QPI-1007 / SBI Biotech - LARVOL DELTA

Results From a Randomized Multicenter Double-Masked Sham-Controlled Phase 2/3 Trial of QPI-1007, a siRNA Against Caspase-2, for Acute NAION (AAO 2024) - "Results for the primary endpoint (at 6 months) and additional secondary endpoints will be presented at the meeting. Conclusion As NAION is the most common acute optic neuropathy in those over 50, and with no treatment and a poor prognosis, a treatment for NAION would be of great public health consequence."

Clinical • P2/3 data • Ocular Inflammation • Ophthalmology • Optic Neuritis

Caspase inhibition: From cellular biology and thanatology to potential clinical agents (PubMed, Med Sci (Paris)) - "The Pan-caspase inhibitor emricasan reached clinical phase III and was proven to be safe but failed to demonstrate efficacy against NASH. Contrary to initial assumptions, selective Caspase-3 inhibitors have not reached the clinical level, while QPI-1007, a siRNA directed against Caspase-2, is currently undergoing a multicentric phase III clinical study for the treatment of ischemic optic neuropathies."

Clinical • Journal • Hepatology • Immunology • Inflammation • Non-alcoholic Steatohepatitis • Ocular Inflammation • Ophthalmology • Optic Neuritis • CASP3

Controversies on neuroprotection therapy in non-arteritic anterior ischaemic optic neuropathy. (PubMed, Br J Ophthalmol) - "Unfortunately, in spite of all the experimental and clinical research on neuroprotection agents in NA-AION so far, we have no scientifically proven evidence of neuroprotection agents showing any benefit in the human clinical studies so far."

Journal • Review • Cardiovascular • Gene Therapies • Ocular Inflammation • Optic Neuritis • Pain

The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market. (PubMed) - Pharm Res - "...The siRNAs in focus are PF-04523655, TKM-080301, Atu027, SYL040012, SYL1001, siG12D-LODER (phase 2), QPI-1002, QPI-1007, and patisiran (phase 3)...Miravirsen is an AntimiR-122 for hepatitis C virus infection. The flexibility of RNAi technology is easily understood taking into account: (i) the different drug targets (i.e. p53, caspase 2, PKN3, β2-adrenergic receptor, mutated KRAS, microRNAs); (ii) therapeutic conditions, including ophthalmic diseases, kidney injury, amyloidosis, pancreatic cancer, viral hepatitis; and (iii) routes of administration (ocular, intravenous, subcutaneous, intratumoral). Although some issues are still matters of concern (delivery, toxicity, cost, and biological barriers), RNAi definitively opens a wide avenue for drug development."

Journal • Review • Biosimilar • Gastrointestinal Cancer • Hepatitis C Virus • Immunology • Oncology • Ophthalmology • Pancreatic Cancer • Renal Disease

Progress on Ocular siRNA Gene Silencing Therapy and Drug Delivery Systems. (PubMed, Fundam Clin Pharmacol) - "RNA interference (RNAi) is being increasingly used in the treatment of these disorders with siRNA drugs, bevasiranib, AGN211745 and PF-04523655 for AMD, and SYL040012 and QPI-1007 for glaucoma. Compared with the non-viral vectors, viral vectors have limited payload capacity and potential immunogenicity. This review summarizes the progress of the ocular siRNA gene silencing therapy by focusing on siRNA drugs for AMD and glaucoma already used in clinical research, the main routes of drug delivery, and the non-viral vectors for siRNA drugs."

Journal • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Gene Therapies • Genetic Disorders • Glaucoma • Macular Degeneration • Ophthalmology • Rare Diseases • Retinal Disorders

Phase 2/3, Randomized, Double-Masked, Sham-Controlled Trial of QPI-1007 in Subjects With Acute Nonarteritic Anterior Ischemic Optic Neuropathy (NAION) (clinicaltrials.gov) - P2/3; N=732; Terminated; Sponsor: Quark Pharmaceuticals; Trial completion date: Oct 2020 ➔ Jul 2019; Recruiting ➔ Terminated; Trial primary completion date: Oct 2020 ➔ Jul 2019; An interim analysis did not warrant to continue enrollment

Clinical • Trial completion date • Trial primary completion date • Trial termination