Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the be­gin­ning of 2015, Deer­field Man­age­ment co-led a $10 mil­lion Se­ries C for a pri­vate gene ther­a­py start­up, re­shap­ing the com­pa­ny and bring­ing in new lead­ers to pave way for an IPO just a year lat­er.

Fast for­ward four more years and the start­up, AveX­is, is now a sub­sidiary of No­var­tis mar­ket­ing the sec­ond-ever gene ther­a­py to be ap­proved in the US.

For its part, Deer­field has al­so grown more com­fort­able and am­bi­tious about the nascent field. And the in­vest­ment firm is now putting down its biggest bet yet: a $1.1 bil­lion con­tract de­vel­op­ment and man­u­fac­tur­ing fa­cil­i­ty to pro­duce every­thing one needs for cell and gene ther­a­py — faster and bet­ter than how it’s cur­rent­ly done.

“What we saw dif­fer­ent­ly here is not just the ob­vi­ous — that there’s such a out­sized lev­el of de­mand for the amount of sup­ply — but we al­so saw the need to cre­ate an en­tire ecosys­tem” for gene ther­a­py play­ers, Alex Kar­nal, part­ner and man­ag­ing di­rec­tor at Deer­field, told End­points News. “This is the first time they’re gonna have a place where they can call home.”

Just how big is the de­mand? Days ago John Chimin­s­ki, who’s lead­ing Catal­ent in bulk­ing up its own gene ther­a­py unit, told an End­points au­di­ence that the num­ber of projects in the pipeline is ex­pect­ed to surge from 300 to­day to 1,100 by 2026 — trans­lat­ing to a vol­ume of 2.5 mil­lion to 4 mil­lion liters of vi­ral vec­tors need­ed. That’s up from 300,000 liters now.

Tony Khoury

Deer­field is build­ing its new Cen­ter for Break­through Med­i­cines at a for­mer Glax­o­SmithK­line cam­pus at King of Prus­sia, PA, now run by The Dis­cov­ery Labs. Span­ning 680,000 square feet, the site will con­sist of some­where be­tween 75 to 100 suites spread around 26 in­ter­con­nect­ed build­ings.

Start­ing from an old lab space with much of the equip­ment still in­tact means the cen­ter can hit the ground run­ning, with the site ex­pect­ed to be par­tial­ly func­tion­al by the end of this year and ful­ly up and run­ning in 2021. And do­ing so on the out­skirts of Philadel­phia — where some of the ear­li­est work in cell and gene ther­a­py were done by pi­o­neers such as Carl June and Jim Wil­son — on a sprawl­ing 1.6 mil­lion square feet com­plex de­signed to oth­er biotech star­tups should make it ap­peal­ing to the 2,000 sci­en­tists, man­u­fac­tur­ing ex­perts, tech­ni­cians and sup­port staff Deer­field plans to re­cruit.

With Tony Khoury, a key con­sul­tant for AveX­is, on board as a di­rec­tor and Paragon Bio founder Mar­co Chacón as chair at the Dis­cov­ery Labs, Kar­nal feels con­fi­dent about cast­ing a wide net and train­ing a whole co­hort of cell and gene ther­a­py spe­cial­ists on site. In fact, Deer­field has seen con­sid­er­able in­ter­est in the 24 hours they’ve an­nounced the project.

Which is good, be­cause the King of Prus­sia site is on­ly step 1. Deer­field is plot­ting three more across the US.

Mar­co Chacón

It’s not just about ex­pand­ing ca­pac­i­ty or even hav­ing the first end-to-end pro­duc­tion fa­cil­i­ty. Ac­cord­ing to Kar­nal, they will in­vest in op­ti­miz­ing the process of pro­duc­ing vi­ral vec­tors — from the most com­mon AAV to lentivirus — to both ad­dress the po­ten­cy and yield.

“The tragedy in the mar­ket­place is that the pu­rifi­ca­tion process­es are still in their in­fan­cy; we’re on­ly get­ting yields that are 10 to 20% on av­er­age from a good run,” he said. “You start with 10 to the X virus­es but then af­ter you pu­ri­fy it you lose 70 to 80% of that batch, that’s just — that’s like liq­uid gold be­ing wast­ed.”

The hope is to dou­ble the cur­rent num­bers.

Most of the ini­tial set­up will be geared to­wards gene ther­a­py — one out of 26 build­ings will be re­served for cell ther­a­py — but Kar­nal said they can keep it flex­i­ble for cus­tomers’ needs, re­gard­less of com­pa­ny lo­ca­tion, tar­get tis­sues, or de­liv­ery meth­ods. Each suite can pro­duce around 8 to 12 batch­es every year, and com­pa­nies will have the op­tion to take one whole build­ing for them­selves. The to­tal num­ber of cus­tomers will de­pend on all those vari­ables.

“Gen­er­al­ly speak­ing a batch is pret­ty con­sis­tent­ly priced, what’s not con­sis­tent is how many pa­tients you can serve,” he said.

A num­ber of oth­er com­pa­nies, both drug­mak­ers and con­trac­tors, are rush­ing to serve the same, ever-ex­pand­ing pa­tient pool. No­var­tis and Pfiz­er have com­mit­ted $500 mil­lion and $600 mil­lion on their own pro­duc­tion ca­pa­bil­i­ties, re­spec­tive­ly, and then there’s the CD­MOs like Catal­ent and Ther­mo Fish­er, which have grown their gene ther­a­py teams through bil­lion-dol­lar ac­qui­si­tions of Paragon and Bram­mer Bio. Biotechs big and small are jump­ing in­to the game; Pitts­burgh-based Krys­tal Biotech has just bro­ken ground on a sec­ond com­mer­cial fa­cil­i­ty in Find­ley Town­ship near Ohio.

“We want all the play­ers in the mar­ket­place to make it and be wild­ly suc­cess­ful be­cause the re­al­i­ty of it is even with us and every­body that ex­ists to­day, the de­mand far ex­ceeds the sup­ply still,” Kar­nal said.

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

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.

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.

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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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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