Dif­ferin mak­er Gal­der­ma wins FDA ap­proval for a new retinoid for ac­ne

Da­ta sug­gest that in 2016 the bur­den of ac­ne vul­garis — the chron­ic in­flam­ma­to­ry skin dis­ease — bal­looned to rough­ly $3 bil­lion in health­care costs and lost pro­duc­tiv­i­ty in the Unit­ed States. Now those pesky blem­ish­es have a new foe in the form of a top­i­cal retinoid from Gal­der­ma.

Gal­der­ma on Fri­day said it had se­cured the ap­proval of tri­farotene, a top­i­cal retinoid cream. Retinoids are a class of med­ica­tions de­rived from vi­t­a­min A that have long played in key role in ac­ne ther­a­py. Retinoids in­ter­act with retinoic acid re­cep­tor (RAR) and retinoid X re­cep­tor (RXR) — mem­bers of a su­per­fam­i­ly of lig­and-ac­ti­vat­ed tran­scrip­tion fac­tors — to re­duce vis­i­ble le­sions as well as in­hib­it the de­vel­op­ment of mi­cro­come­dones and new le­sions.

The mak­er of Dif­ferin — a pop­u­lar over-the-counter top­i­cal retinoid — will make the new treat­ment, chris­tened Ak­lief, avail­able in No­vem­ber. It is the first top­i­cal treat­ment specif­i­cal­ly stud­ied and shown to help both fa­cial and trun­cal (chest, shoul­ders, and back) ac­ne.

Ak­lief is en­gi­neered to se­lec­tive­ly tar­get RARγ, ver­sus ex­ist­ing first- and third-gen­er­a­tion top­i­cal retinoids, which tar­get both RARβ and RARγ. It was test­ed in twin 12-week stud­ies: PER­FECT 1 and PER­FECT 2 in­volv­ing more than 2,400 pa­tients be­tween 2015 to 2017. In both anatom­ic re­gions,  the prod­uct was sig­nif­i­cant­ly su­pe­ri­or to the ve­hi­cle in suc­cess rates and in the re­duc­tion of in­flam­ma­to­ry and non­in­flam­ma­to­ry le­sion counts.

The im­por­tance of retinol, or vi­t­a­min A, was dis­cov­ered dur­ing World War I — sub­se­quent re­search showed that its de­fi­cien­cy gives rise to xe­ro­sis (dry skin) and fol­lic­u­lar hy­per­k­er­ato­sis (ker­atin buildup around the hair fol­li­cles). But be­fore the com­pound could en­ter the zeit­geist, sci­en­tists worked on syn­the­siz­ing com­pounds sim­i­lar to vi­t­a­min A with an im­proved safe­ty and ef­fi­ca­cy pro­file — and these were even­tu­al­ly in­tro­duced for the treat­ment of der­matoses.

Five years af­ter the Swiss con­glom­er­ate Nestlé swal­lowed the der­ma­tol­ogy com­pa­ny Gal­der­ma, it un­veiled plans to ex­it the skin health busi­ness to re­turn to its roots in food and nu­tri­tion. Ear­li­er in 2019, Nestlé di­vulged it was in ex­clu­sive ne­go­ti­a­tions with a con­sor­tium led by EQT and a whol­ly owned sub­sidiary of the Abu Dhabi In­vest­ment Au­thor­i­ty (ADIA) to sell Nestlé Skin Health in the re­gion of CHF 10.2 bil­lion (about $10.3 bil­lion). Last week, the sale was con­sum­mat­ed. An in­de­pen­dent Gal­der­ma is now run by Flem­ming Orn­skov, who led Shire be­fore it was swal­lowed by Japan’s Take­da.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

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

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotechs that fit that general description.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

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Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.

Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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