Take­da inks up to $500M col­lab­o­ra­tion with ex­tra-cel­lu­lar ma­trix biotech for NASH

With Take­da’s mass, over-the-counter sell-off near­ing com­ple­tion, the Japan­ese drug­mak­er is beef­ing up the dis­cov­ery en­gine it wants to be the core part of the post-Shire com­pa­ny. On Tues­day, that meant their most re­cent for­ay in­to NASH — a dis­ease that they have tried with lit­tle ad­vanced suc­cess to de­vel­op drugs for in the past, but which re­mains a gold-mine for any com­pa­ny that can find an ef­fec­tive ther­a­py.

Take­da signed a col­lab­o­ra­tion to­day with Lon­don-based biotech En­gi­tix to de­vel­op drugs for ad­vanced fi­brot­ic liv­er dis­eases, in­clud­ing NASH. Up­front pay­ments weren’t dis­closed but En­gi­tix will be el­i­gi­ble for up to $500 mil­lion in mile­stones.

The deal emerges out of En­gi­tix’s on­go­ing work with Take­da. Take­da spon­sored the 4-year-old start­up with a Lab­Cen­tral “Gold­en Tick­et” last year, giv­ing them ac­cess to lab space, lab per­son­nel, and de­vel­op­ment pro­grams in Cam­bridge. The idea now is to com­bine En­gi­tix’s ex­tra-cel­lu­lar ma­trix plat­form with Take­da’s ex­pe­ri­ence in gas­troen­terol­o­gy.

En­gi­tix is based on the idea that com­pa­nies of­ten fail to trans­late their pre­clin­i­cal work be­cause they use cell lines that don’t take in­to ac­count that ma­trix — the net­works of pro­teins and car­bo­hy­drates that pass be­tween cells, com­mu­ni­cat­ing and as­sur­ing the whole sys­tem func­tions. So far, they’ve built ECM mod­els by tak­ing re­sect­ed tis­sues, break­ing them down “de­cel­lu­lar­iz­ing” them — lit­er­al­ly re­mov­ing the cells — and then lat­er grow­ing cell lines in what’s left­over.

Al­though the com­pa­ny is al­so fo­cused on sol­id tu­mors, the first pub­li­ca­tion on their plat­form, in Sci­en­tif­ic Re­ports, was a pa­per on the first suc­cess­ful de­cel­lu­lar­iza­tion and re­pop­u­la­tion of a hu­man liv­er ex­tra-cel­lu­lar ma­trix.  The com­pa­ny’s lead in­di­ca­tions are in pri­ma­ry scle­ros­ing cholan­gi­tis and NASH, al­though both re­main in the dis­cov­ery phase.

The deal hard­ly marks Take­da’s first ear­ly-stage biotech tie-in for NASH. In 2016, they teamed up for an undis­closed sum with the RNA biotech Arc­turus on NASH and GI dis­cov­ery, and ten months lat­er, they signed an up to $470 mil­lion deal with He­mo­S­hear to use their dis­ease mod­el­ing plat­form for liv­er tar­gets.

So far, those have yet to yield any clin­i­cal can­di­dates or named pre-clin­i­cal can­di­dates, ac­cord­ing to the com­pa­ny’s of­fi­cial pipeline and the data­base on clin­i­cal­tri­als.gov. Still, Take­da opt­ed to ex­pand both part­ner­ships in 2018 and 2019, adding in last year’s He­mo­S­hear re­lease that their ef­forts have yield­ed sev­er­al pre­clin­i­cal can­di­dates.

With the ma­jor clin­i­cal NASH can­di­dates tak­ing hit af­ter hit, the Japan­ese drug­mak­er may have plen­ty of time to de­vel­op those. Lead­er­ship has been seek­ing to move to­ward in­no­va­tion and high-prof­it prod­ucts. The first drug for a dis­ease es­ti­mat­ed to af­fect mil­lions of Amer­i­cans would cer­tain­ly fit the bill.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.