Fi­bro­cell Sci­ence sees light at the end of the tun­nel with Cas­tle Creek deal to de­vel­op 'but­ter­fly' dis­ease gene ther­a­py

Em­bat­tled cell and gene ther­a­py com­pa­ny Fi­bro­cell Sci­ence — which last year ini­ti­at­ed a re­view of strate­gic al­ter­na­tives — has found a way for­ward by join­ing forces with Jeff Aronin’s Cas­tle Creek Phar­ma­ceu­ti­cals, shep­herd­ing its lead gene ther­a­py for “but­ter­fly” dis­ease in­to late-stage de­vel­op­ment.

Jeff Aronin

Aronin, for­mer chief of the con­tro­ver­sial Marathon Phar­ma­ceu­ti­cals, al­ready has one drug in the Cas­tle Creek pipeline for the dis­ease. He raised $71.8 mil­lion last Oc­to­ber to re­pur­pose an oral or­phan drug called di­ac­ere­in (ap­proved to treat joint swelling or pain in the EU, but re­strict­ed due to the risks of di­ar­rhea and liv­er prob­lems) in­to a top­i­cal treat­ment for epi­der­mol­y­sis bul­losa sim­plex, a form of rare ge­net­ic con­di­tion known as “but­ter­fly skin.”

Fi­bro­cell’s ther­a­py, FCX-007, is de­signed to en­code the gene for type VII col­la­gen (COL7).  The pro­posed late-stage tri­al — planned to be­gin in the sec­ond quar­ter — is ex­pect­ed to en­roll 15-20 pa­tients.

Un­der the deal, FCX-007 will be ex­clu­sive­ly li­censed to Cas­tle Creek in the Unit­ed States for $7.5 mil­lion up­front. Fi­bro­cell will al­so get $2.5 mil­lion for the first pa­tient en­rolled in the Phase III tri­al and an­oth­er $30 mil­lion up­on ap­proval. Cas­tle Creek is in charge of all de­vel­op­ment ex­pens­es up to $20 mil­lion pri­or to the sub­mis­sion of the mar­ket­ing ap­pli­ca­tion — but if R&D costs ex­ceed that, Cas­tle Creek will take on a fur­ther 70% of the ad­di­tion­al costs, with Fi­bro­cell pick­ing up the rest of the bill.

If ap­proved, Fi­bro­cell will re­tain sole own­er­ship of the Rare Pe­di­atric Dis­ease Pri­or­i­ty Re­view Vouch­er (PRV), which may be grant­ed up­on FCX-007 ap­proval. Cas­tle will pay Fi­bro­cell a 30% share of the gross prof­its from FCX-007 sales. In ad­di­tion, Fi­bro­cell is al­so el­i­gi­ble to re­ceive up to $75 mil­lion in sales mile­stones.

But Fi­bro­cell was de­vel­op­ing FCX-007 in part­ner­ship with In­trex­on and as such, the com­pa­ny will pay them the lat­ter half of all up­front, mile­stone, and prof­it share pay­ments from Cas­tle Creek. Still, it will have enough cash to fund its op­er­a­tions in­to the third quar­ter of 2020.

Fi­bro­cell’s shares $FC­SC shot up near­ly 61% to $2.86 in ear­ly Mon­day trad­ing.


Im­age: Shut­ter­stock

Ken Frazier appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

Who’s next in line to suc­ceed Ken Fra­zier as CEO of the Keytru­da-blessed Mer­ck?

When Mer­ck waved off a loom­ing forced re­tire­ment for Ken Fra­zier last Sep­tem­ber, the board cit­ed flex­i­bil­i­ty in CEO tran­si­tion as a key fac­tor in the de­ci­sion. Hav­ing Fra­zier — who’s al­so chair­man of the com­pa­ny — around be­yond his 65th birth­day in 2019 would en­sure they in­stall the best per­son at the best time, they said.

The board has ev­i­dent­ly be­gun that process with a clear pref­er­ence for in­ter­nal can­di­dates, sources told Bloomberg. CFO Robert Davis, chief mar­ket­ing of­fi­cer Michael Nal­ly, and chief com­mer­cial of­fi­cer Frank Clyburn are all in the run­ning, ac­cord­ing to an in­sid­er.

In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

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 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.

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 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.

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.”

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.