Jazz Phar­ma’s $1B bet pays off with ac­cel­er­at­ed lung can­cer ap­proval

Jazz Phar­ma­ceu­ti­cals’ $1 bil­lion bet last year looks to be pay­ing off so far: for them and their part­ner.

Sev­en months af­ter the Irish bio­phar­ma li­censed Phar­ma­Mar’s small cell lung can­cer drug lur­binecte­din, the FDA has grant­ed it ac­cel­er­at­ed ap­proval. The drug, which was sub­mit­ted two days be­fore Jazz signed on in De­cem­ber and lat­er grant­ed pri­or­i­ty re­view, will be mar­ket­ed as Zepzel­ca.

Zepzel­ca won’t en­ter the mar­ket un­til Ju­ly, at which point Jazz will face the dif­fi­cult hur­dles of com­mer­cial­iz­ing a drug in a pan­dem­ic, but the ap­proval should trig­ger an im­me­di­ate pay­off for Phar­ma­Mar. Al­though the ex­act terms aren’t spelled out, the orig­i­nal li­cens­ing agree­ment in­clud­ed “up to $250 mil­lion up­on the achieve­ment of ac­cel­er­at­ed and/or full reg­u­la­to­ry ap­proval of lur­binecte­din by FDA with­in cer­tain time­lines.” That deal al­so in­clud­ed $200 mil­lion up­front and $550 mil­lion in com­mer­cial mile­stones.

It’s a bit of re­demp­tion for the Span­ish bio­phar­ma. Orig­i­nal­ly found­ed in 1986 and lat­er re­spon­si­ble for the sar­co­ma drug Yon­delis, Phar­ma­Mar’s stock crashed in 2018 af­ter Zepzel­ca failed in a late-stage ovar­i­an can­cer study.

Still, that left a pair of tri­als on small cell lung can­cer. And at AS­CO in 2019, Phar­ma­Mar an­nounced the Phase II study had shown a 35.2% over­all re­sponse rate as a sec­ond-line ther­a­py — and 45% for the co­hort they de­ter­mined most sen­si­tive to treat­ment. Those pa­tients saw a pro­gres­sion-free sur­vival of 4.6 months and an over­all sur­vival of 11.9 months.

That may not trans­late in­to ac­cel­er­at­ed ap­proval for every in­di­ca­tion, but small cell lung can­cer has for years been a dif­fi­cult field for drug de­vel­op­ment. Be­fore 2018, when Bris­tol My­ers Squibb’s im­munother­a­py Op­di­vo was ap­proved for third-line pa­tients, no drug had been ap­proved for the tu­mor type in over a decade, ac­cord­ing to the Lung Can­cer Re­search Foun­da­tion. Since then, Keytru­da won ap­proval as a third-line treat­ment, and Tecen­triq and Imfinzi were cleared as a first-line ther­a­pies.

This is Jazz’s sec­ond ap­proval in a lit­tle over a year, af­ter their fol­low-on nar­colep­sy drug Sunosi was OK’ed last March. It is their third can­cer ap­proval. Ac­cel­er­at­ed ap­provals can be con­tin­gent on a con­fir­ma­to­ry tri­al, and a Phase III study is al­ready un­der­way.

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.

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.

Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.

Brian Stuglik, Verastem CEO

The du­velis­ib hot pota­to is tossed to a new own­er as Ve­rastem looks to re­or­ga­nize around the pipeline

When Infinity put up duvelisib for a no-money-down instant deal, the biotech was looking for a quick exit from a clinical disaster. AbbVie had walked away from their alliance after looking at how the data stacked up in a crowded field.

And while it was approvable, it wasn’t looking pretty to anyone who thought in commercial terms.

One Big Pharma’s trash, though, was seen as a biotech treasure as a deeply troubled Verastem stepped up to grab the PI3K-delta/gamma — promising to run it across the goal lines at the FDA. And they did just that, only with little to show for it.

Eric Shaff (Seres)

Af­ter a 4-year so­journ, a strug­gling mi­cro­bio­me pi­o­neer claims a break­out PhI­II come­back. And they're tak­ing it straight to the FDA

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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Gallia Levy, Spark CMO (Roche)

Spark Ther­a­peu­tics nabs new CMO from Genen­tech, fill­ing a ma­jor post-merg­er de­par­ture

Spark Therapeutics is getting a new CMO from their new owners.

The gene therapy company-turned-subsidiary has named Gallia Levy, who had been running rare blood disorders — including clinical development for their blockbuster-potential hemophilia antibody Hemlibra for Roche’s big biotech sub Genentech — to run medical affairs.

The appointment is a fitting one. Roche spent $4.8 billion to acquire Spark last year in large part to get their hands on their hemophilia gene therapy, SPK-8011, and expand the toe-hold Hemlibra gave them in an crowded hemophilia space.  It’s also a somewhat ironic appointment: The FTC held up the Spark acquistions for nearly a year, reportedly over concerns about the anti-trust implications of Roche owning both a top chronic treatment in Hemlibra and a top one-time treatment in Spark’s gene therapy.

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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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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Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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