The neurodegenerative disease landscape is undergoing a radical transformation. Breakthroughs in cell regeneration, AI diagnostics, and novel therapies are igniting a new era of Alzheimer’s and dementia treatments. Eli Lilly’s Donanemab has shown significant potential to improve Alzheimer’s disease symptoms. Consequently, it is on track for FDA approval in Q2 2024. Donanemab targets plaques in the brain caused by beta-amyloid protein, the same mode of action as the 2023 FDA-approved Lecanemab.

Why It Matters

The emergence of treatments like Lecanemab and Donanemab mark a critical development for over 55 million patients living with dementia globally.

• The number of Alzheimer’s disease patients is projected to double in the next two decades.
• Donanemab aims to prevent the progression of the disease rather than reverse existing damage. Subsequently, it is primarily suitable for patients in early to mild stages of the disease.
• Advancements in neurogenesis-based therapies are gaining attention for Alzheimer’s and dementia treatments. They are aiming not only to manage symptoms but to potentially reverse some aspects of cognitive decline. Therefore, these medications are suitable for patients at more advanced stages.

State of Discovery

Biogen’s Aducanumad (Aduhelm) and Eisai/Biogen’s Lecanemab (Leqembi) target amyloid plaques to slow disease progression. These were the first significant FDA-approved therapies for Alzheimer’s in over 20 years. However, it was unclear whether Aducanumad slowed down the loss of memory and thinking so many doctors refused to prescribe it and Medicare declined to cover it. Biogen stated in early 2024 it would discontinue commercialization of Aducanumad to focus on Leqembi and other treatments in development.

What We’re Watching

• TauRX Pharmaceuticals is pursuing FDA approval for their second-generation tau aggregation inhibitor. It has shown encouraging outcomes in Phase III trials, including slowed cognitive decline and a favorable safety profile.
• Neuralstem’s small molecule NSI-189 has demonstrated potential in preclinical studies for ameliorating cognitive decline.
• Brainstorm Cell Therapeutics’ treatment ‘NurOwn’ has entered Phase II clinical trials. This signals the exciting potential for neurogenesis and neuronal regeneration approaches. These therapies may hold the key to promoting the restoration of cognitive function.

AI Breakthroughs

AI technologies are revolutionizing Alzheimer’s disease research, helping to advance the discovery of new diagnostics, treatments, and biomarkers.

• Winterlight Labs uses AI technology to analyze narrative speech for early detection of Alzheimer’s Disease.
• Cognetivity Neurosciences is utilizing AI to assess cognitive function. They recently partnered with UC Health’s Gardner Neuroscience Institute to screen patients for early signs of disease.
• Insilico Medicine, in collaboration with a Cambridge (UK) research group, is developing an AI-driven tool for identifying therapeutic targets.
• Lastly, a team at Massachusetts General Hospital is advancing Alzheimer’s detection by training an AI model to recognize patterns in brain MRIs.

Bench to Bedside

Biopharmaceutical Commercialization Immersion is our 8-hour on-demand course that explores the strategic aspects of bringing a drug to market and maximizing its commercial potential. It showcases the different phases of the product life cycle and the real-world decisions that have a profound impact on a drug’s success. Learn how therapeutic antibodies, such as Lecanemab and Donanemab, and small molecule drugs like tau aggregate inhibitors go from bench to bedside.

The Bottom Line

Advancements in early-stage treatments for neurodegeneration are showing significant promise. The integration of AI for early detection could critically improve the clinical benefits of these Alzheimer’s and dementia treatments. Therapies aimed at promoting neurogenesis, represent a potentially transformative approach for Alzheimer’s patients. Particularly, these drugs offer the possibility of not just halting but reversing aspects of cognitive decline.