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.

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

For a pharma company with about $5 billion in revenue, a couple of respectably sized blockbuster drugs on the market and some high-profile partners like Amgen, Belgium’s UCB has kept an unusually low profile on the pipeline side of things over the years.
Until now.
Just days after striking a $2.1 billion deal to buy Ra Pharmaceuticals and its C5 rival to Soliris, UCB is posting positive top-line Phase III results for a dual IL-17 inhibitor that it’s steering into one of the most competitive commercial spaces in the industry. And despite plenty of obvious challenges as they struggle to roll out Evenity with Amgen and patent expirations loom on its franchise drugs, including Cimzia, the company just may be ready to tackle some of the biggest players on the planet.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.
We don’t know the PASI90 and IGA scores — but UCB knows that with the kind of heavyweight competition it faces with Novartis and others, marginal gains for patients won’t stack up. So we’ll be watching for the hard numbers. And there’s another head-to-head with Cosentyx that will play a big role in pushing up analysts’ projections on peak sales, which currently fall well short of blockbuster status.
UCB hasn’t exactly been in the spotlight for the last few years, but it’s in a position now that the company has to win some respect in R&D, with blockbuster projects that can keep investors’ attention at a time the industry is experiencing booming R&D development efforts around the planet.
It hasn’t been easy. There was a setback on a lupus drug partnered with Biogen. But there have been some advances, with a deal to buy Proximagen’s NDA-ready nasal spray therapy USL261, designed as a rescue therapy for acute repetitive seizures, for $150 million in cash and another $220 million in sales and regulatory milestones. There was even a report that the company was kicking the deflated tires at Acorda, though nothing came of that.
Late last year UCB also committed to spend up to £200 million on a new R&D hub in the UK.
That may not translate into a lot of excitement right now, but they’re trying. And there’s a subtle promise that more deals may be in the works.

For years, the idea of protein degradation — utilizing the cell’s natural garbage disposal system to mark problematic proteins for destruction — remained an elegant but technically difficult concept. But now established as a promising clinical strategy, with major biopharma players such as Bayer, Gilead and Vertex trying to grab a foothold via partnership deals, a San Diego startup is looking to exploit it and push its limits.