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.

Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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UCB bags a ri­val to Soliris in $2.1B buy­out deal — but will an in­creas­ing­ly vig­i­lant FTC sign off?

UCB is buying out Ra Pharma $RARX, announcing an acquisition deal that rings up at $48 a share, or $2.1 billion net of cash, and puts them toe-to-toe with Alexion on a clinical showdown.

Ra shares closed at $22.70 on Wednesday.

There’s a small pipeline in play at Ra, but UCB is going for the lead drug — a C5 inhibitor called zilucoplan in Phase III for myasthenia gravis (MG) looking to play rival to Alexion’s Soliris. Soliris has the market advantage, though, with a much earlier approval in MG in late 2017 that UCB feels confident in challenging.

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A new play­er is tak­ing the field in a push for a he­mo­phil­ia A gene ther­a­py, and it’s a big one

BioMarin, the execs at Spark (and buyer-to-be Roche) as well as the Sangamo/Pfizer team have a new rival striding onto the hemophilia block. And it’s a big one.

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Stuck with a PhI­II gene ther­a­py fail­ure at 96 weeks, Gen­Sight prefers the up­beat as­sess­ment

Two years after treatment, the best thing that GenSight Biologics $SIGHT can say about their gene therapy for vision-destroying cases of Leber Hereditary Optic Neuropathy is that it’s just a bit better than a placebo — just maybe because one treatment can cover both eyes.

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George Scangos / Credit: Cornell University

ARCH, Soft­Bank-backed Vir Biotech­nol­o­gy un­der­whelms with $143 mil­lion IPO

George Scangos went back to Wall Street, and came back 700 million pennies short.

Scangos’ vaunted startup Vir Biotechnology raised $143 million in an IPO they hoped would earn $150 million. Shares were priced at $20, the low-end of the $20-$22 target.

Launched with backing from ARCH Venture’s Robert Nelsen, Masayoshi Son’s SoftBank Vision Fund, and the Bill & Melinda Gates Foundation, the infectious disease startup was one of a new wave of well-resourced biotechs that emerged with deep enough coffers to pursue a full R&D line rather than slowly build their case by picking off a single lead program.