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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

Endpoints News

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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115 mil­lion and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS–CoV-2, and to prevent new ones.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

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Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.