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…

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

Pfizer and Merck KGaA have detailed the Phase III data that inspired FDA regulators to designate Bavencio a “breakthrough” for first-line advanced bladder cancer and offered an early glance at how the PD-L1 can help patients with a rare gynecological cancer — carving out niches in the checkpoint space for itself after being shut out of numerous others.

In JAVELIN Bladder 100, Bavencio led to a 31% reduction in risk of death compared to standard care alone. It also extended median survival by more than seven months — a historic feat in this setting, according to investigators at Queen Mary University of London.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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