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

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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The fu­ture of mR­NA, J&J's vac­cine ad­comm, Mer­ck­'s $1.85B au­toim­mune bet and more

Welcome to the third installment of Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If this report was helpful in recapping it all for you, please do share it with your colleagues.

Get ready for FDA’s third Covid-19 vaccine

On the heels of a ringing endorsement from FDA reviewers earlier in the week, J&J‘s single-dose vaccine — which proved 66% effective at preventing symptomatic Covid-19, and 85% effective at stopping severe disease 28 days after administration — the advisory committee convened by the agency voted unanimously to recommend its emergency use authorization. It was “a relatively easy call,” according to one of the committee members — although that doesn’t mean they didn’t have questions. Jason Mast has the highlights from the discussion, including new information from the company, on this live blog.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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CEO Fred Aslan (Artiva)

NK cell ther­a­py play­er Arti­va makes some more noise, pulling in $120M Se­ries B less than a month af­ter Mer­ck deal

Not even one month after Big Pharma took notice of Artiva when Merck signed a collaboration worth nearly $2 billion in milestones, the off-the-shelf NK cell biotech already has its next big fundraise.

Artiva returns from the venture well Friday with a $120 million Series B round, money they will use to get their first program into the clinic and to file INDs for another two candidates. The raise marks the latest development in a rapidly expanding footprint for Artiva, which, in addition to the Merck deal last month, has now raised almost $200 million since its Series A last June.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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