226 articles
Milademetan
Milademetan (DS-3032b, RAIN-32) is an oral spiro-oxindole MDM2-p53 interaction inhibitor from Daiichi Sankyo licensed to Rain Therapeutics; its phase III MANTRA trial in dedifferentiated liposarcoma failed, and it remains unapproved.
Momelotinib
Momelotinib (OJJAARA, Omjjara) is an oral JAK1/JAK2/ACVR1 inhibitor approved in 2023 for intermediate- or high-risk myelofibrosis with anemia, distinguished from other JAK inhibitors by improving hemoglobin and transfusion independence via hepcidin suppression.
Motesanib
Motesanib (AMG 706), an oral tyrosine kinase inhibitor targeting VEGFR 1-3, PDGFR, Kit, and Ret, developed by Amgen and Takeda for thyroid cancer and NSCLC; phase 3 trials failed and development was discontinued.
Napabucasin
Napabucasin (BBI608) is an oral small-molecule anticancer drug that inhibits the STAT3 pathway, tested in phase 3 colorectal, gastric, and pancreatic cancer trials, all of which missed their survival endpoints.
Naquotinib
Naquotinib (ASP8273), Astellas' third-generation irreversible mutant-selective EGFR tyrosine kinase inhibitor for EGFR-mutated NSCLC including T790M; phase 3 SOLAR trial stopped for futility in 2017, development discontinued, never approved.
NAVIGATE trial
NAVIGATE (NCT02576431) is Bayer's phase 2 basket trial of larotrectinib in patients 12 and older with advanced NTRK fusion-positive solid tumors; key trial behind Vitrakvi's 2018 tumor-agnostic approval.
Navitoclax
Navitoclax (ABT-263, A-855071.0) is an oral Bcl-2 family inhibitor targeting Bcl-2, Bcl-XL, and Bcl-w, studied in lymphoma, CLL, and solid tumors; development limited by Bcl-XL-driven thrombocytopenia, leading to venetoclax.
Navtemadlin
Navtemadlin, formerly AMG-232 and KRT-232, is an investigational oral MDM2 inhibitor that restores p53 activity in cancer cells, developed by Amgen and now held by Kartos Therapeutics.
Niraparib
Niraparib (Zejula; Akeega combination) is an oral PARP-1/PARP-2 inhibitor approved in 2017 as maintenance therapy for HRD-positive ovarian, fallopian tube, and peritoneal cancer, and in 2025 with abiraterone for BRCA2-mutated prostate cancer.
Nirogacestat
Nirogacestat (Ogsiveo, PF-03084014) is SpringWorks' oral gamma secretase inhibitor blocking Notch signaling, approved by the FDA on November 27, 2023 as the first systemic therapy for progressing desmoid tumors.
NRC-AN-019
NRC-AN-019 (AN-019, CAS 879507-25-2) is Natco Pharma's imatinib analogue Bcr-Abl tyrosine kinase inhibitor for imatinib-resistant CML; FDA orphan drug designation 2011 for CML, glioma and pancreatic cancer; Phase I completed in India.
Obinutuzumab/venetoclax regimen
The obinutuzumab/venetoclax regimen is a fixed-duration combination of the antibody obinutuzumab and the oral BCL-2 inhibitor venetoclax, used to treat previously untreated chronic lymphocytic leukemia.
Olmutinib
Olmutinib (BI 1482694, HM61713, brand Olita) is an oral, irreversible, mutant-selective third-generation EGFR tyrosine kinase inhibitor for T790M-positive NSCLC, approved in South Korea in 2016 but discontinued after fatal skin toxicity and inferior results versus osimertinib.
Olutasidenib
Olutasidenib (Rezlidhia) is an oral mutant IDH1 inhibitor approved by the FDA in December 2022 for relapsed or refractory AML with a susceptible IDH1 mutation, dosed 150 mg twice daily, with 35% CR+CRh and a boxed warning for differentiation syndrome.
Olverembatinib
Olverembatinib (奥雷巴替尼, Henglike, HQP1351, formerly GZD824) is an oral third-generation BCR-ABL1 tyrosine kinase inhibitor for T315I-mutated and TKI-resistant CML, approved in China since November 2021 and in global phase 3 development.
Opnurasib
Opnurasib (JDQ443, NVP-JDQ-443) is a Novartis investigational covalent KRAS G12C inhibitor binding the Switch II pocket with a novel H95-sparing mode, in phase III (KontRASt-02) versus docetaxel in NSCLC.
Oritinib mesylate (SH-1028)
Oritinib mesylate (SH-1028, rilertinib, 圣瑞沙) is Nanjing Sanhome's irreversible third-generation EGFR TKI for NSCLC; NMPA approved it in 2024 for T790M-positive second-line and first-line EGFR-mutated disease.
Osimertinib regimen
The osimertinib regimen is an oral treatment plan for EGFR-mutated non-small cell lung cancer, built around osimertinib (Tagrisso), a third-generation EGFR inhibitor taken as an 80 mg tablet once daily.
Pacritinib
Pacritinib (VONJO), a JAK2/FLT3/IRAK1 kinase inhibitor approved by FDA in 2022 for myelofibrosis with platelets below 50 × 10^9/L, dosed 200 mg twice daily, developed through PERSIST-1, PERSIST-2, and PAC203 trials.
Pamiparib
Pamiparib (BGB-290) is BeiGene's oral PARP1/PARP2 inhibitor for HRD cancers; conditionally approved in China for germline BRCA ovarian cancer and studied with tislelizumab, temozolomide, and surufatinib.
Parsaclisib
Parsaclisib (INCB050465, IBI-376) is Incyte's investigational next-generation PI3Kδ inhibitor for B-cell malignancies; IC50 1 nM, 10,000-fold selective, EMA application withdrawn 2022, phase 3 myelofibrosis study failed.
Pegargiminase
Pegargiminase (ADI-PEG 20, pegylated arginine deiminase), Polaris Pharmaceuticals' PEGylated bacterial enzyme that depletes blood arginine to kill ASS1-deficient cancers; positive phase 3 ATOMIC-Meso trial in mesothelioma, trials in HCC, melanoma, sarcoma.
Pelcitoclax
Pelcitoclax (APG-1252), an investigational Ascentage Pharma BH3 mimetic, dual Bcl-2/Bcl-xL inhibitor designed as a prodrug to reduce platelet toxicity, in trials for SCLC, NSCLC, and other cancers.
Perifosine
Perifosine (KRX-0401, D-21266), an oral alkylphospholipid Akt inhibitor derived from miltefosine, tested in phase 3 trials for multiple myeloma and colorectal cancer; development ended after failed endpoints and withdrawn orphan designations.
Pevonedistat
Pevonedistat (MLN4924, TAK-924) is a first-in-class small-molecule NEDD8-activating enzyme inhibitor studied in AML, MDS, and CMML with azacitidine; its phase 3 PANTHER trial missed event-free survival.
Pexidartinib
Pexidartinib, sold as Turalio, is an oral tyrosine kinase inhibitor targeting CSF1R, approved in 2019 as the first systemic treatment for symptomatic tenosynovial giant cell tumor that surgery cannot improve, under a liver-safety REMS program.
Pirtobrutinib
Pirtobrutinib (Jaypirca, LOXO-305) is an oral, non-covalent, reversible BTK inhibitor by Eli Lilly for mantle cell lymphoma and CLL/SLL, active against C481-mutant BTK; FDA approved January 27, 2023.
Porfiromycin
Porfiromycin (methylmitomycin C, Promycin, ENT-50825, NSC-56410) is an N-methyl mitomycin C analogue from Streptomyces ardus, a hypoxia-selective bioreductive DNA-cross-linking antibiotic tested in phase III head and neck cancer trials but never FDA approved.
Poziotinib
Poziotinib (HM781-36B, NOV-120101) is an oral irreversible pan-HER tyrosine kinase inhibitor developed for EGFR and HER2 exon 20 insertion-mutant cancers; ZENITH20 showed modest efficacy and marked toxicity, and it was never FDA approved.
Pracinostat
Pracinostat (SB939), an oral pan-HDAC inhibitor cancer drug candidate with FDA orphan drug status for AML; phase 3 PRIMULA showed no survival benefit over azacitidine alone.