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.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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Rare dis­ease drug­mak­ers to Con­gress: Don't gut the ac­cel­er­at­ed ap­proval path­way

The controversy over the FDA’s accelerated approval pathway is heating up.

Last week, the FDA’s top oncology official Rick Pazdur said the pathway is “under attack,” largely due to the agency’s recent accelerated approval of Biogen’s controversial Alzheimer’s drug and the surrogate endpoint used in that decision. In the meantime, three accelerated approval indications have been pulled since July 1 (two from Bristol Myers Squibb and one from Merck in recent weeks), even as Pazdur called on critics of the pathway to not miss the more positive, big picture, with some cancer drugs proving to be enormously helpful and approved years before their confirmatory trials were completed.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.