As­traZeneca, Am­gen whip up some block­buster ex­cite­ment with a pos­si­ble game-chang­ing asth­ma drug

As­traZeneca and Am­gen rolled out Phase IIb da­ta on their late-stage drug teze­pelum­ab late on Wednes­day, spot­light­ing some dra­mat­ic re­sults for a ther­a­py they be­lieve could have megablock­buster po­ten­tial for the broad-based treat­ment of un­con­trolled asth­ma.

Bot­tom line: Treat­ment-re­sis­tant pa­tients in the drug arm tak­ing a med­ley of dos­es saw their an­nu­al ex­ac­er­ba­tion rate slashed any­where from 61% to 71%.

De­vel­oped at As­traZeneca and bound for a com­mer­cial part­ner­ship, re­searchers say the drug was ef­fec­tive be­cause it tar­gets TSLP, an ep­ithe­lial cy­tokine stoked by lung pathogens such as al­ler­gens and virus­es. TSLP in turn trig­gers a ram­page of in­flam­ma­to­ry cy­tokines such as IL-4, IL-5 and IL-13.

By mov­ing up­stream from those tar­gets, the phar­ma gi­ants be­lieve that they may be on to a break­through ap­proach that can leap the more sin­gu­lar­ly-tar­get­ed ri­vals com­ing in­to the mar­ket, seiz­ing bil­lions in new rev­enue.

Bahi­ja Jal­lal

“This broad bio­mark­er re­sponse is un­prece­dent­ed among res­pi­ra­to­ry bi­o­log­ics and re­flects our com­mit­ment to lead­ing res­pi­ra­to­ry sci­ence for un­met med­ical needs,” says Bahi­ja Jal­lal, who runs As­traZeneca’s big sub­sidiary Med­Im­mune, where the re­search work is be­ing done.

That broad-based ap­proach could have a big im­pact on oth­er in­jectable res­pi­ra­to­ry drugs such as GSK’s Nu­cala, Te­va’s Cinqair as well as As­traZeneca’s own ben­ral­izum­ab — which it doesn’t share with Am­gen — and Dupix­ent from Re­gen­eron and Sanofi. Ben­ral­izum­ab is cur­rent­ly un­der re­view and is giv­en a good shot at an ap­proval.

Leerink’s Sea­mus Fer­nan­dez was ex­cit­ed by the da­ta. So ex­cit­ed that he be­lieves these part­ners have a shot at grab­bing an ac­cel­er­at­ed ap­proval on the Phase IIb da­ta while launch­ing Phase III as a con­fir­ma­to­ry study — an ap­proach that could start de­liv­er­ing up to $4.5 bil­lion a year in rev­enue with­in 4-5 years.

His com­ment:

Based on the strength of these da­ta, we be­lieve this tri­al may qual­i­fy as a reg­is­tra­tional study (with the need for a con­fir­ma­to­ry Phase III tri­al for ap­proval), sim­i­lar to the ap­proach that SNY/REGN are tak­ing with Dupix­ent. How­ev­er, this may be de­pen­dent on whether the FDA will want to see ad­di­tion­al dose rang­ing stud­ies to ad­dress ques­tions re­gard­ing safe­ty and the lack of a clear ef­fi­ca­cy dose re­sponse.

Dupix­ent, of course, has al­ready been rack­ing up rev­enue with an es­tab­lished safe­ty pro­file, mak­ing a quick, added OK much eas­i­er for reg­u­la­tors. In this case, reg­u­la­tors are much more like­ly to raise some barbed ques­tions about con­cerns this drug has spurred about its im­pact on the im­mune sys­tem. That is­sue was touched on by Elis­a­beth Bel of Am­s­ter­dam’s Aca­d­e­m­ic Med­ical Cen­ter, who wrote a piece in the NE­JM not­ing the need for safe­ty stud­ies that could care­ful­ly ex­am­ine the in­ter­play be­tween drug and im­mune sys­tem.

Nev­er­the­less, As­traZeneca and Am­gen now have a late-stage drug they can tout as a po­ten­tial block­buster in an in­tense­ly com­pet­i­tive field — ex­act­ly what the biotech or­dered for these com­pa­nies as they work to build new rev­enue with game chang­ing ther­a­pies.

That’s a def­i­nite plus.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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Amit Munshi, Arena

One of Are­na's top drugs flops in a PhI­Ib study for IBS pain. But re­searchers tease out a pos­si­ble path for­ward as CEO ex­plores 's­trate­gic op­tion­s'

Four years ago, when Arena CEO Amit Munshi cut its ties to a troubled weight drug and doubled down on the pipeline, a cannabinoid receptor 2 agonist figured prominently in the biotech’s future. On Tuesday evening, however, Munshi’s high hopes for the drug took a nasty hit after it failed a Phase IIb study for patients with irritable bowel syndrome pain.

Put through a randomized pace with 273 patients, researchers said it flat failed the primary endpoint among the large group with abdominal pain. But they quickly went on to highlight subgroup data, always a tricky and controversial ploy, where they spotlighted a positive p value for patients with moderate to severe pain who received the high dose of the drug — one of 3 provided in the study.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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CEO Marco Taglietti (Scynexis)

'N­ev­er been more ur­gent:' Scynex­is looks to tack­le su­per­bug cri­sis with late-stage read­out for an­ti­fun­gal hope­ful

As the superbug crisis heats up around the world, Scynexis says it has new data from two interim analyses that prove its antifungal has the potential to treat a broad range of infections.

“The need for new anti-infectives capable of fighting the most resistant pathogens has never been more urgent as we confront the ongoing COVID-19 global pandemic,” CEO Marco Taglietti said in a statement.

A spot­light schiz­o­phre­nia drug in Neu­ro­crine's $2B Take­da deal flunks its first ma­jor test. But it's not giv­ing up yet

When Takeda spun out a pipeline of experimental psychiatry drugs to Neurocrine in a $2 billion deal amid a post-merger shakeout, R&D chief Andy Plump described the therapies as “very interesting but still difficult.”

On Tuesday, we got some idea of how difficult.

San Diego-based Neurocrine revealed that one of the three spotlight clinical programs they’d acquired failed the primary endpoint in a Phase II trial for schizophrenia, registering a negative outcome on the change from baseline in the positive and negative syndrome scale/negative symptom factor score (PANSS NSFS).

Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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Fi­bro­Gen shares skid low­er as a sur­prise ad­comm rais­es risks on roxa OK

FibroGen will likely have to delay its US rollout for roxadustat once again.

In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.

Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.