AMG-319 is an oral, small molecule inhibitor of PI3Kdelta which is now being tested in phase II for the treatment of head and neck squamous cell carcinoma and in phase I for lymphoid malignancy.

Ajulemic acid, designated as Resunab™, is being developed by Corbus Pharmaceuticals, for the treatment of cystic fibrosis, systemic sclerosis, systemic lupus erythematosus.Ajulemic acid (AJA) is a first-in-class, synthetic, orally active, cannabinoid-derived drug that preferentially binds to the CB2 receptor and is nonpsychoactive. In preclinical studies, and in Phase 1 and 2 clinical trials, AJA showed a favorable safety, tolerability, and pharmacokinetic profile. It also demonstrated significant efficacy in preclinical models of inflammation and fibrosis. It suppresses tissue scarring and stimulates endogenous eicosanoids that resolve chronic inflammation and fibrosis without causing immunosuppression. AJA is currently being developed for use in 4 separate but related indications including systemic sclerosis (SSc), cystic fibrosis, dermatomyositis (DM), and systemic lupus erythematosus. Phase 2 clinical trials in the first 3 targets demonstrated that it is safe, is a potential treatment for these orphan diseases and appears to be a potent inflammation-resolving drug with a unique mechanism of action, distinct from the nonsteroidal anti-inflammatory drug (NSAID), and will be useful for treating a wide range of chronic inflammatory diseases.

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.

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.