Eli Lil­ly has suf­fered through the biggest set­backs in late-stage drug de­vel­op­ment work for Alzheimer’s — pil­ing up high-pro­file fail­ures for sema­gace­s­tat, solanezum­ab and just months ago lan­abece­s­tat af­ter it be­came ap­par­ent that the BACE ap­proach was a non-starter in symp­to­matic pa­tients. To­day, it’s sig­nal­ing that the com­pa­ny is now ready to fold their hand ear­ly if it doesn’t look like they have a rea­son­able chance to go on to win the pot.

Out in the quar­ter­ly cleanup went a Phase II BACE com­bo in­clud­ing an an­ti­body for N3PG. And the com­pa­ny al­so axed what was termed an SEL BACE 1 Phase I study. N3PG will con­tin­ue on its own.

“There are many vari­abil­i­ties at play with the BACE class and re­cent find­ings from Lil­ly and oth­ers have chal­lenged the BACE hy­poth­e­sis,” notes a spokesper­son from Eli Lil­ly in an email fol­lowup with me. “As a field we are still eval­u­at­ing the class and will con­tin­ue to have ro­bust sci­en­tif­ic dis­cus­sions about its vi­a­bil­i­ty.”

Mer­ck dealt a de­ci­sive blow to BACE with back-to-back Phase III stud­ies in which the drug did what was ex­pect­ed in terms of turn­ing the tap off on amy­loid be­ta, the long­time cul­prit for a dis­ease that wipes out mem­o­ry. With clear ev­i­dence that the strat­e­gy is a non-starter, Lil­ly and its part­ners at As­traZeneca agreed to kill off their late-stage pro­gram for lan­abece­s­tat af­ter re­view­ing the re­sults to date.

But BACE isn’t quite fin­ished yet. 

Back in ear­ly June, Ei­sai and Bio­gen claimed a sig­nif­i­cant suc­cess for their BACE drug, elen­be­ce­s­tat. But while the bio­mark­er for amy­loid be­ta re­duc­tion was hit, the re­searchers failed to see a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in the de­cline of cog­ni­tion. That didn’t stop them from main­tain­ing that they had tracked a re­sponse “sug­gest­ing a de­lay of clin­i­cal symp­tom de­cline in ex­plorato­ry end­points.”

It wasn’t ex­act­ly a big win, rais­ing fresh doubts for two com­pa­nies that con­tin­ue to have a big stake in the amy­loid be­ta the­o­ry.

Ei­sai and Bio­gen have had their hands full de­fend­ing their da­ta on a dif­fer­ent drug, BAN2401, af­ter a change­up in the study de­sign drew a host of crit­ics sug­gest­ing that they had tilt­ed the tri­al to­ward a pos­i­tive out­come. And the crit­ics haven’t backed off af­ter a re­cent at­tempt to de­fend those re­sults.

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?

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.