First Patients Dosed in Trial of Two-Drug Combination Therapy for Alzheimer's Disease

The first patients were dosed in a Phase 2 clinical trial to assess AMX0035 (Amylyx Pharmaceuticals, Inc., Cambridge, MA) in patients with Alzheimer’s disease. AMX0035 is a two-drug combination therapy in development to treat patients with late, mild cognitive impairment or early dementia.  

The 24-week PEGASUS trial (NCT03533257), which will enroll 100 participants, is a 3:2, randomized, placebo-controlled study to evaluate the safety, tolerability, and activity of AMX0035. The therapy combines two existing compounds—sodium phenylbutyrate and tauroursodeoxycholic acid—that act synergistically to prevent the nerve cell death and neurotoxic inflammation that are characteristic of Alzheimer’s disease.

“The rationale for combination therapies has already been demonstrated in diseases like HIV/AIDS, cancer, and heart disease,” said Maria C. Carrillo, PhD, chief science officer, Alzheimer’s Association. “We believe this type of approach will also work for Alzheimer’s and other dementias and are encouraged by the launch of this combination therapy trial.”