John Oyler, Founder & CEO of BeiGene, at the US-China Biopharma Innovation and Investment Summit in Shanghai on October 23, 2018; Credit: Endpoints News, PharmCube

BeiGene scores first Chi­na OK with PD-1 — to be man­u­fac­tured by Boehringer In­gel­heim

On De­cem­ber 17, 2018, Jun­shi Bio­sciences notched the land­mark ap­proval for a made-in-Chi­na PD-(L)1 check­point in­hibitor by Chi­nese reg­u­la­tors. Bare­ly a year lat­er, the fourth home­grown PD-1 is mak­ing its way to a mar­ket that has since seen sev­er­al more firsts even whilst get­ting crowd­ed.

The ap­proval for BeiGene’s tislelizum­ab just be­fore 2019 end­ed is al­so mark­ing a new his­toric event: the first time a for­eign part­ner will be pro­duc­ing the mar­ket­ed drug un­der a re­formed con­tract man­u­fac­tur­ing reg­u­la­to­ry sys­tem. Boehringer In­gel­heim, a part­ner since 2013, will be sup­ply­ing the PD-1 an­ti­body for com­mer­cial use us­ing a Shang­hai fa­cil­i­ty.

“This is an im­por­tant mile­stone, not on­ly to en­sure the sup­ply of med­i­cines for pa­tients in Chi­na, but al­so for the rapid­ly emerg­ing Chi­nese bio­phar­ma­ceu­ti­cal Re­search & De­vel­op­ment land­scape,” said BeiGene CEO John Oyler in a state­ment.

In 2014 Chi­na’s drug reg­u­la­tor — since re­named the Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion — be­gan pi­lot­ing a new Mar­ket­ing Au­tho­riza­tion Hold­er sys­tem un­der which drug­mak­ers with­out man­u­fac­tur­ing ca­pa­bil­i­ties can still ap­ply for mar­ket­ing ap­proval. This move opened up the op­tion of en­list­ing con­tract man­u­fac­tur­ers and sig­ni­fied a com­plete break from the pre­vi­ous sys­tem, where de­vel­op­ers must have man­u­fac­tur­ing au­tho­riza­tion to mar­ket a drug.

While BeiGene does have its own bi­o­log­ics man­u­fac­tur­ing plant in Guangzhou, it’s al­so inked a sep­a­rate deal with Catal­ent to pro­duce its first com­mer­cial drug, zanubru­ti­nib (re­cent­ly chris­tened Brukin­sa in the US).

SVB Leerink an­tic­i­pates BeiGene will launch tislelizum­ab in ear­ly 2020. The ini­tial in­di­ca­tion is third-line re­lapsed/re­frac­to­ry Hodgkin’s lym­phoma, fa­mil­iar ter­rain for any­one who’s been fol­low­ing In­novent’s Tyvyt or Jiang­su Hen­grui’s cam­re­lizum­ab. But BeiGene will have top-line Chi­nese da­ta in non-small cell lung can­cer com­ing lat­er this year, and is plot­ting sup­ple­men­tal OKs for urothe­lial can­cer, he­pa­to­cel­lu­lar car­ci­no­ma and more.

“This is BeiGene’s first in­ter­nal­ly dis­cov­ered drug ap­proved in Chi­na,” the an­a­lysts not­ed. “The ap­proval was based on re­sults from a sin­gle-arm piv­otal Phase 2 tri­al, in which tislelizum­ab demon­strat­ed a 76.9% ob­jec­tive re­sponse rate (ORR) via in­de­pen­dent re­view com­mit­tee (IRC) as­sess­ment, in­clud­ing a 61.5% com­plete re­sponse (CR) rate.”

Their mod­el es­ti­mates $85 mil­lion in 2020 Chi­na rev­enue for tislelizum­ab, part­ly from “mean­ing­ful off-la­bel sales” — as the re­cent Na­tion­al Re­im­burse­ment Drug List up­date fea­tured In­novent’s PD-1 for Hodgkin’s lym­phoma (at about $1,100 per month) but not oth­er check­points in larg­er in­di­ca­tions.

At one point Cel­gene was part­nered on this drug as part of a port­fo­lio swap which had BeiGene in charge of mar­ket­ing Abrax­ane, Revlim­id and Vi­daza in Chi­na, but cut it loose as soon as it got gob­bled up by Op­di­vo mak­er Bris­tol-My­ers Squibb. BeiGene’s de­vel­op­ment suc­cess­es bode well for Am­gen, which re­cent­ly paid $2.7 bil­lion for a rough­ly 20% stake in the biotech and en­trust­ed its R&D team with some of its own projects.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.