Phase II trial of anti-amyloid AR1001 signals little promise for patients with Alzheimer’s disease, says GlobalData

Following the news that AriBio announced the completion of a Phase II clinical trial of AR1001;

Sarah Elsayed, MBA, Neurology Analyst at GlobalData, a leading data and analytics company, offers her view:

“Given the complexity in developing disease-modifying treatments (DMTs) in the Alzheimer’s Disease (AD) market, coupled with the high rate of late-stage clinical trial failures, the advancement of AR1001 in trials  signals a little promise for patients, and provides guidance for future AD trials.

“Although the preliminary results of this study are expected to be announced by the end of Q1 2021, the company is planning to initiate a global Phase III trial in 2022. This means that the drug filing in the US might be expected in Q1 2024 – if it succeeded in both trials. This will be an uphill struggle for AR1001. Pipeline agents targeting amyloid beta (Aβ), have repeatedly failed in previous trials which has led to suggestions to shift focus from the amyloid hypothesis to combination therapies. However, some have argued that anti-amyloid treatment should be only studied in the very early stages of AD, which seems to be the case in AR1001 Phase II trial.

“Key Opinion Leaders (KOLs) interviewed by GlobalData did not believe there will ever be one product that can cure AD. Rather, they consistently expressed that the future of AD treatment will entail combinatory use of preventative, symptomatic, and disease-modifying products. Consequently, AriBio must overcome some common challenges in the future trials regarding aspects of trial design, biomarkers and targeting the optimal phase of the disease in AD patients.

“Benefits of AR001 include a mechanism of action (MOA) that is novel compared to other treatments available in the landscape, and its oral administration will have positive impact on patient compliance. The drug acts by inhibiting phosphodiesterase 5 (PDE5) protein, which is believed to have benefits against the accumulation of Aβ peptides, the main pathological hallmark of AD. Therefore, it had showed potential evidence to promote neuronal cell survival in AD during early-stage studies. Considering that there are no approved treatments that can halt or slow down the progression of neurodegeneration in AD, AR1001 still has an opportunity to be this much-needed therapy should it proves to be effective and tolerable.”

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