Alzheimer’s Drug Development Overview

June is Alzheimer's & Brain Awareness Month — and the spotlight is on innovation. Let’s take a closer look at where we stand with Alzheimer’s disease (AD): the burden, the breakthroughs, and what’s next in the pipeline.

First, a few facts (via Alzheimer’s Association & Alzheimer’s Disease International):

• AD kills more people than breast and prostate cancer combined.
• In the US alone, over 7 million people live with AD
• Two-thirds of people living with AD are women.
• The global cost of dementia has surpassed $1.3 trillion, expected to hit $2.8 trillion by 2030.

Drug development has been slow, but the tide may be turning. From 2003 to 2021, no new AD therapies were approved in the US or EU. The reentry point came with Biogen’s aducanumab, followed by two more amyloid-targeting agents in the US. However, only one of those has been approved by the EMA (Leqembi, 2023). While these treatments have opened new doors, their risk profile (notably ARIA – amyloid-related imaging abnormalities) and costly monitoring requirements highlight the ongoing need for safer, more effective, and affordable options.

What’s exciting now?

Global pipeline (excluding China & Russia): 473 drugs in total

• 296 in Preclinical
• 80 in Phase 1
• 76 in Phase 2
• 19 in Phase 3
• 2 already in Pre-registration!

Two front-runners worth watching closely:

1. TauRx is seeking UK approval for hydromethylthionine mesylate (HMTM) – the only oral tau-targeting (tau aggregation inhibitor to be exact) therapeutic to finish Phase 3 trials. If approved, it could offer a new option focused on slowing disease progression.
2. Anavex Life Sciences’ blarcamesine (ANAVEX 2-73) – EMA is currently reviewing its marketing authorization application. This small molecule activates the SIGMAR1 receptor, aiming to restore cellular homeostasis and boost autophagy – mechanisms beyond amyloid and tau.

Not just amyloid anymore: Phase 3 candidates show MoA diversity.

From tau aggregation inhibitors to glucagon-like peptide-1 (GLP-1) receptor agonists, tyrosine kinase inhibitors, and even gingipain inhibitors, the field is expanding beyond the traditional beta-amyloid focus.

Most trials target mild to moderate AD, where disease-modifying potential is still within reach.

So what’s the takeaway?

The Alzheimer’s pipeline is evolving – not just in size, but in scientific strategy. It’s a promising sign that our collective understanding of AD is deepening, and with it, the hope for more effective and accessible treatments. P.S. maybe then the Likelihood of Approval of drugs for AD in Phase 1 will become higher than just 2%.

Want to explore what this means for your pipeline, investment, or portfolio? Get in touch today!