As gener­ic en­try looms for its cash cow, Jazz wins FDA nod for fol­low-up sleep dis­or­der drug

Af­ter a 3-month de­lay at­trib­uted to la­belling dis­cus­sions, Jazz Phar­ma­ceu­ti­cals $JAZZ has notched an ap­proval for its wake-pro­mot­ing drug sol­ri­amfe­tol, now chris­tened Sunosi.

Like Xyrem — the block­buster nar­colep­sy drug that ac­counts for the bulk of Jazz’s sales — Sunosi is de­signed to treat ex­ces­sive day­time sleepi­ness for pa­tients with ob­struc­tive sleep ap­nea (OSA) as well as nar­colep­sy. In 2018, Xyrem sales reg­is­tered at $1.4 bil­lion, trans­lat­ing to over 70% of to­tal rev­enue. But with Sunosi in tow, to­tal prod­uct sales are ex­pect­ed to jump sub­stan­tial­ly.

Jazz bought Sunosi from Aer­i­al Phar­ma in 2014 af­ter the drug had passed muster in 2 Phase II tri­als for $125 mil­lion up­front. It’s the first dual-act­ing dopamine and nor­ep­i­neph­rine re­up­take in­hibitor to be ap­proved by the FDA, and al­though the com­pa­ny is un­clear as to how it works, the drug has im­proved pa­tients’ over­all clin­i­cal con­di­tion com­pared to a place­bo across four Phase III stud­ies.

Bruce Cozadd

“With this ap­proval, a new, day­time med­i­cine that can pro­vide sus­tained wake­ful­ness through­out the day will be avail­able for pa­tients,” CEO Bruce Cozadd said in a state­ment. “The FDA ap­proval of Sunosi al­so rep­re­sents an im­por­tant mile­stone for Jazz as we con­tin­ue to of­fer new treat­ment op­tions that ad­dress un­met needs for peo­ple liv­ing with chron­ic, and of­ten de­bil­i­tat­ing, sleep dis­or­ders.”

Sunosi is ap­proved with once-dai­ly dos­es of 75 mg and 150 mg for pa­tients with nar­colep­sy and dos­es of 37.5 mg, 75 mg, and 150 mg for pa­tients with OSA — but no­tably not 300 mg, Umer Raf­fat of Ever­core ISI wrote in a note.

[In] our opin­ion, not get­ting 300 mg ap­proved might be the sin­gle most im­por­tant thing that makes this la­bel as clean as it is. As you scroll to page 8 of (the) la­bel, there is a ta­ble on BP and HR da­ta on dos­es in­clud­ing 300 mg dose — and you can clear­ly see why FDA did NOT ap­prove that dose de­spite bet­ter ef­fi­ca­cy. In fact, la­bel men­tions “dosages above 150 mg do not con­fer in­creased ef­fec­tive­ness suf­fi­cient to out­weigh AE”.

By not hav­ing 300 mg, the BP and HR events mere­ly be­came a warn­ing, and NOT a black­box. And…al­though la­bel rec­om­mends as­sess­ing BP and HR be­fore ini­ti­at­ing and dur­ing treat­ment, (the) re­al­i­ty is that many nar­colep­sy and OSA pa­tients have mul­ti­ple CV risk fac­tors

Jazz is wait­ing for a DEA sched­ul­ing be­fore it can launch the drug, a process that’s es­ti­mat­ed to take 90 days. Mean­while, the com­pa­ny will be hir­ing 50 more sales reps for their 95-mem­ber sleep team, ac­cord­ing to Cowen an­a­lysts.

By mak­ing some cross-tri­al com­par­isons be­tween Sunosi and cur­rent stan­dard of care Provig­il/Nu­vig­il (which re­cent­ly went gener­ic), they gave the Jazz drug a ring­ing en­dorse­ment as a best-in-class agent.

“Im­por­tant­ly, our con­sul­tant checks and sur­vey re­sults in­di­cate that Sunosi ap­pears to be a need­ed treat­ment op­tion and a like­ly $500 mil­lion+ long du­ra­tion prod­uct,” they wrote. “The bot­tom line is that sol­ri­amfe­tol ap­pears to be the most ef­fec­tive wake-pro­mot­ing agent ever de­vel­oped for EDS.”

Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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UCB bags a ri­val to Soliris in $2.1B buy­out deal — but will an in­creas­ing­ly vig­i­lant FTC sign off?

UCB is buying out Ra Pharma $RARX, announcing an acquisition deal that rings up at $48 a share, or $2.1 billion net of cash, and puts them toe-to-toe with Alexion on a clinical showdown.

Ra shares closed at $22.70 on Wednesday.

There’s a small pipeline in play at Ra, but UCB is going for the lead drug — a C5 inhibitor called zilucoplan in Phase III for myasthenia gravis (MG) looking to play rival to Alexion’s Soliris. Soliris has the market advantage, though, with a much earlier approval in MG in late 2017 that UCB feels confident in challenging.

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A new play­er is tak­ing the field in a push for a he­mo­phil­ia A gene ther­a­py, and it’s a big one

BioMarin, the execs at Spark (and buyer-to-be Roche) as well as the Sangamo/Pfizer team have a new rival striding onto the hemophilia block. And it’s a big one.

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Stuck with a PhI­II gene ther­a­py fail­ure at 96 weeks, Gen­Sight prefers the up­beat as­sess­ment

Two years after treatment, the best thing that GenSight Biologics $SIGHT can say about their gene therapy for vision-destroying cases of Leber Hereditary Optic Neuropathy is that it’s just a bit better than a placebo — just maybe because one treatment can cover both eyes.

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George Scangos / Credit: Cornell University

ARCH, Soft­Bank-backed Vir Biotech­nol­o­gy un­der­whelms with $143 mil­lion IPO

George Scangos went back to Wall Street, and came back 700 million pennies short.

Scangos’ vaunted startup Vir Biotechnology raised $143 million in an IPO they hoped would earn $150 million. Shares were priced at $20, the low-end of the $20-$22 target.

Launched with backing from ARCH Venture’s Robert Nelsen, Masayoshi Son’s SoftBank Vision Fund, and the Bill & Melinda Gates Foundation, the infectious disease startup was one of a new wave of well-resourced biotechs that emerged with deep enough coffers to pursue a full R&D line rather than slowly build their case by picking off a single lead program.