A Phase 1 Multicenter Open-Label Dose-Escalation Study to Assess Safety Tolerability and Efficacy of EXS74539 as Monotherapy in Participants with Select Solid Tumors

Brief description of study

This is a Phase 1 study testing a new oral drug called EXS74539 in patients with small cell lung cancer (SCLC) and other selected solid tumors. EXS74539 is an experimental cancer drug that is being developed as an LSD1 inhibitor. LSD1 (lysine-specific demethylase 1) is a protein that helps control which genes are turned on or off in cells. EXS74539 works by blocking LSD1, which may slow down or stop cancer growth. The main goals are to evaluate the drug's safety, understand how well patients tolerate it, and learn how it moves through and affects the body. Researchers also want to find the maximum tolerated dose (MTD), which is the highest dose that can be given safely without causing unacceptable side effects.

The study uses a dose-escalation design, meaning different groups of patients will receive increasing doses of EXS74539. Patients will take the drug once daily for 3 days, followed by 4 days off treatment, with this schedule repeating every 28 days. During the first treatment cycle, patients will be closely monitored for dose-limiting toxicities (DLTs), which are serious side effects that may prevent further dose increases. All participants will have frequent medical checkups and testing to monitor their health and how their bodies respond to the study drug.

A Safety Review Committee (SRC) will regularly review safety information and decide whether it is safe to increase, decrease, or maintain the dose for future patients. In addition to evaluating safety, researchers will look for early signs that EXS74539 may help slow, control, or shrink tumors. The results of this study will help identify the best dose to test in future clinical trials and determine whether EXS74539 shows promise as a new cancer treatment.


Clinical Study Identifier: s25-00553
ClinicalTrials.gov Identifier: NCT07517198
Principal Investigator: Salman Rafi Punekar.
Other Investigators: Elaine Shum, Yoke Wah Khoo, David Mendoza, Eneil De La Pena, Janice Mehnert, Maya Dimitrova, Elise M Zahriah, Joshua K Sabari, Keriann M Scavone, Natalia Cherkassky, Rajwanth Veluswamy, Paul E. Oberstein, Kathleen M Lutz, David R. Wise, Jessica Wang, Michelle Diane Swanson Lightfoot, Mariam M Cheaib, Zujun Li, Ian W Tattersall, Samantha VanMaanen, Bhavana Pothuri, Elise Roughan, Kristen Spencer, Kirby Rodriguez, Melissa L Martinez, Kathleen M Madden, Nancy Chan, Maria Francis, Ruth A White, Matthew A. Ingham, Douglas K Marks.


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