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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Fireside chat between Hal Barron and John Carroll, UKBIO19

It’s time we talked about bio­phar­ma — live in Lon­don next week

Zoom can only go so far. And I think at this stage, we’ve all tested the limits of staying in touch — virtually. So I’m particularly happy now that we’ve revved up the travel machine to point myself to London for the first time in several years.

Whatever events we have lined up, we’ve always built in plenty of opportunities for all of us to get together and talk. For London, live, I plan to be right out front, meeting with and chatting with the small crowd of biopharma people we are hosting on October 12 at Silicon Valley Bank’s London headquarters. And there’s a lengthy mixer at the end I’m most looking forward to, with several networking openings between sessions.

Take­da to pull key hy­poparathy­roidism drug from the mar­ket af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024, but the entire inventory will be available until depleted or expired, a company spokesperson said via email.

Pfizer and BioNTech's original Marvel comic book links evolving Covid vaccine science to Avengers' evolving villain-fighting tools.(Source: Pfizer LinkedIn post)

Pfiz­er, BioN­Tech part­ner with Mar­vel for Avengers and Covid-fight­ing com­ic book

Pfizer and BioNTech are collaborating with Marvel to celebrate “everyday” people getting Covid-19 vaccines in a custom comic book.

In the “Everyday Heroes” digital comic book, an evolving Ultron, one of the Avengers’ leading villains, is defeated by Captain America, Ironman and others. The plotline and history of Ultron is explained by a grandfather who is waiting with his family at a clinic for Covid-19 vaccinations.

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Kaile Zagger, Infinant Health CEO

UC Davis mi­cro­bio­me spin­out re­brands in­fant sup­ple­ment busi­ness with na­ture fo­cus

When Kaile Zagger took the helm of UC Davis spinout Evolve Biosystems several months ago, the company billed itself as a probiotic maker.

However, she believes the company’s Evivo supplement designed to help infants develop a healthy gut microbiome is “so much more” — and that, she said, calls for a rebrand.

Evolve has, well, evolved into Infinant Health, the company announced on Monday. The new name is a mash-up of the words “infant” and “infinite,” representing the company’s goal of expanding beyond infant care. While its sole product, Evivo, is intended for newborns, Infinant is “quickly developing” an option for kids through the age of two.

FDA+ roundup: Ad­comm date set for Cy­to­ki­net­ics heart drug; New gener­ic drug guid­ance to re­duce fa­cil­i­ty de­lays

The FDA on Wednesday set Dec. 13 as the day that its Cardiovascular and Renal Drugs Advisory Committee will review Cytokinetics’ potential heart drug, meaning regulators aren’t likely to meet the Nov. 30 PDUFA date that was previously set.

The drug, known as omecamtiv mecarbil, read out its first Phase III in November 2020, hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as the key to breaking into the market, failing to significantly differ in reducing cardiovascular death from placebo.

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Bob Azelby, Eliem Therapeutics CEO

Eliem says ear­li­er drug ex­po­sure is­sues have been re­solved, drops one epilep­sy in­di­ca­tion

After being forced to delay two Phase IIa trials and blaming CMC issues on a Phase Ib miss, Eliem Therapeutics believes it’s now in the clear.

The Seattle and UK-based biotech put out word Wednesday morning about how it conducted new early-stage studies to confirm why low exposure issues arose during the Phase Ib. After researchers compared the results of the studies, Eliem found “no meaningful difference” between them and ruled out CMC as the reason for the foiled Phase Ib study, the company said in a press release.

GSK touts topline win for PD-1 in head-to-head with Keytru­da — while steer­ing next big check­point drug in­to PhI­II

GSK is claiming a win for what it calls the largest head-to-head trial pitting a PD-1 against Merck’s best-selling Keytruda in a type of lung cancer, as its Jemperli met the primary endpoint of objective response rate.

In a separate positive move, GSK says it’s moving both arms of the COSTAR Lung trial into Phase III to test Jemperli as well as the TIM-3 inhibitor cobolimab.

Hesham Abdullah, GSK’s global head of oncology development, said in a statement that the two trials “support the ambition for dostarlimab to become the backbone of our ongoing immuno-oncology-based research and development programme when used alone and in combination with standard of care and future novel cancer therapies, particularly in patients with currently limited treatment options.”

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