Zejula (niraparib) / Medison, GSK, ZAI Lab, J&J, Takeda - LARVOL DELTA

Modulating NPC1L1 to Potentiate PARP Inhibitor-Induced Ferroptosis and Immune Response in Triple-Negative Breast Cancer. (PubMed, Pharmaceutics) - "Combinatorial treatment with Niraparib and either Ezetimibe (an NPC1L1 inhibitor) or AZD5363 (an AKT inhibitor) synergistically enhanced TNBC cell death by promoting ferroptosis through glutathione depletion and lipid peroxidation. Furthermore, NPC1L1 inhibition amplified PARP inhibitor-induced immune responses, increasing CD8+ T cell infiltration and cytotoxicity in tumors. In conclusion, our findings establish NPC1L1 as a critical mediator of PARP inhibitor efficacy and propose dual targeting of lipid metabolism, providing a new therapeutic approach for the combination treatment of TNBC."

Journal • Breast Cancer • Immune Modulation • Immunology • Metabolic Disorders • Oncology • Solid Tumor • Triple Negative Breast Cancer • BRCA • CD8 • RELA

Exploring program-cell death patterns to predict prognosis and sensitivity of cervical cancer immunotherapy via multi-omics analysis and clinical samples. (PubMed, Discov Oncol) - "This study establishes CDI as the PCD-based immune signature for CC, enabling precise prognosis prediction and ICI candidate selection. The CDI framework provides actionable insights for combination therapies targeting PCD-immune interplay, with translational potential for personalized oncology."

IO biomarker • Journal • Tumor mutational burden • Cervical Cancer • Oncology • Solid Tumor • CD46 • CTLA4 • GAPDH • PD-1 • PGK1 • PLAU • TMB

In Vitro Drug Profiling to Guide Treatment for Relapse/Refractory AML (ASH 2024) - "Significant correlation was observed among drugs of the same classes, for example between inhibitors of PARP (e.g. niraparib-talazoparib, r=0.78, p=1.3e-22), proteasome (e.g. bortezomib-ixazomib, r=0.90, p=4.2e-36), JAK (ruxolitinib-tofacitinib, r=0.91, p=8.3e-35), MEK (cobimetinib-trametinib, p=0.93, p=8.8e-47) and CDK (abemaciclib-palbociclib, p=0.56, p=2.7e-10), confirming that the readout is biologically meaningful. Intriguingly, there were unexpected correlations between specific pairs of drugs of different classes, for instance homoharringtonine (protein translation inhibitor)-abemaciclib (CDK inhibitor) (r=0.65, p=4.3e-17) and between specific gene mutations and drug sensitivity was observed, e.g. sensitivity of CEBPAbZIP mutated samples to PARP inhibitors (p=0.00156), and of AML with inv(16) to MEK inhibitors (p=0.0016)...Drug response to daunorubicin showed good prediction of chemo-resistance in patients who had non-remission after "7+3" (ROC curve AUC..."

Preclinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • ANXA5 • FLT3

Early Access Program in oncology: Retrospective study at a Portuguese hospital (ECOP 2024) - "During the study period were submitted 163 EAP requests for abiraterone, amivantamab, bevacizumab, durvalumab, encorafenib, enfortumab, everolimus, erdafitinib, lenvatinib, lurbinectedin, niraparib, nivolumab, olaparib, pembrolizumab, pertuzumab, ramucirumab, sacituzumab govitecan, selpercatinib, trifluridine/tipiracil, trametinib+dabrafenib, trastuzumab-deruxtecan and tucatinib. Conclusion Most cases correspond to metastatic disease, EAPs facilitate timely access to innovative therapies for patients with high unmet medical needs. The majority of situations were financed, which confirms the importance of EAPs in an era where oncology is constantly innovating."

Retrospective data • Gastrointestinal Disorder • Oncology

Niraparib plus PD-1 inhibitor for patients previously treated with immune checkpoint inhibitor for solid tumors with homologous recombination repair gene mutation (IMAGENE): A phase II basket study. (ASCO 2025) - P | "Patients were treated on a 21-day cycle with niraparib (200 mg orally daily) and nivolumab/pembrolizumab (240 mg/body intravenously every 2 weeks or 3 mg/kg every 3 weeks, respectively). Niraparib combined with PD-1 inhibitor showed modest activity even in heavily pretreated patients with HRR gene-mutated cancers who progressed on ICI therapy. Furthermore, patients with specific cancer type had promising benefit from this combination therapy, warranting further investigation in specific populations."

Checkpoint inhibition • Clinical • IO biomarker • P2 data • Pan tumor • Anemia • Bladder Cancer • Gastric Cancer • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor • BRCA1 • BRCA2 • HRD

Exceptional Response in a Patient with mRCC Through Precision-Guided Treatment Involving Immunotherapy Rechallenge with Temsirolimus and Bevacizumab. (PubMed, J Immunother Precis Oncol) - "The patient with metastatic renal cell carcinoma (mRCC) presented here demonstrated an absence of a favorable response accompanied by adverse events after receiving dual immunotherapy with nivolumab plus ipilimumab in combination with a poly (adenosine diphosphate-ribose) polymerase inhibitor, niraparib...The decision was made to transition the patient's treatment to temsirolimus plus bevacizumab, with the rechallenge of immunotherapy with pembrolizumab...This case study underscores the mounting significance of precision oncology in the management of mRCC, thereby suggesting that mammalian target of rapamycin inhibitor may augment the efficacy of immunotherapy in select patients based on their genomic findings. A digital poster of this case is included in the supplemental materials."

Journal • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

Niraparib Plus Aromatase Inhibitors for Hormone Receptor-Positive/HER2-Negative Advanced Breast Cancer with a Germline BRCA Mutation (Multidisciplinary Digital Publishing Institute) - P2 | N=14 | LUZERN (NCT04240106) | "Between June 2020 and November 2022, 14 patients were enrolled in cohort A (n = 6 for stage I, n = 8 for stage II) and no patients were enrolled in cohort B. One patient was excluded from the efficacy analysis due to no prior AI treatment. Nearly all patients (92.9%) previously received a cyclin-dependent kinase 4/6 inhibitor, but no patients had received prior platinum-based chemotherapy. Median follow-up was 16.7 months (range: 13.2–18.2). The CBR was 46.2% (95% CI: 19.2–74.9), meeting the primary endpoint. Median progression-free survival was 5.5 months (95% CI: 1.9–8.5), and median overall survival was 18.1 months (95% CI: 9.7–NE)."

P2 data • HER2 Negative Breast Cancer • Hormone Receptor Positive Breast Cancer

Peripheral Neuropathy in Patients With Ovarian Cancer Administrating Poly ADP-Ribose Polymerase Inhibitors: A Real-World Study Based on Bayesian Disproportionality Analysis of the US Food and Drug Administration Adverse Event Reporting System. (PubMed, Clin Ther) - "For patients with EOC, prudent surveillance of peripheral neuropathy is warranted when administrating niraparib. Certainly, more large-scale and long-term follow-up period studies were entailed."

Adverse events • Journal • Real-world evidence • Epithelial Ovarian Cancer • Fallopian Tube Cancer • Oncology • Ovarian Cancer • Pain • Peritoneal Cancer • Solid Tumor

NIRAPK : Study of the Relationship(s) Between Clinical, Biological and Pharmacokinetic Metrics and Toxicities When Niraparib is Used as Maintenance Treatment for Ovarian Cancer Patients. (clinicaltrials.gov) - P4 | N=12 | Terminated | Sponsor: Hospices Civils de Lyon | N=42 ➔ 12 | Trial completion date: May 2026 ➔ Apr 2025 | Recruiting ➔ Terminated | Trial primary completion date: May 2026 ➔ Apr 2025; End of inclusion period.

Enrollment change • Platinum sensitive • Trial completion date • Trial primary completion date • Trial termination • Epithelial Ovarian Cancer • Oncology • Ovarian Cancer • Solid Tumor • BRCA

Niraparib and Selenium for the Treatment of Recurrent BRCA Negative Platinum Resistant Ovarian Cancer (clinicaltrials.gov) - P1/2 | N=0 | Withdrawn | Sponsor: City of Hope Medical Center | N=45 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Platinum resistant • Trial withdrawal • Fallopian Tube Cancer • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor • BRCA

Folate deficiency anemia in patients on PARP inhibition: Insights from a large single-center study. (ASCO 2025) - "Of these, 16 (3.1%) experienced new FD to a nadir folate of < 2.2 - < 3.3 ng/mL within a median of 16.1 (IQR 8.0-38.2) weeks of olaparib (N = 15) or niraparib (N = 1) initiation. This study is the first to systematically demonstrate the prevalence of FD anemia in association with PARPi use. In the United States population, FD is present in only < 0.1% of adults. Within our cohort of PARPi recipients, 3.1%—and potentially many more given another 29.7% with new unexplained macrocytic anemia—experienced clinically significant FD anemia on PARPi."

Clinical • Anemia • Hematological Disorders • Oncology • Pancreatic Cancer • BRCA1 • BRCA2

Cardiac adverse events with PARP inhibitors: Real world pharmacovigilance study using FAERS database. (ASCO 2025) - " This study evaluates real-world data on PARPi-associated cardiac events using the FDA Adverse Event Reporting System (FAERS) database.The FAERS database was queried on January 25, 2025, using product-specific terms ("Olaparib," "Rucaparib," "Rucaparib camsylate," "Niraparib," "Niraparib Tosylate monohydrate," "Talazoparib," and "Talazoparib Tosylate"), identifying 1,925 cardiac events. Higher RORs for arrhythmias were observed with Niraparib and Talazoparib compared to other drugs in FAERS. While these findings suggest potential safety signals, they do not establish causality and warrant larger studies to evaluate these associations. Baseline demographics for all cardiac events and ROR for specific cardiac events divided in 5 subgroups for PARPi."

Adverse events • Clinical • Real-world • Real-world evidence • Cardiovascular • Oncology • BRCA1 • BRCA2

Molecular docking investigating methotrexate as a potential target for addressing cardiovascular disease risk associated with cancer. (ASCO 2025) - " The molecular docking modeling and analysis was performed using AutoDock Tool 4.2 version software to model the binding interaction between RAGE and each of the four anticancer drugs studied: MTX, Niraparib, Abemaciclib, and Saracatinib. The molecular docking findings show that among the anticancer drug interactions studied, MTX had the strongest binding interaction with RAGE. This suggests that MTX may potentially reduce the progression of atherosclerosis and CVD risk among cancer survivors by inhibiting RAGE."

Atherosclerosis • Cardiovascular • Oncology

Real-world pharmacovigilance study of myelotoxicity with PARP inhibitors in breast and ovarian malignancies. (ASCO 2025) - " We queried the FAERS database for Olaparib, Rucaparib, Niraparib, Talazoparib, and AEs reported between 2016-2024 were categorized. Olaparib has the highest incidence for myelotoxicity, AML and MDS among the different PARPi in breast and ovarian cancer. Close monitoring is essential for patients receiving PARPi, with early recognition and management of myelosuppression crucial to preventing progression to hematological malignancies. Table 1: Myelotoxicity outcomes with different PARP inhibitors"

Adverse events • Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Breast Cancer • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Ovarian Cancer • BRCA1 • BRCA2

Unveiling the power of PARP inhibitors: A meta-analysis on newly diagnosed advanced ovarian cancer maintenance therapy. (ASCO 2025) - "PARPi have demonstrated efficacy as maintenance therapy in patients with newly diagnosed advanced OC. Among patients with BRCAwt tumors, niraparib showed greater effectiveness compared to veliparib."

Metastases • Retrospective data • Oncology • Ovarian Cancer • Solid Tumor • BRCA • HRD

Incidence of myelodysplastic syndrome in patients treated with PARP inhibitors: A systematic review. (ASCO 2025) - "Olaparib was the most common used PARPi (47%) followed by Niraparib (15%), Rucaparib (18%), and Veliparib (19%). This systematic review shows that PARPi treatment is associated with an increased incidence of MDS. There is need for continuous monitoring and risk assessment in patients undergoing PARPi therapy to enhance patient safety and improve treatment outcomes."

Clinical • Review • Breast Cancer • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Ovarian Cancer • Solid Tumor • BRCA • BRCA1 • BRCA2

Ex vivo 3D micro-tumor testing platform for predicting clinical response to platinum-based therapy in patients with high-grade serous ovarian cancer. (ASCO 2025) - "Micro-tumors enriched from ascites were embedded in hydrogel and exposed to carboplatin, paclitaxel and alternative therapies doxorubicin, gemcitabine, topotecan, olaparib and niraparib. An ex vivo 3D micro-tumor testing platform has been established. Results are generated within 2 weeks after sample collection, aligning with the clinical time frame for treatment decision-making. The platform enabled prediction of clinical response to NACT in ovarian cancer patients with high accuracy (CR/PR vs SD/PD p < 0.01) and measured patient-specific responses to second-line therapies."

Preclinical • High Grade Serous Ovarian Cancer • Oncology • Ovarian Cancer • Solid Tumor

Analysis of niraparib-induced changes in glucose levels during three-month therapy in normoglycemic patients with ovarian cancer. (ASCO 2025) - "In view of the observed changes in glycemia during niraparib therapy, it seems essential to recommend monitoring of this parameter during therapy with this PARPi, as in the case of olaparib. 1. Stanislawiak-Rudowicz et al."

Clinical • Hypoglycemia • Oncology • Ovarian Cancer • Solid Tumor

Real world data of maintenance treatment in homologous recombination (HR)–proficient HGSOC. (ASCO 2025) - "In the HRD- population, 24 (33.8%) patients received maintenance treatment with bevacizumab while 31 (43.7%) patients received maintenance treatment with PARP inhibitor niraparib. In HR-proficient patients, first line maintenance treatment was not significantly associated with PFS in multivariate analysis. Maintenance treatment types according to HRD and BRCA mutation status.PARPi: PARP inhibitor."

Clinical • Real-world • Real-world evidence • Gynecologic Cancers • High Grade Serous Ovarian Cancer • Oncology • Ovarian Cancer • BRCA

Outcomes for patients with platinum-sensitive recurrent ovarian cancer treated with prolonged PARP inhibitor therapy. (ASCO 2025) - "Olaparib was most prescribed (n=19, 46.3%) followed by niraparib (n=14, 34.2%). Individuals with PSROC who display a prolonged response (at least 24 months) to PARPi therapy still face a significant recurrence risk of over 60% at five years, suggesting that indefinite treatment may be warranted despite the risks of hematopoietic toxicity. Comparative trial data will be useful to define the optimal duration of therapy."

Clinical • Platinum sensitive • Epithelial Ovarian Cancer • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Ovarian Cancer • Solid Tumor • BRCA • BRCA1 • BRCA2 • HRD • RAD51C

Real-world outcomes among patients with metastatic castration-resistant prostate cancer (mCRPC) receiving guideline-recommended therapies after treatment with 177Lu-PSMA-617: A real-world prostate cancer disease observation (PRECISION) data platform analysis. (ASCO 2025) - "Guideline-recommended therapies included abiraterone, enzalutamide, darolutamide, apalutamide, cabazitaxel, docetaxel, pembrolizumab, sipuleucel-T, niraparib, olaparib, talazoparib, rucaparib, and radium-223. In this real-world analysis, the majority of patients who received guideline-recommended therapies after 177Lu-PSMA-617 achieved at least a PSA50 response, suggesting that 177Lu-PSMA-617 treatment does not preclude response to other subsequent therapies."

Clinical • Metastases • Real-world • Real-world evidence • Castration-Resistant Prostate Cancer • Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor • Urology

Real-world analysis of primary tumor transcriptomes in patients subsequently treated with PARP inhibitors. (ASCO 2025) - P=N/A | "PARP inhibitor (PARPi) use after diagnosis therapy was defined by the presence of one or more NDC-9 codes for olaparib, niraparib, rucaparib and talazoparib. In this real-world study of patients initially presenting with non-metastatic disease who subsequently received PARP inhibitors, transcriptome analysis from the primary tumor revealed the presence of adverse molecular features when disease was still localized. Patients with higher Decipher score, p53 mutation signature and PTEN inactivation tumors at initial testing, could be considered for additional confirmatory genomic or genetic testing and potentially novel trials of PARPi."

Clinical • Real-world • Real-world evidence • Castration-Resistant Prostate Cancer • Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor • CD8 • PTEN • RB1 • TP53

Comparative efficacy of PARP inhibitors in BRCA mutated HER2-negative breast cancer: A systematic review and network meta-analysis of randomized controlled trials. (ASCO 2025) - "Physician's choice chemotherapy (PCT) included capecitabine, eribulin, gemcitabine, or vinorelbine. Veliparib combined with SOC showed the highest OS improvement, while niraparib ranked highest for PFS, followed by talazoparib and olaparib. Further trials are needed to confirm these findings and refine treatment strategies for BRCAm, HER2-BC."

Retrospective data • Review • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • HER2 Positive Breast Cancer • Oncology • Solid Tumor • BRCA • BRCA1 • BRCA2 • HER-2 • HRD

A phase 1 study of PARP inhibitor (niraparib) plus HSP90 inhibitor (pimitespib) in solid tumors: Dose-expansion results from the NiraPim (EPOC2102) study. (ASCO 2025) - P1 | "In cohort A, one patient with hormone receptor-positive breast cancer achieved partial response post-olaparib progression. The dose-expansion part demonstrated a manageable safety profile and potential efficacy at the recommended dose of niraparib plus pimitespib. Clinical benefit was observed in both BRCA-associated cancers resistant to PARP inhibitors and PARP inhibitor-naive non-BRCA associated cancers, supporting further investigation in biomarker-selected populations."

P1 data • Anorexia • Breast Cancer • Fatigue • Genito-urinary Cancer • Hormone Receptor Breast Cancer • Hormone Receptor Positive Breast Cancer • Leiomyosarcoma • Oncology • Ovarian Cancer • Pancreatic Cancer • Prostate Cancer • Sarcoma • Solid Tumor • Urothelial Cancer • BRCA • CDC37 • CDK12 • HRD

A phase 1 study of abemaciclib and niraparib as neoadjuvant therapy in hormone receptor positive and HER2 negative breast cancer. (ASCO 2025) - P1 | "This phase 1 study established abemaciclib 150 mg PO BID and niraparib 100 mg PO daily as the recommended phase 2 dose for this combination. Planned correlative studies are underway. Participant characteristics"

Clinical • P1 data • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • HER2 Positive Breast Cancer • Hormone Receptor Breast Cancer • Hormone Receptor Positive Breast Cancer • Oncology • Ovarian Cancer • Solid Tumor • BRCA • HER-2