#AS­CO21: J&J busts out a small up­date for its an­ti­body-TKI com­bo in lung can­cer — whet­ting ap­petites as FDA thinks it over

In a red-hot EGFR-mu­tat­ed non-small cell lung can­cer mar­ket, John­son & John­son thinks it could have a leader in its bis­pe­cif­ic an­ti­body ami­van­tam­ab. But as com­peti­tors crowd in, J&J is look­ing to back up its case for ap­proval, and it’s rolling out just a taste of that da­ta to back its case in rare mu­ta­tions.

A com­bi­na­tion of J&J’s EGFR/MET bis­pe­cif­ic an­ti­body ami­van­tam­ab and small-mol­e­cule TKI drug laz­er­tinib post­ed a me­di­an du­ra­tion of re­sponse of 9.6 months in pa­tients with NSCLC with ex­on 19 dele­tion or L858R mu­ta­tion that hadn’t pre­vi­ous­ly un­der­gone chemo but pre­vi­ous­ly failed on As­traZeneca’s Tagris­so, ac­cord­ing to co­hort da­ta from the Phase I/II CHRYSALIS study set to be pre­sent­ed at AS­CO.

It’s a mea­ger up­date for J&J’s com­bo af­ter the drugs post­ed a 100% com­plete re­sponse — and whet in­vestors’ ap­petites — at last year’s ES­MO for EGFR-mu­tat­ed NSCLC pa­tients who were treat­ment-naive. In the re­lapsed set­ting, a 45-pa­tient co­hort hit a 36% con­firmed re­sponse rate with 1 com­plete re­sponse and 15 par­tial.

The drug­mak­er filed for ap­proval in the broad­er pa­tient pop­u­la­tion back in De­cem­ber.

Pa­tients in this com­bo co­hort were al­so ge­net­i­cal­ly test­ed for EGFR- or MET-based tu­mor re­sis­tance, with 17 pa­tients iden­ti­fied as hav­ing one or both re­sis­tance mech­a­nisms. Of those, the over­all re­sponse rate was 47% — high­er than the com­bo co­hort on the whole — with a me­di­an du­ra­tion of re­sponse of 10.4 months. The clin­i­cal ben­e­fit re­sponse rate was 82% with a me­di­an PFS of 6.7 months.

Even more im­pres­sive, for the 10 pa­tients in the co­hort whose tu­mors stained high for both MET and EGFR, the over­all re­sponse rate was 90%. The com­pa­ny said it planned to lean on ge­net­ic test­ing in or­der to lo­cate pa­tients with the high­est chance of ben­e­fit­ting from the ami­van­tam­ab-laz­er­tinib com­bo in the Phase III MARI­POSA study cur­rent­ly on­go­ing.

J&J is pri­mar­i­ly set­ting up ami­van­tam­ab for ex­on 20 mu­ta­tions, which have few treat­ment op­tions and have be­come some­what of an arms race for Big Phar­ma. On Wednes­day, Take­da rolled out its own da­ta set for a next-gen TKI in­hibitor look­ing at the same pop­u­la­tion.

Look­ing at the bat­tle against ap­proved and po­ten­tial TKI head-on, J&J al­so rolled out “in­di­rect treat­ment com­par­i­son” show­ing the drug post­ed a 10-month high­er OS com­pared with pa­tients treat­ed with re­al-world ther­a­pies such as TKIs and check­point in­hibitors. It’s all part of an ef­fort, J&J said, to high­light the huge un­met clin­i­cal need in NSCLC that oth­er play­ers haven’t been able to meet.

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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