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

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.