Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an in­dus­try­wide re­view of can­cer drugs with ac­cel­er­at­ed ap­proval, Bris­tol My­ers Squibb had to make the tough call last month to yank an ap­proval for lead­ing I/O drug Op­di­vo af­ter flop­ping a con­fir­ma­to­ry study. Now, a sec­ond Bris­tol My­ers drug is on the chop­ping block.

Bris­tol My­ers has pulled ag­ing HDAC in­hibitor Is­to­dax’s in­di­ca­tion in pe­riph­er­al T cell lym­phoma af­ter a Phase III con­fir­ma­to­ry study for the drug flopped on its pro­gres­sion-free sur­vival end­point, the drug­mak­er said Mon­day.

Is­to­dax, a Cel­gene drug first ap­proved way back in 2009, earned its ac­cel­er­at­ed ap­proval in PT­CL, a form of non-Hodgkin lym­phoma, in 2011, but it took near­ly a decade for the con­fir­ma­to­ry re­sults to ges­tate. At ASH 2020, Bris­tol My­ers rolled out da­ta show­ing Is­to­dax couldn’t ben­e­fit clin­i­cal out­comes when added to a reg­i­men of cy­clophos­phamide, dox­oru­bicin, vin­cristine, and pred­nisone (CHOP).

So late in its life, Is­to­dax is a mi­cro-earn­er for Bris­tol My­ers so pulling one of its two in­di­ca­tions won’t make much of a dif­fer­ence in terms of the drug­mak­er’s topline. But the re­sults are no­table as part of an in­dus­try­wide reck­on­ing for can­cer drugs that earn the FDA’s ac­cel­er­at­ed nod and even­tu­al­ly flop con­fir­ma­to­ry stud­ies.

Just last week, Bris­tol My­ers pulled its US in­di­ca­tion for check­point in­hibitor Op­di­vo as a monother­a­py for he­pa­to­cel­lu­lar car­ci­no­ma in pa­tients pre­vi­ous­ly treat­ed with so­rafenib af­ter an FDA On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee rec­om­mend­ed pulling the nod.

Op­di­vo won an ac­cel­er­at­ed ap­proval as a sec­ond-line treat­ment for HCC back in 2017, be­com­ing the first im­munother­a­py to get OK’d for this in­di­ca­tion. But two years lat­er, a con­fir­ma­to­ry study didn’t meet sta­tis­ti­cal sig­nif­i­cance in its pri­ma­ry end­point of over­all sur­vival. Last March, the drug land­ed an­oth­er, sep­a­rate ac­cel­er­at­ed ap­proval in com­bi­na­tion with Yer­voy for the same set­ting.

Op­di­vo in monother­a­py HCC was one of two in­di­ca­tions ODAC vot­ed against out of a six-in­di­ca­tion re­view. The oth­er in­di­ca­tion re­ceiv­ing a no-vote was Mer­ck’s Keytru­da as a third-line treat­ment for stom­ach can­cer, which Mer­ck opt­ed to with­draw ear­li­er in Ju­ly.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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An­oth­er Cipla site lands a Form 483 over clean­ing is­sues and QC con­trols

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

No­var­tis un­veils 'US-first' strat­e­gy ahead of San­doz spin­off

Weeks after announcing the spinoff of generics arm Sandoz, Vas Narasimhan paints a picture of the new, slimmer Novartis — with a “US-first mindset,” he said at an investor event on Thursday.

The CEO unveiled ambitious plans to become a top-five player in the US by 2027 at Novartis’ “Meet the Management” event in Basel, Switzerland, which means ramping up clinical trials in the states and “building capability and talent, among other things.” The company’s also shooting for a top-three ranking in China.

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