Erik van den Berg AM-Phar­ma

Back in 2015, Pfiz­er put a spot­light on Dutch biotech AM-Phar­ma and its drug can­di­date for sep­sis-as­so­ci­at­ed acute kid­ney in­jury, an­te­ing up $87.5 mil­lion for a mi­nor­i­ty stake in ad­di­tion to an op­tion to gob­ble the whole op­er­a­tion up. The Phase II tri­al that the gi­ant part­ner had bet on end­ed up de­liv­er­ing a mixed win, and Pfiz­er walked away from the deal cit­ing “in­ter­nal strate­gic rea­sons” — leav­ing the small play­er on its own to pull off a large Phase III study and pon­der com­mer­cial­iza­tion.

A year and a half af­ter that da­ta read­out, AM-Phar­ma is go­ing for it. In lieu of one deep-pock­et­ed al­ly, it’s as­sem­bled a group of top-tier Eu­ro­pean in­vestors to back its fi­nal stretch in the clin­ic.

LSP and An­dera Part­ners co-led the €116 mil­lion ($133 mil­lion) round, join­ing found­ing in­vestor For­bion and oth­er ex­ist­ing sup­port­ers: Ys­ios Cap­i­tal, Kur­ma Part­ners, ID In­vest Part­ners, BB Pure­os Bioven­tures and Gilde Health­care.

The Phase III pro­gram will re­cruit 1,400 pa­tients across 12 coun­tries to test whether AM-Phar­ma’s re­com­bi­nant hu­man al­ka­line phos­phatase (re­cAP) can in­deed re­duce the mor­tal­i­ty rate due to kid­ney dam­age in­duced by the body’s over­whelm­ing re­sponse to in­fec­tions.

The 40% re­duc­tion not­ed in the pre­vi­ous Phase II was what gave van den Berg con­fi­dence de­spite the drug not hit­ting the pri­ma­ry end­point of im­prov­ing kid­ney func­tion in 7 days.

“Short term kid­ney func­tion im­prove­ment was re­al­ly more a de­sign for the Phase II study to be able to pick the most op­ti­mal dose,” he said. “It’s kind of a bio­mark­er if you like. For Phase III one has to choose hard clin­i­cal end­points, and the most pre­ferred end­point by the reg­u­la­tors is the mor­tal­i­ty in this set­ting.”

And it’s not hard to see why. With mil­lions of pa­tients suf­fer­ing from acute kid­ney in­jury each oth­er — half of which are as­so­ci­at­ed with sep­sis — and 700,000 deaths around the world an­nu­al­ly, mak­ing a dent in that would mean a block­buster op­por­tu­ni­ty.

Re­cAP works by de­phos­pho­ry­lat­ing sub­stances that can trig­ger the im­mune sys­tem in­to dev­as­tat­ing the kid­ney, thus dex­o­ti­fy­ing them and re­duc­ing the in­flam­ma­tion.

“From our in­ter­ac­tions with FDA and EMA last year, we just need to con­duct one sin­gle pos­i­tive piv­otal study to file for mar­ket ap­proval,” he tells me, adding that the drug has se­cured fast track des­ig­na­tion in the US.

He plans to bring the com­pa­ny’s head­count up from 15 to as many as 30 to com­plete the clin­i­cal work and per­haps be­gin lay­ing some ground­work for launch.

Fol­low­ing the fi­nanc­ing the board will al­so ex­pand, com­pris­ing Mar­ti­jn Klei­jwegt and Fe­lice Ver­duyn van Wee­gen from LSP, Raphael Wis­niews­ki from An­dera, Re­mi Droller from Kur­ma and Geert-Jan Mul­der from For­bion.

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.