Fi­bro­Gen, As­traZeneca score Chi­na ap­proval for ane­mia drug ahead of piv­otal US da­ta

It may be the first time a multi­na­tion­al phar­ma­ceu­ti­cal com­pa­ny As­traZeneca $AZN could be­gin sell­ing a po­ten­tial block­buster med­i­cine in Chi­na, be­fore the Unit­ed States or Eu­rope. Fi­bro­Gen $FGEN and the British drug­mak­er have se­cured Chi­na ap­proval for their oral drug, rox­adu­s­tat, for ane­mic pa­tients with chron­ic kid­ney dis­ease who are de­pen­dent on dial­y­sis.

The drug, which stim­u­lates the pro­duc­tion of red blood cells by mim­ic­k­ing the ef­fect of high al­ti­tude in hu­mans, will be launched in the sec­ond half of next year un­der the Chi­nese brand name: ài ruì zhuó 爱瑞卓. While Fi­bro­Gen will take care of man­u­fac­tur­ing, As­traZeneca is in charge of com­mer­cial­iza­tion. Ap­proval for a larg­er pool of pa­tients — who are not de­pen­dent on dial­y­sis — is ex­pect­ed some­time in 2019, af­ter Chi­nese reg­u­la­tors com­plete an in­spec­tion of tri­al sites, the San Fran­cis­co-based com­pa­ny said on Mon­day.

The ap­proval could open up an es­ti­mat­ed $300-500 mil­lion sales op­por­tu­ni­ty, not­ed Jef­feries’ Michael Yee. “For Chi­na, FGEN has re­ceived $43 mil­lion from AZN al­ready and is en­ti­tled to up to $334 mil­lion of ad­di­tion­al pay­ments, much of which could be paid to FGEN this quar­ter,” he wrote. Over­all, Fi­bro­Gen has rough­ly $900 mil­lion in glob­al reg­u­la­to­ry mile­stones out­stand­ing, the ma­jor­i­ty of which should be re­al­ized over the next 12-18 months, not­ed Leerink’s Ge­of­frey Porges.

Over the past few years, Chi­na’s ver­sion of the FDA in­tro­duced a slate of re­forms to en­tice drug de­vel­op­ers, in­clud­ing over­turn­ing a rule that re­quired drug­mak­ers to con­duct sep­a­rate tri­als in Chi­na be­fore ap­provals were sanc­tioned. How­ev­er, Rox­adu­s­tat — which could even­tu­al­ly be used to treat mil­lions af­flict­ed with CKD in Chi­na — was giv­en pri­or­i­ty re­view by Chi­nese health reg­u­la­tors, who agreed to eval­u­ate the mar­ket­ing ap­pli­ca­tion on a rolling ba­sis, af­ter the drug per­formed well in two piv­otal stud­ies in Chi­na.

Porges es­ti­mat­ed rox­adusat will gen­er­ate sales of $9 mil­lion in Chi­na next year – but ex­pects that num­ber will climb to a hefty $1 bil­lion by 2025.

In the Unit­ed States, the drug is ex­pect­ed to be sub­mit­ted for re­view in the first half of next year, af­ter piv­otal tri­als are com­plet­ed. Top-line ef­fi­ca­cy da­ta from a US tri­al are ex­pect­ed in the com­ing weeks, fol­lowed by key MACE safe­ty da­ta in March/April 2019, Yee not­ed.

Fi­bro­Gen, which orig­i­nal­ly de­vel­oped the drug, has part­nered with As­traZeneca since 2013 to de­vel­op and sell the treat­ment in re­gions in­clud­ing the Unit­ed States, Chi­na, Aus­tralia, New Zealand and South­east Asia. Astel­las is work­ing with Fi­bro­Gen to do the same in ar­eas such as Japan, Eu­rope, the Mid­dle East and South Africa. An ap­pli­ca­tion to mar­ket rox­adu­s­tat in Japan was sub­mit­ted in Oc­to­ber.

Fi­bro­Gen and As­traZeneca are locked in a race with Ake­bia $AK­BA, whose ex­per­i­men­tal drug vadadu­s­tat has a sim­i­lar mech­a­nism of ac­tion. But piv­otal vadadu­s­tat da­ta are not ex­pect­ed un­til 2020. The dom­i­nant drugs for anaemia in the Unit­ed States are red-blood-cell boost­ing ery­thro­poiesis-stim­u­lat­ing agents (ESA) from Am­gen $AMGN.

Healthy kid­neys pro­duce a hor­mone called ery­thro­poi­etin (EPO), which prompts the bone mar­row to make red blood cells that car­ry oxy­gen through­out the body. If dis­eased or dam­aged, kid­neys are un­able to make enough EPO, re­sult­ing in few­er blood cells, and even­tu­al­ly anaemia.

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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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.

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.

Rob Etherington, Clene CEO

Mary­land of­fers loan to Clene de­spite ALS tri­al bumps

Even after Utah-based Clene failed to hit its primary endpoints for its ALS drug last year, the state of Maryland is putting its money at least behind Clene’s manufacturing facility.

The Maryland Board of Public Works has finalized a $3 million, 60-month loan facility with Clene Nanomedicine. The loan was provided by the state’s Neighborhood BusinessWorks program within the Maryland Department of Housing and Community Development.

Armon Sharei, SQZ founder and CEO

SQZ's out­side-the-box man­u­fac­tur­ing method slash­es pro­duc­tion time in ear­ly in study

At ASCO 2021 in June of last year, SQZ Biotech showcased a glimpse of its unorthodox cell therapy manufacturing tech. And on Wednesday, the Watertown, MA, company announced that its first-generation system showed comparable or better performance than a conventional clean-room-based manufacturing process.

The study was non-clinical. Clinical trials are expected by the first half of 2023.

SQZ’s device opens up a temporary window by cell-squeezing to deliver cargoes into cells. Its average processing time was less than six hours per batch, which is more than half the time than conventional methods. The company is planning to use the technology in its first red blood cell derived program for celiac disease. That IND is set to be submitted in the first half of 2023, the company said.

Bobby Sheng, Bora Pharmaceuticals CEO

With new ac­qui­si­tion, Bo­ra to ven­ture in­to bi­o­log­ics

Last week, Taiwan-based CDMO Bora Pharmaceuticals announced that it acquired Eden Biologics. Now, it says that purchase has helped established Bora Biologics, expanding into the biopharmaceutical market.

The acquisition of the company’s assets, which are located in the Hsinchu Biomedical Science Park in Taiwan, is helping Bora build its presence in the biopharma world by expanding production capacity of cell lines for the production of protein drugs. It will also improve the quality control and inspection specifications, as well as cell bank generation. The facility has four 500-liter bioreactors that have been approved by European and Taiwanese regulators.

Paul Chaplin, Bavarian Nordic president and CEO

With mon­key­pox cas­es ris­ing, one Eu­ro­pean coun­try is lock­ing down a small­pox vac­cine con­tract

As the global number of confirmed and suspected monkeypox cases continues to slowly climb, one country is trying to get a head start on potential vaccine stocking.

Bavarian Nordic signed a contract with an undisclosed European nation to supply its smallpox vaccine in response to new cases this month, the company announced Thursday morning. The continent saw its first monkeypox case confirmed about two weeks ago, with both the UK and Portugal seeing cases, according to the Washington Post.

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