Gilead scores a TKO with PhI­II fil­go­tinib da­ta, but still faces some big bouts — and doubts — ahead

Gilead’s new Phase III rheuma­toid arthri­tis da­ta on its JAK1 in­hibitor fil­go­tinib have sparked a huz­zah from the cheer­ing sec­tion of Wall Street an­a­lysts, but there re­main plen­ty of unan­swered ques­tions about the fu­ture of this ther­a­py.

Thurs­day night the big biotech post­ed pos­i­tive da­ta from 2 Phase III stud­ies out­lin­ing a new batch of pos­i­tive ef­fi­ca­cy and safe­ty scores need­ed for an ap­proval of this drug — its oth­er and now even more close­ly watched big late-stage drug in the wake of their selon­sert­ib mis­fire in NASH. But while the drug beat out place­bo hand­i­ly, Gilead has a much high­er mark to hit in a fast-chang­ing field.

The news was a sol­id pos­i­tive for Gilead’s part­ners at Gala­pa­gos $GLPG, though, which saw its stock shoot up 13% on the re­leas­es. Gilead shares were up 2.7% af­ter the bell.

At this point, the more cau­tious ob­servers be­lieve that Gilead may well have a com­pet­i­tive play­er in the field, but the back-to-back pos­i­tive Phase II­Is are no clear-the-bases home run.

A num­ber of an­a­lysts spot­light­ed the new FINCH 1 and FINCH 3 re­sults, with ACR20/50/70 re­sults that over­shad­owed the sug­ar pill re­sponse lev­els as well as a methotrex­ate arm. But their low 100 mg da­ta were al­so gen­er­al­ly in line with Ab­b­Vie’s Hu­mi­ra, set to lose patent pro­tec­tion no lat­er than 2023. And the 200 mg da­ta, while out­per­form­ing Hu­mi­ra, is al­so go­ing to be heav­i­ly scru­ti­nized by reg­u­la­tors alarmed by the se­ri­ous safe­ty is­sues posed by high­er dos­es of the JAK in­hibitors on the mar­ket.

The FDA and EMA both is­sued stark warn­ings re­cent­ly about the dan­gers posed by 10 mg Xel­janz, and Eli Lil­ly was de­nied an ap­proval on the high dose of Olu­mi­ant — af­ter the drug was ini­tial­ly re­ject­ed out­right. And while Gilead re­searchers helped ad­dress the most im­me­di­ate con­cerns about safe­ty with new late-stage da­ta to sup­port the drug’s safe­ty pro­file, the ju­ry will like­ly re­main out on that score.

Bri­an Sko­r­ney

Baird’s Bri­an Sko­r­ney was gen­er­al­ly pleased, but not quite ready to call this an out­right win.

Da­ta re­leased from the FINCH 1 and 3 tri­als of fil­go­tinib in RA show that the med­ica­tion has sim­i­lar ef­fi­ca­cy to Ab­b­Vie’s up­atic­i­tinib. Clean safe­ty should help al­le­vi­ate in­vestor con­cerns sur­round­ing pri­or hic­cups seen with oth­er JAK in­hibitors, as fil­go­tinib was shown to have nu­mer­i­cal­ly low­er rates of CV events as com­pared to place­bo. 

Sko­r­ney is re­serv­ing fi­nal judg­ment un­til he sees da­ta from the MAN­TA study. But the com­pa­ny re­mains in the game for com­pet­ing with Ab­b­Vie, which has dom­i­nat­ed the space for years with its $20 bil­lion drug and next-gen ap­proach.

Jef­feries’ Michael Yee was en­thu­si­as­tic, but al­so con­ced­ed:

We ac­knowl­edge that a key over­hang is the on­go­ing male tox­i­c­i­ty study (MAN­TA), but it could have in­ter­im da­ta in Q3:19 and lead to a faster-than-ex­pect­ed fil­ing short­ly there­after (e.g. file on in­ter­im rather than tri­al com­ple­tion in 2020). Fil­ings are pend­ing post dis­cus­sions with FDA.

Tyler Van Bu­ren

Piper Jaf­fray’s Tyler Van Bu­ren wasn’t call­ing this a best-in-class drug, but he still sees block­buster po­ten­tial in a huge mar­ket.

The mag­ni­tude of the var­i­ous ACR20 treat­ment ben­e­fits ob­served in FINCH1 and FINCH3…are nu­mer­i­cal­ly low­er than tri­al re­sults from com­pet­i­tive agents. How­ev­er, we note that these cross-tri­al com­par­isons are fraught with er­ror and like­ly af­fect­ed by the rel­a­tive high place­bo/MTX rates in the FINCH tri­als. With re­spect to safe­ty, the in­ci­dence of SAEs – in­clud­ing DVTs – ap­pears rel­a­tive­ly low when com­par­ing the events/100 pa­tient years to the reg­is­tra­tional Xel­janz da­ta. All in all, fil­go­tinib ap­pears to be an ef­fec­tive, safe, and com­pet­i­tive oral JAK in­hibitor and we con­tin­ue to mod­el a launch in 2021 with sales sur­pass­ing $1.5B by 2028. Our big­ger con­cerns are (1) if a fil­go­tinib launch can re­al­ly oc­cur in 2021 (giv­en that Man­ta study com­ple­tion is re­quired and tim­ing is un­cer­tain) and (2) the com­pet­i­tive dy­nam­ics when launched as it is the fourth oral JAK to mar­ket.

Gilead still has lots of work to do on this pro­gram, and suc­cess won’t come eas­i­ly.

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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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.

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.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

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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