LMI-070 is an experimental compound being developed by Novartis Pharma as a treatment for spinal muscular atrophy (SMA). It is a small-molecule drug which modifies alternative splicing of the SMN2 gene, bringing about increased levels of SMN protein. LMI-070 originated from a high-throughput phenotypic screening hit, pyridazine 2, and evolved via multiparameter lead optimization. In a severe mouse SMA model, LMI-070 treatment increased full-length SMN RNA and protein levels, and extended survival. LMI-070 is taken orally, usually in a liquid form once a week. It is in phase II clinical trial.

CGC-11047 is a polyamine analog designed to halt cell growth and induce apoptosis of cancer cells. In preclinical models CGC-11047 significantly inhibited tumor development in both lung and prostate cancer models when administered as a single agent. In the lung cancer model, CGC-11047 potentiated the antitumor effect of cisplatin. Although potent activity was observed with CGC-11047 and bevacizumab when administered as single agents in the prostate cancer model, the combination arm significantly enhanced antitumor activity compared with either agent alone. In all experiments, CGC-11047 was well tolerated with no adverse effects on bodyweight gain.

Entospletinib (GS-9973) is an adenosine triphosphate competitive inhibitor of Syk that disrupts kinase activity, which is currently in clinical trials for multiple B-cell malignancies. The most common treatment-emergent serious adverse events included dyspnea, pneumonia, febrile neutropenia, dehydration, and pyrexia.

T-817 is a thiophenylalkyl derivative patented by Toyama Chemical Co., Ltd. as a neuroprotectant for the treatment of Alzheimer's disease. T-817MA protects neurons against amyloid-b peptide- and hydrogen peroxide-induced neurotoxicity and promoted neurite outgrowth in hippocampal slice cultures and neuronal reaggregation culture. T-817MA protected against nitric oxide-induced neurotoxicity in cultured primary neurons. In a preclinical model of Alzheimer's disease, T-817 increases decreased dopamine levels and tyrosine hydroxylase immunostaining in mice's substantia nigra (SNc) and striatum. Systemic administration of T-817 rescues tau-induced synaptic abnormalities.

JNJ-37822681 is a novel, potent, specific, centrally active, dopamine D2 receptor antagonist, which was developed by Johnson & Johnson. This drug is in the phase II of clinical trial for the treatment of schizophrenia. JNJ-37822681 has optimal brain disposition and somnolence was the most frequently reported adverse effect.