Sponsor: Processa Pharmaceuticals (industry)
Phase: 2
Start date: Oct. 2, 2024
Planned enrollment: 90
PCS6422 (eniluracil; 776C85, GW776) is an oral, potent, selective, irreversible inhibitor of dihydropyrimidine dehydrogenase (DPD), the rate‑limiting enzyme that catabolizes 5‑fluorouracil (5‑FU). It has been developed historically to enable predictable oral 5‑FU exposure and more recently to increase the potency of capecitabine by blocking conversion of 5‑FU to inactive catabolites. (pubmed.ncbi.nlm.nih.gov)
Historical (eniluracil + oral 5‑FU)
- Metastatic/advanced colorectal cancer, Phase III (FUMA3008): In 964 treated patients, oral eniluracil/5‑FU did not meet protocol‑specified equivalence to IV 5‑FU/leucovorin for overall survival (median OS 13.3 vs 14.5 months; HR 0.88, 95% CI 0.75–1.03) and had inferior progression‑free survival (median 20.0 vs 22.7 weeks; P=0.01). (ascopubs.org)
- Metastatic colorectal cancer, Phase II (28‑day oral EU/5‑FU): Response rates ~23–28%, median PFS ~22.6 weeks, median OS ~59 weeks, supporting activity but not superior to established regimens of the era. (ascopubs.org)
- Metastatic pancreatic adenocarcinoma, Phase II (SWOG): 6‑month survival 34% (chemo‑naïve) and 29% (pre‑treated); median OS 3.6 and 3.4 months, respectively. (pubmed.ncbi.nlm.nih.gov)
- Advanced/metastatic breast cancer, Phase II (first‑line low‑dose continuous oral EU/5‑FU): Partial response rate 55% in 29 assessable patients; small single‑arm study. (ascopubs.org)
Current (PCS6422 + capecitabine)
- Phase 1b (NCT04861987) in refractory gastrointestinal cancers: Company‑reported preliminary analysis showed partial response (PR) or stable disease (SD) in 66.7% (8/12) of evaluable patients (2 PR, 6 SD); at the capecitabine MTD (225 mg BID after single‑dose PCS6422), all three evaluable patients had progression‑free intervals ~5–7 months. These are small, early findings; peer‑reviewed efficacy data are not yet available. (processapharmaceuticals.com)
- Pharmacokinetic/PD signal in Phase 1b: Single‑dose PCS6422 produced marked DPD inhibition with <10% of 5‑FU metabolized to FBAL vs ~80% historically with standard capecitabine, and extended 5‑FU half‑life to ~3–4 h on day 1; the effect waned by day 7, informing regimen optimization. (processapharmaceuticals.com)
- 2025 ASCO: A trials‑in‑progress poster for a Phase 2 adaptive study in metastatic breast cancer and an online abstract summarizing Phase 1b safety/efficacy were accepted; detailed peer‑reviewed results have not yet been published. (processapharmaceuticals.com)
Historical (eniluracil + oral 5‑FU)
- In the Phase III colorectal study, grade 3/4 granulocytopenia was far less frequent with eniluracil/5‑FU than with bolus IV 5‑FU/LV (5% vs 47%), while grade 3/4 diarrhea occurred in 19% vs 16% (NS). Overall survival non‑equivalence and PFS inferiority tempered enthusiasm. (ascopubs.org)
- A Phase II regimen combining eniluracil/5‑FU/leucovorin showed notable toxicity (grade 4 neutropenia 42%, grade 3–4 diarrhea 30%, 20% febrile neutropenia), underscoring schedule‑ and dose‑dependence of tolerability. (pubmed.ncbi.nlm.nih.gov)
Current (PCS6422 + capecitabine)
- Early Phase 1b readouts suggest exposure‑driven “potency” increases with potentially different toxicity trade‑offs versus standard capecitabine; the company reports a similar or improved side‑effect profile at lower capecitabine doses, but full peer‑reviewed safety data are pending. (processapharmaceuticals.com)
Disclaimer: For PCS6422 + capecitabine, most publicly available data to date are from company communications and meeting abstracts; peer‑reviewed, controlled clinical outcomes have not yet been published. (processapharmaceuticals.com)
Last updated: Oct 2025
Goal: Assess whether adding the DPD inhibitor PCS6422 to capecitabine improves antitumor activity and maintains acceptable safety compared with standard capecitabine alone in patients with advanced or metastatic breast cancer who are not candidates for anthracycline-, taxane-, PD-1–, or PARP inhibitor–based therapies.
Patients: Adults ≥18 years with histologically confirmed unresectable, advanced or metastatic breast cancer, including triple-negative or HR-positive/HER2-negative disease, measurable by RECIST 1.1, ECOG 0–1, adequate organ function, and a life expectancy ≥24 weeks. Patients must have received chemotherapy in the metastatic setting and be resistant or intolerant to taxanes and/or anthracyclines or otherwise not indicated for available therapies. Key exclusions include recent systemic therapy within 21 days or 5 half-lives, recent fluoropyrimidines or DPD inhibitors, known complete/near-complete DPD deficiency (homozygous or compound heterozygous DPYD variants), significant cardiac disease or QTc >480 ms, and pregnancy or breastfeeding.
Design: Adaptive, randomized, open-label, multi-center Phase 2 trial evaluating up to two PCS6422+capecitabine regimens versus standard-dose capecitabine monotherapy. Planned enrollment approximately 90 patients. Allocation is randomized with efficacy and safety comparisons across arms.
Treatments: Experimental arms: Single fixed dose PCS6422 40 mg combined with low-dose capecitabine administered twice daily for 7 days per cycle. Two regimens are explored: capecitabine 150 mg BID (total 300 mg/day) or 225 mg BID (450 mg/day), with protocol text also allowing 75 mg BID (150 mg/day) depending on regimen. Comparator arm: standard capecitabine 1000 mg/m2 BID (2000 mg/m2/day). PCS6422 is an irreversible inhibitor of dihydropyrimidine dehydrogenase (DPD), the primary enzyme that catabolizes 5-FU; by inhibiting DPD, it increases exposure to active 5-FU from capecitabine while potentially reducing toxic catabolites. Early Phase 1b data in gastrointestinal cancers using this Next Generation Capecitabine approach showed disease control in a majority of evaluable patients with predominantly low-grade toxicities and no hand-foot syndrome signals in initial cohorts, supporting further evaluation in breast cancer.
Outcomes: Primary outcomes: objective response rate per RECIST 1.1 and incidence, frequency, duration, and severity of adverse events. Secondary outcomes: disease control rate, duration of response, time to response, and progression-free survival, with follow-up up to 24 weeks post end of treatment and on-treatment tumor assessments approximately every 12 weeks.
Burden on patient: Moderate. The trial uses oral therapy with tumor imaging approximately every 12 weeks, similar to standard of care. Being Phase 2 and open-label, monitoring for safety is expected to include regular clinic visits and labs to assess hematologic, hepatic, and renal function, as well as cardiac eligibility screening, but no protocol-mandated biopsies or intensive pharmacokinetic sampling are described. The addition of PCS6422 does not inherently increase visit frequency beyond routine safety assessments; however, early-cycle safety checks may be more frequent given DPD inhibition and fluoropyrimidine-related risk, contributing to a moderate overall burden.
Last updated: Oct 2025
Inclusion Criteria:
1. Aged ≥18 years at Screening
2. Diagnosis of histologically confirmed breast cancer that is unresectable. The following subsets of breast cancer are included:
1. Patients with triple-negative breast cancer, advanced or metastatic
2. Patients with hormone receptor (HR) positive, ER positive, HER2 negative advanced or metastatic breast cancer
3. Has measurable disease in accordance with RECIST 1.1 obtained by imaging within 28 days prior to C1D1
4. Other therapies are not indicated (eg, resistant or intolerant to taxanes and/or an anthracycline-containing regimen) for treatment of advanced or metastatic breast cancer
5. Has a life expectance of at least 24 weeks
6. Has Eastern Cooperative Oncology Group Performance Status (ECOG PS) 0 or 1 at screening
7. Has adequate bone marrow, liver, and renal function as assessed by the following laboratory requirements conducted within 7 days before C1D1 (Note: labs will also be repeated pre-dose on C1D1 to confirm eligibility): a. Hemoglobin ≥9 g/dL (≥90 g/L) b. Adequate renal function by estimated glomerular filtration rate (eGFR) defined as a creatinine clearance \>50 mL/min (\>0.84 mL/s) (Cockcroft-Gault equation) and normalized to body surface area c. Peripheral absolute neutrophil count (ANC) of ≥1.5×109/L d. Platelet count of ≥100×109/L without growth factor/transfusion e. Total bilirubin \<1.5× upper limit of normal (ULN); or ≤3×ULN if the patient has Gilbert's disease f. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \<2.5×ULN, with liver metastasis \<5×ULN g. International normalized ratio (INR) \<1.5 and prothrombin time (PT) ≤1.5×ULN, unless both of the following conditions are met: i. Patient is receiving anticoagulant therapy, and ii. PT or partial thromboplastin time (PTT) is within therapeutic range of intended use of anticoagulant h. Activated partial thromboplastin time (aPTT) ≤1.5×ULN, unless both of the following conditions are met: i. Patient is receiving anticoagulant therapy, and ii. PT or PTT is within therapeutic range of intended use of anticoagulants
Exclusion Criteria:
1. Received any line of treatment for advanced or metastatic breast cancer within 21 days or 5 half-lives (whichever is longer) prior to randomization
2. Currently receiving any hormone replacement therapy, unless discontinued within 21 days prior to randomization
3. Received IV 5-FU or oral 5-FU analog in the 4 weeks prior to C1D1
4. Received DPD inhibitor within 4 weeks prior to C1D1
5. Has homozygous or compound heterozygous DPYD variants that result in complete or near-complete absence of DPD activity
6. Cardiac:
1. Has history or presence of clinically significant abnormal 12-lead electrocardiogram (ECG) results, in the Medical Monitor or Investigator's opinion
2. Has prolonged QTc (with Fridericia's correction) of \>480 msec performed at Screening
3. Has a history of prolonged QTc interval, ventricular tachycardia/fibrillation or significant ventricular arrhythmia, or Torsades de Pointes, or a history of ventricular ablation for arrhythmia
4. Has congenital long QT syndrome or a family history of long QT syndrome
5. Has other clinically significant cardiac disease including, but not limited to, myocardial infarction, unstable angina, cardiac or other vascular stenting, angioplasty, or surgery ≤12 months prior to randomization, congestive heart failure
* Class II per the New York Heart Association, or history of myocarditis
7. Is pregnant or breastfeeding
Tucson, Arizona, 85711, United States
[email protected] / 520-866-0206
Status: Recruiting
Oxnard, California, 93030, United States
[email protected] / 805-483-1185
Status: Recruiting
Los Angeles, California, 90067, United States
[email protected] / 424-535-1874
Status: Recruiting
Miami, Florida, 33165, United States
[email protected] / 305-400-8899
Status: Recruiting
Tampa, Florida, 33612, United States
[email protected] / 813-745-4933
Status: Recruiting
Dyer, Indiana, 46311, United States
[email protected] / 219-924-8178
Status: Recruiting
Baltimore, Maryland, 21201, United States
[email protected] / 410-328-3546
Status: Recruiting
New Brunswick, New Jersey, 08901, United States
[email protected] / 732-754-7788
Status: Recruiting
Westbury, New York, 11590, United States
[email protected] / No phone
Status: Recruiting
Canton, Ohio, 44718, United States
[email protected] / 330-417-8231
Status: Recruiting
Nashville, Tennessee, 37203, United States
[email protected] / 615-712-3268
Status: Recruiting
Austin, Texas, 78731, United States
[email protected] / 512-427-9400
Status: Recruiting
San Antonio, Texas, 78240, United States
[email protected] / 210-595-5300
Status: Recruiting