Novartis is paying Quark Pharmaceuticals $10 million for an option to obtain exclusive worldwide rights to develop and commercialize the latter’s Phase II-stage siRNA candidate QPI-1002. If Novartis exercises its option Quark could receive another $670 million in option exercise fees and milestone payments, plus additional royalties on product sales. QPI-1002 is an siRNA-based temporary inhibitor of p53, in development for use in the prevention of acute kidney injury in patients undergoing major cardiovascular surgery, and for the prophylaxis of delayed graft function in patients receiving deceased donor kidney transplants.
Quark points out the drug is the first synthetic siRNA to be administered systemically in humans. QPI-1002 has been granted orphan designation by FDA and the European Medicines Agency for the prophylaxis of delayed graft function in kidney transplant patients.
Quark is a clinical-stage pharmaceutical company focusing on the development of RNAi-based therapeutics.
The firm’s clinical pipeline includes the synthetic siRNA QPI-1007, which is designed to temporarily inhibit expression of the pro-apoptotic protein Caspase 2. The candidate is being developed as a neuroprotectant for the treatment of nonarteritic ischemic optic neuropathy (NAION) and other optic neuropathies such as glaucoma that result in the death of retinal ganglion cells. Quark claims QPI-1007 utilizes a novel siRNA structure that preserves activity while attenuating off-target and immunostimulatory effects. The drug is currently being tested in Phase I trials in patients with optic nerve atrophy and NAION.
PF-655 (formerly REDD14NP and RTP801i) is a synthetic siRNA designed to inhibit the expression of RTP801, a target identified by Quark using its BiFAR™ target discovery platform. The drug has been licensed to Pfizer on an exclusive worldwide basis, and is currently undergoing Phase II studies against diabetic macular edema and age-related macular degeneration.
Quark's preclinical pipeline includes potential siRNA candidates in fields as diverse as lung diseases and lung transplantation disorders, acute and chronic neurodegenerative disorders, and hearing loss due to chemotherapy.