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.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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Pascal Soriot, AstraZeneca CEO (AP Images)

Pas­cal So­ri­ot cash­es in As­traZeneca’s chips on Mod­er­na for $1.2B cash in­jec­tion

While still working to prove its own Covid-19 vaccine, AstraZeneca has reportedly capitalized on the success of another.

The company has sold off its 7.7% stake in Moderna and turned it into $1.2 billion in cash, according to the Times, beefing up the reserves just as Pascal Soriot is wrapping up his $39 billion acquisition of Alexion and its rare disease pipeline.

AstraZeneca’s stock sale follows a similar move by Merck in December. But like its pharma brethren, the British giant is keeping its R&D collaborations with Moderna.

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Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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Fi­bro­Gen shares skid low­er as a sur­prise ad­comm rais­es risks on roxa OK

FibroGen will likely have to delay its US rollout for roxadustat once again.

In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.

Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.

In­tro­duc­ing End­pointsF­DA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

UP­DAT­ED: Feds clear the road for J&J to start de­liv­er­ing mil­lions of dos­es of their Covid-19 vac­cine — but frets linger about run­ner-up sta­tus

All the pieces needed to trigger a third wave of Covid-19 vaccine supply to start washing over the US fell neatly into place over the weekend.

After providing for a brief mime of regulatory judiciousness, the FDA stamped their emergency approval on J&J’s Covid-19 vaccine Saturday, adding to the Biden administration’s plan aimed at ending the pandemic in the near term — at least in the US. The CDC came through on Sunday with its stamp of approval and J&J is reportedly expected to start delivering vaccine sometime in the next few days.

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