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.


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Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

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