Fasidotril is a diester prodrug of the active metabolite fasidotrilat. Fasidotrilat inhibited both angiotensin I converting enzyme (ACE, EC 3.4.25.1) and neprilysin (NEP, EC 3.4.24.11, also named neutral endopeptidase, enkephalinase, or atriopeptidase) at nanomolar concentrations (Ki = 9.8 nM against ACE and 5.1 nM against NEP) Fasidotril was being developed for the treatment of myocardial infarction, congestive heart failure and myocardial infarction.

PZ-128 (also known as P1pal7 ) is a cell-penetrating pepducin inhibitor of PAR1 that targets the receptor-G-protein interface on the inside surface of platelets. In preclinical studies, PZ-128 suppresses PAR1 aggregation and arterial thrombosis in guinea pigs and baboons and strongly synergized with oral clopidogrel. PZ-128 shows potent anti-metastatic and anti-angiogenic activity in Breast, Ovarian, and Lung Cancer preclinical studies. In clinical trials, PZ-128 shows a promising antiplatelet activity that provides rapid, specific, dose-dependent, and reversible inhibition of platelet protease-activated receptor-1 through a novel intracellular mechanism.

Stercuronium is a conessine derivative patented by Koninklijke Nederlandsche Gist-en Spiritusfabriek N. V. as a competitive neuromuscular blocking agent with antimuscarinic activity. In preclinical models, Stercuronium produced significantly greater inhibition (P < 0.05) of the bradycardia than of the vasodepressor response produced by carbachol. In guinea-pig atria, the negative chronotropic response to carbachol was inhibited to a similar degree to the negative inotropic response by Stercuronium, whereas in bladder and ileum Stercuronium was 17 fold less active as an antimuscarinic drug. The affinity of Stercuronium for the prejunctional muscarinic receptor on sympathetic nerve endings in the rabbit ear artery was similar to that for the muscarinic receptor mediating negative inotropic responses to carbachol in the rabbit left atrium. Unfortunately, in clinical trials Stercuronium producing marked tachycardia in some patients when used as a muscle relaxant.

QAV-680 is a pyrrolopyridine derivative patented by Novartis Pharma GmbH as chemoattractant receptor (CRTh2) antagonist for the treatment of an inflammatory or allergic condition, particularly an inflammatory or obstructive airways disease. QAV-680 shows low nM potency and excellent selectivity across a range of CRTh2 dependent primary human inflammatory cell assays. In preclinical studies, QAV-680 shows good pharmacokinetic properties (including a suitable escalating dose–exposure relationship) in the rat and mouse and demonstrates efficacy in both mechanistic and allergic disease CRTh2-dependent rodent in vivo models. In 2008-2010 QAV-680 was tested in Phase 2 clinical trials but no further development reports were published.

AKN-028 is an orally bioavailable protein tyrosine kinase inhibitor for FMS-related tyrosine kinase 3 (IC(50)=6 nM) and stem cell factor receptor (SCFR; KIT), with potential antineoplastic activity. FLT3/KIT kinase inhibitor AKN-028 binds to and inhibits both the wild-type and mutated forms of FLT3 and SCFR. Akinion Pharmaceuticals is developing AKN-028 for the treatment of acute myeloid leukaemia. AKN-028 is presently undergoing investigation in a phase I/II study.

CUDC-907 is a small molecule inhibitor of histone deacetylase and PI3 kinase developed by Curis. It is investigated in clinical trials for the treatment of relapsed or refractory lymphomas, thyroid cancer, multiple myeloma, breast cancer and other malignancies.

Pinometostat, also known as EPZ-5676, is a small molecule inhibitor of histone methyltransferase with potential antineoplastic activity. Upon intravenous administration, EPZ-5676 specifically blocks the activity of the histone lysine-methyltransferase DOT1L, thereby inhibiting the methylation of nucleosomal histone H3 on lysine 79 (H3K79) that is bound to the mixed lineage leukemia (MLL) fusion protein which targets genes and blocks the expression of leukemogenic genes. Epizyme is developing pinometostat, a small molecule inhibitor of DOT1L, for the treatment of patients with MLL-r, a genetically defined acute leukemia. Epizyme is conducting a phase 1 clinical trial in pediatric patients. Epizyme is evaluating preclinical combinations of pinometostat with other anti-cancer agents in MLL-r leukemia. Pinometostat is being developed in collaboration with Celgene. Epizyme retains all U.S. rights to pinometostat and has granted Celgene an exclusive license to pinometostat outside of the U.S.