Ab­b­Vie scores speedy re­view for RA drug upadac­i­tinib; Mer­ck KGaA wins CRISPR patent

→ Ab­b­Vie $AB­BV has high hopes, pro­ject­ing peak po­ten­tial sales of $6.5 bil­lion, for its oral JAK1 in­hibitor upadac­i­tinib for rheuma­toid arthri­tis, which is up against well-en­trenched ri­vals. On Tues­day, the drug­mak­er said FDA had re­ward­ed the drug — which is al­so un­der EU re­view — pri­or­i­ty re­view, and that the US health reg­u­la­tor is ex­pect­ed to makes its de­ci­sion by the third quar­ter of 2019.

Pfiz­er has nabbed the Eu­ro­pean green light for its Avastin copy­cat — its sec­ond on­col­o­gy biosim­i­lar in the con­ti­nent— in a slate of can­cer in­di­ca­tions from car­ci­no­ma of the colon or rec­tum to non-small cell lung can­cer. The launch of Zirabev would fol­low a trail blazed by Am­gen and Al­ler­gan with Mvasi, which was ap­proved by the EU last year and by the FDA a few months pri­or.

→ While the CRISPR IP land­scape is as con­tentious as ever with the cus­tom­ary play­ers bat­tling it out, Ger­many’s Mer­ck has swooped in with a patent for proxy-CRISPR, an im­proved ver­sion of tra­di­tion­al CRISPR that makes the gene-edit­ing tech­nique “more ef­fi­cient, flex­i­ble and spe­cif­ic by open­ing the genome for mod­i­fi­ca­tion of DNA.”

In oth­er CRISPR news, Gami­da Cell $GM­DA has joined forces with Ed­i­tas Med­i­cine $ED­IT to ed­it NAM-NK cells — nat­ur­al killer cells that have been ex­pand­ed us­ing Gami­da Cell’s nicoti­namide-based, or NAM, tech­nol­o­gy — to im­prove the treat­ment of blood can­cers and sol­id tu­mors. The fi­nan­cial terms of the deal were not dis­closed. Gami­da Cell’s NAM-NK im­munother­a­py is cur­rent­ly un­der de­vel­op­ment in Phase I tri­als for use pa­tients with re­frac­to­ry non-Hodgkin lym­phoma and mul­ti­ple myelo­ma.

Si­mon Mo­roney

Ger­man can­cer drug­mak­er Mor­phoSys $MOR is los­ing one of its founders. CEO Si­mon Mo­roney, who has led the com­pa­ny for 27 years, has told the com­pa­ny’s board he in­tends to re­tire. Mo­roney will step down when his cur­rent con­tract ex­pires on June 30, 2020, or when a suc­ces­sor is ap­point­ed, whichev­er comes soon­er. In 2017, Trem­fya, sold by J&J $JNJ for use in plaque pso­ri­a­sis, be­came the first drug based on Mor­phoSys’s an­ti­body tech­nol­o­gy to re­ceive reg­u­la­to­ry ap­proval.

Is­raeli biotech Bioblast Phar­ma $ORPN has sold its Tre­halose clin­i­cal de­vel­op­ment pro­grams to See­los Ther­a­peu­tics $SEEL for $3.5 mil­lion in two in­stall­ments, and up to $17 mil­lion in mile­stone pay­ments.

Apart from Ar­a­digm $ARDM and Im­mune Phar­ma­ceu­ti­cals $IM­NPPernix Ther­a­peu­tics $PTX has al­so filed for Chap­ter 11 to un­der­take its re­struc­tur­ing and sale process, which it hopes to con­clude by May.

→ Gilead’s $GILD CAR-T ther­a­py Yescar­ta has notched an­oth­er ap­proval, this time in Cana­da for re­lapsed or re­frac­to­ry large B-cell lym­phoma in adult pa­tients who have un­der­gone at least two sys­temic ther­a­pies.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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Paul Hudson, Sanofi CEO (via Getty)

Sanofi's $20B buy­out of Gen­zyme pays off again with Eu­ro­pean OK for first Nie­mann-Pick drug

Sanofi CEO Paul Hudson has made clear his intention to develop new rare disease drugs and broaden his company’s offerings. That effort leaped forward on Friday with the EMA’s signing off on the company’s — and the EU’s — first drug to treat the non-central nervous system manifestations of the rare and debilitating Niemann-Pick disease.

The enzyme replacement therapy, developed to replace patients’ deficient or defective enzyme, known as acid sphingomyelinase, was first developed by Genzyme, which Sanofi acquired for more than $20 billion in 2011. That acquisition has also helped Sanofi pull in sales in the field of MS.

Proac­tive­ly pre­vent­ing short­ages: New FDA guid­ance spells out which drugs re­quire risk man­age­ment plans

As the majority of drug shortages are still associated with manufacturing-related quality issues, the FDA on Thursday published new draft guidance spelling out how to proactively assess risks to manufacturing processes and supply chains, while understanding the market’s vulnerabilities.

While drug shortages peaked in 2011, the FDA says in its new 18-page draft guidance that the number of new drug shortages “has declined significantly since” that peak, reaching a low in 2015 and 2016, thanks in part to a new law’s enactment, known as FDASIA, which helped the agency better prevent or mitigate drug supply disruptions and shortages, and clarified cGMP requirements.