Trial of AJ201 in SBMA fully enrolled, results expected this year

Marisa Wexler MS avatar

by Marisa Wexler MS |

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A Phase 1b/2a clinical trial testing the experimental therapy AJ201 in people with spinal and bulbar muscular atrophy (SBMA) has finished enrolling patients and topline data are expected in the first half of 2024.

The milestone was announced by Avenue Therapeutics, which last year acquired rights to develop AJ201 in the U.S., European Union and select other regions from the therapy’s original developer AnnJi Pharmaceutical.

“We are pleased with the speed of enrollment in our Phase 1b/2a study evaluating AJ201 for the treatment of SBMA, reflecting the large unmet need in this debilitating neurodegenerative disease,” Alexandra MacLean, MD, Avenue’s CEO, said in a company press release. “We are intently focused on advancing this much needed drug for patients and their caregivers, and we look forward to reporting topline data in the second quarter of 2024.”

The Phase 1b/2a clinical trial (NCT05517603) enrolled 25 adults with SBMA who are being randomly assigned to take AJ201 at 600 mg a day or a placebo for 12 weeks (about three months).

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How does AJ201 work on SBMA?

The study’s main goals are to assess the safety profile and pharmacological properties of AJ201. Scientists will also assess biological data to see if the investigational treatment appears to be working as intended.

SBMA, also called Kennedy’s disease, is a rare type of SMA caused by mutations in the AR gene, which provides instructions for making the androgen receptor (AR) protein. SBMA-causing mutations lead to an abnormal form of the protein being produced that forms clumps in nerve and muscle cells, disrupting their function and leading to progressive cell dysfunction and death that causes the disease’s symptoms.

AJ201 is designed to activate multiple biological pathways that may be beneficial in SBMA by targeting three different proteins —  Nrf1, Hsf1, and Nrf2. By activating them, it’s thought that AJ201 may help cells clear out clumps of abnormal AR protein and help protect cells from oxidative stress, a type of cell damage that may contribute to nerve cell death in many neurological diseases, including SBMA.

A Phase 1 study (NCT04392830) that enrolled 72 healthy adult volunteers found AJ201 was generally well tolerated.

AJ201 has received orphan drug status from the U.S. Food and Drug Administration (FDA) for SBMA, Huntington’s disease, and spinocerebellar ataxia. The designation is granted to treatments that target conditions that impact fewer than 200,000 people in the U.S. and offers various advantages, including exemption from FDA fees and seven years of exclusive market rights upon approval.

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