Phase 2 Safety Trial of AT-1501 Nears Full Enrollment of Patients

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by Vanda Pinto PhD |

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AP-101 | ALS News Today | AL-S Pharma AG | illustration of clinical trial meds

A Phase 2 study of the investigational antibody AT-1501 in treating amyotrophic lateral sclerosis (ALS) has completed patient enrollment in three of its four dosing groups, and is likely to be fully enrolled by year’s end, its developer, Eledon Pharmaceuticals, announced.

A total of 54 ALS patients diagnosed in the previous two years are expected to participate in this safety and tolerability trial (NCT04322149). Recruitment is ongoing at 12 sites in the U.S. and one in Canada (Montreal). Additional contact and location information is available here.

These updates were presented at the 2021 Annual Northeast Amyotrophic Lateral Sclerosis (NEALS) meeting, held virtually Oct. 6–7, in the poster, “A Phase 2 Safety and Tolerability Study of an Anti CD40L Antibody, AT-1501 in Adults with ALS.”

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AT-1501 is a lab-made antibody designed to block a protein called CD40 ligand (CD40L). Binding of CD40L to the CD40 receptor of some immune cells has been shown to promote pro-inflammatory responses.

In animal models of ALS, blocking CD40L was found to delay disease onset and extend survival. A Phase 1 single-ascending dose trial in healthy volunteers and ALS patients reported that AT-1501 was safe and well tolerated at all doses tested.

The ongoing Phase 2 trial is an open-label (no placebo group) trial investigating the safety and tolerability of multiple ascending doses of AT-1501. Each patient will receive six into-the-vein (IV) infusions, given every other week for 11 weeks, and all will be monitored for treatment safety and tolerability for another eight weeks. The trial is expected to last about five months.

The trial was designed to include four groups of patients, each testing one of four doses — 1, 2, 4, and 8 mg/kg — of AT-1501.

According to Eledon, patients in the 1 mg/kg and 2 mg/kg groups (nine in each) have already completed the study, and the group receiving the 4 mg/kg dose has been fully enrolled (18 patients) since July. A total of 18 patients are expected to enroll in the highest dose group by the end of the year.

In addition to safety and tolerability, investigators will also assess AT-1501’s effect on certain markers of inflammation — such as TNF-alfa, MCP-1, IL-6, and IL1-beta — and on the levels of neurofilament heavy and light chain proteins, markers of neurodegeneration.

How treatment affects lung function and the ability to perform daily living activities will also be assessed using the ALS Functional Rating System-Revised (ALSFRS-R) and spirometry tests.