Annovis Seeks Orphan Drug Status for ANVS401 for Down Syndrome Patients

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by Forest Ray PhD |

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ANVS401, orphan drug application

Annovis Bio is seeking orphan drug status for its investigational therapy ANVS401, known as Posiphen, for treating Alzheimer’s disease in people with Down syndrome (DS-AD).

The clinical-stage drug platform company has filed an application with the U.S. Food and Drug Administration (FDA) for such designation, granted to therapies for rare diseases.

“The FDA orphan drug application for ANVS401 to treat DS-AD is an important milestone for Annovis as we focus on building a robust product pipeline focused on novel uses of AVNS401 to treat significant unmet medical needs,” Maria Maccecchini, PhD, CEO of Annovis Bio, said in a press release.

Alzheimer’s disease affects about 30% of people with Down’s syndrome in their 50s, according to current estimates. By the time individuals with Down are in their 60s, the proportion is nearly 50%.

“We are currently evaluating ANVS401 in two Phase 2a clinical trials targeting [Alzheimer’s] and [Parkinson’s],” Maccecchini said. “The data from these studies will guide us in the continuing development of ANVS401 for chronic neurodegenerative diseases — specifically in DS-AD and in [Parkinson’s].”

The two Phase 2a trials are assessing the therapy’s safety and tolerability, as well as changes in disease-related biomarkers. Changes in the participants’ functional impairment, and cognitive, mental, and other neuropsychiatric symptoms also are being evaluated.

The DISCOVER study (NCT02925650) was designed to involve up to 24 adults with early Alzheimer’s at six locations within the United States. It may still be recruiting.

A dose-finding and biomarker evaluation study (NCT04524351) is enrolling up to 68 participants, ages 45 and older, at 13 sites across the U.S. This trial includes both Alzheimer’s and Parkinson’s patients. 

Posiphen is designed to reduce the formation of toxic clumps of misshapen proteins that damage nerves in both Alzheimer’s and Parkinson’s. In Alzheimer’s, it targets the beta-amyloid and tau aggregates, or clumps, that form in patients’ brains. It also targets the alpha-synuclein clumps that form in the brains of people with Parkinson’s.

Annovis hopes Posiphen can prove therapeutic in both disorders.

Interim results from the DISCOVER study showed that Posiphen led to improvements in patients’ speed and coordination. The first three patients recruited into the combined Alzheimer’s and Parkinson’s study recently received their first doses of ANVS401.

Preclinical studies found that Posiphen fully restored function lost to disease. This included memory and learning in mouse models of Down syndrome and Alzheimer’s, and in rat models of traumatic brain injury.

Based on these results, Annovis believes that Posiphen can “change the course” of Alzheimer’s in people with Down syndrome.

Depending on FDA approval of the orphan drug status application, the company hopes to initiate a Phase 3 trial among DS-AD individuals by year’s end.

Orphan drug designation is a way to help promising rare disease therapies reach market faster through certain development incentives. These include additional support from the FDA, tax credits, waivers for FDA administrative fees, and a seven-year period of marketing exclusivity, should the treatment win approval.