{"entity":{"id":"paper-garralda-nat-med","kind":"paper","name":"MYC targeting by OMO-103 in solid tumors: a phase 1 trial","aka":[],"tldr":"Paper cited by one idea page, indexed on Europe PMC as PubMed record 38321218 and published in Nature Medicine; the citing page links this DOI, which is how the record was matched.","summary":"Among the 'most wanted' targets in cancer therapy is the oncogene MYC, which coordinates key transcriptional programs in tumor development and maintenance. It has, however, long been considered undruggable. OMO-103 is a MYC inhibitor consisting of a 91-amino acid miniprotein. Here we present results from a phase 1 study of OMO-103 in advanced solid tumors, established to examine safety and tolerability as primary outcomes and pharmacokinetics, recommended phase 2 dose and preliminary signs of activity as secondary ones. A classical 3 + 3 design was used for dose escalation of weekly intravenous, single-agent OMO-103 administration in 21-day cycles, encompassing six dose levels (DLs). A total of 22 patients were enrolled, with treatment maintained until disease progression. The most common adverse events were grade 1 infusion-related reactions, occurring in ten patients. One dose-limiting toxicity occurred at DL5. Pharmacokinetics showed nonlinearity, with tissue saturation signs at DL5 and a terminal half-life in serum of 40 h. Of the 19 patients evaluable for response, 12 reached the predefined 9-week time point for assessment of drug antitumor activity, eight of those showing stable disease by computed tomography. One patient defined as stable disease by response evaluation criteria in solid tumors showed a 49% reduction in total tumor volume at best response. Transcriptomic analysis supported target engagement in tumor biopsies. In addition, we identified soluble factors that are potential pharmacodynamic and predictive response markers. Based on all these data, the recommended phase 2 dose was determined as DL5 (6.48 mg kg -1).ClinicalTrials.gov identifier: NCT04808362.\n\nIndexed on Europe PMC as PubMed record 38321218 (DOI 10.1038/s41591-024-02805-1). Matched by DOI alone: one idea page cites this DOI among its external links (the pages are listed under Related), and this page was written so that the citation resolves inside OnCo. No figure has been checked by an editor.","asOf":"2026-09-22","links":[{"label":"Nat Med 2024","url":"https://doi.org/10.1038/s41591-024-02805-1"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/38321218/"},{"label":"Europe PMC","url":"https://europepmc.org/article/MED/38321218"}],"tags":["europepmc-ingest"],"related":["idea-drugging-myc"],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["nature-medicine"],"dependsOn":[],"notes":[],"journal":"Nature Medicine","year":2024,"doi":"10.1038/s41591-024-02805-1","pmid":"38321218","authors":"Garralda E, Beaulieu ME, Moreno V, et al.","paperType":"observational","findings":[],"whatItMeans":"One idea page on OnCo cites this paper by its DOI; this record gives the citation a page of its own so a reader can follow it without leaving OnCo. Read the abstract above alongside the citing page listed under Related; the record was created automatically from the Europe PMC entry and its figures have not been checked by hand.","caveats":["Matched to the citing OnCo records by DOI alone; the summary reproduces the Europe PMC abstract and no figure has been verified against the full paper."]},"route":"/key-papers/paper-garralda-nat-med/","neighbours":{"idea":[{"id":"idea-drugging-myc","kind":"idea","name":"Can MYC be drugged directly, and will patients tolerate it?","route":"/ideas/idea-drugging-myc/"}],"journal":[{"id":"nature-medicine","kind":"journal","name":"Nature Medicine","route":"/journals/nature-medicine/"}]}}