Am­gen, As­traZeneca bol­ster their case for break­through asth­ma pro­gram as FDA con­sid­ers tak­ing up a re­view

Just a few days af­ter sub­mit­ting a BLA for their high­ly-tout­ed asth­ma pro­gram, Am­gen and As­traZeneca re­leased new da­ta they hope will con­tin­ue to strength­en their FDA pitch.

The com­pa­nies re­leased fur­ther de­tailed Phase III re­sults for teze­pelum­ab ear­ly Thurs­day morn­ing, and pub­lished the full dataset in the New Eng­land Jour­nal of Med­i­cine. Teze­pelum­ab plus stan­dard of care “demon­strat­ed su­pe­ri­or­i­ty” across the pri­ma­ry and all key sec­ondary end­points com­pared to the stan­dard of care alone, they said.

As part of the new re­sults, Am­gen and As­traZeneca high­light­ed new pre-spec­i­fied, ex­plorato­ry analy­ses re­gard­ing re­duc­tions in an­nu­al­ized asth­ma ex­ac­er­ba­tion rates. Re­searchers ob­served pa­tients from four sub­groups over 52 weeks, di­vid­ing them up based on blood eosinophil count and frac­tion­al ex­haled ni­tric ox­ide lev­els.

Among those with el­e­vat­ed blood eosinophil counts and FeNO lev­els, teze­pelum­ab re­duced AAERs by 77% com­pared to the con­trol — a fig­ure Am­gen and As­traZeneca said was clin­i­cal­ly mean­ing­ful. An­oth­er mea­sure showed the ex­per­i­men­tal drug re­duced the rate of ex­ac­er­ba­tions re­quir­ing hos­pi­tal­iza­tion by 85% over 52 weeks.

The da­ta build up­on the re­sults re­leased by the pair in Feb­ru­ary, where they said teze­pelum­ab plus stan­dard of care cut the rate of se­vere asth­ma at­tacks by 56% at the one-year mark com­pared to the con­trol arm. Teze­pelum­ab is a mon­o­clon­al an­ti­body giv­en once month­ly and al­ready sports the FDA’s break­through ther­a­py des­ig­na­tion.

David Reese

Am­gen and As­traZeneca have been drum­ming up ex­cite­ment over this pro­gram for years, par­tic­u­lar­ly since earn­ing that BTD back in late sum­mer 2018. Back then, As­traZeneca ex­ec Bahi­ja Jal­lal called the re­sults “un­prece­dent­ed” and an­a­lysts pegged peak sales around $4.5 bil­lion.

Mene Pan­ga­los

When topline Phase III re­sults for teze­pelum­ab came out last No­vem­ber, the pair again played up their ex­cite­ment — David Reese, Am­gen’s head of R&D, said he was “ab­solute­ly thrilled” with the study, while Mene Pan­ga­los called the da­ta “ex­cit­ing.”

Some con­cern arose last De­cem­ber, how­ev­er, re­gard­ing a key dif­fer­en­tia­tor from drugs like Dupix­ent. That month, teze­pelum­ab failed in a Phase III tri­al on se­vere asth­ma pa­tients in re­duc­ing the num­ber of oral cor­ti­cos­teroids pa­tients took to con­trol their asth­ma. Am­gen called the re­sults “sur­pris­ing,” point­ing out tri­al de­sign flaws as a pos­si­ble cul­prit.

Mi­na Makar

In an in­ter­view with End­points News on Wednes­day, As­traZeneca’s se­nior VP of US res­pi­ra­to­ry and im­munol­o­gy Mi­na Makar said the part­ners were look­ing at fu­ture clin­i­cal stud­ies to re-ad­dress that failed cor­ti­cos­teroids end­point but not­ed that very few US asth­ma pa­tients are ac­tu­al­ly on chron­ic steroids. Mean­while, in his telling, clin­i­cians are far more con­cerned about get­ting the 80% of se­vere asth­ma pa­tients who are in­el­i­gi­ble for ex­ist­ing bi­o­log­ics on­to ther­a­py — an area the drug­mak­ers think they can make a se­ri­ous dent, even with Dupix­ent on the mar­ket.

But an­a­lysts have proved skep­ti­cal, with Baird’s Bri­an Sko­r­ney not­ing in De­cem­ber that physi­cians may ul­ti­mate­ly pre­fer Dupix­ent over teze­pelum­ab, should it be ap­proved. Nonethe­less, the Am­gen/As­traZeneca pro­gram could still see peak sales as high as $2.5 bil­lion.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

An Alzheimer's dark­horse fails its first big tri­al, but of­fers hope for a long-over­looked hy­poth­e­sis

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

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No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.