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…

Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.

Sen. Elizabeth Warren (D-MA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

Sen. Eliz­a­beth War­ren urges FTC to 'scru­ti­nize' two phar­ma buy­outs

Sen. Elizabeth Warren (D-MA) is calling on senior Federal Trade Commission officials to “closely scrutinize” two proposed pharma mergers.

Warren expressed concern over “rampant consolidation in the pharmaceutical industry,” in particular Amgen’s $28 billion plans to take over Horizon Therapeutics, and Indivior’s proposed acquisition of Opiant for $145 million upfront, in a letter to FTC Chair Lina Khan and Commissioners Alvaro Bedoya and Rebecca Kelly Slaughter earlier this week.