Another Major Drug Trial Failure

[antimatter37]

Biogen stock is down about 30% today as the company abandons its phase three trial for Aducanumab. This was considered to be a potential blockbuster drug for the treatment of AD. The drug is an antibody treatment for the removal of AB. I guess this result should not be a great surprise considering the failure of all other "AB removal" strategies. Sigh.

[broiler_x]

Biogen stock is down about 30% today as the company abandons its phase three trial for Aducanumab. This was considered to be a potential blockbuster drug for the treatment of AD. The drug is an antibody treatment for the removal of AB. I guess this result should not be a great surprise considering the failure of all other "AB removal" strategies. Sigh.

Yeah I saw that. I think they were the last company left trying to treat AD using an anti-amyloid Beta antibody. All the others have failed as well. This came as no surprise to me at all. The bigger surprise is that anyone actually thought this had a chance to work (and obviously a lot of people did, since the stock got hammered so badly on the news - it should have been non-news). I have worked on 2 different anti-ABeta antibody programs at 2 different companies and the last one I personally killed it about 4 years ago. The data from all of the other companies was overwhelmingly negative. I remember a few people at the company thought I was killing it prematurely, but now it looks like a good call. Probably the biggest problem with these drugs is not that they failed, but that they drove a lot of companies away from doing any AD research as it seems like a research black hole. I'm not expecting any help from a miracle pharma drug in my lifetime. The only possibility that I see is perhaps gene therapy to change ApoE4, but that is very far off.

[Fiver]

Disappointing. But now that it resoundingly decided the research community can put the time and money into something else. The CRISPR gene-editing approach looks quite promising.

[mike]

Not sure how CRISPR would work for folks already born... I'm hoping the idea of using a virus to infect us with ApoE2 can help those of us already here - sooner rather than later!

[Fiver]

Hi Mike. It's often a combo approach - viral delivery of CRISPR molecules. Sometimes just one or the other. But, yeah, I agree, some approach to swapping out a problematic gene. Anyway, it will probably take some time to get over this disappointment and refocus.

[mike]

Didn't know "viral delivery of CRISPR molecules" was a thing!? Here is an article discussing both viral and non-viral ways of delivering CRISPR: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6058482/