Re­gen­eron, Sanofi win a de­layed OK for rheuma­toid arthri­tis IL-6 heavy­weight sar­ilum­ab

It took more than six months and like­ly caused more than a few red faces, but Sanofi has dealt with the FDA’s is­sues with its man­u­fac­tur­ing ops and won a green light to start mar­ket­ing the like­ly block­buster sar­ilum­ab along­side its part­ners at Re­gen­eron.

Con­sen­sus peak sales fore­casts on the drug — to be mar­ket­ed as Kevzara — top the $1 bil­lion block­buster mark, mak­ing it a big ad­di­tion for both com­pa­nies.

George Yan­copou­los, Re­gen­eron (Get­ty/Bloomberg)

The part­ners say they set the WAC at $39,000/year for the 200 mg and 150 mg dos­es, which is ap­prox­i­mate­ly “30 per­cent low­er than the WAC for the two most wide­ly used TNF-al­pha in­hibitors.” Those two are Hu­mi­ra – which earned $16 bil­lion last year – and En­brel.

The IL-6 drug whet­ted an­a­lysts’ ex­pec­ta­tions last March when it beat out Ab­b­Vie’s Hu­mi­ra in a head-to-head show­down for rheuma­toid arthri­tis. The Hu­mi­ra da­ta weren’t in reg­u­la­tors’ pack­age for the ap­proval, but you can be sure that pay­ers will give it a good look over once the sales team hits the streets. On the down­side, sar­ilum­ab was linked to a sig­nif­i­cant­ly high­er rate of neu­trope­nia.

Leerink high­light­ed the price com­pe­ti­tion with a nod to the low­er price mod­el.

As im­por­tant­ly, Kevzara is al­so ap­prox­i­mate­ly 15% be­low the price of Actem­ra at the high­est dose and, with dis­counts, should of­fer a cost ef­fec­tive treat­ment op­tion for pay­ers in this set­ting. In a re­cent In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view (ICER) analy­sis, Kevzara did not reach a typ­i­cal $100,000 per qual­i­ty-ad­just­ed life year (QALY) cost ef­fec­tive­ness thresh­old at this lev­el, but nei­ther did any of the oth­er wide­ly pre­scribed bi­o­log­i­cals for this dis­ease, in­clud­ing En­brel, Hu­mi­ra, Cimzia, Oren­cia, Actem­ra and Xel­janz.

Sar­ilum­ab now be­comes the third an­ti­body to win ap­proval from a Re­gen­eron/Sanofi part­ner­ship. The first was the PC­SK9 drug Pralu­ent, which has trig­gered a bruis­ing le­gal bat­tle with Am­gen and has yet to find any re­al trac­tion in the mar­ket, de­spite its dra­mat­ic im­pact on cho­les­terol. The part­ners are now lin­ing up car­dio out­comes da­ta from a big Phase III, hop­ing to push that out front of the com­pe­ti­tion. Then there was an OK ear­li­er this year for Dupix­ent, which a num­ber of fore­casts have pegged as a po­ten­tial megablock­buster.

Rheuma­toid arthri­tis is a tough dis­ease to beat, es­pe­cial­ly when you have to line up against some big ri­vals that are al­ready well en­trenched in the field. J&J and Glax­o­SmithK­line demon­strat­ed that when they came up with mixed re­sults in their head-to-head with sirukum­ab vs. Hu­mi­ra. But it was Eli Lil­ly that turned in the big nasty sur­prise, with the FDA stun­ning an­a­lysts with their re­jec­tion of baric­i­tinib, a drug li­censed from In­cyte.

“In the clin­i­cal tri­al pro­gram, sar­ilum­ab demon­strat­ed sta­tis­ti­cal­ly sig­nif­i­cant, clin­i­cal­ly-mean­ing­ful im­prove­ments in adult pa­tients with rheuma­toid arthri­tis by re­duc­ing signs and symp­toms and im­prov­ing phys­i­cal func­tion, re­sult­ing in sig­nif­i­cant­ly less ra­di­ograph­ic pro­gres­sion of struc­tur­al dam­age of RA,” said Alan Kivitz, an in­ves­ti­ga­tor in the glob­al SAR­IL-RA clin­i­cal pro­gram for sar­ilum­ab. “This is im­por­tant be­cause not all cur­rent­ly avail­able treat­ments work in all pa­tients, and some pa­tients may spend years cy­cling through dif­fer­ent treat­ments with­out achiev­ing their treat­ment goals. Sar­ilum­ab works dif­fer­ent­ly from the most com­mon­ly used bi­o­log­ics, such as those in the an­ti-TNF class, and is a wel­come new op­tion for pa­tients and their physi­cians.”

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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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.

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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