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.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

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Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

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'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

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Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

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Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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