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 

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Sanofi, Cel­lec­tis team up to pair Lem­tra­da with al­lo­gene­ic CAR-Ts; Bris­tol My­ers joins Evotec's dri­ve to ad­vance UK sci­ence

Sanofi is partnering up with Cellectis on their allogeneic CAR-Ts.

Cellectis struck a deal to use the French pharma’s drug alemtuzumab (Lemtrada, an MS agent) as part of its lymphodepletion regimen — used to sideline host immune cells and improve CAR-T cell expansion and persistence.

The Sanofi drug is already used in some clinical trials, and now the pharma giant is on the hook for clinical studies as they engage in negotiations over a commercial pact. — John Carroll

Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

Distribution of Moderna's Covid-19 Vaccine (Photo by Paul Sancya - Pool/Getty Images)

Opin­ion: Ado­les­cents can wait. The US needs to start do­nat­ing Covid-19 vac­cines to needy coun­tries now.

Now that the US is swimming in Covid-19 vaccines and the supply has officially eclipsed the demand, it’s time for America to lead the world and start shipping these excess doses to countries that desperately need them.

Unlike the IP waiver at the World Trade Organization, which Biden now supports and will likely take years to translate into actual shots in arms, the US could easily donate just a tiny fraction of the more than 60 million doses of Pfizer, Moderna and J&J vaccines sitting on American shelves right now.