Eye­ing a fast pitch for quick OK in race with blue­bird, J&J/Leg­end team en­joys a reg­u­la­to­ry em­brace for their ri­val BC­MA CAR-T

J&J and its part­ners at Leg­end just picked up brag­ging rights to the EMA’s VIP pro­gram for their made-in-Chi­na BC­MA CAR-T, of­fer­ing some much-need­ed reg­u­la­to­ry love af­ter a few in­flu­en­tial an­a­lysts turned a cold shoul­der to it last year.

The de­ci­sion to be­stow their Prime des­ig­na­tion on JNJ-68284528 is based on LEG­END-2 da­ta for mul­ti­ple myelo­ma we saw at ASH last year — which is what un­der­whelmed some of the an­a­lysts — as well as some da­ta the in­dus­try has yet to see from a Phase I/II study dubbed CAR­TI­TUDE-1. And J&J plans to take max­i­mum ad­van­tage of the open doors in Am­s­ter­dam as it hus­tles up a fast pitch for a quick ap­proval.

Sjaak Bot

“We hope to bring this im­por­tant ad­vance to pa­tients as quick­ly as pos­si­ble and this PRIME des­ig­na­tion, the first for Janssen, marks an im­por­tant mile­stone to­wards po­ten­tial mar­ket ap­proval,” said Sjaak Bot, J&J’s reg­u­la­to­ry chief in Eu­rope.

That high reg­u­la­to­ry opin­ion for new, un­seen da­ta might bol­ster ex­pec­ta­tions for the drug, which wowed AS­CO back in the sum­mer of 2017 while earn­ing a dis­missal from Nick Leschly. The CEO of blue­bird has his own lead­ing BC­MA CAR-T — bb2121, part­nered with Cel­gene and now head­ed in­to Bris­tol-My­ers Squibb’s pipeline as a top late-stage prospect — which he’s as­sert­ed has been test­ed in sick­er pa­tients than Leg­end re­cruit­ed.

Leschly gained some sup­port for his po­si­tion at ASH, when J&J/Leg­end re­vealed that their pa­tients had had an av­er­age of 3 pre­vi­ous lines of treat­ment. That com­pares poor­ly with blue­bird’s 7 pre­vi­ous lines of treat­ment, though Leg­end in­sist­ed that they were just be­ing con­ser­v­a­tive in track­ing ear­li­er at­tempts to beat can­cer.

No mat­ter, said some blue­bird sup­port­ers. Leg­end’s stel­lar da­ta were based on a less sick pa­tient pop­u­la­tion — ad­van­tage blue­bird. This is a tough crowd, though, as Leschly found out when his own lat­est round of BC­MA CAR-T da­ta was seen as pos­i­tive but a lit­tle light on the de­sired en­durance.

Ad­van­tage J&J/Leg­end. And don’t think any of it was lost on Bris­tol-My­ers or Cel­gene, which has a ma­jor CVR bonus tied to the near-term ap­proval of bb2121 and two oth­er top piv­otal-stage drugs at Cel­gene.

This race has yet to be de­cid­ed as a long line­up of play­ers use new tech­nolo­gies to tar­get BC­MA in the hunt for a slice of a block­buster mar­ket.


Im­age: Shut­ter­stock

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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José Baselga, AstraZeneca cancer chief (Brent N. Clarke/FilmMagic via Getty Images)

As­traZeneca's Calquence nabs an­oth­er win against Im­bru­vi­ca, but Eli Lil­ly is on its heels

Three years after first launching Calquence as a second generation BTK inhibitor, AstraZeneca continues to tout new data to compete with J&J and AbbVie’s first generation blockbuster Imbruvica.

The British pharma announced on Monday that Calquence passed a head-to-head Phase III study against Imbruvica in chronic lymphocytic leukemia, proving non-inferior — i.e. just as good — as the older drug. Although AstraZeneca did not break down any of the numbers, they said the drug proved superior on safety, triggering fewer cases of atrial fibrillation, an irregular heartbeat that can lead to stroke or heart failure.

Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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Roche amps up its bis­pe­cif­ic at­tack on Eylea with more PhI­II da­ta — but just how threat­en­ing is it?

Roche has another stack of data to back up its longer-acting challenger to Eylea — although it’s still far from certain just how much they can threaten Regeneron’s dominance.

The latest Phase III results come from two trials that enrolled 1,329 patients with neovascular age-related macular degeneration. With 45% of people in both studies getting faricimab 16 weeks apart during the first year, the bispecific still induced the same level of gains in visual acuity as Eylea every 8 weeks did, Roche’s Genentech reported.

Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.