Sponsor: Memorial Sloan Kettering Cancer Center (other)
Phase: 1
Start date: April 7, 2026
Planned enrollment: 60
BMS-986504 (formerly MRTX1719) is an investigational oral drug being studied for advanced cancers with homozygous deletion of MTAP. Human results are available from an early-phase, nonrandomized trial; they do not yet establish how it compares with standard treatment. (pmc.ncbi.nlm.nih.gov)
Loss of MTAP causes methylthioadenosine (MTA) to accumulate in tumor cells. BMS-986504 is designed to bind and inhibit the PRMT5–MTA complex, exploiting this vulnerability while having less effect on cells without MTAP deletion. (pmc.ncbi.nlm.nih.gov)
In a phase 1/2 study of heavily pretreated patients with MTAP-deleted advanced solid tumors, the reported objective response rate was 23% and the disease control rate was 70% across evaluated tumor types. Median duration of response was 10.5 months. These figures come from an interim analysis, not a randomized comparison. (ascopubs.org)
A later analysis of the non–small cell lung cancer subgroup reported responses in 10 of 33 evaluable patients (30%). A separate analysis reported responses in 17% of 35 evaluable patients with pancreatic ductal adenocarcinoma; the small subgroups and differing analysis dates warrant caution when comparing rates. (sciencedirect.com)
In the 152-patient interim analysis, most treatment-related adverse events were grade 1 or 2; 13% of patients had a grade 3 or higher treatment-related event, and no grade 5 treatment-related events were reported. Longer follow-up and larger studies are needed to characterize safety more fully. (ascopubs.org)
Last updated: Sep 2026
Goal: Assess the safety of BMS-986504 combined with disease-specific standard therapy and identify a recommended phase 2 dose in MTAP-deleted advanced solid tumors.
Patients: Adults with measurable, metastatic or unresectable diffuse pleural mesothelioma, gastroesophageal carcinoma, or urothelial carcinoma and tumor-tissue confirmation of MTAP loss or deletion. Mesothelioma and gastroesophageal cohorts are generally treatment-naive in the metastatic setting; the urothelial cohort requires at least one prior line of treatment. Gastroesophageal tumors must be PD-L1 CPS ≥1 and HER2-negative. Patients must have adequate organ function and a Karnofsky performance status ≥70 or ECOG performance status of 0.
Design: Nonrandomized phase 1a/1b basket trial with three disease-specific treatment cohorts and no control arm.
Treatments: The mesothelioma cohort receives BMS-986504 with ipilimumab and nivolumab; the gastroesophageal cohort receives it with FOLFOX and nivolumab; and the urothelial cohort receives it with gemcitabine plus cisplatin or carboplatin, followed by BMS-986504 alone. BMS-986504 is an investigational oral inhibitor of the PRMT5–methylthioadenosine complex, designed to exploit methylthioadenosine accumulation caused by MTAP deletion. In a separate early-phase, nonrandomized study of previously treated MTAP-deleted solid tumors, an interim analysis reported a 23% objective response rate and 13% grade ≥3 treatment-related adverse events; these results do not establish the benefit or safety of the combinations in this trial.
Outcomes: The primary outcome is the recommended phase 2 dose, informed by adverse events and dose-limiting toxicities graded using CTCAE v5.0. Secondary outcomes are objective response rate and progression-free survival assessed by RECIST v1.1 or, where applicable for mesothelioma, modified RECIST, over two years.
Burden on patient: Likely moderate to high: combination treatment entails repeated clinic visits for intravenous chemotherapy and/or immunotherapy alongside oral BMS-986504, plus safety monitoring and response scans. Phase 1 dose-finding may require additional assessments, but the supplied information does not specify mandatory research biopsies or intensive pharmacokinetic blood draws.
Last updated: Sep 2026
Inclusion Criteria:
* Documentation of Disease:
* Patients must have pathologic confirmation of one of three diseases:
* Diffuse pleural mesothelioma (DPM)
* Gastroesophageal carcinoma (GEC) including adenocarcinoma or squamous cell carcinoma of the esophagus, gastroesophageal junction, or stomach.
* PD-L1 CPS ≥1 (using clone 73-10, DAKO)
* HER2 overexpression negative (using clone 4B5, Ventana): HER2 IHC 0-1+, or HER2 2+ with ISH showing HER2:CEP17 ratio \<2 and average HER2 copy number \<6.0 signals/cell
* Urothelial carcinoma (UC)
* Archival tissue is acceptable
* Metastatic or advanced/unresectable disease:
* For Diffuse Pleural Mesothelioma (DPM) and Gastroesophageal Carcinoma (GEC )cohorts: no prior systemic treatment for metastatic disease
* Patients with metastatic disease after treatment for localized GEC may have received prior systemic therapy (chemotherapy and/or chemoradiation) if \>6 months have elapsed between the end of therapy and registration.
* One prior cycle of standard-of-care therapy alone without BMS-986504 or other MTAP inhibitors (ipi/nivo for DPM, FOLFOX + nivo for GEC) is acceptable with PI approval.
* For UC cohort: must have received at least 1 prior line of treatment without prior gemcitabine (prior tx with Gem+Platinum in the perioperative setting is permitted if at least 12 months have elapsed from trial enrollment)
* Patients with recurrent disease within 1 year of completion of prior perioperative systemic therapy are eligible with PI approval.
* One prior cycle of standard-of-care therapy alone with gemcitabine + platinum, without BMS-986504 or other MTAP inhibitors, is acceptable with PI approval.
* Confirmation of MTAP deletion by either IHC or NGS:
* MTAP deletion must be detected by either IHC and/or NGS (including FACETS), done on tumor tissue (not blood):
* IHC (using antibody 1813, NBP2-75730, Novus Biologicals)30
* IHC staining showing loss of MTAP expression
* Tissue-based NGS options
* MSK-IMPACT version 7 or beyond showing homozygous MTAP copy number loss
* FACETS showing homozygous deletion
* Other CLIA-approved commercial Template Version: 1-21-25
* Full report must be available for review and confirmation
* Cases with discordant results between NGS and IHC, in which one test shows MTAP loss/MTAP del and the other shows MTAP intact, are acceptable with PI approval
* Measurable disease per RECIST v 1.1 (or, for DPM cohort, by either RECIST v 1.1 or modified RECIST \[mRECIST\] for mesothelioma31)
* No contraindications to receiving other standard-of-care agents per package inserts (and see Appendix IV), and per the discretion of the PI:
* DPM: Ipilimumab + nivolumab
* GEC: FOLFOX (5-FU, leucovorin, and oxaliplatin) + nivolumab
* UC: Gemcitabine + platinum (carboplatin or cisplatin)
* Age ≥ 18
* KPS ≥ 70/ECOG \<1
* Reproductive Status:
* Female participants of child-bearing potential (as assigned at birth) must have a negative highly sensitive urine or serum (as required by local regulations) pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin) within 24 hours prior to the start of study intervention.
* If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
* Female participants of child-bearing potential (as assigned at birth) must agree to use a combination of a hormonal and a non-hormonal contraceptive method or a non-hormonal method alone that is highly effective (with a failure rate of \< 1% per year)during the intervention period and for 14 months (for females) after the last dose of study intervention (or longer if required by institutional guidelines)
* Female participants of child-bearing potential (as assigned at birth) must also agree not to donate eggs (ova, oocytes) for the purpose of reproduction for the same period.
* If needed, these participants should be advised to seek advice about egg donation and cryopreservation of germ cells before treatment.
* Female participants (as assigned at birth) are deemed to be without child-bearing potential if they meet one of the following criteria:
* Postmenopausal for at least 1 year before the screening visit
* Permanently sterile (undergone a hysterectomy, bilateral salpingectomy or bilateral oophorectomy) with surgery at least 1 month before the first dose of study drug or confirmed by follicle stimulating hormone (FSH) test \> 40 mIU/mL and estradiol \< 40 pg/mL (\<140 pmol/L)
* Male participants (as assigned at birth) will be required to always use a latex or other synthetic condom during any sexual activity (eg, vaginal, anal, oral) with a female of childbearing potential, even if the participant has undergone a successful vasectomy or if the partner is pregnant or breastfeeding. Male participants (as assigned at birth) should continue to use a condom during the intervention period and for at least 11 months after the last dose of study intervention (or longer if required by institutional guidelines).
* Male participants must refrain from donating sperm during the intervention period and for at least 11 months after the last dose of study intervention (or longer if required by institutional guidelines).
* If needed, male participants should be advised to seek advice about sperm donation and cryopreservation of germ cells before treatment.
* Individuals of child-bearing potential who are partners of male participants should be advised to use a highly effective method of contraception during the intervention period and for at least 11 months after the last dose of study intervention for the male participant
* Male participants (as assigned at birth) with a pregnant or breastfeeding partner must agree to remain abstinent from sexual activity or use a male condom during any sexual activity (eg, vaginal, anal, oral), even if the participant has undergone a successful vasectomy, during the intervention period and for at least 11 months after the last dose of study intervention
* Breastfeeding partners of male participants (as assigned at birth) should be advised to consult their health care provider about using appropriate highly effective contraception during the time the male participant is required to use condoms
* Recovery from the adverse effects of prior therapy at the time of enrollment to baseline or ≤ Grade 1 (excluding alopecia, peripheral neuropathy, and parameters superseded by other eligibility criteria \[eg, hematology parameters\]). Note: Participants with prior endocrine adverse effects are permitted to enroll if they are stably maintained on appropriate replacement therapy and are asymptomatic.
* Required organ function
* Adequate hematologic function defined as follows:
* Absolute neutrophil count (ANC) ≥ 1,500 cells/mm\^3
* Platelets ≥ 100,000 cells/mm3
* Hemoglobin ≥ 8 g/dl
* Adequate renal function defined as follows:
* Creatinine clearance (CrCL) of ≥50 mL/min by the CKD-Epi creatinine equation
* Adequate hepatic function defined as follows:
* Total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN) (patients with known Gilbert's disease who have bilirubin level ≤ 3 x ULN may be enrolled)
* AST and ALT ≤5 x ULN
* Signed informed consent form (ICF)
Exclusion Criteria:
* Prior treatment with PRMT5i or MAT2Ai
* Symptomatic CNS metastases
* Patients with treated brain metastases are eligible if follow up brain imaging after CNS directed therapy shows no evidence of progression and the patient is on a stable dose of corticosteroids
* Received palliative radiation therapy within 3 days prior to initiation of study treatment or definitive SRS including CNS SRS within 14 days prior to initiation of study treatment
* Patients who have had major surgery within 3 weeks of start of study drug
o Note: procedures such as biopsy, pleural catheter insertion, central venous catheter or other minor procedures are permitted
* Any of the following cardiac abnormalities:
* Unstable angina pectoris or myocardial infarction within 6 months prior to enrollment
* Congestive heart failure ≥ NYHA Class 3 within 6 months prior to enrollment
* Prolonged QTc \> 500 milliseconds or history of Long QT Syndrome
* Child-Pugh class C liver cirrhosis
* Ongoing medical illness not otherwise listed which would preclude study at the discretion of the PI
* Inability to take medications PO (BMS-986504 cannot be taken via gastrostomy tube), refractory nausea and vomiting, malabsorption, biliary shunt, significant bowel resection, or any other condition that significantly affects gut motility or absorption and would preclude adequate absorption of BMS-986504 in the opinion of the treating physician and/or PI
* Ongoing need for a medication that is a strong inhibitor or strong inducer of cytochrome P450 (CYP) 3A4 and/or P-glycoprotein (P-gp) or proton-pump inhibitor that cannot be switched to alternative treatment prior to study entry
* HIV, HBV, or HCV with detectable viral load
* For patients with known HIV, HBV, and/or HCV infection:
* HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial.
* For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable with or without suppressive therapy
* Patients with a history of HCV infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.
* Active infection requiring parenteral antibiotic(s)
* Pregnant or breastfeeding
* Presence of another malignancy that could be mistaken for the malignancy under study during disease assessments.
* Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial.
Basking Ridge, New Jersey, 07920, United States
No email / 646-608-3763
Status: Recruiting
Montvale, New Jersey, 07645, United States
No email / 646-608-3763
Status: Recruiting
Middletown, New Jersey, 07748, United States
No email / 646-608-3763
Status: Recruiting
New York, New York, 10065, United States
No email / 646-608-3763
Status: Recruiting
Uniondale, New York, 11553, United States
No email / 646-608-3763
Status: Recruiting
Commack, New York, 11725, United States
No email / 646-608-3763
Status: Recruiting
Harrison, New York, 10604, United States
No email / 646-608-3763
Status: Recruiting