Mer­ck KGaA adds to gene ther­a­py man­u­fac­tur­ing boom with €100M fa­cil­i­ty in Cal­i­for­nia

Add a new, €100 mil­lion Cal­i­for­nia fa­cil­i­ty to the grow­ing gene ther­a­py in­fra­struc­ture.

Mer­ck KGaA an­nounced they are open­ing a sec­ond gene ther­a­py and vi­ral vec­tor fac­to­ry in Carls­bad, Cal­i­for­nia. The new cen­ter will be just one point in the glob­al sup­ply chain Big Phar­ma is rapid­ly erect­ing to keep man­u­fac­tur­ing ca­pac­i­ty for the new tech­nol­o­gy at pace with clin­i­cal de­vel­op­ment. Over the past year, No­var­tis, PTC Ther­a­peu­tics, Pfiz­er and Ver­tex each an­nounced or opened new fa­cil­i­ties in Switzer­land, North Car­oli­na and New Jer­sey that will help build gene ther­a­py.

In No­vem­ber, Reuters re­port­ed that 11 drug­mak­ers had set aside $2 bil­lion for the man­u­fac­tur­ing ef­fort. They were led by No­var­tis, the gi­ant be­hind the sec­ond FDA-ap­proved gene ther­a­py in Zol­gens­ma which planned to spend $500 mil­lion, and Pfiz­er, which has yet to get a gene ther­a­py ap­proved but will spend $600 mil­lion. Catal­ent and Ther­mo Fish­er, mean­while, each spent over a bil­lion dol­lars ac­quir­ing com­pa­nies in­volved in gene ther­a­py or vi­ral vec­tor man­u­fac­tur­ing.

Mer­ck KGaA last shored up its gene ther­a­py man­u­fac­tur­ing in 2016 — the year be­fore the first gene ther­a­py was ap­proved in the US — when they ex­pand­ed their orig­i­nal Carls­bad fa­cil­i­ty from 44,000 to 65,000 feet. At 140,000 feet, the new fa­cil­i­ty will be more than dou­ble the size of its old­er neigh­bor. Gene ther­a­pies and the vi­ral vec­tors used to de­liv­er them will be cooked up in 1,000 liter biore­ac­tors — met­al vats rough­ly the size of 260 gal­lons of milk. Mer­ck has not said when it is sched­uled for com­ple­tion.

Al­though Mer­ck KGaA lists no gene ther­a­pies in its most re­cent pipeline, the Ger­man drug­mak­er us­es its fa­cil­i­ties to help biotechs fo­cused on the emerg­ing modal­i­ty to man­u­fac­ture their prod­ucts. The list of ap­proved gene ther­a­pies can still be count­ed on one hand, but hun­dreds are now in clin­i­cal tri­als.

Di­ane Blu­men­thal

Man­u­fac­tur­ing emerged as a press­ing bot­tle­neck al­most as soon as Spark Ther­a­peu­tics got Lux­tur­na, a treat­ment for a form of in­her­it­ed blind­ness, ap­proved as the US’ first gene ther­a­py. In an op-ed in STAT last year, Spark’s head of tech­ni­cal op­er­a­tions,  Di­ane Blu­men­thal, de­scribed the process of build­ing in Philadel­phia one of the first in-house gene ther­a­py man­u­fac­tur­ing fa­cil­i­ties.  With reg­u­la­tors in­creas­ing­ly open to ap­prov­ing the treat­ments quick­ly, she en­cour­aged oth­er com­pa­nies to in­vest in man­u­fac­tur­ing in ad­vance of even know­ing if the ther­a­py works, par­tic­u­lar­ly be­cause gene ther­a­py re­quires cus­tomiza­tion in ways few oth­er modal­i­ties do.

And she warned the prob­lem will on­ly get more press­ing.

“There isn’t a gene ther­a­py man­u­fac­tur­ing play­book — yet — to guide the de­vel­op­ment of gene ther­a­pies,” Blu­men­thal wrote. “Man­u­fac­tur­ing a gene ther­a­py is on­ly half the bat­tle. The oth­er half is mak­ing enough of it, do­ing that as ef­fi­cient­ly as pos­si­ble, and get­ting it to the pa­tients who need it. These chal­lenges be­come even more ur­gent to tack­le as the in­dus­try shifts to the next chap­ter in gene ther­a­py de­vel­op­ment, from treat­ments made in small batch­es for small pa­tient pop­u­la­tions to big­ger vol­umes for larg­er rare-dis­ease pop­u­la­tions and com­mer­cial scale.”

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.