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…

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.