News brief­ing: Re­gen­eron/Sanofi post pos­i­tive PhI­II Dupix­ent da­ta in pe­di­atric asth­ma; Vineti scores $33M Se­ries C ex­ten­sion

Re­gen­eron and Sanofi re­vealed pos­i­tive Phase III da­ta for their block­buster Dupix­ent on Tues­day, paving a po­ten­tial path for­ward in pe­di­atric asth­ma.

Dupix­ent, eval­u­at­ed in chil­dren be­tween ages 6 and 11, met its pri­ma­ry end­point of low­er­ing the rate of se­vere asth­ma at­tacks in two pa­tient pop­u­la­tions by 65% and 59% per year com­pared to place­bo. The drug al­so showed im­proved lung func­tion by 10.15 and 10.53 per­cent­age points over base­line, com­pared to 4.83 and 5.32 per­cent­age points for place­bo.

The tri­al en­rolled 408 chil­dren with mod­er­ate-to-se­vere asth­ma who did not see im­prove­ments up­on the stan­dard of care, in this case in­haled cor­ti­cos­teroids. Dur­ing the 52-week treat­ment pe­ri­od, pa­tients re­ceived in­jec­tions of Dupix­ent or place­bo every two weeks, ei­ther at the 100 mg or 200 mg dosage lev­el.

Dupix­ent is al­ready ap­proved to treat asth­ma in pa­tients old­er than 12 as well as a few oth­er in­di­ca­tions — atopic der­mati­tis and chron­ic rhi­nos­i­nusi­tis with nasal polyps.

With Tues­day’s re­sults, Re­gen­eron and Sanofi plan to file for an ap­proval in the pe­di­atric asth­ma field some­time in the first quar­ter of 2021. Dupix­ent is the duo’s biggest sell­er, net­ting more than $1.9 bil­lion in glob­al sales through the first half of 2020. — Max Gel­man

Vineti adds $33M to Se­ries C and Eli Cas­din to its board

Vineti has some new cash — and a new high-pro­file board mem­ber.

Amy DuRoss

The San Fran­cis­co-based com­pa­ny an­nounced a $33 mil­lion ex­ten­sion from its Feb­ru­ary Se­ries C round and that Eli Cas­din, founder of Cas­din Cap­i­tal, has joined its board of di­rec­tors. Vineti plans to use the funds to help boost de­vel­op­ment for its per­son­al­ized late-stage can­cer treat­ments, in­clud­ing CAR-T ther­a­py.

Tues­day’s ex­ten­sion was led by Car­di­nal Health with par­tic­i­pa­tion from Marc Be­nioff and ex­ist­ing Vineti in­vestors, in­clud­ing Canaan, Thresh­old Ven­tures, Sec­tion 32, Cas­din Cap­i­tal, No­var­tis Phar­ma AG, McKesson Ven­tures, and Life­Force Cap­i­tal. Over­all, Vineti has raised more than $115 mil­lion to­tal.

“2020 has dri­ven one key point home — the con­tin­ued suc­cess of ad­vanced ther­a­pies re­lies on stan­dard­ized, de­pend­able in­fra­struc­ture,” Vineti CEO Amy DuRoss said in a state­ment. “Vineti was built for [the] pur­pose to serve per­son­al­ized med­i­cine.” — Max Gel­man

Abi­vax grabs a €15M life­line lead­ing to ul­cer­a­tive col­i­tis da­ta

French biotech Abi­vax has ob­tained a $17.6 mil­lion (€15 mil­lion) loan from Kre­os Cap­i­tal to keep it afloat un­til a key read­out in the sec­ond quar­ter of 2021.

Hart­mut Ehrlich

“We con­tin­ue to fo­cus on the ABX464 Phase 2b ul­cer­a­tive col­i­tis tri­al, with top-line re­sults ex­pect­ed in Q2 2021, while the ABX464 Phase 2a study in rheuma­toid arthri­tis is al­so pro­gress­ing well,” CEO Hart­mut Ehrlich said in a state­ment. “Fur­ther­more, the fi­nanc­ing will be used to ad­vance the projects that are cru­cial for the fu­ture de­vel­op­ment of the Com­pa­ny, such as the prepa­ra­tion of the clin­i­cal Phase 3 in UC and the ini­ti­a­tion of a piv­otal Phase 2b/3 study in Crohn’s dis­ease.”

A small mol­e­cule de­signed to mod­u­late RNA splic­ing, ABX464 is al­so be­ing test­ed in a Phase IIb/Phase III tri­al for Covid-19.

Kre­os’ debt fund­ing is di­vid­ed in­to two tranch­es. The first €10 mil­lion is to be ful­ly drawn im­me­di­ate­ly, while the oth­er €5 mil­lion will be col­lect­ed be­fore No­vem­ber 1. Abi­vax, which is cur­rent­ly fo­cused on chron­ic in­flam­ma­to­ry dis­eases, said it will be look­ing for oth­er fi­nanc­ing op­tions. — Am­ber Tong

Twist and Neo­gene to work to­geth­er on CAR-T and TCR can­di­dates 

Twist Bio­science and Neo­gene Ther­a­peu­tics are join­ing forces to work on CAR-T and TCR ther­a­pies for can­cer pa­tients.

Emi­ly Lep­roust

Un­der the strate­gic part­ner­ship, Twist will cre­ate a TCR li­brary a self-pro­claimed “li­brary of li­braries” to aid Neo­gene’s search for en­gi­neered TCRs against tar­gets in can­cer. Twist will al­so use the li­brary to dis­cov­er an­ti­bod­ies for fu­ture Neo­gene CAR-T can­di­dates.

The com­pa­nies kept the fi­nan­cial terms of the deal un­der wraps, but said that Twist will get tech­nol­o­gy ac­cess fees, and mile­stones and roy­al­ties for any an­ti­bod­ies or TCRs that come from the part­ner­ship. Neo­gene gets ex­clu­sive rights to the syn­thet­ic TCR li­brary for tar­get­ing vi­ral and neo-anti­gens in on­col­o­gy.

“Putting our plat­forms to­geth­er, we be­lieve we will be able to ex­pe­dite the iden­ti­fi­ca­tion and ge­net­ic en­gi­neer­ing of TCR genes to cre­ate per­son­al­ized T cell ther­a­pies for can­cer, bring­ing new hope to ad­dress the cur­rent lim­i­ta­tions of treat­ments avail­able to­day,” Twist CEO and co-founder Emi­ly Lep­roust said in a state­ment. — Nicole De­Feud­is 

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Thomas Gad, Y-mAbs Therapeutics founder and interim CEO

FDA re­jects Y-mAbs’ neu­rob­las­toma drug af­ter tak­ing is­sue with clin­i­cal tri­al de­sign

Uncertainty about clinical trial evidence has led the FDA to hand down a complete response letter for Y-mAbs’ neuroblastoma drug, casting a cloud on the future of a candidate that had gone through a long development journey in a rare pediatric cancer.

Y-mAbs said it’s disappointed “but not surprised” given that the agency’s oncology drug advisory committee had voted 16-0 against its drug’s approval a few weeks ago.

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Philip Tagari switch­es Am­gen's dis­cov­ery lab for in­sitro's ma­chine learn­ing tools; CEO Joaquin Du­a­to to chair J&J's board

In February, Philip Tagari will take a few days of retirement and then immediately return to industry. He won’t be leading the therapeutics discovery unit for a large biopharma, though.

He’ll trade in his Amgen hat for chief scientist at a machine learning startup that has reeled in hundreds of millions in capital to lay the groundwork for a much-hyped new model of drug discovery that aims to speed up the time to new clinical assets.

Raul Rodriguez, Rigel Pharma CEO

Rigel Phar­ma scores FDA ap­proval for leukemia, kick­ing off show­down with Servi­er in IDH1

When Rigel Pharma bought olutasidenib from Forma Therapeutics, it acquired a drug that already secured a PDUFA date at the FDA — for February 2023. But regulators are ready to give their OK sooner than that.

The FDA has approved the IDH1 inhibitor as a treatment for adult patients with relapsed or refractory acute myeloid leukemia who have a susceptible IDH-1 (isocitrate dehydrogenase-1) mutation as detected by an FDA-greenlit test. Rigel will market it as Rezlidhia.

Tim Pearson, Carrick Therapeutics CEO

Pfiz­er backs $60M in­fu­sion in­to Car­rick, teams up on breast can­cer treat­ment

In a big week for Carrick Therapeutics, the company announced $60 million in funding for its lead breast cancer drug and development of a second program, as well as a collaboration with Pfizer for combo development.

The $35 million from Pfizer comes with an agreement under which Pfizer will support Carrick’s Phase II study of samuraciclib in combination with Pfizer’s Faslodex for advanced breast cancer. Along with the investment, Adam Schayowitz, vice president and development head of breast cancer, colorectal cancer and melanoma at Pfizer global product development, will join Carrick’s scientific advisory board.

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with Rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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