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.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.