Af­ter thy­roid eye drug scores in PhI­II test, Hori­zon Phar­ma read­ies mar­ket­ing ap­pli­ca­tion

Less than two years af­ter Hori­zon Phar­ma $HZNP pro­cured phase III-ready thy­roid eye dis­ease (TED) drug tepro­tu­mum­ab for $145 mil­lion up­front by ac­quir­ing a unit of Nar­row Riv­er, biotech vet­er­an David Mad­den’s in­vest­ment firm — the bet has paid off, as the au­toim­mune in­flam­ma­to­ry dis­or­der drug passed its late-stage test on Thurs­day.

Tim Wal­bert, Hori­zon CEO

In TED pa­tients, the eye mus­cles and fat­ty tis­sue be­hind the eye be­come in­flamed, caus­ing the eyes to ap­pear to be ‘bulging’ and eye­lids to be­come swollen and red, even­tu­al­ly cul­mi­nat­ing in eye mis­align­ment, dou­ble vi­sion and in some cas­es even blind­ness — but there is lim­it­ed win­dow of up to three years dur­ing which the con­di­tion can be treat­ed with­out sur­gi­cal in­ter­ven­tion.

Tepro­tu­mum­ab is a mon­o­clon­al an­ti­body de­signed to in­hib­it in­sulin-like growth fac­tor 1 re­cep­tor (IGF-1R). In the 83-pa­tient phase III tri­al, 82.9% of pa­tients treat­ed with tepro­tu­mum­ab ex­pe­ri­enced a mean­ing­ful im­prove­ment in prop­to­sis or bulging of the eye (a 2 mm or more re­duc­tion (p<0.001)) com­pared to 9.5% of pa­tients on the place­bo — meet­ing the main goal of the study. All the sec­ondary end­points were al­so met, the com­pa­ny said.

The rare dis­ease drug­mak­er is work­ing on sub­mit­ting a mar­ket­ing ap­pli­ca­tion for tepro­tu­mum­ab by the mid­dle of 2019, it said. The FDA has grant­ed the ex­per­i­men­tal TED treat­ment — which was orig­i­nal­ly de­vel­oped by Gen­mab and sub­se­quent­ly li­censed from Roche — all the bells and whis­tles: break­through ther­a­py, or­phan drug and fast track des­ig­na­tions that should make a speedy re­view like­ly.

Ray­mond Dou­glas

“If ap­proved, tepro­tu­mum­ab would give physi­cians the first med­i­cine shown to re­duce prop­to­sis dur­ing ac­tive thy­roid eye dis­ease, in ad­di­tion to treat­ing oth­er painful symp­toms,” said Ray­mond Dou­glas, the study’s co-prin­ci­pal in­ves­ti­ga­tor and di­rec­tor of the or­bital and thy­roid eye dis­ease pro­gram at the Cedars-Sinai Med­ical Cen­ter, in a state­ment.

If the BLA is ac­cept­ed, the drug is po­si­tioned for launch in the first half of next year — giv­en the pos­si­bil­i­ty of pri­or­i­ty re­view, Jef­feries an­a­lyst David Stein­berg wrote in a note, adding that there are rough­ly 15,000 to 20,000 ac­tive TED US pa­tients el­i­gi­ble for treat­ment.

Stein­berg es­ti­mat­ed $500 mil­lion in peak sales of the drug by 2024, but added that Hori­zon man­age­ment has fore­cast $750 mil­lion, giv­en the drug has se­cured or­phan sta­tus and is dis­ease-mod­i­fy­ing — po­ten­tial­ly paving the way for ro­bust pric­ing.

Hori­zon cur­rent­ly sells the drug with the high­est price tag in the Unit­ed States — Ac­tim­mune.

“Giv­en the strong ex­e­cu­tion in the re­launch of Krys­texxa (a drug for chron­ic gout) and now on the cusp of in­tro­duc­ing an­oth­er long du­ra­tion or­phan drug with tepro, we in­creas­ing­ly see HZNP be­com­ing a leader in the rare dis­ease area,” Stein­berg wrote.

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.”