Phase 1 Trial / MTI-301

December 2025

Modulation Therapeutics is pleased to announce the activation of a Phase 1 Clinical Trial to evaluate the safety, tolerability, pharmacokinetics and activity of MTI-301 in patients with advanced solid tumor malignancy.

MTI-301 is an orally administered novel small molecule drug that inhibits an enzyme called stearoyl CoA desaturase 1 (SCD1). SCD1 promotes monounsaturated fatty acid (MUFAs) synthesis in cancer cells.  Tumor growth, metastasis, and drug resistance in multiple cancer types have been associated with MUFAs. In pre-clinical studies, MTI-301 has been shown to inhibit the activity of SCD1 and reduced tumor growth and/or spread.

The MTI-301 Phase 1 clinical trial entitled “Phase 1 trial of SCD I: A First in Human Dose Regimen-Finding Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Activity of MTI-301 in Patients with Advanced Malignancy" is supported in part by a NIH Grant (R44CA272064).

More information on the Phase 1 clinical trial with MTI-301 can be found at: "www.clinicaltrials.gov" (Trial Identifier: NCT06911008); “www.cancer.gov” (Trial Identifier; NCI-2025-01930; “www.mayo.edu/research/clinical-trials” (Trial Identifier MC210103).

MTI-201 Phase I Clinical Trial

Targeted Alpha Particle Radiotherapy for Metastatic Uveal Melanoma

This study will enroll patients with metastatic uveal melanoma that have failed at least one form of therapy from a single academic medical center in the United States. All participants will be informed about the study and potential risks and required to provide written informed consent prior to undergoing study-related procedures. A continual reassessment method (CRM) design will be used for this clinical trial. The study proposes single patient cohorts with dose escalation starting at 4.7 microCi of 225Ac-MTI-201 after each cohort in the absence of safety concerns (2- fold increases for doses and lower dose increases between higher doses). Dose Limiting Toxicities will be assessed using the CTCAE version 5.0 criteria.

The participants who meet the eligibility requirements will be administered a single intravenous dose of 225Ac-MTI-201. After study treatment at the Moffitt Cancer Research Center in Tampa, Florida, the study participants will stay overnight at the study center, undergo study procedures(i.e. vital signs, physical exam, multiple blood and urine sample collections) and will be scheduled to return to the clinic at 48 hours and for additional appointments of weekly clinic visits during the first month and on Week 9 for health status assessments, including physical exams, complete blood chemistry, and EKG. Tumor measurements every 8 weeks in first year post-injection; extended to 12 weeks in year 2; every 16 weeks in year 3, and 24 weeks in years 4 and 5. The clinic visits will involve seeing a study doctor plus radiological tests (such as MRI and/or CT scans) to see how the metastatic uveal melanoma has responded to the study drug.

Learn more about the trial

Modulation Therapeutics Announces receipt of a peer reviewed Fast Track SBIR award

February 2023

Modulation Therapeutics Announces receipt of a peer reviewed Fast Track SBIR award from the National Institutes of Health and the National Cancer Institute to complete research with MTI-301, a First in Class SCD-1 inhibitor for treating multiple cancers.

The FastTrack SBIR award will be funded in 2 milestone driven phases totaling up to $2.4M supporting both preclinical and first in human phase I clinical studies to assess the safety, tolerability, and pharmacokinetics of MTI-301, a Stearoyl CoA desaturase 1 (SCD1) inhibitor, as a therapeutic strategy for patients with advanced solid tumors in breast, liver, gastrointestinal and genitourinary cancers.