Em­maus who? A biotech you nev­er heard of just won an his­toric drug OK for sick­le cell dis­ease

Af­ter pur­su­ing an R&D odyssey that stretched out more than 15 years, lit­tle Em­maus Life Sci­ences in Tor­rance, CA has just won an FDA OK for En­dari, the first new drug ap­proved for sick­le cell dis­ease in close to 20 years.

Op­er­at­ed by Yu­ta­ka Ni­ihara, Em­maus gained the back­ing of a large ma­jor­i­ty of out­side ex­perts brought in by the agency to re­view the drug. “Un­til now, on­ly one oth­er drug was ap­proved for pa­tients liv­ing with this se­ri­ous, de­bil­i­tat­ing con­di­tion,” not­ed the FDA’s Richard Paz­dur in mark­ing the OK.

While the piv­otal da­ta from the tri­al — which was start­ed in 2010 — was marred by a high num­ber of dropouts from both the drug and place­bo arms of the study, Em­maus sig­nif­i­cant­ly cut the num­ber of days pa­tients with this drug spent in the hos­pi­tal, as well as the rate of po­ten­tial­ly lethal cas­es of acute chest syn­drome.

I queried a spokesman for the com­pa­ny to see if they had a com­mer­cial or­ga­ni­za­tion or plans to sell the drug them­selves and got this state­ment in re­turn:

En­dari is a sig­nif­i­cant mile­stone for the sick­le cell pa­tient com­mu­ni­ty. It is the first ad­vance­ment in treat­ment of this dev­as­tat­ing dis­ease in near­ly 20 years and the first-ever treat­ment op­tion for chil­dren. We ex­pect to be­gin mar­ket­ing En­dari in the fourth quar­ter of 2017 and have plans to price En­dari at a very rea­son­able amount to en­sure pa­tient ac­cess. We will ap­ply for Eu­ro­pean mar­ket ap­proval in late 2017 and we’re work­ing with var­i­ous groups and reg­u­la­to­ry en­ti­ties to pro­vide ac­cess to En­dari in the Mid­dle East and Africa.

Late Fri­day the com­pa­ny added this: “Cur­rent­ly we are an­tic­i­pat­ing that the list price range will be ap­prox­i­mate­ly $11,000 to $18,000 per year de­pend­ing on dos­ing.”

The drug, though, is not in the least bit new. It’s bet­ter known as L-glu­t­a­mine, an old, cheap over-the-counter drug which the com­pa­ny sells as Nu­tre­Store for short bow­el syn­drome as well as a nu­tri­tion­al sup­ple­ment. The biotech re­port­ed to the SEC that it net­ted on­ly $107,000 in sales rev­enue on it for the first quar­ter af­ter ac­cu­mu­lat­ing a to­tal deficit of $113 mil­lion. In SEC fil­ings, the com­pa­ny has not­ed that its own in­ter­nal fi­nan­cial re­port­ing process suf­fers from ma­te­r­i­al weak­ness­es.

In one of many odd facts I’ve col­lect­ed about this com­pa­ny, new FDA chief Scott Got­tlieb was on the board at Em­maus in the sec­ond half of 2015, one of many board post­ings he took over the past decade. Ni­ihara’s bio al­so cites a role as a pro­fes­sor at the David Gef­fen School of Med­i­cine at UCLA. A spokesper­son for the school tells me he’s a vol­un­teer clin­i­cal pro­fes­sor at the school of med­i­cine who works at Har­bor-UCLA Med­ical Cen­ter.

At one point Ni­ihara had struck a deal to merge in­to a trou­bled Generex $GNBT, which re­cent­ly com­plet­ed a 1-for-1000 re­verse stock split to re­solve its pen­ny stock sta­tus. In ear­ly Jan­u­ary, Ni­ihara took the post of ex­ec­u­tive chair­man at the com­pa­ny while it was still be­ing trad­ed on the pink sheets. But ac­cord­ing to doc­u­ments filed with the SEC, Generex nev­er was able to re­cap­i­tal­ize in or­der to com­plete the $225 mil­lion ac­qui­si­tion deal. Generex to­day has a mar­ket cap of on­ly $3 mil­lion.

En­dari’s OK marks the 24th new drug ap­proval at the FDA for the year, which is al­ready past the mark post­ed for all of a lack­lus­ter 2016.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

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While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.