PL-3994 is a novel natriuretic cyclic peptide. It is a receptor-A (NPR-A) and receptor-C (NPR-C) agonist. PL-3994 has high affinity for recombinant human, dog, or rat NPR-As. PL-3994 produced concentration-dependent relaxation of pre-contracted guinea-pig trachea. PL-3994 elicited a potent, concentration-dependent but small relaxation of pre-contracted human precision-cut lung slices. Intratracheal PL-3994 produced a dose-dependent inhibition of the bronchoconstrictor response evoked by aerosolized methacholine, but was without significant effect on cardiovascular parameters. PL-3994 was resistant to degradation by human neutral endopeptidase. PL-3994 was well tolerated in subjects with controlled hypertension. A phase I and phase II trial have been successfully completed with PL-3994 with no safety concerns identified. In phase II study in subjects with hypertension who were receiving >1 antihypertensive medications, a significant reduction in blood pressure compared with placebo was observed. It appears that PL-3994 works synergistically with ACE inhibitors.

Othera Pharmaceuticals originally developed OT-730 (now known as QLT 091568) as an oculoselective beta-blocker for reducing the elevated intraocular pressure associated with glaucoma. OT-730 was involved in phase II clinical trial in patients with ocular hypertension or open-angle glaucoma. However, these studies were discontinued. At December 30, 2009, QLT Inc. acquired OT-730.

SOR-C13, is a novel, short, synthetic peptide developed from the C-terminal region of soricidin, a proprietary 54 amino acid peptide, discovered by Soricimed Biopharma found in the saliva of the Northern Short-tailed Shrew. SOR-C13 binds with high affinity and selectivity - and disrupts the function of - TRPV6, a calcium channel over-expressed in solid tumor cancers. TRPV6 plays a central role in a biochemical cascade that results in the upregulation of an array of pro-cancerous genes. TRPV6 is considered to be an important target for novel anticancer therapy. SOR-C13 is the first highly specific TRPV6 inhibitor to be identified and to be taken into clinical development. SOR-C13 was effective in inhibition of tumors in animal xenograft models of human ovarian and breast cancer. The ongoing phase I trial studies the side effects and best dose of SOR-C13 in treating patients with solid tumors. The FDA has awarded orphan drug status to SOR-C13 for the treatment of ovarian cancer and for the treatment of pancreatic cancer.

ACT-451840 is a new compound with potent activity against sensitive and resistant Plasmodium falciparum strains. ACT-451840 has been used in trials studying malaria. ACT-451840 appears to have a distinct mode of action that sets it apart from current antimalarial drugs, including artemisinins. It has a rapid onset of action as well as activity on all blood-borne (erythrocytic) stages, which is retained against multiple resistant parasites, including artemisinin-resistant strains. Unlike the majority of antimalarial agents, ACT-451840 has the potential to block disease transmission due to its activity on gametocytes. he antimalarial activity of ACT-451840 was studied in an experimental induced blood stage malaria clinical trial performed in collaboration with MMV Medicines for Malaria Ventures at the laboratories of Professor James McCarthy, MBBS, MD of the Royal Brisbane and Women's Hospital in Herston, Australia. In the trial, ACT-451840 was safe and well tolerated and showed clinical efficacy against the early stages of P. falciparum infections. The PK/PD model developed from this proof-of-concept study with eight healthy subjects enabled prediction of therapeutic effects, with cure rates following 1 week of therapy (single daily doses) predicted to be equivalent to artesunate monotherapy.

Mitoglitazone (previously known as MSDC-0160 or CAY-10415) is a mTOT (mitochondrial target of thiazolidinediones) modulator that targets the mitochondrial pyruvate carrier (MPC), which is a key controller of cellular metabolism. MSDC-0160 is modulated MPC and act as insulin sensitizers without activating PPAR gamma. (Mitoglitazone exhibits very low binding affinity and activity at PPARγ). Mitoglitazone has been used in trials phase II studying the treatment of Type 2 Diabetes and Alzheimer's disease; the treatment for diabetes was discontinued. In addition, MSDC-0160 has demonstrated significant neuroprotective effects in the En1+/- mouse model of Parkinson’s disease via modulation of the mTOR-autophagy signaling cascade.