Phase 2 Trial of Tauroursodeoxycholic Acid for Alzheimer Disease Completes Enrollment

  • Alzheimer Disease
  • Early onset Alzheimer disease
  • Taurursodiol-phenylbutyrate

Enrollment for the phase 2 Pegasus trial (NCT03533257) assessing tauroursodeoxycholic acid (TUDCA) /sodium phenylbutyrate (PB) combination (AMX0035; Amylyx Pharmaceuticals, Cambridge, MA) in individuals with Alzheimer disease (AD) has been completed, and 96 of the previously planned 100 participants have received a dose.

The Pegasus trial will provide data on the safety and tolerability profile of TUDCA/PB in individuals with AD, biological activity on biomarkers related to disease processes, and preliminary information on cognitive and functional effects of TUDCA/PB. Trial collaborators include the Alzheimer Association, the Alzheimer Drug Discovery Foundation (ADDF) and Cure Alzheimer Fund.

In April 2019, the Pegasus trial’s planned enrollment was doubled to approximately 100 participants with additional funding support from Cure’s Alzheimer Fund. The trial was first supported in 2017 through the Alzheimer Combination Therapy Opportunities (ACTO) program, a joint research funding initiative created by the Alzheimer Association and the ADDF.

Pegasus is a 3:2 randomized double-blind multicenter placebo-controlled study evaluating the safety, tolerability, and activity of TUDCA/PB in participants with late mild cognitive impairment or early dementia of AD over 24 weeks. The biomarker-focused trial design will assist in understanding the effects of TUDCA/PB and its potential for treating AD.

“AD is a complex, progressive brain disorder with no disease-modifying treatments that impact the neurodegenerative process,” said Steven E. Arnold, MD, Translational Neurology head of the Interdisciplinary Brain Center at MA General Hospital and Harvard Medical School and the Pegasus trial’s principal investigator. “Due to COVID-19, we must pay close attention to the safety of older adults whose associated health conditions and age may pose a risk. We are pressing ahead safely as we expect the trial will generate meaningful insights about the ability of AMX0035 to target mechanisms involved in the progression of Alzheimer.”

“AD remains one of the most critically unmet needs in healthcare and we desperately need new treatments for patients,” said Rudolph Tanzi, PhD, Kennedy professor of Neurology, Massachusetts General Hospital and Harvard Medical School, chair of the Amylyx Scientific Advisory Board and chair of the Cure Alzheimer Fund Research Leadership Group. “AMX0035 recently showed benefit in patients with ALS, and the same mechanism of neuroprotection may also be helpful in AD. We are very fortunate that the Pegasus trial has continued, despite COVID-19, and eagerly await data in Q1 2021.”

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