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.”

The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

Pfizer and Merck KGaA have detailed the Phase III data that inspired FDA regulators to designate Bavencio a “breakthrough” for first-line advanced bladder cancer and offered an early glance at how the PD-L1 can help patients with a rare gynecological cancer — carving out niches in the checkpoint space for itself after being shut out of numerous others.

In JAVELIN Bladder 100, Bavencio led to a 31% reduction in risk of death compared to standard care alone. It also extended median survival by more than seven months — a historic feat in this setting, according to investigators at Queen Mary University of London.

Look­ing to move past an R&D fi­as­co, Ipsen poach­es their new CEO from Sanofi

Ipsen has turned to another Paris-based biopharma company for its next CEO.

Sanofi Pasteur chief David Loew — who’s been leading one of the most advanced efforts to develop vaccines for Covid-19 — is making the journey to Ipsen, 5 months after David Meek jumped ship to run a startup in late-stage development.

Loew arrives as Ipsen works to get back on track with their rare bone disease drug palovarotene, picked up in the $1.3 billion Clementia buyout, which was slammed with a partial hold after researchers observed cases of “early growth plate closure” in patients under the age of 14. But they are pushing ahead with the over-14 crowd after writing down slightly more than half of its initial development.

Iron­wood, Ab­b­Vie kick de­layed-re­lease Linzess for­mu­la­tion to the curb af­ter tri­al fail­ure

The delayed-release formulation of Ironwood and Allergan’s bowel drug Linzess will not see the light of day.

The experimental drug, MD-7246, failed to help patients with abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) in a mid-stage study, prompting the partners to abandon the therapy.

First approved in 2012, Linzess (known chemically as linaclotide) enhances the activity of the intestinal enzyme guanylate cyclase-C to increase the secretion of intestinal fluid and then transit through the intestinal tract, as well as reduce visceral pain, to relieve pain and constipation associated with IBS.