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

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.