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.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

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Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.