Presented at the 30th International Symposium on ALS/MND in Perth, Australia on December 4, 2019.

Background: Trial NCT01906658 was a small, 8-week randomized, open-label trial to evaluate the safety and tolerability of 4 Acthar® gel (repository corticotropin injection, RCI) dosing regimens (16U and 24U daily and 56U and 80U twice weekly) to inform dose selection for future RCI studies in ALS. The study included an optional 28- week open-label extension period plus a 3-week treatment taper and 1-week follow up. The study also investigated the rate of ALSFRS-R change as an exploratory endpoint.

Conclusion: No differences over predicted mortality were apparent in either the full analysis set (FAS) (n = 43) or any of the treatment regimens (n = 10 or 11). Likewise, no group showed significant improvement in VC. The RCI 16U group showed a trend towards a slight improvement, with a possible treatment effect of 1.63% at 8 weeks and 2.28% at 36 weeks and a time to 50% VC Kaplan-Meier curve better than predicted. No patient in the Acthar® 16U group reached the 50% VC threshold.

In contrast, both the FAS and the RCI 16U groups achieved statistical significance for the ALSFRS-R outcome. For the FAS, the possible treatment effect was 1.08pts (week 8) and 3.22pts (week 36). The main driver was the RCI 16U group, which had possible treatment effects of 1.57pts and 4.33pts at 8 and 36 weeks. These results should be interpreted cautiously as the predictions have a tendency for type 1 errors.

Authors: Danielle Beaulieu1, Jonavelle Cuerdo1, Albert A. Taylor1, Susan VanMeter2, Enxu Zhao2, Mike Keymer1, David L. Ennist1
1Origent Data Sciences, Inc., Vienna, VA, USA
2Mallinckrodt Pharmaceuticals, Bedminster, NJ USA

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