The top 10 prospec­tive block­buster drug launch­es slat­ed for 2018 — Eval­u­ate

Im­age: Gilead’s John Mil­li­gan. Get­ty


Every­body in bio­phar­ma talks about un­met med­ical needs when they re­view their late-stage pipeline, but it’s the mar­ket po­ten­tial that will ei­ther whip up in­vestors or leave them cold about any in­no­va­tion. The top 10 prospec­tive drug launch­es loom­ing in 2018 — as se­lect­ed by Eval­u­ate in its 2018 pre­view — un­der­scores all the po­ten­tial of the would-be block­busters that al­ways dom­i­nate bio­phar­ma R&D news.

So what will we be look­ing out for?

Some very well es­tab­lished mar­ket­ing com­pa­nies are in the mix for a 2018 block­buster launch. There are new ad­vances in HIV and di­a­betes, and it’s not the least bit un­ex­pect­ed to find that 6 of the 10 are ei­ther new can­cer ther­a­pies or drugs for rare dis­eases — two fields where the FDA is now square­ly be­hind rapid launch­es.

The po­ten­tial sales fig­ures are pro­ject­ed for 2022 by Eval­u­ate.

1 Bicte­gravir/F/TAF — Gilead

Peak sales: $5.05 bil­lion
Cat­e­go­ry: HIV

The scoop: The HIV triplet that Gilead has poised for the mar­ket­place with a Feb­ru­ary 12 PDU­FA date il­lus­trates a few things about the com­pa­ny and its po­si­tion in the mar­ket. Gilead has been a dom­i­nant play­er in HIV for years now, and wasn’t about to let Glax­o­SmithK­line just walk away with any part of their rev­enue with their new­ly ap­proved two drug com­bo Ju­lu­ca — which it­self is a land­mark achieve­ment in the dri­ve to make these cock­tail ther­a­pies eas­i­er than ever to re­main com­pli­ant with. Gilead’s un­der­stand­ing of the HIV mar­ket is now part of its DNA, and an­a­lysts are quick to give it high marks for be­ing com­plete­ly prepped on the roll­out when that ap­proval comes through — prob­a­bly soon­er than lat­er.


2 Semaglu­tide — No­vo Nordisk

Peak sales: $2.7 bil­lion
Cat­e­go­ry: Di­a­betes

The scoop: Just a few days ago the FDA hit the green light for No­vo Nordisk’s semaglu­tide, a once-week­ly GLP-1 di­a­betes drug that came through a ma­jor Phase III pro­gram with fly­ing col­ors. In fact, their drug — to be sold as Ozem­pic — beat out Trulic­i­ty in a head-to-head study, which will put con­sid­er­able pres­sure on Eli Lil­ly’s big new drug. Tapped as a block­buster with more than $2 bil­lion in peak sales po­ten­tial, No­vo has al­so start­ed up a huge obe­si­ty tri­al, in­ter­est­ed in see­ing if it can get pa­tients to shed the weight that can cause di­a­betes in the first place. That makes this drug a clear and present threat to a whole slate of drugs on the mar­ket, none of which are do­ing very well.


3 Epaca­do­stat — In­cyte

Peak sales: $1.94 bil­lion
Cat­e­go­ry: On­col­o­gy

Hervé Hop­penot

The scoop: The leader in the IDO1 field is sub­ject­ed to a close-up with every new cut of the da­ta. That hasn’t al­ways gone ac­cord­ing to plan at In­cyte, but re­searchers have stuck close enough to the path to stay on track with fore­casts of a ma­jor block­buster fu­ture in can­cer. The im­por­tance of this drug for In­cyte can’t be over­es­ti­mat­ed. And every move from a com­peti­tor, in­clud­ing the pow­er­house team at Bris­tol-My­ers Squibb, which paid $1.25 bil­lion to get their hands on theirs, gets equal scruti­ny. Get­ting to the top of the field is one thing, stay­ing there will be some­thing else. One part of In­cyte’s plan for main­tain­ing its po­si­tion is by com­bin­ing its drug with sev­er­al dif­fer­ent check­points. And In­cyte CEO Hervé Hop­penot even re­cent­ly went out and bought one of his own, af­ter the first deal left him with a check­point with an odd­ly check­ered his­to­ry in the clin­ic.


4 Ro­va-T — Ab­b­Vie

Peak sales: $1.44 bil­lion
Cat­e­go­ry: On­col­o­gy

The scoop: Ab­b­Vie paid $5.8 bil­lion in cash for this drug, promis­ing up to $4 bil­lion more in mile­stones to ac­quire the lit­tle-known biotech uni­corn Stem­cen­tryx. And with mon­ey like that on the ta­ble, ex­pec­ta­tions are run­ning high. Com­bined with Bris­tol-My­ers’ Op­di­vo and Yer­voy, Ab­b­Vie is bet­ting that it has a win­ner in small cell lung can­cer, though its first cut of the ear­ly da­ta last year failed to im­press any­one out­side of the com­pa­ny. The drug drops a cy­to­tox­ic bomb right on DLL3-ex­press­ing can­cer cells com­mon in SCLC.


5 Ozan­i­mod — Cel­gene

Peak sales: $1.27 bil­lion
Cat­e­go­ry: Mul­ti­ple scle­ro­sis

The scoop: Cel­gene ex­ecs are not the shy type when it comes to tout­ing an ex­per­i­men­tal drug’s sales po­ten­tial. Their ini­tial mark: $4 bil­lion to $6 bil­lion. That num­ber has been com­ing down, though. Still, Ge­of­frey Porges re­mained a big be­liev­er at the end of Oc­to­ber as Cel­gene rolled out ap­prov­able num­bers. His es­ti­mate: $2.9 bil­lion in peak sales.


6 Apa­lu­tamide — J&J

Peak sales: $1.24 bil­lion
Cat­e­go­ry: On­col­o­gy

Rich Hey­man

The scoop: This drug (ARN-509) has been on my radar for years. I was fol­low­ing it close­ly when J&J came in and scooped up Rich Hey­man’s Aragon in one of its block­buster deals, with $650 mil­lion for the up­front alone. It’s thrilling to see it head­ed to reg­u­la­tors, even though we still haven’t seen the da­ta. J&J is the on­ly top 10 phar­ma com­pa­ny on this list, and it wouldn’t be here with­out the biotech team at Aragon. It al­so wouldn’t be here with­out an ag­gres­sive busi­ness de­vel­op­ment team that struck a line­up of bil­lion-dol­lar deals.


7 Elagolix — Ab­b­Vie

Peak sales: $1.21 bil­lion
Cat­e­go­ry: Women’s health

The scoop: I got a chance to pick through the Phase III da­ta last May, so no big sur­prise that this qual­i­fies as Ab­b­Vie’s sec­ond drug on the top 10. The oral go­nadotropin-re­leas­ing hor­mone (GnRH) re­cep­tor an­tag­o­nist demon­strat­ed some clear, dose-de­pen­dent re­spons­es that eas­i­ly out­paced the place­bo arms in two Phase III tri­als, each with more than 800 women en­rolled in them. Both stud­ies tracked clin­i­cal re­spons­es for dys­men­or­rhea — acute and po­ten­tial­ly dis­abling men­stru­al pain — as well as non­men­stru­al pelvic pain. Ab­b­Vie has been lin­ing up block­busters for the day — prob­a­bly some years from now — when it won’t have the Hu­mi­ra gold mine to re­ly on any­more. This drug looks like it should make that port­fo­lio.


8 AVXS-101 — Avex­is

Peak sales: $1.14 bil­lion
Cat­e­go­ry: Rare dis­eases

The scoop: Avex­is has a piv­otal study in the works for this drug, but it won’t be wait­ing for the da­ta be­fore seek­ing an ap­proval. A few weeks ago we saw some ex­cel­lent da­ta from a tiny study of 15 in­fants treat­ed with this gene ther­a­py for spinal mus­cu­lar at­ro­phy. And the agency might well be in a mood to hur­ry it out to save pa­tients fac­ing a lethal dis­ease. The ther­a­py pos­es a di­rect threat to Bio­gen’s new fran­chise for Spin­raza, priced at $750,000 for the first year — one of the top 10 most ex­pen­sive ther­a­pies in the world.


9 Lanadelum­ab — Shire

Peak sales: $1.12 bil­lion
Cat­e­go­ry: Rare dis­eases

Flem­ming Orn­skov

The scoop: Back in May we got a peek at some stel­lar Phase III da­ta for this drug, an­oth­er rare dis­ease ther­a­py like­ly to de­but with a six-fig­ure stick­er price. Shire paid close to $6 bil­lion for Dyax, large­ly on the promise of the next-gen HAE ther­a­py. And CEO Flem­ming Orn­skov added a $4 CVR worth $656 mil­lion if the drug is ap­proved. That pay­off could be loom­ing in 2018 if the da­ta pass­es muster with reg­u­la­tors. Orn­skov is trans­form­ing Shire in­to a rare dis­ease play­er with glob­al heft. This drug will keep the com­pa­ny point­ed in that di­rec­tion.


10 Epid­i­olex — GW Phar­ma

Peak sales: $960 mil­lion
Cat­e­go­ry: Rare dis­eases

The scoop: This is close enough to a bil­lion dol­lars to qual­i­fy for the block­buster mar­ket. The GW crew work­ing on epid­i­olex has been on a roll all year long. They get start­ed with a slate of up­beat da­ta in March, when the drug scored with pos­i­tive piv­otal da­ta for Dravet syn­drome, a rare form of se­vere epilep­sy. A few months lat­er, it was Lennox-Gas­taut syn­drome. Not all an­a­lysts have been this con­ser­v­a­tive with peak sales es­ti­mates, with some break­ing the $2 bil­lion mark. But this biotech ap­pears poised for a trans­for­ma­tion­al year.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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UP­DAT­ED: CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.

The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.

CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.