GSK's asth­ma drug Nu­cala gets speedy re­view for rare blood dis­or­der

Glax­o­SmithK­line’s asth­ma drug Nu­cala may not be on the same tier as ri­val Re­gen­eron’s Dupix­ent, but it has scored a speedy re­view for use in a rare blood dis­or­der.

First ap­proved in 2015 for se­vere eosinophilic asth­ma, Nu­cala (known chem­i­cal­ly as mepolizum­ab) tar­gets IL-5. The British drug­mak­er on Wednes­day dis­closed that the mon­o­clon­al an­ti­body se­cured pri­or­i­ty re­view from the FDA for pa­tients with hy­per­e­osinophilic syn­drome (HES) — a group of life-threat­en­ing in­flam­ma­to­ry dis­or­ders char­ac­ter­ized by a per­sis­tent over­pro­duc­tion of a type of white blood cell called eosinophils.

The US drug reg­u­la­tor has laid out the red car­pet for the use of the drug in HES, al­so hav­ing grant­ed fast track and or­phan drug des­ig­na­tions for the in­di­ca­tion. Last year, how­ev­er, the agency had a de­cid­ed­ly con­trast­ing view on Nu­cala’s use in pa­tients with COPD, af­ter it failed one late-stage test but cleared an­oth­er. Af­ter an ex­pert pan­el vot­ed 16 to 3 against ap­proval, the FDA spurned the ap­pli­ca­tion, as wide­ly an­tic­i­pat­ed.

For HES, Nu­cala was test­ed in a 108-pa­tient late-stage study. The drug in­duced a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 56% in HES flares, char­ac­ter­ized by a wors­en­ing of symp­toms or ex­ag­ger­at­ed eosinophil lev­els re­quir­ing an es­ca­la­tion in ther­a­py over the 32-week pe­ri­od, ver­sus 28% in the place­bo group — meet­ing the main goal of the study. It al­so met a host of sec­ondary tri­al end­points, such as the risk of first HES flare over the study pe­ri­od, the an­nu­al­ized rate of HES flares and fa­tigue scores.

Last month, in a late-stage tri­al the bi­o­log­ic gen­er­at­ed a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in chron­ic rhi­nos­i­nusi­tis pa­tients with nasal polyps, an­oth­er in­di­ca­tion Dupix­ent is ap­proved for.

Nu­cala record­ed £210 mil­lion ($258 mil­lion) in first-quar­ter sales, ben­e­fit­ing from at-home use ap­pli­ca­tion launch­es world­wide as Covid-19 di­min­ished the abil­i­ty of pa­tients to vis­it health­care fa­cil­i­ties. Dupix­ent gen­er­at­ed $855 mil­lion over the same pe­ri­od.

“Nu­cala is not a ma­jor com­pet­i­tive threat to Re­gen­eron’s Dupix­ent—the like­ly pre­ferred asth­ma drug near term giv­en physi­cians’ pref­er­ence for Dupix­ent’s safe­ty and ef­fi­ca­cy. In our pri­or sur­vey work, physi­cians in­di­cat­ed that they ex­pect their us­age of Nu­cala to de­cline,” Cred­it Su­isse an­a­lyst Evan Seiger­man wrote in a note last month.

Nu­cala is be­lieved to work by pre­vent­ing IL-5 from bind­ing to its re­cep­tor on the sur­face of eosinophils di­min­ish­ing blood eosinophils, but not erad­i­cat­ing them al­to­geth­er. Re­gen­eron and Sanofi flag­ship Dupix­ent tar­gets IL-4 and IL-13.

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

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.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Sanofi and GSK reach deal with Cana­da for 72 mil­lion vac­cine dos­es

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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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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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.

J&J re­leas­es PhI­II safe­ty blue­print for Covid-19 vac­cine tri­al. How does it stack up to Mod­er­na, Pfiz­er and As­traZeneca?

Along with the initiation of its Phase III Covid-19 vaccine study announced Wednesday morning, Johnson & Johnson has also released its trial protocol, giving an inside look at how the company is conducting its late-stage research.

The move comes after the other three companies conducting Phase III’s in the US — Moderna, Pfizer and AstraZeneca — each disclosed their own trial blueprints within the last week. Though the release of such protocols is typically done after trials have been completed, drug developers had come under intensifying pressure to do so given the approaching November presidential election.

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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Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.