Jazz Phar­ma­ceu­ti­cals gets FDA OK for cat­a­plexy drug as it pre­pares to re­place old cash cow

Fol­low­ing an FDA ap­proval just last month for its small cell lung can­cer drug, Jazz Phar­ma­ceu­ti­cals keeps on rolling.

The Irish phar­ma an­nounced Wednes­day that Xy­wav, treat­ing cat­a­plexy and ex­ces­sive day­time sleepi­ness as­so­ci­at­ed with nar­colep­sy in pa­tients old­er than sev­en years, has been giv­en the green light by US reg­u­la­tors. This is the third FDA ap­proval grant­ed to Jazz in the last 16 months and the sec­ond nar­colep­sy-re­lat­ed drug af­ter Sunosi was OK’ed in March 2019.

Jazz plans to launch the new drug by the end of 2020 af­ter Risk Eval­u­a­tion and Mit­i­ga­tion Strat­e­gy im­ple­men­ta­tion.

Cat­a­plexy is the sud­den loss of mus­cle tone nor­mal­ly trig­gered by strong emo­tions, and it is com­mon­ly ex­pe­ri­enced in those who suf­fer from nar­colep­sy. Jazz has made these dis­or­ders cen­tral to their port­fo­lio. In ad­di­tion to Sunosi, the phar­ma al­ready has a block­buster nar­colep­sy drug on the mar­ket in Xyrem, which ac­count­ed for $1.4 bil­lion in 2018 sales or 70 per­cent of the com­pa­ny’s to­tal rev­enue.

Al­though Xyrem has been a boon, con­cerns arose due to the drug’s high sodi­um con­tent as a sodi­um oxy­bate. Xy­wav, which like Xyrem is an oxy­bate-based drug, con­tains 92 per­cent less sodi­um than its pre­de­ces­sor, and Jazz hopes the new treat­ment will re­place Xyrem as the stan­dard of care.

Richard Bo­gan

“Xy­wav is still an oxy­bate mol­e­cule, so it works in the same mech­a­nism of ac­tion of sodi­um oxy­bate,” Richard Bo­gan, lead in­ves­ti­ga­tor of the Phase III tri­al, told End­points News. “We ex­pect it to be as equal­ly ef­fec­tive [as Xyrem] and that’s what the clin­i­cal tri­als demon­strat­ed, that it was ef­fi­ca­cious.”

Jazz notes that Xy­wav and Xyrem have the same oxy­bate con­cen­tra­tion. But Xy­wav in­cludes a mix of cal­ci­um, mag­ne­sium, potas­si­um and sodi­um cations, Bo­gan said, and most pa­tients were able to tran­si­tion from one med­i­cine to the oth­er with min­i­mal side ef­fects.

“We were able to use what we call asym­met­ric dos­ing so we could ad­just the first and the sec­ond dose, which is kind of a new method­ol­o­gy that’s now ac­cept­able to the FDA that we had used clin­i­cal­ly in the past,” Bo­gan said. “The im­por­tant mes­sage is that those pa­tients who were on Xyrem, we just con­vert­ed them the next day to the same dose of Xy­wav, and then we have a pe­ri­od of time where we could tweak the dose if we want­ed to.”

Again like Xyrem, Xy­wav al­so comes with a black box warn­ing as a cen­tral ner­vous sys­tem de­pres­sant and for po­ten­tial abuse and mis­use. As such, it’s on­ly avail­able through a re­strict­ed REMS pro­gram. Giv­en that the FDA had al­ready ac­cu­mu­lat­ed lots of ap­plic­a­ble da­ta from pre­vi­ous Xyrem test­ing, and due to the sim­i­lar­i­ties of the drugs, Jazz and Bo­gan are con­fi­dent in Xy­wav’s safe­ty and ef­fi­ca­cy pro­files.

The Xy­wav com­pound, dubbed JZP-258, is al­so in the Phase III clin­i­cal tri­al stage for the treat­ment of id­io­path­ic hy­per­som­nia, or ex­ces­sive sleepi­ness. This dif­fers from nar­colep­sy in that pa­tients with IH do not ex­pe­ri­ence the sud­den on­set of REM sleep cy­cles more than once per sleep pe­ri­od.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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