­tion of Em­maus and Generex re­sult­ing from the trans­ac­tions con­tem­plat­ed in the LOI, which pre­vent­ed fur­ther ne­go­ti­a­tion and agree­ment on key ma­te­r­i­al terms of the for­mal pur­chase agree­ment pro­vid­ed for in the LOI.

Generex says it couldn’t com­plete the deal as planned. From their fil­ing:

The LOI was pred­i­cat­ed up­on a time­line for the im­ple­men­ta­tion of Generex’s re­or­ga­ni­za­tion plan, which in­clud­ed, among oth­er things, the ac­qui­si­tion of the Em­maus cap­i­tal stock, the ac­qui­si­tion of the cap­i­tal stock of Hema Di­ag­nos­tic Sys­tems, Inc., a re­verse stock split, an in­crease in the au­tho­rized num­ber of shares of Generex com­mon stock (to be ap­proved at a stock­hold­ers’ meet­ing), and rais­ing cap­i­tal….(T)he Com­pa­ny sub­se­quent­ly en­coun­tered unan­tic­i­pat­ed reg­u­la­to­ry and op­er­a­tional is­sues that de­layed the im­ple­men­ta­tion of the re­or­ga­ni­za­tion plan…

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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