Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation - Takeaways - 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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  • 1

    Sonlicromanol is a promising treatment for mitochondrial disease linked to the m.3243A>G mutation, targeting metabolic and inflammatory pathways.

  • 2

    The phase 2b study included a randomized controlled trial followed by a 52-week open-label extension to assess safety and efficacy.

  • 3

    Patients reported good tolerability and favorable safety, with pharmacokinetic results consistent with earlier studies.

  • 4

    Statistically significant improvements were observed in various cognitive and quality of life measures during the extension study.

  • 5

    Sonlicromanol demonstrated a favorable benefit/risk ratio and was effective in patients with more severe baseline symptoms.

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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