Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation - Summary - MDSpire

Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation

  • By

  • Jan Smeitink

  • Just van Es

  • Brigitte Bosman

  • Mirian C H Janssen

  • Thomas Klopstock

  • Grainne Gorman

  • John Vissing

  • Gerrit Ruiterkamp

  • Chris J Edgar

  • Evertine J Abbink

  • Rob van Maanen

  • Oksana Pogoryelova

  • Claudia Stendel

  • Almut Bischoff

  • Ivan Karin

  • Mahtab Munshi

  • Anne Kümmel

  • Lydia Burgert

  • Christianne Verhaak

  • Herma Renkema

  • November 6, 2024

  • 0 min

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Objective:

To evaluate the efficacy and safety of sonlicromanol in adults with the m.3243A>G mutation and primary mitochondrial disease.

Key Findings:
  • Sonlicromanol was well-tolerated with a favorable safety profile.
  • No statistically significant change in the primary endpoint of cognition score was observed.
  • Treatment effects were noted in patients with more affected baseline scores (P = 0.0338).
  • Significant improvements were observed in multiple patient-reported outcomes during the EXT study, including BDI (P = 0.0143) and TAP (P = 0.0102).
Interpretation:

Sonlicromanol demonstrated potential efficacy in improving symptoms related to mitochondrial disease in patients with the m.3243A>G mutation, particularly in those with more severe baseline impairments.

Limitations:
  • Small sample size with only 27 patients randomized, which may limit the generalizability of the findings.
  • Primary endpoint did not reach statistical significance.
Conclusion:

Sonlicromanol shows promise as a treatment for mitochondrial disease linked to the m.3243A>G mutation, particularly in patients with more severe symptoms, warranting further investigation.

Original Source(s)

Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation

  • by Jan Smeitink, Just van Es, Brigitte Bosman, Mirian C H Janssen, Thomas Klopstock, Grainne Gorman, John Vissing, Gerrit Ruiterkamp, Chris J Edgar, Evertine J Abbink, Rob van Maanen, Oksana Pogoryelova, Claudia Stendel, Almut Bischoff, Ivan Karin, Mahtab Munshi, Anne Kümmel, Lydia Burgert, Christianne Verhaak, Herma Renkema

  • November 6, 2024

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