Asapiprant, also known as S-555739, a selective Prostaglandin D2 receptor 1 antagonist, participated in phase III of clinical trials in Japan and in phase II of trials in the USA for patients with Allergic rhinitis.

AZD-4547 is an orally bioavailable inhibitor of the fibroblast growth factor receptor (FGFR) with potential antineoplastic activity. FGFR inhibitor AZD4547 binds to and inhibits FGFR, which may result in the inhibition of FGFR-related signal transduction pathways, and, so, the inhibition of tumor cell proliferation and tumor cell death. FGFR, up-regulated in many tumor cell types, is a receptor tyrosine kinase essential to tumor cellular proliferation, differentiation and survival. AZD-4547 is a selective FGFR inhibitor targeting FGFR1/2/3 with IC50 of 0.2 nM/2.5 nM/1.8 nM in cell-free assays, weaker activity against FGFR4, VEGFR2 (KDR), and little activity observed against IGFR, CDK2, and p38. Compared to FGFR1-3, AZD-4547 displays weaker activity against FGFR4 with IC50 of 165 nM. AZD-4547 only inhibits recombinant VEGFR2 (KDR) kinase activity with IC50 of 24 nM, in the in vitro selectivity test against a diverse panel of representative human kinases. AZD-4547 is under clinical investigation for the treatment of FGFR-dependent tumors. It is in phase II clinical studies for the treatment of breast cancer; gastric cancer; lung cancer; oesophageal cancer and in phase II/III clinical studies for the treatment of non-small cell lung cancer.

Pavinetant, also known as MLE-4901, AZD-4901, AZD-2624, is a small molecule, orally active, selective neurokinin-3 (NK3) receptor antagonist developed for schizophrenia, hot flashes, and Polycystic ovary syndrome. The development for schizophrenia was discontinued due to lack of effectiveness. Phase II trials for polycystic ovary syndrome and for hot flashes showed the clinical risks exceeded benefits, in addition, abnormal liver function was observed, and that is why these studies were also discontinued.

GKT137831, a novel pyrazolopyridinedione derivative, a dual inhibitor of NADPH oxidases (NOX) 1 and NOX4, reduces inflammation in the ischemic retina by dampening the pro-inflammatory phenotype of retinal immune cells as well as macroglial Müller cells and neurons. In patients with diabetic kidney disease, GKT137831 demonstrated an excellent safety profile and statistically significant reduction in both liver enzyme and inflammatory marker levels in multiple phase I and phase II clinical studies. GKT137831 may be of significant benefit for patients with systemic sclerosis as studies in experimental models show that the compound may reduce the abnormal growth of connective tissue (fibrosis) and improve survival. GKT137831 was granted Orphan Drug designation for the treatment of systemic sclerosis from the FDA and the EMA. GKT-137831 acts as a Nox4 and Nox1 inhibitor ((Ki 100–150 nM). GKT-137831 attenuates hypoxia-induced pulmonary vascular cell proliferation. Also a potent inhibitor of fibrosis and hepatocyte apoptosis.

Voxtalisib (SAR245409, XL765) is an orally available inhibitor of PI3K and the mammalian target of rapamycin (mTOR), which are frequently activated in human tumors and play central roles in tumor cell proliferation. Exelixis discovered Voxtalisib internally and out-licensed the compound to Sanofi. Voxtalisib is being evaluated by Sanofi as a single agent and in multiple combination regimens in a variety of cancer indications. Clinical trials have included a single agent phase 2 trial in Non-Hodgkin’s lymphoma, combination phase 1b/2 trials with temozolomide in patients with glioblastoma, with letrozole in hormone receptor positive breast cancer, with bendamustine and/or rituximab in lymphoma or leukemia, and a phase 1 trial in combination with a MEK inhibitor. Voxtalisib is a highly selective, potent and reversible ATP-competitive inhibitor of pan-Class I PI3K (α, β, γ, and δ) and mTORC1/mTORC2. It is orally active, highly selective over 130 other protein kinases. In cellular assays, XL765 inhibits the formation of PIP3 in the membrane, and inhibits phosphorylation of AKT, p70S6K, and S6 phosphorylation in multiple tumor cell lines with different genetic alterations affecting the PI3K pathway.