Arimoclomol citrate is an experimental drug developed by a biopharmaceutical company CytRx Corporation. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS. The European Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) granted orphan drug designation to arimoclomol as a potential treatment for Niemann-Pick type C in 2014 and 2015 respectively. Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases.

Taurolidine [bis(1,1-dioxoperhydro-1,2,4-thiadiazinyl-4)-methane (TRD)], a product derived from the aminosulfoacid taurin, was first described as an anti-bacterial substance. Taurolidine is a small dimeric molecule with molecular weight 284. It comprises the semiconditional amino acid taurine. Taurolidine was originally designed as a broad-spectrum antibiotic. Taurolidine has a broad antimicrobial spectrum of activity that is effective against aerobes and anaerobes, Gram-negative and Gram-posi-tive bacteria as well as yeasts and moulds in vitro. Taurolidine is also effective against methicillin-resistant and vancomycin-resistant bacteria (MRSA, VISA and VRE). It was mainly used in the treatment of patients with peritonis as well as antiendoxic agent in patients with systematic inflammatory response syndrome. It has been shown to be an effective bactericidal agent against both aerobic and anaerobic bacteria. It is currently licensed for intraperitoneal use in several European countries for the treatment of peritonitis. The compound appears to be nontoxic and has an excellent safety record since its initial introduction over 30 years ago. Taurolidine also possesses antiadherence properties and has been shown in vivo to reduce the extent and severity of postoperative peritoneal adhesions. It also possesses a strong anti-inflammatory action. This action appears, at least in part, to arise through its ability to inactivate endotoxin. Inflammation-induced tumor development is well described in the literature. Taurolidine’s anti-inflammatory and antiadherence properties prompted an investigation to examine whether it has a role in antitumor therapy. Taurolidine induces cancer cell death through a variety of mechanisms. It appears to act through enhancing apoptosis, inhibiting angiogenesis and tumor adherence, downregulating proinflammatory cytokine and endotoxin levels, and stimulating the immune system in response to surgically induced trauma. Taurolidine is currently in preclinical development for neuroblastoma. In February 23, 2018 the U.S. Food and Drug Administration (FDA) granted orphan drug designation to taurolidine for the treatment of neuroblastoma. Taurolidine is a key component in the Neutrolin®, a novel anti-infective solution for the reduction and prevention of catheter-related infections and thrombosis in patients requiring central venous cathers in end stage renal disease. Neutrolin contains a mix of Taurolidine, Citrate and Heparin. Neutrolin is designed to: 1) Aid in the prevention of Catheter-Related Bloodstream Infections (CRBIs) and 2) Prevent catheter dysfunction (due to blood clotting).

Pacritinib (SB1518), discovered in Singapore at the labs of S*BIO Pte Ltd., is an oral tyrosine kinase inhibitor (TKI) with activity against two important activating mutations: Janus Associated Kinase 2 (JAK2) and FMS-like tyrosine kinase 3 (FLT3). The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development as well as inflammatory cytokine expression and immune responses. Activating mutations of JAK2 are implicated in certain blood-related cancers, including myeloproliferative neoplasms (MPNs), leukemia and certain solid tumors. FLT3 is a gene commonly found mutated in patients with acute myeloid leukemia (AML). Pacritinib has demonstrated encouraging results in Phase 1 and 2 studies for patients with myelofibrosis and may offer an advantage over other JAK inhibitors through effective treatment of symptoms while having less treatment-emergent thrombocytopenia and anemia than has been seen in currently approved and in-development JAK inhibitors. Pacritinib is acquired by Cell Therapeutics, Inc. (CTI) and Baxter international and could effectively address an unmet medical need for patients living with myelofibrosis who face treatment-emergent thrombocytopenia on marketed JAK inhibitors. Currently Pacritinib is undergoing preregistration for myelofibrosis.