Richard Gonzalez, AbbVie CEO (AP Images)

Ab­b­Vie chas­es its next gold­en goose in a post-Hu­mi­ra fu­ture with new ul­cer­a­tive col­i­tis da­ta for Rin­voq

Still bathing in the af­ter­glow of its big-mon­ey ac­qui­si­tion of Al­ler­gan, Ab­b­Vie faces the dark cloud of a post-Hu­mi­ra fu­ture — and the dead­line is ap­proach­ing fast. Key to Ab­b­Vie’s plan are two im­munol­o­gy launch­es with ag­gres­sive clin­i­cal dreams; one of those, JAK in­hibitor Rin­voq, is now one step clos­er to an­oth­er Hu­mi­ra-match­ing ap­proval.

Ab­b­Vie’s Rin­voq (upadac­i­tinib) best­ed place­bo in help­ing ul­cer­a­tive col­i­tis pa­tients achieve clin­i­cal re­mis­sion at the eight-week mark and sig­nif­i­cant­ly re­duced key symp­to­matic bio­mark­ers, in­clud­ing gas­troin­testi­nal in­flam­ma­tion, ac­cord­ing to da­ta from the Phase III U-Achieve study un­veiled Wednes­day.

Twen­ty-six per­cent of pa­tients dosed with a once-dai­ly, 45-mg dose of Rin­voq hit clin­i­cal re­mis­sion af­ter two months com­pared with just 5% on place­bo, Ab­b­Vie said. Rin­voq al­so in­duced gas­troin­testi­nal im­prove­ment in 36% of pa­tients; mean­while, just 7% pa­tients on place­bo hit that mark.

Rin­voq al­so aced its oth­er sec­ondary end­points over place­bo, in­clud­ing achiev­ing clin­i­cal re­sponse in pa­tients and re­duc­ing swelling of the mu­cos­al lin­ing. U-Achieve is one of two in­duc­tive stud­ies Ab­b­Vie has planned for Rin­voq in UC along with three piv­otal tri­als for a pos­si­ble reg­u­la­to­ry sub­mis­sion.

Rin­voq’s UC quest is part of a wide-rang­ing clin­i­cal pro­gram for the drug that in­cludes on­go­ing late-stage tri­als for un­ap­proved in­di­ca­tions in atopic der­mati­tis, pso­ri­at­ic arthri­tis, ax­i­al spondy­loarthri­tis, Crohn’s dis­ease, gi­ant cell ar­teri­tis and Takaya­su’s ar­teri­tis.

The end goal is sim­ple and dri­ven by a loom­ing axe in Ab­b­Vie’s port­fo­lio: Rin­voq must even­tu­al­ly snare enough in­di­ca­tions to make up for the sales loss of megablock­buster Hu­mi­ra, which is fac­ing US biosim­i­lar com­pe­ti­tion start­ing in 2023.

That time­line would give Rin­voq, which orig­i­nal­ly notched an FDA ap­proval to treat mod­er­ate-to-se­vere rheuma­toid arthri­tis back in Au­gust 2019, rough­ly three years to gain enough mo­men­tum to be­gin off­set­ting Hu­mi­ra’s ex­pect­ed de­cline. But it won’t be a sure-fire path to suc­cess: Rin­voq sports a JAK class warn­ing la­bel for se­ri­ous in­fec­tions, ma­lig­nan­cy and throm­bo­sis that has spooked many rheuma­tol­o­gists off the drugs and could make a ma­jor mar­ket en­try in­to any of those new in­di­ca­tions a dif­fi­cult road.

Luck­i­ly, Ab­b­Vie isn’t putting all its eggs in­to one bas­ket as it looks in­to the post-Hu­mi­ra fu­ture. The Chica­go drug­mak­er al­so sports re­cent pso­ri­a­sis launch Skyrizi, which it hopes to pair up with Rin­voq as a one-two punch to match Hu­mi­ra high­est sales peak. Ab­b­Vie is so gung-ho about the pair’s chances that CEO Rick Gon­za­lez posit­ed in Jan­u­ary at the JP Mor­gan Health­care Con­fer­ence that the two drugs could hit a com­bined $20 bil­lion in an­nu­al sales if they snag all of Hu­mi­ra’s ap­provals and add more — atopic der­mati­tis, for in­stance.

Mean­while, Ab­b­Vie is still in the process of di­gest­ing its $63 bil­lion ac­qui­si­tion of Al­ler­gan, which brought aes­thet­ic/ther­a­peu­tic block­buster Botox in­to the fold. Al­ler­gan came in sport­ing boom­ing an­tipsy­chot­ic Vray­lar as well, tack­ing a sec­ond growth dri­ver on­to the new com­pa­ny’s lapel.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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UP­DAT­ED: New Kaiser analy­sis shows how lim­it­ing price ne­go­ti­a­tions to tar­get­ed drugs may bet­ter fo­cus up­com­ing leg­is­la­tion

As Congress considers whether to adopt sweeping new legislation to lower prescription drug prices across the board, the Kaiser Family Foundation is out with a new report on Monday showing how a more targeted approach on a subset of drugs might be a more efficient way to save government funds.

“This analysis shows that Medicare Part D and Part B spending is highly concentrated among a relatively small share of covered drugs, mainly those without generic or biosimilar competitors,” wrote Juliette Cubanski, deputy director of the program on Medicare policy at KFF, and Tricia Neuman, SVP of KFF. “Focusing drug price negotiation or reference pricing on a subset of drugs that account for a disproportionate share of spending would be an efficient use of administrative resources, though it would also leave some potential savings on the table.”

When is a drug re­al­ly a de­vice? Court knocks down FDA ap­peal in try­ing to sort that grey area

It’s always a surprise when a court has to step in to tell the FDA that it erred in performing one of its main duties: classifying whether a medical product is drug or a device.

But that’s what the US Court of Appeals for the District of Columbia did on Friday, making clear to the world’s top drug regulator that Genus Medical Technologies’ contrast agent barium sulfate (also known as Vanilla SilQ) should not be considered a drug, as the FDA had said, but a medical device.

FDA lays the ham­mer on Emer­gen­t's Bal­ti­more plant af­ter J&J de­ba­cle, halt­ing all pro­duc­tion in un­usu­al move

Emergent BioSolutions has had a tough month: First, the CDMO ruined 15 million doses of J&J’s Covid-19 vaccine in March and then suffered the ignominy of the FDA seizing the reins. Now, as the agency receives a full accounting of the site’s problems, Emergent has slammed the brakes on all production at the FDA’s behest.

Emergent will cease manufacturing at its Baltimore plant until the FDA’s inspection and remediation of any findings is complete, the company said in a statement. Emergent will also quarantine existing materials that have already been manufactured. That stoppage started on Friday, four days after the initiation of the FDA’s inspection.

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Ovid cuts its loss­es on con­tro­ver­sial An­gel­man syn­drome drug, mak­ing its crush­ing de­vel­op­ment halt per­ma­nent

After a turbulent couple of years, Ovid is officially doing away with a program it had once championed in the face of heavy analyst skepticism.

Ovid has discontinued development of OV101, or gaboxadol, in Angelman syndrome and will not pursue further clinical trials for Fragile X syndrome, the company announced Monday morning. The news comes after Ovid had previously paused development in Angelman when the compound flunked a Phase III trial in December.

FDA slaps a hold on Mof­fitt’s next-gen CAR-T as reg­u­la­tors de­mand an­oth­er de­lay on clin­i­cal work — shares crater

Close to a year-and-a-half after tapping the brakes on one of its preclinical programs to do some added genetic engineering work on their next-gen CAR-T, Anixa $ANIX Therapeutics says the regulatory light is flashing red on their IND.

The San Jose, CA-based biotech — which changed its name from ITUS in 2018 — explained in late 2019 that they were taking a knee for at least a year so that researchers could go back and amp up the expression of follicle stimulating hormone on T cells to improve targeting of the FSH receptor on a specific set of ovarian cells. That required new vector engineering work by their partners at Moffitt Cancer Center.

Tillman Gerngross (Adagio)

Till­man Gern­gross' Covid-19 an­ti­body moon­shot scores $336M with the help of new ace CFO. Is an IPO next?

Less than a year into its existence, serial biotech entrepreneur Tillman Gerngross’ antibody play Adagio has raced ahead into a pivotal trial for its lead drug for Covid-19 on the back of some very promising preclinical data. Now, crossover investors led by Peter Kolchinsky at RA are rolling up the Brinks truck — and that could spell an IPO in the offing for Adagio.

Adagio has bagged $336 million as part of a Series C round led by RA Capital to advance lead single-shot antibody ADG20 through a pivotal Phase I/II/III trial for the treatment of mild to moderate Covid-19 patients at high risk of infection, the biotech said Monday.

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Q1: A flood of in­vestor cash drove biotech's num­bers to new record highs, and the tor­rent of cash is mov­ing up­stream fast

If you thought biotech was booming last year, wait until you get a load of the numbers from Q1 2021.

On virtually every level, with one exception, the money engine was working around the clock in the first 3 months of this year. Venture capital has reached such a fever peak that the average B round now weighs in at an average mega-weight value of $100 million. The money flow is also finding its way to the mouth of the R&D river, where discovery work now merits the big bucks instead of cautionary seed funds.

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