AMPHOTERICIN B CHOLESTERYL SULFATE COMPLEX (AMPHOTEC) is an antifungal medicine. AMPHOTEC® is a sterile, pyrogen-free, lyophilized powder for reconstitution and intravenous (IV) administration. AMPHOTEC consists of a 1:1 (molar ratio) complex of amphotericin B and cholesteryl sulfate. AMPHOTEC is indicated for the treatment of invasive aspergillosis in patients where renal impairment or unacceptable toxicity precludes the use of amphotericin B deoxycholate in effective doses, and in patients with invasive aspergillosis where prior amphotericin B deoxycholate therapy has failed. The active ingredient of AMPHOTEC, amphotericin B, is a polyene antibiotic that acts by binding to sterols (primarily ergosterol) in cell membranes of sensitive fungi, with subsequent leakage of intracellular contents and cell death due to changes in membrane permeability. Amphotericin B also binds to the sterols (primarily cholesterol) in mammalian cell membranes, which is believed to account for its toxicity in animals and humans. AMPHOTEC is active against Aspergillus spp (A. fumigatus, A. flavus), Candida spp (C. albicans, C. krusei, C. parapsilosis, C. tropicalis), Cryptococcus neoformans, and Blastomyces dermatitidis. Active against most fungi with the notable exceptions of Candida lusitaniae, Trichosporon beigelii, Aspergillus terreus (some isolates), Pseudallescheria boydii, Malassezia furfur and Fusarium spp. The lipid formulations are designed to reduce binding of amphotericin to mammalian cell membranes, therefore reducing toxicities.

Linarotene is arotinoid derivative. It is an antikeratolytic agent. Linarotene had been in preclinical phase by Ortho Pharmaceutical (Johnson & Johnson) and BASF Pharma for the treatment of skin disorders. However, this research was discontinued.

Aftobetin, also known as ANCA11 and NCE-11, is a novel amyloid–binding compound applied topically in the form of an ophthalmic ointment. Aftobetin offers a promising way for a noninvasive eye-scan to diagnose Alzheimer’s disease early.

Malethamer is the generic name of a new synthetic hydrocarbon copolymer with extraordinary water-binding properties, being able to absorb 200 times its weight of water. It is physically and chemically binds poliovirus 1 and certain bacterial enterotoxins. Ethylene-maleic anhydride copolymers and their derivatives have come to be considered for a number of applications generally involving the formation of insoluble complexes with proteins and viruses. The copolymer is not absorbed from the gastrointestinal tract and is pharmacologically inert. Malethamer, because of its water absorbing quality, is an effective compound for the symptomatic control of diarrhea, both organic and functional.

Scyllo-inositol (ELND005) is an inositol isoform. Inositol is a derivative of cyclohexane with six hydroxyl groups, making it a polyol. It also is known as a sugar alcohol, having exactly the same molecular formula as glucose or other hexoses. Scyllo-inositol (ELND005) is a naturally occurring plant sugar alcohol found most abundantly in the coconut palm. It appears to accumulate in a number of human tissues and biofluids through dietary consumption. It has traditionally been considered to be a B vitamin although it has an uncertain status as a vitamin and a deficiency syndrome has not been identified in man. Although scyllo-inositol (ELND005) at pharmacologic doses may alter myo-inositol levels and indirectly affect phosphatidyl-inositol signaling, its main effects are thought to be binding and inhibition of beta-amyloid 42 peptide aggregation and formation of beta-amyloid fibrils. In transgenic animals, scyllo-inositol (ELND005) reduced brain beta-amyloid concentrations and plaque burden, preserved synaptic density, and improved learning deficits. Scyllo-inositol (ELND005) also appears to neutralize toxic effects of beta-amyloid oligomers, including amelioration of oligomer-induced synaptic loss, long-term potentiation inhibition, and memory deficits. Scyllo-inositol (ELND005) is an attractive candidate as a potential disease-modifying oral treatment for Alzheimer disease.

Dexmecamylamine (TC-5214, also known, as S isomer of mecamylamine) is a positive allosteric modulator of α4β2 neuronal nicotinic receptors, rather than an open-channel blocker. It was evaluated as a potential adjunct treatment for patients with major depressive disorder (MDD). TC-5214 was generally well tolerated, however, the studies were not supportive of an antidepressant effect for TC-5214 in patients with MDD and inadequate response to prior antidepressant therapy. The Phase 2b clinical trial of TC-5214 for the treatment for overactive bladder (OAB) revealed the high dose of TC-5214 demonstrated mixed results on the co-primary endpoints by providing a statistically significant reduction in micturition frequency and an improvement that did not reach statistical significance on episodes of urinary incontinence. As a consequence of these results, this drug was discontinuing further development of TC-5214 in OAB. The study for using TC-5214 in patients with refractory hypertension was also terminated.

GPX-150 is an anthracycline compound that is being tested for treatment in patients with soft-tissue sarcomas. This doxorubicin (DOX) analog does not show the cumulative dose-dependent cardiotoxicity of DOX. It works by reducing the formation of reactive oxygen species and the cardiotoxic metabolite, doxorubiciniol, the two pathways that are linked to the irreversible cardiotoxicity seen with DOX. Phase 1 clinical trials showed no irreversible, cumulative dose-dependent cardiotoxicity. A phase 2 study investigating the safety and efficacy of GPX-150 in patients with soft tissue sarcoma has been completed. No patients developed any evidence of irreversible, cumulative dose-dependent chronic cardiotoxicity. Toxicities reported include grade 3 anemia, neutropenia, and grade 4 leukopenia.

Vidofludimus (SC12267; 4SC-101) is a novel oral immunomodulator inhibiting dihydroorotate dehydrogenase (DHODH) and the expression of proinflammatory cytokines including interleukin-17 (IL17A and IL17F) and interferon-gamma. This drug is in the clinical trial for the treatment of inflammatory bowel diseases and Rheumatoid arthritis.