Phase 2 Trial of AT-1501 in Slowing Progression Nears Full Enrollment

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by Teresa Carvalho |

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Eledon Pharmaceuticals announced that its Phase 2 study of AT-1501 in people with amyotrophic lateral sclerosis (ALS) should be fully enrolled by year’s end.

Top-line trial results are expected between April and September 2022.

“Enrollment in our ALS study is progressing well and we anticipate completing enrollment in the 4th quarter,” David-Alexandre C. Gros, MD, CEO of Eledon, said in a press release.

The trial (NCT04322149), which enrolled its first patient late last year, is recruiting up to 54 ALS patients across multiple sites in the U.S. and one in Montreal, Canada. Contact and location information is available here.

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AT-1501 is a lab-made antibody that targets a protein called CD40 ligand (CD40L), which has a key role in controlling the immune response. Researchers have observed that the CD40L pathway is excessively active in people with ALS.

While the exact mechanism of action of AT-1501 in ALS is not completely known, it is believed that it can block or delay the activation of the inflammatory immune response by blocking CD40L. In this way, AT-1501 could delay ALS onset and slow its progression, improving patient outcome.

The ongoing Phase 2 trial is an open-label, multiple-dose study assessing the safety and tolerability of AT-1501, as well as determining the levels of biomarkers of inflammation and ALS progression.

Patients are given one of four ascending doses of AT-1501, administered into a vein (IV infusion). Each participant will receive six infusions, given every other week for 11 weeks, and all will be monitored for treatment safety and tolerability for another eight weeks. The study, which lasts a total of 19 weeks (about five months), is expected to finish in March 2022.

A Phase 1 single-ascending dose trial in healthy volunteers and ALS patients, which finished in 2019, found that AT-1501 was safe and well tolerated at all doses tested.

The Food and Drug Administration (FDA) designated AT-1501 an orphan drug in 2018 for its potential to slow ALS onset and progression. This status provides benefits and incentives to advance its development, such as waiving FDA application fees and assistance in clinical trial design. Should it be approved, the therapy will also have seven years of U.S. market exclusivity.

AT-1501 is also being tested for its potential to treat other disorders, including autoimmune diseases like IgA nephropathy, an autoimmune kidney disorder, and to prevent transplant rejection.

Eledon Pharmaceuticals was previously known as Novus Pharmaceuticals; it took over developing AT-1501 in 2019 when it acquired Anelixis Therapeutics.