As­traZeneca caps its big win in lung can­cer with pos­i­tive sur­vival da­ta for Imfinzi — and what a re­lief it is

As­traZeneca capped its big win on the land­mark PA­CIF­IC study to­day with the news that its PD-L1 check­point Imfinzi de­liv­ered a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in over­all sur­vival for a key seg­ment of the lung can­cer mar­ket. 

Pas­cal So­ri­ot

The OS score dur­ing an in­ter­im analy­sis is for pa­tients whose stage III non-small cell lung can­cer hasn’t pro­gressed af­ter chemora­di­a­tion and whose tu­mors can’t be sur­gi­cal­ly re­moved. The da­ta here — specifics will fol­low at a con­fer­ence — will help As­traZeneca con­sol­i­date a cru­cial lead spot for it­self, which As­traZeneca CEO Pas­cal So­ri­ot has boast­ed will put the phar­ma gi­ant in the lead for “half the pool in lung can­cer.”

The FDA has al­ready come through with an OK for As­traZeneca here, un­der­scor­ing that reg­u­la­tors aren’t wait­ing for every i to be dot­ted and every t crossed be­fore get­ting new can­cer drugs to pa­tients and doc­tors.

Dave Fredrick­son

The im­por­tance of the very re­al suc­cess here for So­ri­ot is hard to over­state. The CEO has to cap­i­tal­ize on ad­vances for the can­cer drug port­fo­lio to prove to in­vestors that new drugs can fi­nal­ly pro­vide the fi­nan­cial turn­around that As­traZeneca has to have as the Crestor fran­chise melts away. PA­CIF­IC is blaz­ing a path for a would-be block­buster that can let So­ri­ot prove that.

Just a few days ago Dave Fredrick­son, who heads up the on­col­o­gy busi­ness unit for As­traZeneca, told an­a­lysts that PA­CIF­IC had swift­ly opened up a valu­able mar­ket seg­ment demon­strat­ing some pow­er­ful po­ten­tial. He not­ed:

This has re­sult­ed in a true in­flec­tion point for Imfinzi with sales now at $62 mil­lion in the first quar­ter and re­al­ly the over­whelm­ing ma­jor­i­ty of these com­ing from the lung can­cer in­di­ca­tion. As you saw from a slide that Pas­cal showed in Feb­ru­ary when we launched, we had about 3,500 pa­tient in­fu­sions per month and you could see that we’ve now dou­bled in the most re­cent month to 7,000 pa­tient in­fu­sions per month, re­al­ly show­ing that the un­der­ly­ing pa­tient de­mand is the dri­ver of these sales. 

Sean Bo­hen

The suc­cess in this seg­ment gives As­traZeneca’s can­cer team added cred­i­bil­i­ty for un­der­stand­ing how to score a strate­gic ad­vance in an in­tense­ly com­pet­i­tive field, with Mer­ck and Bris­tol-My­ers tus­sling for dom­i­nance in front­line lung can­cer. Com­ing on the heels of the em­bar­rass­ing first-round MYS­TIC fail­ure, it al­so of­fered an en­cour­ag­ing en­dorse­ment for the com­pa­ny’s R&D com­pe­tence.

As­traZeneca CMO Sean Bo­hen said: “The read­out of pos­i­tive over­all sur­vival da­ta at the in­ter­im analy­sis of the PA­CIF­IC tri­al pro­vides ad­di­tion­al com­pelling ev­i­dence of the clin­i­cal ben­e­fit that Imfinzi can of­fer pa­tients in this ear­li­er stage of lung can­cer.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: Iver­ic Bio, un­de­terred by loom­ing PDU­FA for com­peti­tor, touts sub­group da­ta on GA drug

CHICAGO — While its competitor is on the cusp of likely securing the first FDA nod, Iveric Bio is trudging ahead with its potential treatment for geographic atrophy, an advanced form of AMD, and has new data to support its upcoming NDA filing.

The biotech said its drug was more favorable than sham across all subgroups in the second Phase III study of the investigational complement C5 protein inhibitor, performing similarly to a previous, smaller late-stage trial.

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#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: In bid for first FDA nod in ge­o­graph­ic at­ro­phy, Apel­lis claims an­oth­er first in eye dis­ease field

CHICAGO — Eight weeks before patients and industry find out if the FDA approves the first treatment for geographic atrophy, an advanced form of age-related macular degeneration, the biotech behind the drug is out with some new data on a secondary endpoint.

In what study investigator Charles Wykoff called the “first direct evidence of function preservation by slowing GA growth” in an investigational treatment, Apellis Pharmaceuticals’ drug pegcetacoplan led to less loss of retinal sensitivity versus sham  within 250 microns on either side of the GA lesion over 24 months.

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