PF-03715455 is a potent p38 inhibitor, showing some selectivity for p38-alpha over p38-beta. PF-03715455 inhibited IL-6, MCP-1, MIP1β, CC16 and CRP levels in plasma of healthy subjects. PF-03715455 was optimized for delivery by inhalation. PF-03715455 substantially reduced tobacco-smoke--induced lung neutrophilia and inflammatory damage to the lungs. PF-03715455 had been in phase II clinical trial for the treatment of asthma and phase I clinical trial for the treatment of chronic obstructive pulmonary disease. However, these investigations were discontinued.

Lestaurtinib (CEP-701, KT-5555) is an orally bio-available polyaromatic indolocarbazole alkaloid derived from K-252a. Lestaurtinib is a multi-targeted tyrosine kinase inhibitor which has been shown to potently inhibit FLT3 at nanomolar concentrations in preclinical studies, leading to its rapid development as a potential targeted agent for treatment of AML. Phase I studies have shown lestaturtinib to be an active agent particularly when used in combination with cytotoxic drugs. Currently, Phase II and Phase III studies are underway aiming to establish the future of this agent as a treatment option for patients with FLT3-ITD AML.

Filgotinib (GLPG0634) is a highly selective JAK1 inhibitor. GLPG0634 is a promising drug candidate for the future treatment of autoimmune and inflammatory disorders. It is in phase III clinical trials (initiated mid-2016) for the treatment of rheumatoid arthritis, Crohn's disease and ulcerative colitis. Most common adverse events observed were infections, gastrointestinal disorders and nervous system disorders.

FIN-6 (PATIDEGIB, IPI-926), a semisynthetic derivative of alkaloid cyclopamine, is a G protein-coupled receptor Smoothened (Smo) inhibitor with antineoplastic activity. Smo is a key signaling transmembrane protein in the Hedgehog signaling pathway which plays an important role in the proliferation of neuronal precursor cells in the developing cerebellum and other tissues. FIN-6 (PATIDEGIB, IPI-926) is under development for Gorlin syndrome, basal cell carcinomas, and other potential indications.

Alkeus Pharma's lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to prevent the formation of these toxic vitamin A dimers in the eye. ALK-001 is a chemically-modified vitamin A, in which 3 hydrogen atoms have been replaced by 3 deuterium atoms at carbon number 20. Replacing the retina's vitamin A with ALK-001 slows the formation of toxic vitamin A dimers. ALK-001 is in phase II clinical trials for the treatment of Stargardt's disease. The compound was co-developed by Alkeus Pharmaceuticals and Columbia University.

DSM265 is an experimental antimalarial that selectively inhibits the parasite dihydroorotate dehydrogenase (DHODH). DSM265 is the first DHODH inhibitor to reach clinical development for treatment of malaria. DSM265 is highly selective toward DHODH of the malaria parasite Plasmodium, efficacious against both blood and liver stages of P. falciparum, and active against drug-resistant parasite isolates. The only possible DSM265-related adverse event was a moderate transient elevation in serum bilirubin.