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RTX-111

Development stage: Unknown
Lead developer: Repeat Therapeutics
Modality: Small Molecules
Administration: Oral

Overview

**RTX-111** is an oral investigational fixed-dose combination therapy developed by Repeat Therapeutics for **C9orf72-mediated amyotrophic lateral sclerosis**. It comprises three small-molecule medications, including the two-component regimen RTX-101 and RTX-010. Repeat Therapeutics describes RTX-111 as designed to restore cellular balance and reduce neurotoxic stress in motor neurons; however, the individual active ingredients and specific molecular targets have not been publicly disclosed. RTX-111 is being evaluated in Phase 1 safety, tolerability, and pharmacokinetic studies in healthy adults and in adults with C9orf72-mediated amyotrophic lateral sclerosis. ([anzctr.org.au](https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?ACTRN=12625001215415))

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