RARE DISEASE
Primary peritoneal carcinoma
Primary peritoneal carcinoma
Primary peritoneal carcinoma
Synonyms: EOPPC, Extra-ovarian primary peritoneal carcinoma, PPC, Primary peritoneal serous carcinoma, Serous surface papillary carcinoma
Synonyms: EOPPC, Extra-ovarian primary peritoneal carcinoma, PPC, Primary peritoneal serous carcinoma, Serous surface papillary carcinoma
Synonyms: EOPPC, Extra-ovarian primary peritoneal carcinoma, PPC, Primary peritoneal serous carcinoma, Serous surface papillary carcinoma
Drug discovery
7
drugs
With orphan designations
Overview
Primary peritoneal carcinoma (PPC) is a rare, aggressive malignancy arising from the peritoneal lining, clinically and histologically resembling ovarian serous carcinoma [1][3][6]. It predominantly affects postmenopausal women (median age 60–72), with BRCA1/2 mutations increasing lifetime risk by ~5% even after oophorectomy [1][3][15]. Diagnosis requires exclusion of ovarian primaries, and most present with advanced-stage disease (Stage III/IV), ascites, and elevated CA-125 [4][6][11]. Treatment mirrors ovarian cancer, combining cytoreductive surgery and platinum-taxane chemotherapy, but prognosis remains poor (median survival 12–17 months) [1][5][13].
Burden
Prognosis: Median survival 12–17 months, 5-year survival ~26% [1][4][11].
Morbidity: Advanced-stage diagnoses (70–80%) cause ascites, bowel obstruction, and cachexia, requiring palliative interventions (paracentesis, stenting) [8][15][17].
Recurrence: >75% relapse within 2 years, necessitating secondary-line therapies (gemcitabine, pegylated liposomal doxorubicin) [8][18].
Therapies
First-line: Cytoreductive surgery (optimal debulking) with hyperthermic intraperitoneal chemotherapy (HIPEC) [3][9][17], followed by platinum-paclitaxel systemic chemotherapy [8][13].
Targeted therapies: PARP inhibitors (olaparib, niraparib) for BRCA-mutated cases and bevacizumab for advanced/metastatic disease [8][13][17].
Advanced disease: Neoadjuvant chemotherapy for unresectable tumors, with interval debulking if responsive [5][8][13].
Categories: rare abdominal surgical diseases, rare neoplastic diseases, rare transplant-related disorders
Research Papers
652 drug discovery papers about Primary peritoneal carcinoma, with 4 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
652 drug discovery papers about Primary peritoneal carcinoma, with 4 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-06-09 | Single-cell transcriptomics of heterogeneous patient-derived organoids reveals novel therapeutic targets in high-grade serous ovarian cancer
Abstract High-grade serous ovarian carcinoma (HGSOC) is characterized by widespread peritoneal dissemination and poor long-term survival, largely driven by metastatic relapse following initial response to chemotherapy. Defining the molecular programs that enable tumor progression from the primary ovarian site to metastatic niches remains a key challenge. Here, we leverage patient-derived organoids (PDOs) coupled with single-cell RNA sequencing (scRNA-seq) to interrogate tumor evolution and identify regulators of metastatic competence in HGSOC. We profiled PDOs and matched formalin-fixed paraffin-embedded (FFPE) tumor samples from ovarian and omental disease sites across seven patients. Single-cell transcriptomic analysis revealed conserved and patient-specific cellular states and enabled reconstruction of inferred trajectories of tumor progression. Comparative trajectory analysis identified gene expression programs associated with metastatic transition from ovarian to omental tumors. Among these, the heparan sulfate proteoglycan AGRIN emerged as a consistently upregulated gene along the metastatic axis. Cell-cell communication analyses suggested that AGRIN -mediated signaling involves both epithelial tumor cells and stromal components, implicating the extracellular matrix in shaping metastatic behavior through mechanotransduction and integrin-associated pathways. Functional validation using genetic depletion of AGRIN in ovarian cancer cell lines demonstrated reduced migratory and invasive capacity, supporting a causal role for AGRIN in promoting metastatic phenotypes. Together, these findings identify AGRIN as a regulator of metastatic competence in HGSOC and highlight extracellular matrix–associated signaling as a key driver of disease progression. More broadly, this study demonstrates that PDO-based single-cell transcriptomic approaches can uncover actionable regulators of metastasis and provide a scalable framework for therapeutic target discovery across cancer types. Significance Patient-derived organoids analyzed by single-cell transcriptomics reveal dynamic tumor evolution and uncover AGRIN as a regulator of metastatic competence in HGSOC, demonstrating the utility of living tumor models for therapeutic target discovery.
2026-05-28 | Study design of OMAHA-015: A phase 2 basket study of steroidogenesis inhibitor opevesostat in participants with selected solid tumors.
TPS3183 Background: Opevesostat (MK-5684; ODM-208), an oral steroidogenesis inhibitor, selectively inhibits CYP11A1, blocking synthesis of all steroid hormones and their precursors, and may suppress steroid-driven tumor growth. The randomized, open-label, phase 2 OMAHA-015 trial (NCT06979596) is designed to evaluate the efficacy and safety of opevesostat compared with standard of care in breast, ovarian, and endometrial cancers. Methods: Cohort A will enroll participants (pts) with locally advanced unresectable or metastatic hormone receptor–positive (estrogen receptor and/or progesterone receptor ≥1% expression)/HER2-negative breast cancer (IHC 0, 1+ or 2+/ISH-) and disease progression on or after ≥1 prior endocrine-based therapy in the metastatic setting. Exclusion criteria include any line of cytotoxic chemotherapy (chemo) or PARP inhibitor in the inoperable or noncurative advanced/metastatic setting and prior treatment with both fulvestrant and exemestane in the metastatic setting. Cohort B will enroll pts with platinum-sensitive, histologically confirmed high-grade epithelial ovarian (including high-grade serous or predominantly serous, high-grade endometrioid, clear cell, or malignant mixed Müllerian tumors [carcinosarcoma]), fallopian tube, or primary peritoneal carcinomas. Pts must have received 4-8 cycles of platinum-based doublet chemo in a third-line setting for ovarian cancer with ≤9 weeks between the last dose of third-line chemo and randomization. Cohort C will enroll pts with histologically confirmed primary advanced or recurrent low-grade endometrioid carcinoma (FIGO grade 1/2, or well/moderately differentiated), with known pMMR status, and wild type p53 expression. Pts must either be treatment naive or have received 1 prior line of platinum-based therapy in the advanced/metastatic or adjuvant/neoadjuvant setting. In cohort A, ~80 pts will be randomized 1:1 to arm 1: opevesostat 5 mg PO twice daily (BID) + daily corticosteroids or arm 2: physician’s decision of fulvestrant 500 mg IM (D1 and D15 of cycle 1 and D1 of each cycle thereafter) or exemestane 25 mg PO daily. Randomization will be stratified by 1 line versus ≥2 lines of prior endocrine therapy. In cohort B, ~90 pts will be randomized 1:1 to arm 3: opevesostat 5 mg PO BID + daily corticosteroids; or arm 4: observation. In cohort C, ~80 pts will be randomized 1:1 to arm 5: opevesostat 5 mg PO BID + daily corticosteroids; or arm 6: physician’s choice of megestrol acetate 80 mg PO BID, alternating megestrol + tamoxifen 20 mg PO BID, or letrozole 2.5 mg PO daily. The primary end point is progression-free survival per RECIST v1.1 assessed by blinded independent central review (BICR). Secondary end points include overall survival, clinical benefit rate (cohort A only), objective response rate, and duration of response per RECIST v1.1 assessed by BICR, and safety. The study is currently enrolling. Clinical trial information: NCT06979596 .
2026-05-27 | Azenosertib plus paclitaxel for platinum-resistant ovarian cancer: Results from a phase 1b study.
5529 Background: Most patients (pts) with ovarian cancer develop platinum-resistant disease following surgery and platinum-based chemotherapy and have a poor prognosis. Paclitaxel (PAC) monotherapy is the most efficacious chemotherapy for platinum-resistant ovarian cancer (PROC) but clinical benefits require further improvement. Azenosertib (Azeno) is a potentially best-in-class, novel, selective and orally bioavailable small molecule inhibitor of the WEE1 tyrosine kinase. Herein we report results from a Phase 1b study of Azeno plus PAC for PROC. Methods: MUIR (NCT04516447) is an open-label Phase 1b study evaluating the safety, efficacy and preliminary clinical activity of Azeno plus chemotherapy (Part 1 dose escalation) for PROC. Eligible pts were aged ≥18 years, had ECOG PS score ≤2, histologically/cytologically confirmed high-grade serous epithelial ovarian, fallopian tube, or peritoneal carcinoma, and measurable disease per RECIST v1.1; pts had received 1‒4 prior lines of systemic therapy and had platinum-resistant disease. Pts were assigned to 1 of 4 cohorts: carboplatin, gemcitabine, pegylated liposomal doxorubicin, or PAC (80 mg/m 2 IV on Days 1, 8 and 15 of 28-day cycles). Azeno plus PAC is the focus of this analysis where Azeno was orally administered 200 mg (continuously) or 200, 250 and 300 mg (intermittently 5 days on, 2 days off [5:2]) for 28-day cycles. Primary objectives were safety and tolerability. Clinical activity was a key secondary objective assessed by objective response rate (ORR) and duration of response (DOR) per RECIST v1.1, and progression-free survival. Exploratory objectives included analysis of baseline tumor and plasma biomarkers with optional biopsies/blood plasma. Results: As of December 1, 2025, 46 pts received Azeno (continuously 200 mg, n=7 and intermittently 5:2: 200 mg, n=15; 250 mg, n=12; 300 mg, n=12) plus PAC. Median age was 66 years (range 45‒83), median number of prior lines of therapy was 2 (range, 1‒4), and all pts received prior PAC. Most common treatment related adverse events (TRAEs) were fatigue (61%), anemia (59%), nausea (52%), and neutropenia (50%); most frequent grade ≥3 TRAEs were neutropenia (30%) and anemia (20%). Serious TRAEs occurred in 20% of pts. Overall, confirmed ORR was 39.1% (95% CI, 25.1‒54.6) with a median DOR of 5.6 months (95% CI, 5.6‒9.2) (Table). Conclusions: Azeno plus PAC showed promising clinical activity and tolerability in pts with PROC. ORR and median DOR were improved when compared historically with PAC monotherapy supporting the continued evaluation of this regimen in PROC and other indications. Clinical trial information: NCT04516447 . OR and median DOR. Azeno 5:2 Azeno 5:2 Azeno 5:2 Azeno 200 mg + PAC (n=7) 200 mg + PAC (n=15) 250 mg + PAC (n=12) 300 mg + PAC (n=12) All(N=46) ORR, %(95% CI) 57.1(18.4‒90.1) 20.0(4.3‒48.1) 50.0 (21.1‒78.9) 41.7 (15.2‒72.3) 39.1 (25.1‒54.6) Median DOR, months(95% CI) 7.8(5.6‒NE) 5.6(3.7‒NE) 9.2 (3.8‒NE) 5.6 (5.2‒NE) 5.6(5.6‒9.2) NE, not evaluable.
2026-05-27 | Dose-dense weekly versus standard three-weekly paclitaxel in combination with carboplatin for resectable stage II-IV epithelial ovarian carcinoma: A retrospective cohort study.
5568 Background: Dose-dense weekly paclitaxel combined with carboplatin has demonstrated variable efficacy in first-line treatment of resectable epithelial ovarian cancer across different populations. While Japanese and selected real-world studies suggest improved outcomes, large Western trials have failed to confirm a survival advantage. Data from South Asian populations remains limited. This study compared progression-free survival (PFS), overall survival (OS), and toxicity profiles of dose-dense versus standard three-weekly paclitaxel plus carboplatin in a real-world, resource-constrained setting. Methods: This single-center retrospective cohort study included women aged ≥20 years with resectable, FIGO stage II–IV epithelial ovarian, fallopian tube, or primary peritoneal carcinoma treated between January 2015 and December 2018. Patients received first-line carboplatin at an AUC of 5 (Area Under Curve 5) with either dose-dense weekly paclitaxel (80 mg/m² on days 1, 8, and 15) or standard three-weekly paclitaxel (175 mg/m²). Kaplan–Meier methods were used to estimate PFS and OS, and comparisons were performed using the log-rank test. Cox proportional hazards models were applied for univariate and multivariate analyses. Results: Seventy-two patients were included, 36 in each arm. The majority presented with stage III disease (63.9% in the dose-dense arm vs. 52.8% in the standard arm). High-grade serous carcinoma was the predominant histology in both groups (83% vs. 75%). The distribution of primary versus interval debulking surgery and rates of residual disease were similar between the two arms. The dose-dense regimen was associated with significantly improved PFS compared with the standard schedule (mean PFS 25 vs. 18 months; 95% CI: 23.1-28.4; log-rank p = 0.01); median PFS was 32 months in the dose-dense arm, whereas it could not be estimated in the standard arm. OS favoured the dose-dense group (mean OS of 56 vs. 50 months), although this difference was not statistically significant (p = 0.27). In the multivariate analysis of the dose-dense cohort, elevated baseline Ca-125 (HR = 6.80; 95% CI: 1.38–34.20; p = 0.01) and cytoreduction type (HR = 13.30; 95% CI: 1.90–90.20; p = 0.008) were independent predictors of PFS but not for OS. Rates of grade ≥3 treatment-related adverse events were comparable between arms, while sensory neuropathy occurred more frequently with dose-dense therapy (39% vs. 28%). Conclusions: This real-world analysis supports the use of dose-dense weekly paclitaxel combined with carboplatin for improved PFS, findings consistent with some regional studies. While OS data showed a non-significant trend favouring the dose-dense regimen with some risk of neuropathy, they highlight the need for larger prospective regional studies to better define patient populations most likely to benefit.
2026-05-27 | Preliminary efficacy and safety of first-line anlotinib plus carboplatin/paclitaxel induction therapy followed by anlotinib maintenance in advanced ovarian cancer: Results from the ALTER-GO-010 phase II multicenter trial.
5576 Background: Several studies have shown that antiangiogenic drugs combined with chemotherapy as first-line treatment, followed by maintenance therapy with antiangiogenic agents, significantly improve outcomes for ovarian cancer patients. Anlotinib, a highly effective multi-target tyrosine kinase inhibitor targeting VEGFRs, FGFRs, PDGFRs, and c-Kit, has been approved for multiple tumor types in China. The ALTER-GO-010 study is a single-arm, multicenter phase II trial designed to assess the efficacy and safety of anlotinib in combination with carboplatin/paclitaxel as first-line induction therapy, followed by anlotinib maintenance, in patients with newly diagnosed advanced ovarian cancer. Methods: Eligible patients with newly diagnosed FIGO stage III–IV primary epithelial ovarian, fallopian tube, or primary peritoneal cancer (ECOG PS 0–1) who have undergone primary or interval debulking surgery will receive 6-8 cycles of chemotherapy (paclitaxel 175 mg/m² + carboplatin AUC 5 every 3 weeks) combined with anlotinib (12 mg orally once daily on days 1–14, 21-day cycle; omitted during the first cycle to prevent delayed wound healing). Following chemotherapy completion, anlotinib will be continued as maintenance monotherapy until disease progression, unacceptable toxicity, or death. Exclusion criteria include prior anti-angiogenic therapy or major surgery within 28 days before anlotinib initiation. The primary endpoint is progression-free survival (PFS). Key secondary endpoints include overall survival, and safety profile. Results: As of the data cutoff date (November 30, 2025), 54 patients had received at least one dose of the study drug and were included in the analysis. The median age was 56 years, 98.11% had high-grade serous carcinoma and 1.89% had endometrioid carcinoma. 96.23% had FIGO stage III and 3.77% had stage IV. The data maturity for PFS analysis was 40.7%. The median PFS was 20.76 months (95% CI: 11.58-29.95). Patients without prior history of neoadjuvant therapy had longer mPFS than those with prior therapy. The median OS was not reached. The most common all-grade treatment emergent adverse events (TEAEs) were white blood cell deceased, anemia, neutrophil count decreased, platelet count decreased and lymphocyte count decreased. Grade ≥3 TEAEs occurred in 75.5% of patients during the chemotherapy phase and 22.7% during maintenance. No treatment-related deaths were reported. Conclusions: The preliminary efficacy and safety data suggest that anlotinib plus carboplatin/paclitaxel induction therapy, followed by anlotinib maintenance, is a promising regimen for newly diagnosed advanced ovarian cancer, with a median PFS of 20.76 months and a favorable safety profile. Clinical trial information: NCT04807166 .
2026-06-09 | Single-cell transcriptomics of heterogeneous patient-derived organoids reveals novel therapeutic targets in high-grade serous ovarian cancer
Abstract High-grade serous ovarian carcinoma (HGSOC) is characterized by widespread peritoneal dissemination and poor long-term survival, largely driven by metastatic relapse following initial response to chemotherapy. Defining the molecular programs that enable tumor progression from the primary ovarian site to metastatic niches remains a key challenge. Here, we leverage patient-derived organoids (PDOs) coupled with single-cell RNA sequencing (scRNA-seq) to interrogate tumor evolution and identify regulators of metastatic competence in HGSOC. We profiled PDOs and matched formalin-fixed paraffin-embedded (FFPE) tumor samples from ovarian and omental disease sites across seven patients. Single-cell transcriptomic analysis revealed conserved and patient-specific cellular states and enabled reconstruction of inferred trajectories of tumor progression. Comparative trajectory analysis identified gene expression programs associated with metastatic transition from ovarian to omental tumors. Among these, the heparan sulfate proteoglycan AGRIN emerged as a consistently upregulated gene along the metastatic axis. Cell-cell communication analyses suggested that AGRIN -mediated signaling involves both epithelial tumor cells and stromal components, implicating the extracellular matrix in shaping metastatic behavior through mechanotransduction and integrin-associated pathways. Functional validation using genetic depletion of AGRIN in ovarian cancer cell lines demonstrated reduced migratory and invasive capacity, supporting a causal role for AGRIN in promoting metastatic phenotypes. Together, these findings identify AGRIN as a regulator of metastatic competence in HGSOC and highlight extracellular matrix–associated signaling as a key driver of disease progression. More broadly, this study demonstrates that PDO-based single-cell transcriptomic approaches can uncover actionable regulators of metastasis and provide a scalable framework for therapeutic target discovery across cancer types. Significance Patient-derived organoids analyzed by single-cell transcriptomics reveal dynamic tumor evolution and uncover AGRIN as a regulator of metastatic competence in HGSOC, demonstrating the utility of living tumor models for therapeutic target discovery.
2026-05-28 | Study design of OMAHA-015: A phase 2 basket study of steroidogenesis inhibitor opevesostat in participants with selected solid tumors.
TPS3183 Background: Opevesostat (MK-5684; ODM-208), an oral steroidogenesis inhibitor, selectively inhibits CYP11A1, blocking synthesis of all steroid hormones and their precursors, and may suppress steroid-driven tumor growth. The randomized, open-label, phase 2 OMAHA-015 trial (NCT06979596) is designed to evaluate the efficacy and safety of opevesostat compared with standard of care in breast, ovarian, and endometrial cancers. Methods: Cohort A will enroll participants (pts) with locally advanced unresectable or metastatic hormone receptor–positive (estrogen receptor and/or progesterone receptor ≥1% expression)/HER2-negative breast cancer (IHC 0, 1+ or 2+/ISH-) and disease progression on or after ≥1 prior endocrine-based therapy in the metastatic setting. Exclusion criteria include any line of cytotoxic chemotherapy (chemo) or PARP inhibitor in the inoperable or noncurative advanced/metastatic setting and prior treatment with both fulvestrant and exemestane in the metastatic setting. Cohort B will enroll pts with platinum-sensitive, histologically confirmed high-grade epithelial ovarian (including high-grade serous or predominantly serous, high-grade endometrioid, clear cell, or malignant mixed Müllerian tumors [carcinosarcoma]), fallopian tube, or primary peritoneal carcinomas. Pts must have received 4-8 cycles of platinum-based doublet chemo in a third-line setting for ovarian cancer with ≤9 weeks between the last dose of third-line chemo and randomization. Cohort C will enroll pts with histologically confirmed primary advanced or recurrent low-grade endometrioid carcinoma (FIGO grade 1/2, or well/moderately differentiated), with known pMMR status, and wild type p53 expression. Pts must either be treatment naive or have received 1 prior line of platinum-based therapy in the advanced/metastatic or adjuvant/neoadjuvant setting. In cohort A, ~80 pts will be randomized 1:1 to arm 1: opevesostat 5 mg PO twice daily (BID) + daily corticosteroids or arm 2: physician’s decision of fulvestrant 500 mg IM (D1 and D15 of cycle 1 and D1 of each cycle thereafter) or exemestane 25 mg PO daily. Randomization will be stratified by 1 line versus ≥2 lines of prior endocrine therapy. In cohort B, ~90 pts will be randomized 1:1 to arm 3: opevesostat 5 mg PO BID + daily corticosteroids; or arm 4: observation. In cohort C, ~80 pts will be randomized 1:1 to arm 5: opevesostat 5 mg PO BID + daily corticosteroids; or arm 6: physician’s choice of megestrol acetate 80 mg PO BID, alternating megestrol + tamoxifen 20 mg PO BID, or letrozole 2.5 mg PO daily. The primary end point is progression-free survival per RECIST v1.1 assessed by blinded independent central review (BICR). Secondary end points include overall survival, clinical benefit rate (cohort A only), objective response rate, and duration of response per RECIST v1.1 assessed by BICR, and safety. The study is currently enrolling. Clinical trial information: NCT06979596 .
2026-05-27 | Azenosertib plus paclitaxel for platinum-resistant ovarian cancer: Results from a phase 1b study.
5529 Background: Most patients (pts) with ovarian cancer develop platinum-resistant disease following surgery and platinum-based chemotherapy and have a poor prognosis. Paclitaxel (PAC) monotherapy is the most efficacious chemotherapy for platinum-resistant ovarian cancer (PROC) but clinical benefits require further improvement. Azenosertib (Azeno) is a potentially best-in-class, novel, selective and orally bioavailable small molecule inhibitor of the WEE1 tyrosine kinase. Herein we report results from a Phase 1b study of Azeno plus PAC for PROC. Methods: MUIR (NCT04516447) is an open-label Phase 1b study evaluating the safety, efficacy and preliminary clinical activity of Azeno plus chemotherapy (Part 1 dose escalation) for PROC. Eligible pts were aged ≥18 years, had ECOG PS score ≤2, histologically/cytologically confirmed high-grade serous epithelial ovarian, fallopian tube, or peritoneal carcinoma, and measurable disease per RECIST v1.1; pts had received 1‒4 prior lines of systemic therapy and had platinum-resistant disease. Pts were assigned to 1 of 4 cohorts: carboplatin, gemcitabine, pegylated liposomal doxorubicin, or PAC (80 mg/m 2 IV on Days 1, 8 and 15 of 28-day cycles). Azeno plus PAC is the focus of this analysis where Azeno was orally administered 200 mg (continuously) or 200, 250 and 300 mg (intermittently 5 days on, 2 days off [5:2]) for 28-day cycles. Primary objectives were safety and tolerability. Clinical activity was a key secondary objective assessed by objective response rate (ORR) and duration of response (DOR) per RECIST v1.1, and progression-free survival. Exploratory objectives included analysis of baseline tumor and plasma biomarkers with optional biopsies/blood plasma. Results: As of December 1, 2025, 46 pts received Azeno (continuously 200 mg, n=7 and intermittently 5:2: 200 mg, n=15; 250 mg, n=12; 300 mg, n=12) plus PAC. Median age was 66 years (range 45‒83), median number of prior lines of therapy was 2 (range, 1‒4), and all pts received prior PAC. Most common treatment related adverse events (TRAEs) were fatigue (61%), anemia (59%), nausea (52%), and neutropenia (50%); most frequent grade ≥3 TRAEs were neutropenia (30%) and anemia (20%). Serious TRAEs occurred in 20% of pts. Overall, confirmed ORR was 39.1% (95% CI, 25.1‒54.6) with a median DOR of 5.6 months (95% CI, 5.6‒9.2) (Table). Conclusions: Azeno plus PAC showed promising clinical activity and tolerability in pts with PROC. ORR and median DOR were improved when compared historically with PAC monotherapy supporting the continued evaluation of this regimen in PROC and other indications. Clinical trial information: NCT04516447 . OR and median DOR. Azeno 5:2 Azeno 5:2 Azeno 5:2 Azeno 200 mg + PAC (n=7) 200 mg + PAC (n=15) 250 mg + PAC (n=12) 300 mg + PAC (n=12) All(N=46) ORR, %(95% CI) 57.1(18.4‒90.1) 20.0(4.3‒48.1) 50.0 (21.1‒78.9) 41.7 (15.2‒72.3) 39.1 (25.1‒54.6) Median DOR, months(95% CI) 7.8(5.6‒NE) 5.6(3.7‒NE) 9.2 (3.8‒NE) 5.6 (5.2‒NE) 5.6(5.6‒9.2) NE, not evaluable.
2026-05-27 | Dose-dense weekly versus standard three-weekly paclitaxel in combination with carboplatin for resectable stage II-IV epithelial ovarian carcinoma: A retrospective cohort study.
5568 Background: Dose-dense weekly paclitaxel combined with carboplatin has demonstrated variable efficacy in first-line treatment of resectable epithelial ovarian cancer across different populations. While Japanese and selected real-world studies suggest improved outcomes, large Western trials have failed to confirm a survival advantage. Data from South Asian populations remains limited. This study compared progression-free survival (PFS), overall survival (OS), and toxicity profiles of dose-dense versus standard three-weekly paclitaxel plus carboplatin in a real-world, resource-constrained setting. Methods: This single-center retrospective cohort study included women aged ≥20 years with resectable, FIGO stage II–IV epithelial ovarian, fallopian tube, or primary peritoneal carcinoma treated between January 2015 and December 2018. Patients received first-line carboplatin at an AUC of 5 (Area Under Curve 5) with either dose-dense weekly paclitaxel (80 mg/m² on days 1, 8, and 15) or standard three-weekly paclitaxel (175 mg/m²). Kaplan–Meier methods were used to estimate PFS and OS, and comparisons were performed using the log-rank test. Cox proportional hazards models were applied for univariate and multivariate analyses. Results: Seventy-two patients were included, 36 in each arm. The majority presented with stage III disease (63.9% in the dose-dense arm vs. 52.8% in the standard arm). High-grade serous carcinoma was the predominant histology in both groups (83% vs. 75%). The distribution of primary versus interval debulking surgery and rates of residual disease were similar between the two arms. The dose-dense regimen was associated with significantly improved PFS compared with the standard schedule (mean PFS 25 vs. 18 months; 95% CI: 23.1-28.4; log-rank p = 0.01); median PFS was 32 months in the dose-dense arm, whereas it could not be estimated in the standard arm. OS favoured the dose-dense group (mean OS of 56 vs. 50 months), although this difference was not statistically significant (p = 0.27). In the multivariate analysis of the dose-dense cohort, elevated baseline Ca-125 (HR = 6.80; 95% CI: 1.38–34.20; p = 0.01) and cytoreduction type (HR = 13.30; 95% CI: 1.90–90.20; p = 0.008) were independent predictors of PFS but not for OS. Rates of grade ≥3 treatment-related adverse events were comparable between arms, while sensory neuropathy occurred more frequently with dose-dense therapy (39% vs. 28%). Conclusions: This real-world analysis supports the use of dose-dense weekly paclitaxel combined with carboplatin for improved PFS, findings consistent with some regional studies. While OS data showed a non-significant trend favouring the dose-dense regimen with some risk of neuropathy, they highlight the need for larger prospective regional studies to better define patient populations most likely to benefit.
2026-05-27 | Preliminary efficacy and safety of first-line anlotinib plus carboplatin/paclitaxel induction therapy followed by anlotinib maintenance in advanced ovarian cancer: Results from the ALTER-GO-010 phase II multicenter trial.
5576 Background: Several studies have shown that antiangiogenic drugs combined with chemotherapy as first-line treatment, followed by maintenance therapy with antiangiogenic agents, significantly improve outcomes for ovarian cancer patients. Anlotinib, a highly effective multi-target tyrosine kinase inhibitor targeting VEGFRs, FGFRs, PDGFRs, and c-Kit, has been approved for multiple tumor types in China. The ALTER-GO-010 study is a single-arm, multicenter phase II trial designed to assess the efficacy and safety of anlotinib in combination with carboplatin/paclitaxel as first-line induction therapy, followed by anlotinib maintenance, in patients with newly diagnosed advanced ovarian cancer. Methods: Eligible patients with newly diagnosed FIGO stage III–IV primary epithelial ovarian, fallopian tube, or primary peritoneal cancer (ECOG PS 0–1) who have undergone primary or interval debulking surgery will receive 6-8 cycles of chemotherapy (paclitaxel 175 mg/m² + carboplatin AUC 5 every 3 weeks) combined with anlotinib (12 mg orally once daily on days 1–14, 21-day cycle; omitted during the first cycle to prevent delayed wound healing). Following chemotherapy completion, anlotinib will be continued as maintenance monotherapy until disease progression, unacceptable toxicity, or death. Exclusion criteria include prior anti-angiogenic therapy or major surgery within 28 days before anlotinib initiation. The primary endpoint is progression-free survival (PFS). Key secondary endpoints include overall survival, and safety profile. Results: As of the data cutoff date (November 30, 2025), 54 patients had received at least one dose of the study drug and were included in the analysis. The median age was 56 years, 98.11% had high-grade serous carcinoma and 1.89% had endometrioid carcinoma. 96.23% had FIGO stage III and 3.77% had stage IV. The data maturity for PFS analysis was 40.7%. The median PFS was 20.76 months (95% CI: 11.58-29.95). Patients without prior history of neoadjuvant therapy had longer mPFS than those with prior therapy. The median OS was not reached. The most common all-grade treatment emergent adverse events (TEAEs) were white blood cell deceased, anemia, neutrophil count decreased, platelet count decreased and lymphocyte count decreased. Grade ≥3 TEAEs occurred in 75.5% of patients during the chemotherapy phase and 22.7% during maintenance. No treatment-related deaths were reported. Conclusions: The preliminary efficacy and safety data suggest that anlotinib plus carboplatin/paclitaxel induction therapy, followed by anlotinib maintenance, is a promising regimen for newly diagnosed advanced ovarian cancer, with a median PFS of 20.76 months and a favorable safety profile. Clinical trial information: NCT04807166 .
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Drug Discovery Landscape
7 orphan drug designations for Primary peritoneal carcinoma, including 2 approved therapies.
7 orphan drug designations for Primary peritoneal carcinoma, including 2 approved therapies.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Gleolan | small molecules | FDA | 2024-02-15 | — | NX Development Corp. |
olaparib [Lynparza] | small molecules | FDA | 2018-05-15 | 2017-08-17 | AstraZeneca Pharmaceuticals LP |
antibody drug conjugate targeting dipeptidase 3 (DPEP3) | antibodies | FDA | 2018-01-08 | — | AbbVie Stemcentrx, LLC |
pelareorep | gene therapies | FDA | 2015-02-24 | — | Oncolytics Biotech, Inc. |
bevacizumab [Avastin] | antibodies | FDA | 2010-11-02 | 2014-11-14 | Genentech, Inc. |
patupilone | small molecules | FDA | 2010-02-02 | — | Novartis Pharmaceuticals Corporation |
Patupilone | small molecules | EMA | 2009-11-05 | — | Novartis Europharm Limited |
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