No­var­tis con­fi­dent­ly breaks out mid-stage da­ta on Xo­lair com­peti­tor

Late last year, No­var­tis in­di­cat­ed its ex­per­i­men­tal ther­a­py lige­lizum­ab was Phase III ready af­ter it out­per­formed Xo­lair — the com­pa­ny’s Roche-part­nered chron­ic spon­ta­neous ur­ticaria ther­a­py that has lost patent pro­tec­tion — in a Phase II study.  On Tues­day, the Swiss drug­mak­er broke out some of the mid-stage da­ta that trig­gered its con­fi­dence.

Er­ic Hugh­es

In the 382-pa­tient tri­al, pa­tients were ei­ther giv­en lige­lizum­ab at a dose of 24 mg, 72 mg, or 240 mg, oma­l­izum­ab (Xo­lair) at a dose of 300 mg, or place­bo, ad­min­is­tered sub­cu­ta­neous­ly every 4 weeks for a pe­ri­od of 20 weeks, or a sin­gle 120-mg dose of lige­lizum­ab. Pa­tients in the study were al­ready on an­ti­his­t­a­mines.

At week 12, a to­tal of 30%, 51%, and 42% of the pa­tients treat­ed with 24 mg, 72 mg, and 240 mg, re­spec­tive­ly, of lige­lizum­ab had com­plete con­trol of hives, ver­sus 26% of the pa­tients in the oma­l­izum­ab group and no pa­tients in the place­bo group — sug­gest­ing a dose-re­sponse re­la­tion­ship.

In ad­di­tion, about 30%, 44%, and 40% of the pa­tients treat­ed with 24 mg, 72 mg, and 240 mg, re­spec­tive­ly, of lige­lizum­ab had com­plete con­trol of symp­toms, ver­sus 26% of the pa­tients in the oma­l­izum­ab group and no pa­tients in the place­bo group.

The block­buster bi­o­log­ic Xo­lair, which is ap­proved for chron­ic spon­ta­neous ur­ticaria (CSU) as well as al­ler­gic asth­ma, is vul­ner­a­ble to com­pe­ti­tion af­ter los­ing patent pro­tec­tion, par­tic­u­lar­ly in the Unit­ed States and Eu­rope.

“Around half of pa­tients on cur­rent stan­dard-of-care treat­ment, in­clud­ing oma­l­izum­ab, for CSU con­tin­ue to have un­con­trolled symp­toms,” said Er­ic Hugh­es, No­var­tis’  glob­al de­vel­op­ment unit head for im­munol­o­gy, he­pa­tol­ogy and der­ma­tol­ogy, in a state­ment.

Both No­var­tis $NVS and Roche $RHB­BY have con­tin­gency plans in place for even­tu­al Xo­lair com­pe­ti­tion in pa­tients with chron­ic spon­ta­neous ur­ticaria (CSU), an un­pre­dictable skin con­di­tion that is char­ac­ter­ized by spon­ta­neous swelling and itchy hives. No­var­tis has lige­lizum­ab, while Roche is de­vel­op­ing a BTK in­hibitor, fene­bru­ti­nib, which it is cur­rent­ly be­ing test­ed in a Phase II CSU tri­al. A host of drug­mak­ers are al­so work­ing on ri­val treat­ments.

Lige­lizum­ab, mean­while, is cur­rent­ly be­ing in­ves­ti­gat­ed in an on­go­ing late-stage pro­gram which in­cludes twin Phase III tri­als PEARL 1 and PEARL 2 that are re­cruit­ing more than 2,000 pa­tients.

Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

J&J’s Rem­i­cade — the poster child for how to block biosim­i­lars — fi­nal­ly set­tles Pfiz­er suit

Biosimilars have proven time and again (although mostly in Europe) that competition works to bring down the cost of a once-pricey biologic, and can even expand its use.

J&J’s Remicade, however, has always proven to be an outlier.

Back in 2016, Pfizer won FDA approval for its infliximab biosimilar, known as Inflectra, but when the launch foundered, the company sued J&J, claiming that the company’s plan to block biosimilar competition worked incredibly well. Pfizer even went on to win FDA approval for a second infliximab biosimilar in 2017, known as Ixifi, but decided to never launch it.

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Seth Lederman, Tonix Pharmaceuticals CEO

Small, strug­gling biotech winds up with a 3X los­er as an­oth­er PhI­II of its lead drug col­laps­es

Little Tonix Pharmaceuticals has run into another brick wall as its lead drug — a reformulated muscle relaxant originally approved 44 years ago — has failed another Phase III study, sending shares back into penny stock territory.

Three years after going down in their first Phase III trial of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for symptoms of PTSD, the biotech — which had been encouraged by a breakthrough designation at the FDA — reported late Friday the drug also failed its second late-stage challenge for pain associated with fibromyalgia. Outside data monitors recommended the Phase III trial be halted for futility after deciding interim data made it unlikely the drug would pass muster.

An­oth­er one bites the dust: Bris­tol My­ers Squibb pulls 'dan­gling' ac­cel­er­at­ed ap­proval for Op­di­vo in liv­er can­cer

Bristol Myers Squibb has agreed to pull a second-line liver cancer indication for its blockbuster Opdivo as a monotherapy, becoming the second PD-(L)1 indication to bite the dust after the FDA’s oncology adcomm reviewed six “dangling” accelerated approvals in April.

The outside experts voted against two of the six indications discussed at the meeting, including Opdivo as a monotherapy for hepatocellular carcinoma (HCC) patients who have previously been treated with sorafenib, and Merck’s Keytruda as a third-line treatment for stomach cancer. The adcomm voted 5 to 4 not to maintain Opdivo’s indication, after it failed to show clinical benefit in a confirmatory trial.

Pascal Soriot, AstraZeneca CEO (Raphael Lafargue/Abaca/Sipa via AP Images)

Covid-19 roundup: Fau­ci warns that US is head­ed in wrong di­rec­tion, rec­om­mends boost­er; As­traZeneca-Pfiz­er com­bo proves ef­fec­tive in South Ko­rea

A combination of unvaccinated Americans and the Delta variant has led a frustrated NIAID director Anthony Fauci to say the US is headed in the wrong direction, he said on CNN’s show “State of the Union.”

Booster shots may be required for those with suppressed immune systems and public health officials are considering a mask recommendation for those who are already vaccinated, the Associated Press reported. More than 163 million people are vaccinated, but that number is less than half of the US population. And 57% of those who are eligible for the vaccine have been inoculated.

Hervé Hoppenot, Incyte CEO (Jeff Rumans for Endpoints News)

FDA un­sur­pris­ing­ly brings down the ham­mer on In­cyte's PD-1 — draw­ing a line for fu­ture ac­cel­er­at­ed ap­provals

It appears the PD-(L)1 honeymoon is finally over.

Incyte $INCY revealed late Friday the FDA has slammed its PD-1 retifanlimab — which was under priority review for locally advanced or metastatic squamous cell carcinoma of the anal canal — with a complete response letter, demanding “additional data” to show clinical benefit.

On one hand, the rejection should come as no surprise: Regulators spelled out the problems they saw with Incyte’s data package in no uncertain terms, raising concerns about the low response rates, lack of diversity and dearth of safety data in the single-arm trial. During the ensuing adcomm, the FDA’s cancer czar, Richard Pazdur, suggested the whole episode underscores the need to “reassess” how drugs get approved under the accelerated approval pathway without randomized studies.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; and more

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If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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