Fi­deli­ty, Val­or pool $71.8M for Jeff Aron­in's plan to re­for­mu­late a gener­ic for but­ter­fly skin dis­ease at Cas­tle Creek Phar­ma


Jeff Aronin is back.

To be sure, Aronin didn’t ex­act­ly go away. The for­mer Marathon Phar­ma­ceu­ti­cals CEO mere­ly sold off a con­tro­ver­sial drug, shut­tered his em­bat­tled com­pa­ny, and ducked from the scan­dal (we’ll ex­plain). But he held on as chair­man and chief ex­ec­u­tive of Paragon Bio­sciences, which has con­tin­ued in­cu­bat­ing rare dis­ease ven­tures like Marathon.

For the first time since last May, though, Aronin is now ready to hus­tle an­oth­er re­pur­posed or­phan drug through the reg­u­la­to­ry process — with $71.8 mil­lion he just raised.

The drug, di­ac­ere­in, is a IL-1β in­hibitor used in a num­ber of coun­tries to treat joint swelling or pain, al­though its use has been re­strict­ed in the EU due to the risks of di­ar­rhea and liv­er prob­lems. It is not ap­proved in the US.

Michael Der­by

Cas­tle Creek Phar­ma, a der­ma­tol­ogy-fo­cused biotech in Paragon’s port­fo­lio of six, wants to turn this gener­ic com­pound — usu­al­ly tak­en oral­ly — in­to a top­i­cal oint­ment for the treat­ment of epi­der­mol­y­sis bul­losa sim­plex, a sub­type of the al­ready rare ge­net­ic con­di­tion known as “but­ter­fly skin.”

The com­pa­ny is go­ing to con­tin­ue a Phase II/III study with the mon­ey Paragon just raised from old sup­port­er Fi­deli­ty Man­age­ment & Re­search Com­pa­ny and new in­vestor Val­or Eq­ui­ty Part­ner on its be­half, hav­ing ob­tained the FDA’s rare pe­di­atric dis­ease des­ig­na­tion a few months ago.

Sound fa­mil­iar? It should.

Es­sen­tial­ly, Marathon ran on the same mod­el — buy­ing an old gener­ic steroid avail­able for decades in coun­tries like Cana­da and the UK along with the old da­ta pack­age, then gain­ing an FDA ap­proval through the or­phan chan­nel — un­til a price goug­ing scan­dal blew it up. Where­as some DMD pa­tients in the US had been buy­ing the drug, called de­flaza­cort, from a UK sup­pli­er for lit­tle more than $1,000 a year, Marathon at­tempt­ed to charge a list price of $89,000, pro­vok­ing out­rage from DMD pa­tients and sen­a­tors alike. Marathon swift­ly sold the drug, now Em­flaza, to PTC Ther­a­peu­tics and closed its doors.

The key dif­fer­ence here, of course, is that Cas­tle Creek is go­ing af­ter a dif­fer­ent in­di­ca­tion than the gener­ic has been used to treat. The com­pa­ny, split be­tween of­fices in Par­sip­pa­ny, NJ and Chica­go, has not brought up price in its dis­cus­sions so far.

In an ear­li­er Phase II clin­i­cal tri­al in­volv­ing 17 young EBS pa­tients, the drug was shown to help 40% of them re­duce the num­ber of blis­ters in treat­ed ar­eas by 60%, com­pared to 15% for place­bo.

Michael Der­by, pres­i­dent and gen­er­al man­ag­er of the CNS unit at Marathon, is the co-founder and CEO of Cas­tle Creek.

“We are fo­cused on de­vel­op­ing CCP-020 as an im­por­tant treat­ment for EBS and look for­ward to tar­get­ing the new in­vest­ment from Fi­deli­ty and Val­or to­wards the late-stage de­vel­op­ment of this in­ves­ti­ga­tion­al drug,” he said in a state­ment.


Im­age: Jeff Aronin.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

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I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.