#AS­CO17 high­lights: Roche ush­ers ear­ly-stage bis­pe­cif­ic in­to view; Agios, Cel­gene un­veil more AG-221 da­ta; Plen­ty more com­ing at AS­CO

Roche has an ear­ly-stage T cell bis­pe­cif­ic — which binds si­mul­ta­ne­ous­ly to T cells and tu­mor cells — called CEA-TCB (RO6958688; RG7802) that it is bull­ish about. It re­leased a very ear­ly look at Phase I da­ta for AS­CO, look­ing at monother­a­py as well as a com­bo with its check­point Tecen­triq. The da­ta demon­strate: “In the monother­a­py, out of 31 pa­tients with mCRC treat­ed with CEA-TCB dos­es of 60mg or above, 14 pa­tients (45%) showed ei­ther par­tial re­sponse (n=2, 6%) or sta­ble dis­ease (n=12, 39%). For the com­bi­na­tion, of 25 pa­tients treat­ed with dos­es of 5–160mg of CEA-TCB, 11 pa­tients with MSS mCRC were treat­ed at dos­es shown to in­duce tu­mour le­sion in­flam­ma­tion (80 and 160 mg). Nine of these pa­tients (82%) showed ei­ther a par­tial re­sponse (n=2, 18%) or sta­ble dis­ease (n=7, 64%) in this dif­fi­cult-to-treat pop­u­la­tion.”

Agios and Cel­gene delve deep­er in­to AG-221 da­ta

Agios and its big part­ner Cel­gene have al­ready filed for an ap­proval of AG-221, which they now pre­fer to call enasi­denib. Pre­sum­ably we’ll get a bet­ter look at its da­ta — and its prospects — at the con­fer­ence. In the mean­time it of­fers an up­date on their Phase I dose es­ca­la­tion study:

For R/R AML pts, over­all re­sponse rate (ORR) was 40.3%, in­clud­ing 34 (19.3%) com­plete re­mis­sions (CR; Table). Re­sponse was as­so­ci­at­ed with cel­lu­lar dif­fer­en­ti­a­tion, typ­i­cal­ly with no ev­i­dence of apla­sia. Me­di­an over­all sur­vival (OS) for R/R AML pts was 9.3 months (mos). For pts who at­tained CR, OS was 19.7 mos. Pts who had re­ceived ≥2 pri­or AML reg­i­mens (n=94; 53%) had me­di­an OS of 8.0 mos.

So far, so good.

A snap­shot of Five Prime’s cabi­ral­izum­ab da­ta

Michael Schmidt at Leerink of­fered this quick look at Phase I/II da­ta from Five Prime’s cabi­ral­izum­ab. He not­ed:

Re­call, this study ad­vanced in­to Phase II in May 2016 and in April 2017 en­roll­ment of 30 PVNS pa­tients com­plet­ed. Ab­stract #11078 high­lights ac­tiv­i­ty at the 4mg/kg dose (that was cho­sen for Phase II) with 1 par­tial re­sponse (PR) seen in 3 pa­tients in the Phase I por­tion of the study. One pa­tient dis­con­tin­ued based on tri­al rues fol­low­ing cre­a­tine ki­nase (CK) el­e­va­tion, an asymp­to­matic lab­o­ra­to­ry anom­aly and on-tar­get ef­fect of macrophage in­hi­bi­tion. In the Phase II por­tion, FPRX re­port­ed 4 PRs in 7 evalu­able pa­tients. An­oth­er 6 pa­tients in the Phase II por­tion have re­ceived treat­ment but were not evalu­able at time of ab­stract sub­mis­sion. Pos­i­tive func­tion­al sta­tus im­prove­ments were al­so not­ed in ob­jec­tive re­spon­ders. Man­age­ment pre­vi­ous­ly em­pha­sized the im­por­tance of qual­i­ty of life and func­tion im­prove­ments (e.g., pain) for PVNS pa­tients.

The good stuff is be­ing held back for AS­CO

You’re go­ing to have to wait for AS­CO for a lot of things, in­clud­ing more ma­ture da­ta on blue­bird bio’s an­ti-BC­MA CAR-T bb2121 Phase I re­lapsed/re­frac­to­ry mul­ti­ple myelo­ma study.

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:

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,100+ biopharma pros reading Endpoints daily — and it's free.

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.
Endpoints News

Basic subscription required

Unlock this story instantly and join 53,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,100+ biopharma pros reading Endpoints daily — and it's free.

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.”