ATL1102 for multiple sclerosis

What was ATL1102 for MS?

ATL1102 is an experimental therapy for relapsing forms of multiple sclerosis (MS), but its development appears to have been stopped.

The therapy, developed by Antisense Therapeutics, now Percheron Therapeutics, was designed to be given via twice-weekly subcutaneous, or under-the-skin, injections. In a Phase 2a clinical trial, ATL1102 significantly reduced the number of brain lesions, suggesting it could help limit disease activity.

After completing the MS trial, the company decided to shift its focus to Duchenne muscular dystrophy (DMD), a genetic disorder that causes progressive muscle weakness, indicating the future of ATL1102 for MS would depend on the drug’s success in DMD. However, a Phase 2b trial in DMD was stopped in December 2024 after failing to meet its main goal. Percheron’s website currently makes no mention of ATL1102, suggesting the program has been fully discontinued.

ATL1102 is an antisense oligonucleotide that was designed to reduce the production of CD49d, a component of the adhesion molecule VLA-4 found on the surface of immune cells. VLA-4 helps immune cells move from the bloodstream into the brain and spinal cord, where they can trigger inflammation and cause damage to nerve fibers in MS.

By lowering CD49d levels, ATL1102 was expected to prevent immune cells from reaching the brain and spinal cord, helping to reduce inflammation. It targeted the same biological pathway as Tysabri (natalizumab), an approved therapy for relapsing forms of MS that blocks VLA-4 activity to reduce disease activity.

Therapy snapshot

Treatment name ATL1102
Administration Subcutaneous injection
Clinical testing Development appears to have been discontinued

 

ATL1102 in MS clinical trials

ATL1102 was investigated in a Phase 2a trial (ACTRN12608000226303) in Australia, which enrolled 77 adults with relapsing-remitting MS (RRMS). Participants received ATL1102 or a placebo, given three times in the first week and twice weekly for the next seven weeks, and were then followed for another eight weeks without treatment. Results showed:

  • ATL1102 significantly reduced by 54.4% the cumulative number of new active lesions at weeks 4, 8, and 12 — an assessment that combines the number of active lesions as well as new or enlarging lesions at all three time points.
  • The cumulative number of lesions with active inflammation at the three assessments was also significantly lower in the ATL1102 group, by 66.7%.
  • Relapse rates and disability levels were similar in both groups.

Following the promising Phase 2a data, the company started making plans for additional clinical trials of ATL1102 in MS, including a Phase 2b trial for people with RRMS and secondary progressive MS. However, no further updates were provided about any of these studies since 2017.


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FAQs about ATL1102

ATL1102 is an investigational therapy that was expected to reduce inflammation and disease activity in people with relapsing forms of multiple sclerosis. However, despite promising results in an early Phase 2a clinical trial, the therapy’s development seems to have been discontinued.

While ATL1102 significantly reduced the number of lesions in a Phase 2a trial, and a Phase 2b trial was authorized by the U.S. Food and Drug Administration in 2017, the medication is no longer listed in the developer’s website, suggesting its development program has been fully discontinued.

Clinical trials of ATL1102 in multiple sclerosis did not include participants who were pregnant or breastfeeding, so it is not known whether the treatment is safe in these situations.

In a Phase 2a clinical trial, reductions in active or new or enlarging lesions were evident as early as after eight weeks of treatment with ATL1102. However, individual responses to treatment can vary, so patients should always ask their doctor how a medication might affect them.

Neither hair loss nor weight gain were reported as side effects of ATL1102 in multiple sclerosis clinical trials. Patients who experience unexpected side effects while on a new medication should talk with their healthcare team.