teprasiran (QPI-1002) / Novartis, SBI Biotech - LARVOL DELTA

Teprasiran, A Small Interfering RNA, for the Prevention of Acute Kidney Injury in High-Risk Patients Undergoing Cardiac Surgery: A Randomized Clinical Study. (PubMed, Circulation) - P2, P3 | "A Phase 3 study with a MAKE90 primary outcome which has recently completed enrollment was designed based on these findings (NCT03510897). Clinical Trial Registration: URL: https://clinicaltrials.gov/ Unique Identifier: NCT02610283."

Clinical • Journal • Acute Kidney Injury • Cardiovascular • Nephrology • Renal Disease • CST3

QPI-1002 Phase 3 for Prevention of Major Adverse Kidney Events (MAKE) in Subjects at High Risk for AKI Following Cardiac Surgery (clinicaltrials.gov) - P3; N=1043; Terminated; Sponsor: Quark Pharmaceuticals; Active, not recruiting ➔ Terminated; Trial primary completion date: Jul 2021 ➔ Oct 2020; Study completed day 90 for primary endpoint but was terminated early for one year follow up due to results not meeting efficacy outcome at Day 90.

Adverse events • Clinical • Trial primary completion date • Trial termination • Acute Kidney Injury • Cardiovascular • Nephrology • Renal Disease

The growth of siRNA-based therapeutics: updated clinical studies. (PubMed, Biochem Pharmacol) - "This review provides a brief overview of mechanisms of siRNA action, physiological barriers to its delivery and activity, and the most common chemical modifications and delivery platforms used to overcome these barriers. Furthermore, this review presents comprehensive profiles of the three approved siRNA drugs (patisiran, givosiran, and lumasiran) and the seven other siRNA candidates in Phase 3 clinical trials (vutrisiran, nedosiran, inclisiran, fitusiran, teprasiran, cosdosiran, and tivanisiran), summarizing their modifications and delivery strategies, disease-specific mechanisms of action, updated clinical trial status, and future outlooks."

Clinical • Journal • Review • Dyslipidemia • Metabolic Disorders

QPI-1002 Phase 3 for Prevention of Major Adverse Kidney Events (MAKE) in Subjects at High Risk for AKI Following Cardiac Surgery (clinicaltrials.gov) - P3; N=1043; Active, not recruiting; Sponsor: Quark Pharmaceuticals; Recruiting ➔ Active, not recruiting

Adverse events • Clinical • Enrollment closed • Acute Kidney Injury • Cardiovascular • Nephrology • Renal Disease

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

I5NP for prophylaxis of delayed graft function in kidney transplantation (clinicaltrials.gov) - P1/2, N=366; Sponsor: Quark Pharmaceuticals; Active, not recruiting; Primary completion date: Jun 2013 -> Nov 2013.

Trial primary completion date • Renal Disease

"AKI development and 1 year mortality as well as renal outcomes. Compared to PLACEBO. High risk population #Teprasiran #KidneyWk #AKIoutcomes" (@kidney_doctor)

Clinical • Renal Disease

ReGIFT: QPI-1002 for Prevention of Delayed Graft Function in Recipients of an Older Donor Kidney Transplant (clinicaltrials.gov) - P3; N=594; Completed; Sponsor: Quark Pharmaceuticals; Active, not recruiting ➔ Completed; Trial completion date: Jul 2019 ➔ Jan 2019

Clinical • Trial completion • Trial completion date

Acute kidney injury: emerging pharmacotherapies in current clinical trials. (PubMed, Pediatr Nephrol) - "Here we review emerging pharmacotherapies for AKI that are currently in clinical trials. Categorized by their general mechanism of action, the therapies discussed include anti-inflammatory agents (recAP, AB103, ABT-719), antioxidants (iron chelators, heme arginate), vasodilators (levosimendan), apoptosis inhibitors (QPI-1002), and repair agents (THR-184, BB-3, mesenchymal stem cells)."

Journal