Results from Phase 1 HATTR Amyloidosis Trial Presented at Recent Meeting

According to a story from, the clinical company Prothena Corporation plc recently presented encouraging results from its phase 1 clinical trial. This trial was evaluating its investigational product candidate PRX004 as a treatment for neuropathy associated with hATTR amyloidosis, a rare disease. The findings were presented at the 2021 Virtual Annual Meeting of the American Academy of Neurology. The results suggest that this therapy could slow the progression of neuropathy in people living with the disease.

About HATTR Amyloidosis

HATTR Amyloidosis is a disease which is characterized by the buildup of amyloid fibrils in body tissue. Amyloid fibrils are a type of abnormal protein. The disease is caused by a heritable genetic mutation. Symptoms of the illness include swelling, heart failure, irregular heartbeat, shortness of breath, fatigue, weight loss, easy bruising and bleeding, stroke, lung problems, enlarged liver, and changes in skin color. Treatment approaches for hATTR amyloidosis include liver transplant; This can actually cure the disease, but the procedure carries substantial risks and side effects. This form of amyloidosis has a better prognosis than some of the more common types with some patients surviving for over a decade. It is estimated to affect around 50,000 people worldwide. To learn more about amyloidosis, click here.

About The Trial

This phase 1 trial was a dose escalation study which included 21 patients. There were a total of six different dose cohorts in the trial: 0.1, 0.3, 1, 3, 10, and 30 mg/kg. There was also a long-term extension portion of the study which patients could opt for once they had completed the dose escalation cycle; ultimately 17 of the patients would opt for the extension portion. At nine months, seven of the patients in the study were considered evaluable. 

These patients all saw a slowing of neuropathy symptom progression as determined by Neuropathy Impairment Score (NIP) at nine months. Treated patients saw their score increase by a mean of 1.29 points, compared to 9.2 points in a placebo group. Three of the seven patients actually saw their neuropathy improve, losing a mean of 3.33 points to their NIS. All of these patients also saw improved cardiac systolic function.

About PRX004

Overall, the results bode well for the continued development of PRX004 in this disease. It is a monoclonal antibody designed to clear excess amyloid protein (which appears as extracellular deposits) as found in ATTR amyloidosis. 

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