Veledimex is an oral activator ligand for a proprietary gene therapy promoter system, and a moderate inhibitor of and substrate for CYP3A4/5. Veledimex controls the expression of the target gene. The amount of gene product produced by the system and the duration of the effect is dependent on veledimex dose level and duration of dosing. Nonclinical studies demonstrated that intratumoral administration of Ad-RTS-IL12 along with oral administration of veledimex elicited dose-dependent anti-tumor effects in murine melanoma, breast cancer and glioma models which correlated with increased plasma exposure of veledimex. The FDA granted Fast Track designation for Ad-RTS-hIL-12 plus veledimex for the treatment of recurrent or progressive glioblastoma multiforme in adults. Ad-RTS-hIL-12 is an inducible adenoviral vector encoding human pro-inflammatory cytokine interleukin-12 (IL-12), which is under the transcriptional control of the RheoSwitch Therapeutic System. Veledixmex is an oral activator ligand. Data previously presented suggest that Ad-RTS-hIL-12 with 20 mg veledimex improves the median overall survival from 6 to 9 months seen with available therapies to 12.7 months, with further improvement in median overall survival to 17.8 months in a subset of subjects with reduced cumulative steroid exposure during the active dosing period of veledimex. Veledimex has been used in trials studying the treatment of glioblastoma multiforme, metastatic breast cancer, and anaplastic oligoastrocytoma.

Evodenoson (ATL-313) is an agonist of adenosine A2A receptor. It exerts anti-inflammatory activity through activation of adenosine A2A receptors on CD4+ T cells and neutrophils. Evodenoson demonstrates efficacy in animal models of sepsis, myocardial infarction, allograft rejection and enteritis.

Esuberaprost (314d) is an active enantiomer of beraprost, a prostacyclin analog. The pharmacologic action of esuberaprost is mediated by specifically binding to PGI2 receptors in smooth muscle cells (the blood vessel’s endothelium) and platelets. Upon binding, the compound widened blood vessels, preventing the formation of blood clots, and lowering blood pressure in the lungs. As a result, it was expected to improve the symptoms of pulmonary arterial hypertension. United Therapeutics announced it has ended its Phase 3 BEAT trial testing esuberaprost as an add-on therapy to its product Tyvaso (inhaled treprostinil) for clinically symptomatic patients with pulmonary arterial hypertension (PAH). According to a company press release, the study failed to reach its main endpoint of delaying the time to first clinical worsening events.

Dioxaphetyl butyrate is a synthetic narcotic analgesic and spasmolytic agent that has no accepted medicinal value in the United States. This opioid drug under international control according to the UN Single Convention 1961 and its amendments, Schedule I.

Gadocoletic acid (also Gadoletic acid trisodium salt, or B22956/1) is a magnetic resonance contrast agent. Based on results from animal imaging experiments and pharmacokinetic data it was suggested that gadocoletic acid trisodium salt has strong potential for clinical use in Magnetic Resonance Coronary Angiography and Myocardial Perfusion Imaging. The small molecules of gadocoletic acid are bound after injection to large human serum albumin molecules in coronary vessels with the result of high vessel/muscle contrast. The ability of B229563− (anion) to bind to more than one site on the albumin molecule allows a positive correlation between dose and blood relaxation rate enhancement at doses higher than 0.05 mmol/kg, the dose that produces roughly a total plasma concentration equimolar to the albumin concentration at equilibrium distribution. Gadocoletic acid is thought to be highly efficacious in inversion recovery-prepared 3D gradient-recalled echo, navigator echo-gated coronary angiography in humans already at doses below 0.1 mmol/kg.

Sodium aceneuramate is a sodium salt of aceneuramic acid (sialic acid). It is an effective inhalant expectorant. Inhalation of sodium aceneuramate repaired inflammation in the airway, and caused bronchitic rabbits to produce sputa with a low viscosity, similar to normal air-way secretions. Sodium aceneuramate protected the mucociliary transport impaired bycigarette smoke in a dose-dependent manner. The results suggest that sodium aceneuramate may participate in the defense mechanism in the airway against irritant gases. Sodium aceneuramate inhibited bronchial anaphylaxis and the release of histamine into bronchoalveolar lavages. Sodium aceneuramate has an action which elevates the viscoelasticity of secretions in the respiratory tract.

METHYLENETETRAHYDROFOLIC ACID, L-(+), an endogenous biomodulator that was developed under the brand name modufolin or arfolitixorin by the Isofol Medical. Arfolitixorin is developed to increase the efficacy of standard of care chemotherapy for advanced colorectal cancer. This drug is currently being studied in a global phase 3 clinical trial. Besides, the drug successfully completed phase II as rescue therapy for osteosarcoma. 21 May 2019 Isofol Medical has received clinical patent protection for the arfolitixorinin USA. It relates to a method of increasing blood concentration of deoxyuridine, a blood biomarker for inhibition of tumor growth in human cancer treatment.

9cUAB30 is a synthetic analog of 9-cis-RA with little or no RAR-binding activity relative to 9-cis-RA and other RA. 9cUAB30, is a selective rexinoid for the retinoid X nuclear receptors (RXR). Retinoid 9cUAB30 binds to and activates retinoid X receptor (RXR) homodimers and/or and retinoic acid receptor (RAR)/RXR heterodimers, which may result in the dissociation of corepressor protein and the recruitment of coactivator protein, followed by transcription of downstream target genes into mRNAs and protein translation. Gene transcription regulated by these transcription factors may result in inhibition of cell proliferation, induction of cell differentiation, and apoptosis of both normal cells and tumor cells. 9cUAB30 displays substantial chemopreventive capacity with little toxicity and is being translated to the clinic as a novel cancer prevention agent. 9cUAB30 has been assessed in vitro with human cell cultures. Human hepatocytes demonstrated no signs of cytotoxicity with treatment of 9cUAB30 up to 50 umol/L, although when human breast cancer cells were treated with 9cUAB30, they showed a significant inhibition of cell proliferation and apoptotic levels 2.5to 3.5 times the levels of untreated cells. 9cUAB30 inhibits telomerase and induces apoptosis in HL60 cells.