Zuretinol (QLT091001, 9-cis-retinol) is a retinoid. Retinoids (vitamin A and its analogs) are essential dietary substances that are needed by mammals for reproduction, normal embryogenesis, growth, vision, and maintaining normal cellular differentiation and the integrity of the immune system. Within cells, retinoids regulate gene transcription acting through ligand-dependent transcription factors, the retinoic acid receptors (RARs), and the retinoid X receptors (RXRs). All-trans-retinoic acid binds only to RARs with high affinity, whereas its 9-cis isomer binds with high affinity to both RARs and RXRs. The actions of all-trans- and 9-cis-retinoic acid in regulating cellular responses are distinct and not interchangeable. Zuretinol is a retinal derivative for treatment of visual disorders. It is a synthetic retinoid replacement for 11-cis-retinal. It is an investigational product under development for the treatment of retinal diseases caused by gene mutations that interfere with the availability of 11-cis-retinal. The therapeutic strategy with Zuretinol is to facilitate recovery or restoration of visual function by acting as a replacement for missing 11-cis-retinal and restoring a key biochemical component of the visual (retinoid) cycle. Novelion Therapeutics is currently developing QLT091001 for the treatment of Inherited Retinal Disease caused by retinal pigment epithelium protein 65 (“RPE65”) and lecithin:retinol acyltransferase (“LRAT”) gene mutations, which include Leber Congenital Amaurosis (“LCA”) and Retinitis Pigmentosa (“RP”). QLT091001 has received orphan drug designations for the treatment of LCA (due to inherited mutations in the LRAT and RPE65 genes) and RP (all mutations) by the U.S. Food and Drug Administration (the “FDA”), and for the treatment of LCA and RP (all mutations) by the European Medicines Agency (the “EMA”).

Morpholine salicylate is a derivative of salicylic acid. It is a non-steroidal anti-inflammatory drug and was marketed under a tradename Retarcyl, Dolical and Deposal.

Dichloroacetic acid, often abbreviated DCA (dichloroacetate), is an acid analog of acetic acid in which two of the three hydrogen atoms of the methyl group have been replaced by chlorine atoms. The salts and esters of dichloroacetic acid are called dichloroacetates. Salts of DCA are used as drugs since they inhibit the enzyme pyruvate dehydrogenase kinase. Early reports of its activity against brain cancer cells led patients to treat themselves with DCA, which is commercially available in non-pharmaceutical grade. A phase 1 study in 5 patients concluded that DCA was safe, but wasn't designed to establish effectiveness. DCA was approved for use in Canada in 1989 (as a topical formulation for the treatment of warts and for cauterization and removal of a wide variety of skin and tissue lesions), but was cancelled post market. DCA is a noncompetitive inhibitor of the endoplasmic reticulum enzyme HMG CoA reductase, which catalyzes the rate limiting step in cholesterol biosynthesis. DCA has been researched in adults, children, animals, and cells as a monotherapy as well as in combination with other therapies for the treatment of severe metabolic disorders including diabetes and hypercholesterolemia, lactic acidosis, certain heart conditions, and cancer. DCA has been prescribed to reduce tumour size and tumour markers, prevent angiogenesis, reduce cancer related symptoms, manage pain, and aid in palliation.

Ornithine is an amino acid produced in the urea cycle by the splitting off of urea from arginine. It is a central part of the urea cycle, which allows for the disposal of excess nitrogen. Ornithine is also a precursor of citrulline and arginine. Arginine stimulates the pituitary release of growth hormone. Burns or other injuries affect the state of arginine in tissues throughout the body. As de novo synthesis of arginine during these conditions is usually not sufficient for normal immune function, nor for normal protein synthesis, ornithine may have immunomodulatory and wound-healing activities under these conditions (by virtue of its metabolism to arginine).

2,5-Dimethyl-N-Phenyl-3H-diazaphophol-4-imine is a quinonoid tautomer of GABAA and GABAB agonist progabide. According to quantum mechanical calculations, a quinonoid form is predominant in polar solvents, while aromatic tautomer is prevalent in apolar solvents. Progabide is a prodrug of gamma-aminobutyric acid and was investigated for the treatment of epilepsy, Parkinson's disease, schizophrenia, clinical depression, anxiety disorder, and other diseases. Progabide was marketed in France by Sanofi Aventis under tradename Gabrene for use in monotherapy and also as adjunctive therapy for generalized tonic-clonic, myoclonic, partial seizures, and for Lennox‐Gastaut syndrome, in both children and adults.

Oleanolic acid or oleanic acid is a naturally occurring pentacyclic triterpenoid. It is widely distributed in food and plants where it exists as a free acid or as an aglycone of triterpenoid saponins. Oleanolic acid protects the liver from acute chemically induced liver injury, fibrosis and cirrhosis caused by chronic liver diseases. Its possess cytotoxic activity against tumor cell lines

Almasilate is an antacid compound, consisting of a crystalline aluminum/magnesium silicate polymer. It guffers gastric acid by binding hydrogen ions within the polymer. It has been used in peptic ulcers and dyspepsia and marketed under the tradename Malinal, however, its therapeutic efficacy was lower than that of other approved antacids.

Sodium dehydroacetate, a water-soluble antiseptic, is a food and feed additive with antimicrobial effects. Recently published studies have shown that sodium dehydroacetate in patients with leg ulcers could cause allergic contact dermatitis.

Alpha-linolenic acid (ALA), an 18-carbon omega-3 essential fatty acid, is the precursor of eicosapentaenoic acid (EPA) and docosahexaenoic acid (DHA). ALA cannot be synthesized by humans and therefore must be entirely acquired from exogenous sources. Evidence for the essentiality of ALA was first provided by a study showing that ALA supplementation reversed the abnormal neurologic signs observed in a 6-year-old girl who suffered from sensory loss and visual complications. Most of the ALA is catabolized via beta-oxidation for energy generation, and a small proportion of it undergoes conversion to produce another two potent members of omega-3 PUFA family: EPA and DHA. Delta 6 desaturase (D6D) enzyme is responsible the conversion of ALA to DHA. Although not conclusive, it was suggested, that the benefits associated with ALA seem to stem mainly from EPA and DHA, and as major consequence of ALA deficiency it appears that EPA and DHA are not adequately produced.