Aca­cia Phar­ma did well on the R&D front — by 2017, it had con­duct­ed four pos­i­tive piv­otal clin­i­cal tri­als for its lead drug, Barhem­sys. Lat­er that year, it sub­mit­ted an FDA ap­pli­ca­tion to mar­ket the drug for res­cue treat­ment of pa­tients who de­vel­op post­op­er­a­tive nau­sea & vom­it­ing (PONV), de­spite hav­ing been giv­en pri­or antiemet­ic pro­phy­lax­is — on­ly to re­ceive a re­jec­tion in Oc­to­ber 2018.

The US agency made its de­ci­sion based on a pre-ap­proval in­spec­tion of a fa­cil­i­ty run by the con­tract man­u­fac­tur­er of the drug’s main in­gre­di­ent — amisul­pride — and not on clin­i­cal or non-clin­i­cal da­ta in the ap­pli­ca­tion, as­sured Aca­cia in a press re­lease, adding that the FDA had asked for no ex­tra stud­ies or da­ta analy­ses re­lat­ed to the treat­ment.

With re­newed en­thu­si­asm lat­er that month, Aca­cia said its con­tract man­u­fac­tur­er had agreed to “in­sti­tute a cor­rec­tive and pre­ven­tive ac­tion plan that will rec­ti­fy the de­fi­cien­cy iden­ti­fied as quick­ly as pos­si­ble. We con­tin­ue to plan for a launch in the first half of 2019,” Aca­cia chief Ju­lian Gilbert said in a state­ment.

Ju­lian Gilbert

Aca­cia then re­sub­mit­ted its mar­ket­ing ap­pli­ca­tion in De­cem­ber, in­di­cat­ing that the FDA’s con­cerns out­lined in the com­plete re­sponse let­ter had been re­solved. But on Fri­day, the com­pa­ny re­ceived an­oth­er re­jec­tion, with the FDA flag­ging the same con­cerns.

“We are on track to com­plete the qual­i­fi­ca­tion of an al­ter­na­tive sup­pli­er of amisul­pride and plan to en­gage with FDA as soon as pos­si­ble to de­ter­mine the most rapid route to ob­tain­ing ap­proval,” Gilbert said in a state­ment on Fri­day.

Barhem­sys is de­signed to help the 30-40% of sur­gi­cal pa­tients who suf­fer from PONV de­spite pri­or pro­phy­lax­is, as well as for com­bi­na­tion pro­phy­lax­is in high-risk pa­tients.

Cam­bridge, UK-based Aca­cia Phar­ma, which al­so has US op­er­a­tions in In­di­anapo­lis, was found­ed in 2007. It has an­oth­er amisul­pride-based drug — APD403 — in mid-stage de­vel­op­ment for chemother­a­py in­duced nau­sea & vom­it­ing.

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.