Danielle Beaulieu, Jonavelle Cuerdo, Mark Schactman, David L. Ennist, Albert Taylor

The poster will be presented at the MDA Clinical & Scientific Conference on March 13-16 2022.

Background:ALS drug development has been plagued by high clinical trial failure rates. Subgroup analysis is
a key tool used to account for patient heterogeneity, but current methods fall short. DEC analysis
systematically groups and analyzes patients based on predicted disease path, creating more
homogeneous patient subgroups with reduced noise around the endpoint.

Conclusions: DEC analysis organizes clinical trial participants into subgroups that are more homogeneous than
the full trial population:

  • Method reveals “hot spots” of detectable treatment effects that could form the basis for a
    subsequent successful clinical trial
  • Numerous ways to implement this approach are envisioned:
    • Retrospective rescue of drugs that failed late-stage clinical development
    • Prospective use in platform and adaptive trials
    • Use of DEC analysis post-hoc as a complement to or in place of current subgroup analysis
      methods (2)
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