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.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Robert Gould, Fulcrum Therapeutics CEO

Ful­crum stum­bles in PhII of old GSK drug, send­ing shares tum­bling

Investors are selling off shares of Fulcrum Therapeutics $FULC after their lead drug failed in a Phase II trial.

The company, founded three years ago on new research techniques such as CRISPR screening, isolated a gene called DUX4 they believed to have a central role in facioscapulohumeral muscular dystrophy, where patients’ muscle dies and is replaced by fat. And to target it, they licensed a GlaxoSmithKline drug that had failed as a cardio drug.

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Eisai moves to 200 Metro Blvd. by late 2021 (ON3)

Ei­sai is cre­at­ing a new US cor­po­rate, R&D HQ in Roche’s old Nut­ley, NJ cam­pus

Eight years after Roche pulled up stakes from Nutley, NJ in a major R&D reorganization, Japan’s Eisai is moving its US corporate and research hub into their old campus.

Now the ON3 property, Eisai — a longtime Biogen partner focused on neurodegenerative disorders like Alzheimer’s — will bring together a staff of up to 1,200 employees. And execs are pitching the move to the New Jersey campus as a cultural game-changer.

Lig­and scoops up Pfenex for up to $516M, adding pro­teins to their an­ti­body chick­ens and de­liv­ery tech

The technology hunting folks over at Ligand Pharmaceuticals have picked up a new one from across town, for a significant price.

Ligand has acquired fellow San Diego-based biotech Pfenex and their protein expression platform for $438 million cash, plus $78 million in contingent value agreements should an undisclosed milestone be hit before the end of next year.  The deal pays $12 per share, or $4.34 more than what Pfenex had been trading at before the announcement.