Let the joust­ing be­gin: Which top bio­phar­ma com­pa­nies are look­ing to make a splash at ASH?

Any­one who fol­lows on­col­o­gy knows the field has at­tract­ed so much at­ten­tion and re­search mon­ey that it sparks a con­tin­u­al erup­tion of bio­phar­ma news as com­pa­nies joust over block­buster mar­kets. That’s kept me busy this year with a string of glob­al con­fabs — as AS­CO be­comes just one big event in a fren­zied com­pe­ti­tion.

John Car­roll

So now I’ll be at­tend­ing ASH in At­lanta in ear­ly De­cem­ber for the first time. What’s on my radar? I turned to Leerink to­day for some sug­ges­tions, and boiled them down here.

Ap­provals for No­var­tis $NVS and Gilead/Kite $GILD for their first CAR-T ap­pli­ca­tions are a his­toric first step, but this tech­nol­o­gy is just now be­gin­ning to write its first com­mer­cial chap­ter. Juno $JUNO is now back af­ter get­ting de­railed with the dis­as­ter that JCAR015 be­came. And in fig­ur­ing out what went wrong there, they are promis­ing to come up with the next big thing in CAR-T: JCAR017. The ther­a­py has pro­duced some jaw-drop­ping ef­fi­ca­cy re­sults and a much im­proved safe­ty pro­file, and now we all want to see every up­date to de­ter­mine if Juno can leapfrog the lead­ers.

No­var­tis, the big phar­ma com­pa­ny that fig­ured out how to work like a biotech on this one, will be of­fer­ing some im­por­tant de­tails on Kym­ri­ah for DL­B­CL, where it’s look­ing to go head-to-head with Gilead’s Yescar­ta. So the up­date on that front from the Gilead/Kite team will earn a close in­spec­tion.

Then there’s Pfiz­er $PFE, an­oth­er of the Big Phar­ma play­ers that has made on­col­o­gy a top pri­or­i­ty. The gi­ant bagged rights to Cel­lec­tis’ $CLLS UCART19 in ALL, and a pre­sen­ta­tion in At­lanta will be close­ly fol­lowed. Cel­lec­tis’ re­cent clin­i­cal hold for its own off-the-shelf ap­proach to CAR-T — an­oth­er po­ten­tial leapfrog ther­a­py — will keep the Paris-based biotech in the in­dus­try’s eye.

Blue­bird bio $BLUE is seem­ing­ly al­ways at ASH and al­ways has some­thing in­ter­est­ing to say. In this case it’s the “Lenti­Glo­bin gene ther­a­py pro­gram in trans­fu­sion-de­pen­dent be­ta-tha­lassemia and se­vere sick­le cell dis­ease, as well as up­dat­ed re­sults of the bb2121 an­ti- BC­MA CAR-T Phase I study in pa­tients with re­lapsed/re­frac­to­ry mul­ti­ple myelo­ma,” to quote Leerink.

Blue­bird, by the way, just an­nounced that it has dosed the first pa­tient in its North­star-3 (HGB-212) tri­al, a Phase III study of Lenti­Glo­bin.

Cel­gene $CELG has had its ups and downs this year, with some stun­ning and un­char­ac­ter­is­tic set­backs that have trig­gered a small rout on their stock, ding­ing a large swath of big biotechs in the process. A good show­ing in At­lanta would go a long way to earn­ing back some of the re­spect it’s long en­joyed. Leerink’s as­sess­ment of what to watch for on their close­ly watched ap­pear­ance at ASH:

(O)ral pre­sen­ta­tions from the bb2121 Phase I study in mul­ti­ple myelo­ma, and from the phase I TRAN­SCEND study of JCAR017 in NHL. CELG pre­pares to ini­ti­ate a piv­otal Phase III tri­al with bb2121 in myelo­ma in the com­ing months. CELG al­so will re­port sev­er­al sets of Phase I da­ta from next-gen CelMod CC-122 in NHL in­di­ca­tions.

I’m al­so pluck­ing Alex­ion $ALXN out of the mix. Their next-gen PNH drug is about the on­ly im­por­tant pro­gram they have in the pipeline, for now, and the ear­ly-stage snap­shot on ALXN1210 will get some at­ten­tion as well.

This next part is web on­ly. I’m round­ing out my list now with sug­ges­tions com­ing in over e-mail along with some sug­ges­tions via an­a­lysts re­marks. 

Spark $ONCE has one of those land­mark drugs we all like to watch in Lux­tur­na, and an­a­lysts at Jef­feries say they’ll be on hand to make sure that the biotech demon­strates con­sis­tent­ly pos­i­tive da­ta on the lead­ing drug in the gene ther­a­py field. “At ASH, we ex­pect SPK-8011 to show gen­er­al con­sis­ten­cy of 11-14% and 20-25%, with the third co­hort ide­al­ly show­ing 50-100% high­er lev­els than the pri­or co­hort,” says an up­beat Michael Yee.

I al­so plan to talk to Ax­el Hoos at GSK about their ear­ly-stage BC­MA-tar­get­ing ther­a­py, an an­ti­body drug con­ju­gate dubbed GSK2857916. Just a few days ago GSK flagged the FDA’s break­through drug des­ig­na­tion, which will like­ly keep it on fast-track de­vel­op­ment. And the phar­ma gi­ant is like­ly to use this pro­gram to high­light a re­turn to late-stage de­vel­op­ment in on­col­o­gy.

Al­so adding Re­gen­eron, which has Phase I tri­als of their an­ti-PD-1 cemi­plimab and CD20xCD3 bis­pe­cif­ic REGN1979 in b-cell lym­phomas, specif­i­cal­ly B-cell non-Hodgkin lym­phoma.

There’s al­so Gami­da Cell, which will have the full Phase II da­ta avail­able on NiCord, now in late-stage de­vel­op­ment.

That’s it for now. These are on­ly the high­lights I’m ze­ro­ing in on. But if you have sug­ges­tions for any­thing im­por­tant I missed, let me know. You can email me at john@end­pointsnews.com.


Im­age: The Geor­gia World Con­gress Cen­ter in At­lanta 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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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.

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.

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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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.

Sanofi aligns it­self with Google to stream­line drug de­vel­op­ment

Tech­nol­o­gy is bleed­ing in­to health­care, and big phar­ma is rid­ing the wave. Sanofi $SNY ap­point­ed its first chief dig­i­tal of­fi­cer this Feb­ru­ary, fol­low­ing the foot­steps of its peers. By May, the French drug­mak­er and some of its big phar­ma com­pa­tri­ots joined forces with Google par­ent Al­pha­bet’s Ver­i­ly unit to aug­ment clin­i­cal tri­al re­search. On Tues­day, the Parisian com­pa­ny tied up with Google to ac­cess its cloud com­put­ing and ar­ti­fi­cial in­tel­li­gence tech to spur the de­vel­op­ment of new ther­a­pies.

UP­DAT­ED: Roche fields first ap­proval for Ro­z­lytrek in the run-up to a show­down with Bay­er, Pfiz­er

While it’s wait­ing to hear back from FDA reg­u­la­tors, Roche is be­gin­ning the vic­to­ry lap for en­trec­tinib in Japan.

Roche is giv­ing Bay­er a run for their mon­ey with this tu­mor-ag­nos­tic drug, which tar­gets NTRK gene fu­sions. Now dubbed Ro­z­lytrek, it’s sanc­tioned to treat adult and pe­di­atric pa­tients in Japan with neu­rotroph­ic ty­ro­sine re­cep­tor ki­nase fu­sion-pos­i­tive, ad­vanced re­cur­rent sol­id tu­mors.

Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”