Jazz Phar­ma­ceu­ti­cals push­es to ex­pand la­bel on Xy­wav; FDA eyes Oc­to­ber for de­ci­sion on Seagen's lat­est ADC

Eight and a half months since its last OK for Xy­wav, Jazz Phar­ma­ceu­ti­cals has won pri­or­i­ty re­view for a new in­di­ca­tion with the drug.

The FDA grant­ed the ex­pe­dit­ed in­spec­tion Mon­day morn­ing, Jazz an­nounced, as the biotech looks to ex­pand Xy­wav in­to adult id­io­path­ic hy­per­som­nia pa­tients. Reg­u­la­tors have set a PDU­FA date of Aug. 12.

Back in Ju­ly 2020, Xy­wav notched its first ap­proval to treat cat­a­plexy and ex­ces­sive day­time sleepi­ness as­so­ci­at­ed with nar­colep­sy in pa­tients old­er than 7. At the time, the drug joined Sunosi and Xyrem in the com­pa­ny’s sleep­ing dis­or­der-re­lat­ed port­fo­lio.

Jazz’s new ap­pli­ca­tion, which will be of­fi­cial­ly sub­mit­ted to the FDA on Tues­day, is based on a Phase III tri­al that wrapped up in De­cem­ber. The study, look­ing at adults with id­io­path­ic hy­per­som­nia, met its pri­ma­ry end­point of im­prove­ments in the Ep­worth Sleepi­ness Scale (ESS) at a p-val­ue of p<0.0001.

Al­though Xyrem has been a block­buster for the com­pa­ny, con­cerns arose due to the drug’s high sodi­um con­tent as a sodi­um oxy­bate. Xy­wav is sim­i­lar­ly an oxy­bate-based drug, but con­tains 92 per­cent less sodi­um than its pre­de­ces­sor. — Max Gel­man

FDA eyes Oc­to­ber for de­ci­sion on Seagen’s lat­est ADC

About a year af­ter get­ting a nod in ad­vanced breast can­cer, Seagen could be on its way to its fourth drug ap­proval, this time in cer­vi­cal can­cer.

The FDA hand­ed Seagen and Gen­mab an Oct. 10 PDU­FA date for their an­ti­body-drug con­ju­gate (ADC) ti­so­tum­ab ve­dotin, the com­pa­nies said on Fri­day. They filed for a BLA back in Feb­ru­ary to treat re­cur­rent or metasta­t­ic cer­vi­cal can­cer pa­tients whose dis­ease pro­gressed on or af­ter chemother­a­py.

Clay Sie­gall

“It’s a very good step and an­oth­er op­tion for pa­tients in a dis­ease with a grim prog­no­sis,” CEO Clay Sie­gall told End­points News back in Sep­tem­ber.

The sub­mis­sion was based on Phase II da­ta read out at vir­tu­al ES­MO 2020, which showed a 24% ob­jec­tive re­sponse rate last­ing a me­di­an of 8.3 months. It was a sin­gle-arm tri­al, but Seagen com­pared the re­sults to da­ta sug­gest­ing ex­ist­ing ther­a­pies “typ­i­cal­ly” have a re­sponse rate of less than 15% and pa­tients sur­vive for 6 to 9.4 months. By con­trast, me­di­an sur­vival in the study was 12.1 months, with 79% of pa­tients alive af­ter 6.

Seagen’s last two ap­provals came in the span of four months. In De­cem­ber 2019, the FDA OK’d its blad­der drug Pad­cev, which was de­vel­oped with Japan-based Astel­las. Then, the fol­low­ing April, it ap­proved Tukysa, an oral ki­nase in­hibitor.

Ti­so­tum­ab ve­dotin came out of a pact Seagen ini­tial­ly inked with Gen­mab back about a decade ago. Seagen — then called Seat­tle Ge­net­ics — agreed to give Gen­mab rights to its ADC tech back in 2010. A year lat­er, they struck a deal giv­ing Seagen an opt-in on ti­so­tum­ab ve­dotin. The ear­ly da­ta were good enough to per­suade Seagen to share the de­vel­op­ment costs and any prof­its 50/50. — Nicole De­Feud­is 

Tre­ovir has sights set on PhII for pe­di­atric glioblas­toma can­di­date

Tre­ovir is hur­ry­ing its glioblas­toma can­di­date in­to Phase II af­ter a Phase I tri­al showed the treat­ment more than dou­bled the typ­i­cal sur­vival rate in chil­dren with high-grade glioma, the com­pa­ny said on Mon­day.

“It is our goal that the Phase 2 study will sup­port mar­ket ap­proval of G207 to treat pe­di­atric re­cur­rent high-grade gliomas and our com­pa­ny’s mis­sion is clear: We want to com­mer­cial­ize G207 and pro­vide hope to the chil­dren and fam­i­lies who cur­rent­ly have no ef­fec­tive ther­a­peu­tic op­tions,” CEO Michael Chris­ti­ni said in a state­ment.

The Phase I dose-es­ca­la­tion study en­rolled 12 pa­tients be­tween the ages of 7 and 18 with pro­gres­sive high-grade glioma. Par­tic­i­pants got the on­colyt­ic HSV im­munother­a­py, dubbed G207, ei­ther alone or in com­bi­na­tion with ra­di­a­tion.

No dose-lim­it­ing tox­ic ef­fects or se­ri­ous ad­verse events were not­ed, Tre­ovir said, though 20 Grade 1 ad­verse events were “pos­si­bly re­lat­ed” to the can­di­date. Re­searchers not­ed ra­di­ograph­ic, neu­ropatho­log­i­cal or clin­i­cal re­spons­es in 11 of the 12 pa­tients, and me­di­an over­all sur­vival was 12.2 months. About 36% of the pa­tients sur­vived longer than 18 months, ac­cord­ing to the biotech.

Tre­ovir plans to start a Phase II tri­al lat­er this year. G207 is al­so be­ing stud­ied at the Uni­ver­si­ty of Al­aba­ma-Birm­ing­ham and Chil­dren’s of Al­aba­ma in re­cur­rent pe­di­atric brain tu­mors, in­clud­ing medul­loblas­toma. — Nicole De­Feud­is 

Mirum and Vivet col­lab­o­rate on liv­er dis­ease gene ther­a­py pro­grams

Mirum has picked up ex­clu­sive li­cense op­tions on a cou­ple of pre­clin­i­cal gene ther­a­py pro­grams.

The Fos­ter City, CA-based biotech is team­ing up with French out­fit Vivet Ther­a­peu­tics to po­ten­tial­ly de­vel­op and com­mer­cial­ize gene ther­a­py pro­grams for pro­gres­sive fa­mil­ial in­tra­hep­at­ic cholesta­sis, sub­types 3 and 2, the pair an­nounced Mon­day. No fi­nan­cial terms were dis­closed.

Chris Peetz

Vivet will re­main re­spon­si­ble for the pre­clin­i­cal stud­ies for the two pro­grams, known as VTX-803 and VTX-802. Once those have been com­plet­ed, Mirum can ex­er­cise op­tions to lead clin­i­cal tri­als and com­mer­cial­iza­tion. Mirum is still pro­vid­ing fund­ing for the pro­grams un­til that time.

The big idea be­hind the gene ther­a­pies is to cor­rect the de­fec­tive MDR3 trans­porter and bile salt ex­port pump (BSEP) func­tions for PF­IC3 and PF­IC2, re­spec­tive­ly. That, in turn, may ul­ti­mate­ly pro­vide a cure for pa­tients liv­ing with the rare liv­er dis­eases.

“We are en­cour­aged by the ex­per­tise and ded­i­ca­tion of the Vivet team to de­vel­op next-gen­er­a­tion gene ther­a­pies that have the po­ten­tial to trans­form the lives of pa­tients and their fam­i­lies,” Mirum CEO Chris Peetz said in a state­ment. “There are clear syn­er­gies in our com­bined mis­sions to help the peo­ple who need it the most.” — Max Gel­man

VBI Vac­cines re­ports ear­ly da­ta from Brii-part­nered Hep B vac­cine

He­pati­tis B vac­cine mak­er VBI Vac­cines has new da­ta out for their chron­ic HBV shot, and though the sam­ple size is small, the biotech is hop­ing they’re strong enough to keep push­ing for­ward.

In a Phase Ib/IIa tri­al from 33 evalu­able pa­tients, VBI ob­served a res­tim­u­la­tion of T cell ac­tiv­i­ty in re­act­ing to he­pati­tis B anti­gens rang­ing from 50% to 78% across all co­horts, com­pared to no de­tectable re­sponse in the con­trol group. The fig­ures in­clude pa­tients tak­ing 20 µg or 40 µg dos­es, with or with­out an ad­ju­vant. P-val­ues were not re­port­ed.

T cell and an­ti­body re­spons­es were al­so con­sis­tent in both dos­ing groups, VBI added. VBI is de­vel­op­ing the can­di­date with Brii Bio­sciences. — Max Gel­man

ICER launch­es pi­lot pro­gram to up­date past as­sess­ments us­ing re­al-world da­ta

ICER is re­vis­it­ing its 2018 as­sess­ments of three pro­phy­lac­tic treat­ments for hered­i­tary an­gioede­ma (HAE) us­ing re­al-world ev­i­dence col­lect­ed through a part­ner­ship with health care tech com­pa­ny Ae­tion.

The in­sti­tute said on Mon­day that up­dates are com­ing in Ju­ly for its as­sess­ments of Take­da’s Takhzy­ro and Cin­ryze, as well as CSL Behring’s Hae­gar­da. Back in 2018, ICER said all three treat­ments ex­ceed­ed the thresh­old for cost-ef­fec­tive­ness ($50,000 to $150,000 per qual­i­ty-ad­just­ed life year gained).

“As ICER not­ed through­out our ear­li­er as­sess­ment of these HAE ther­a­pies, our eco­nom­ic mod­els were very sen­si­tive to small changes in as­sump­tions about the fre­quen­cy of at­tacks, the amount of on-de­mand treat­ment re­quired, and the ex­act dos­ing reg­i­mens of pro­phy­lac­tic ther­a­py,” Jon Camp­bell, ICER’s se­nior VP of health eco­nom­ics, said in a state­ment. Now, he said, they’re try­ing to use re­al-world da­ta to close some of these “in­for­ma­tion gaps.”

“By defin­ing the base­line de­mo­graph­ics and uti­liza­tion char­ac­ter­is­tics of pa­tients who start HAE pro­phy­lax­is, eval­u­at­ing HAE at­tack rates be­fore ini­ti­a­tion of pro­phy­lax­is, and up­dat­ing our health cost as­sump­tions ac­cord­ing­ly, ICER aims to fur­ther clar­i­fy the over­all val­ue of these HAE ther­a­pies in re­al-world set­tings,” Camp­bell said. — Nicole De­Feud­is 

Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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Mer­ck­'s wom­en's health spin­out snags mid-stage can­di­date for preterm birth; Keytru­da nails down TNBC ap­proval af­ter March CRL

Nearly two months after spinning out from Merck, women’s health business Organon has struck its first half-billion-dollar deal.

Organon $OGN has promised $25 million upfront and another $475 million in biobucks for worldwide rights to ebopiprant, ObsEva’s investigational treatment for preterm labor. Ebopiprant, a selective prostaglandin F2α (PGF2α) receptor antagonist, was originally licensed from Merck KGaA in 2015. The candidate works by reducing inflammation and uterine contractions.

Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; 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.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Frank Pallone (Kevin Dietsch/Pool via AP Images)

House com­mit­tee seeks more from FDA on in­spec­tion back­log, when to restart work on for­eign sites

House Energy & Commerce committee leaders are raising fresh questions about the FDA’s ability to conduct foreign manufacturing site inspections and bring down its growing backlog.

“While we understand that the emergence of COVID-19 required the agency to suspend in-person inspection activities temporarily, we remain concerned that more than one year into the pandemic, the strategy for resuming all inspections and addressing the backlog of delayed inspections remains unclear,” E&C chair Frank Pallone (D-NJ) and a group of five other bipartisan leaders of the committee wrote to FDA acting commissioner Janet Woodcock.