From part­ner to knight in shin­ing ar­mor: Cas­tle Creek to buy Fi­bro­cell

In April, Cas­tle Creek swooped in to part­ner with the em­bat­tled gene and cell ther­a­py Fi­bro­cell to shep­herd its lead gene ther­a­py for a type of “but­ter­fly” dis­ease in­to late-stage de­vel­op­ment. Now, the New Jer­sey-based der­ma­tol­ogy com­pa­ny is ac­quir­ing its part­ner in a deal worth $63.3 mil­lion.

Cas­tle Creek CEO Greg Wu­jek

Penn­syl­va­nia-based Fi­bro­cell last year ini­ti­at­ed a re­view of strate­gic al­ter­na­tives, in­clud­ing a sale.

Its lead drug FCX-007 is en­gi­neered to treat the un­der­ly­ing cause of re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa, which is caused by the de­fi­cien­cy of the pro­tein COL7. Cells are ex­tract­ed from the pa­tient, ge­net­i­cal­ly mod­i­fied, and then used to treat wounds by lo­cal in­jec­tion, avoid­ing sys­temic dis­tri­b­u­tion.

A late-stage study for FCX-007 was kicked off in Ju­ly, and if all goes well, a mar­ket­ing ap­pli­ca­tion for the treat­ment is ex­pect­ed to be sub­mit­ted in 2021, Fi­bro­cell said on Thurs­day. The com­pa­ny, which al­so counts In­trex­on $XON as a part­ner, has an ex­per­i­men­tal gene ther­a­py FCX-013 in ear­ly-stage de­vel­op­ment for mod­er­ate to se­vere lo­cal­ized scle­ro­der­ma.

Epi­der­mol­y­sis Bul­losa (EB) is a group of ge­net­ic skin con­di­tions that cause the skin to blis­ter and tear due to min­i­mal con­tact — in­fants born with the dis­ease are called ‘but­ter­fly chil­dren’ as their skin is con­sid­ered as frag­ile as a wing of a but­ter­fly.

Cas­tle Creek Phar­ma­ceu­ti­cals — one of for­mer Marathon chief Jeff Aronin’s port­fo­lio com­pa­nies un­der his flag­ship in­vest­ment en­gine Paragon Bio­sciences — has its own EB drug in de­vel­op­ment: CCP-020 is a late-stage top­i­cal oint­ment un­der de­vel­op­ment for use in epi­der­mol­y­sis bul­losa sim­plex. The drug is a re­pur­posed an oral or­phan treat­ment called di­ac­ere­in, which is ap­proved to treat joint swelling or pain in the EU, but its use is re­strict­ed due to the risks of di­ar­rhea and liv­er prob­lems.

“Fol­low­ing our li­cens­ing agree­ment to de­vel­op and com­mer­cial­ize FCX-007, our ex­pe­ri­ence work­ing to­geth­er on rare der­ma­to­log­i­cal con­di­tions caused us to quick­ly re­al­ize that Cas­tle Creek and Fi­bro­cell could achieve even greater syn­er­gies by com­bin­ing the com­pa­nies in­to one,” said Greg Wu­jek, CEO of Cas­tle Creek Phar­ma­ceu­ti­cals, in a state­ment.

Cas­tle Creek has agreed to pay $3 per Fi­bro­cell share $FC­SC, which is a near­ly 64% pre­mi­um to the com­pa­ny’s Thurs­day clos­ing. The deal, in which Cas­tle Creek will ab­sorb Fi­bro­cell’s debt, is ex­pect­ed to close by the fourth quar­ter.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

News brief­ing: PureTech plans Nas­daq de­but with sec­ondary list­ing; GoodRx prices $725M IPO

London Stock Exchange-listed PureTech Health announced Wednesday that it’s looking to extend to Nasdaq. But due to its “strong cash position,” the biotech says it isn’t issuing any new shares in the potential secondary listing.

The company’s shares closed at £256.50 Tuesday on the London Stock Exchange. Its candidate LYT-100 is currently in Phase I development for various indications, including lymphatic flow disorders and fibrotic and inflammatory disorders. PureTech is expecting a Phase Ib readout in lymphatic flow disorders later this year, and is also planning to launch a Phase II study for the drug to treat respiratory conditions experienced after Covid-19.

Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to announce their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

J&J re­leas­es PhI­II safe­ty blue­print for Covid-19 vac­cine tri­al. How does it stack up to Mod­er­na, Pfiz­er and As­traZeneca?

Along with the initiation of its Phase III Covid-19 vaccine study announced Wednesday morning, Johnson & Johnson also released its trial protocol, giving an inside look at how the company is conducting its late-stage research.

The move comes after the other three companies conducting Phase III’s in the US — Moderna, Pfizer and AstraZeneca — each disclosed their own trial blueprints within the last week. Though the release of such protocols is typically done after trials have been completed, drug developers had come under intense pressure after a brief safety scare in an AstraZeneca trial and amid growing concern of a politically motivated vaccine authorization.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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