Gilteritinib, also known as ASP2215, is a potent FLT3/AXL inhibitor, which showed potent antileukemic activity against AML with either or both FLT3-ITD and FLT3-D835 mutations. In in vitro, among the 78 tyrosine kinases tested, Gilteritinib inhibited FLT3, LTK, ALK, and AXL kinases by over 50% at 1 nM with an IC50 value of 0.29 nM for FLT3, approximately 800-fold more potent than for c-KIT, the inhibition of which is linked to a potential risk of myelosuppression. Gilteritinib inhibited the growth of MV4-11 cells, which harbor FLT3-ITD, with an IC50 value of 0.92 nM, accompanied with inhibition of pFLT3, pAKT, pSTAT5, pERK, and pS6. Gilteritinib decreased tumor burden in bone marrow and prolonged the survival of mice intravenously transplanted with MV4-11 cells. In previous preclinical studies, gilteritinib has demonstrated superior antitumor effects when given in combination with AraC and either DNR or IDR compared with combination chemotherapy. In November 2018, the FDA approved gilteritinib for treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

Ceftolozane is a novel a cephalosporin-class antibacterial drug. In combination with a beta-lactamase inhibitor tazobactam (ZERBAXA, ceftolozane/tazobactam ) ceftolozane, is currently indicated for the treatment of the adult patients with complicated intra-abdominal infections caused by designated Gram-negative and Gram-positive microorganisms and complicated urinary tract infections caused by certain Gram-negative bacteria, including those caused by multi-drug resistant Pseudomonas aeruginosa. To reduce the development of drug-resistant bacteria and maintain the effectiveness of ZERBAXA and other antibacterial drugs, ZERBAXA should be used only to treat infections that are proven or strongly suspected to be caused by susceptible bacteria. Safety and effectiveness in pediatric patients have not been established.

Mirabegron (trade name Myrbetriq in the US and Betmiga in Europe) is a drug for the treatment of overactive bladder (OAB). It was developed by Astellas Pharma and was approved in the United States in July 2012. Originally developed as a treatment for diabetes, the development of mirabegron was later refocused to OAB. Mirabegron is an orally bioavailable agonist of the human beta-3 adrenergic receptor (ADRB3), with muscle relaxing, neuroprotective and potential antineoplastic activities. Upon oral administration, mirabegron binds to and activates ADRB3, which leads to smooth muscle relaxation. Mirabegron also restores sympathetic stimulation in mesenchymal stem cell (MSC) niches, inhibits JAK2-mutated hematopoietic stem cell (HSC) expansion and blocks the progression of myeloproliferative neoplasms (MPNs). Lack of sympathetic stimulation of the MSC and HSC niche is associated with the development of MPNs.

Tamsulosin, a sulfamoylphenethylamine-derivative alpha-adrenoceptor blocker with enhanced specificity for the alpha-adrenoceptors of the prostate, is commonly used to treat benign prostatic hyperplasia (BPH). The drug is commercially available in a racemic mixture of 2 isomers, and is pharmacologically related to doxazocin, prazosin, and terazosin. However, unlike these drugs, tamsulosin has a higher affinity for the alpha-1A- adrenergic receptors, which are located in vascular smooth muscle. Studies show that tamsulosin has about 12 times greater affinity for alpha-1 adrenergic receptors in the prostate than those in the aorta, which may result in a reduced incidence of adverse cardiovascular effects. Tamsulosin is sold under the trade name Flomax.

Amenamevir is an antiviral agent discovered by Astellas Pharma Inc. and is considered to exert anantiviral effect by inhibiting the activity of the helicase-primase complex, which is involved in DNA replication of herpes virus. It is a helicase-primase inhibitor against herpes simplex virus 1 (HSV-1), HSV-2, and varicella zoster virus. Amenamevir inhibited the in vitro replication of HSV-1 with a mean 50% effective concentration (EC(50)) of 14 ng/ml. Amenamevir was approved in Japan for the treatment of Herpes zoster.

ASP-3026 is a ALK tyrosine kinase receptor inhibitor which was developed by Astellas Pharma for the treatment of cancer conditions. The drug is being tested in phase I of clinical trials in patients with solid tumors, B-cell lymphoma and non-small cell lung carcinoma. The drug was discontinued for strategic reasons.

OBP-801 (spiruchostatin A) is an inhibitor of histone deacetylase (HDAC) enzymes, with potential antineoplastic activity. OBP-801 was originally identified as an enhancer of PAI-1 gene expression and was established as a new HDAC inhibitor by a p21 promoter reporter screen. Upon administration, OBP-801 inhibits the activity of HDACs; this results in an accumulation of highly acetylated chromatin histones, the induction of chromatin remodeling and an altered pattern of gene expression. This leads to selective transcription of tumor suppressor genes, tumor suppressor protein-mediated inhibition of tumor cell division and induction of tumor cell apoptosis. This may inhibit proliferation of susceptible tumor cells. HDAC, which is upregulated in many tumor cell types, deacetylates chromatin histone proteins. OBP‑801 induces M‑phase arrest and apoptosis in rhabdomyosarcoma cells. OBP‑801 is expected to show anticancer effect by promoting an expression of tumor suppressor genes in cancer cell and inducing apoptotic and autophagic cell death. The results of pre-clinical studies on OBP-801 indicated the most potent HDAC inhibitory activity as compared to other HDAC inhibitors including and Zolinza® and Istodax®, and its efficacy on a wide range of cancers is expected. Furthermore, Oncolys has been exploring the potential ophthalmic use of OBP-801 in collaboration with Kyoto Prefectural University of Medicine. OBP-801 has been used in trials studying the treatment of solid tumor.

Naquotinib (ASP8273) is an orally available, irreversible, mutant-selective, epidermal growth factor receptor (EGFR) inhibitor, with potential antineoplastic activity. Naquotinib was found by mass spectrometry to covalently bind to a mutant EGFR (L858R/ T790M) via cysteine residue 797 in the kinase domain of EGFR with long-lasting inhibition of EGFR phosphorylation for 24 h. In the NSCLC cell lines harboring the above EGFR mutations, Naquotinib had IC50 values of 8-33 nM toward EGFR mutants, more potently than that of WT EGFR (IC50 value of 230 nM). In mouse xenograft models, Naquotinib induced complete regression of the tumors after 14 days of treatment. ASP8273 even showed activity in mutant EGFR cell line which is resistant to other EGFR TKIs. Naquotinib is in phase III clinical trials for the oral treatment of EGFR mutated non-small cell lung cancer (NSCLC).

Sepantronium bromide (YM155) is a selective survivin suppressant that exhibits potent antitumor activities by inducing apoptosis and autophagy in various types of cancer. Sepantronium bromide inhibited the growth of various human cancer cell lines in vitro with GI50 values in the low nM range. Sepantronium bromide blocked the growth of 119 human cancer cell lines, with the greatest inhibition in lines derived from non-Hodgkin's lymphoma, hormone-refractory prostate cancer, ovarian cancer, sarcoma, non-small-cell lung cancer, breast cancer, leukemia, and melanoma, with an average GI50 of 15 nM. Sepantronium bromide inhibited the growth of tumor cell lines regardless of their p53 status and demonstrated significant antitumor activity in 5 mice xenograft models. It also caused tumor regressions in vivo, possibly by its effects in reducing intratumoral survivin expression levels and increasing apoptosis. Sepantronium Bromide had been in phase II clinical trials by Astellas for the treatment of prostate cancer, melanoma, non-Hodgkin's lymphoma, breast cancer, diffuse large B cell lymphoma, non-small cell lung cancer (NSCLC) and other solid tumors. This compound had also been in clinical trials by National Cancer Institute (NCI) for the treatment of solid tumors (phase I) and advanced non-small cell lung cancer (NSCLC) (phase II). However, all these researches about this compound for all indications were discontinued.

Peficitinib (formerly known as ASP015K) is a pyrrolo[2,3-b]pyridine derivative orally administered once-daily JAK inhibitor in development for the treatment of Rheumatoid Arthritis. In preclinical studied Peficitinib inhibited JAK1 and JAK3 with IC50 of 3.9 and 0.7 nM, respectively. Peficitinib also inhibited IL-2-dependent T cell proliferation in vitro and STAT5 phosphorylation in vitro and ex vivo. Furthermore, Peficitinib dose-dependently suppressed bone destruction and paw swelling in an adjuvant-induced arthritis model in rats via prophylactic or therapeutic oral dosing regimens.In clinical trials, Peficitinib treatment prescribed at 50, 100 and 150 mg amounts each showed statistically significantly higher ACR20 response rates compared to the placebo and response rates increased up to the 150 mg dosage. Adverse events included neutropenia, headache, and abdominal pain. The treatment-emergent adverse events occurring more frequently in the Peficitinib group compared with the placebo group included diarrhea, nasopharyngitis, and increased serum creatine phosphokinase activity. No cases of serious infections were reported. Herpes zoster occurred in four patients (two each in the peficitinib 25 and 100 mg cohorts). The authors concluded that treatment with peficitinib as monotherapy for 12 weeks in Japanese patients with moderate to severe RA is efficacious and showed an acceptable safety profile.