High Purity 95% White Powder Fosgonimeton CAS 2093305-05-4 Peptide

Fosgonimeton More Info

Fosgonimeton is an investigational small molecule prodrug designed to positively modulate the hepatocyte growth factor (HGF) system, an endogenous repair mechanism for a healthy nervous system. It is being explored for its potential to treat neurodegenerative diseases like Alzheimer's, Parkinson's, and dementia with Lewy bodies.

Mechanism of Action

Fosgonimeton is converted into its active metabolite, fosgo-AM (also known as ATH-1001), which crosses the blood-brain barrier and enhances the interaction of HGF with its receptor tyrosine kinase MET. This activation induces downstream signaling through PI3k/Akt and MAPK pathways and augmenting NMDA receptor-mediated long-term potentiation through protein kinase C. By positively modulating the HGF/MET system, fosgonimeton aims to promote neuronal health and function, potentially addressing neurodegeneration in dementia.

Preclinical Studies

Preclinical studies of fosgo-AM have demonstrated neurotrophic and neuroprotective effects in primary rat neurons, reversing scopolamine-induced learning deficits in rats. It has also shown the ability to protect cortical neurons from amyloid-ß-induced toxicity.

Clinical Trials

Fosgonimeton has been evaluated in multiple clinical trials:
Phase 1 Trial: A Phase 1 clinical trial found fosgonimeton to be generally safe and well-tolerated in both healthy volunteers and individuals with Alzheimer's disease. The most common side effects were mild pain and itching at the injection site.
Phase 2 ACT-AD Trial: In the Phase 2 ACT-AD trial, fosgonimeton was well-tolerated, with no serious adverse events reported. However, a higher incidence of mild-to-moderate adverse events, such as injection site reactions, transient eosinophilia, and dizziness, were observed in participants treated with fosgonimeton compared to placebo. AEs led to an overall discontinuation rate of 14.3%.
Phase 2/3 LIFT-AD Trial: Top-line data from the Phase 2/3 LIFT-AD trial showed that fosgonimeton failed to significantly improve cognitive and daily functioning in adults with mild to moderate Alzheimer’s disease, despite being safe and well-tolerated.

Safety and Tolerability

Fosgonimeton has generally been well-tolerated in clinical trials, with a favorable safety profile. The most common side effects are injection site reactions, which are generally mild and resolve on their own. Serious adverse events and deaths were not reported in the ACT-AD trial.

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