Quercis Announces Special Protocol Assessment Agreement with U.S. Food and Drug Administration
ZUG, Switzerland, May 4, 2021 /PRNewswire/ -- Quercis Pharma AG, a private, clinical stage biopharmaceutical company leveraging its novel antithrombotic platform, today announces that it has entered into a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA) for the protocol titled, "A Randomized, Placebo-Controlled, Double-Blind Phase 3 Trial Comparing, Relative to Placebo, the Effect of Isoquercetin on Thromboembolic Events in Patients with Metastatic Pancreatic Cancer (CATIQ P3)."
Isoquercetin, the Company's lead product candidate, employs a novel pathway that is based on a new mechanism of action to prevent or reverse thrombus formation with a profile that potentially conveys substantially lowered bleeding risk compared to current standard of care. These mechanisms are related to key thrombotic pathways, including the inhibition of soluble P-selectin, which is known to help prevent or reduce thrombotic events.
"Venous thromboembolism (VTE) is an important cause of morbidity and mortality among patients with cancer. Besides an increased risk of VTE, advanced cancer patients have a significantly increased bleeding risk," said Jeffrey Zwicker, M.D., Associate Professor of Medicine at Harvard Medical School, Chief of Benign Hematology, Beth Israel Deaconess Medical Center. "In the Phase 2 multi-center study, we observed that isoquercetin reduced platelet-dependent thrombin generation in advanced cancer patients without an apparent increased risk of serious bleeding. We look forward to evaluating the efficacy and safety of isoquercetin in Phase 3 trials."
Under the agreed upon SPA, the Phase 3 study will be a randomized placebo-controlled trial in which patients will receive either 1000 mg or 2000 mg of oral isoquercetin (formulated with vitamin C and vitamin B3) or matching placebo capsules daily for 16 weeks. The agreed upon primary endpoints of the study are the comparison of plasma D-dimer at baseline and at day 56 and the development of any symptomatic proximal or distal deep vein thrombosis (DVT), symptomatic pulmonary embolism (PE) or fatal PE, asymptomatic proximal DVT at the end of treatment period. There is a planned interim analysis of the primary endpoint for futility, dose selection and sample size reassessment following the randomization of 180 patients and after 26 primary endpoint events have accrued. If futility is passed, the best performing isoquercetin dose will be selected and a sample size reassessment will be performed. After the interim, the trial will enroll an additional 160-300 patients who will be randomized on a 1:1 basis to the selected isoquercetin dose or placebo. The Company also plans to initiate a second randomized placebo-controlled Phase 3 trial with a similar design in glioblastoma patients, and on the basis of both trials it plans to pursue FDA approval for the prevention of VTE in all cancer types.
"This SPA marks a meaningful milestone, as it underscores our alignment with the FDA on the agreed upon protocol for this important registrational study and brings us one step closer to bringing this potentially life-saving therapeutic to cancer patients in danger of developing VTEs," stated Stefan Wohlfeil, M.D., Chief Medical Officer of Quercis Pharma. "With study start-up activities already under way, we look forward to initiating this Phase 3 study of isoquercetin in metastatic pancreatic patients and to having its outcome replicate the results from our Phase 2 clinical study."
Results from an earlier Phase 2 clinical trial validated protein disulfide isomerase (PDI) as a viable therapeutic target for anticoagulant therapy. The data showed that at 1000 mg daily, isoquercetin inhibited plasma PDI activity and significantly reduced plasma D-dimer levels along with soluble P-selectin and platelet-dependent thrombin generation. In a cancer population considered at high risk for thrombosis and hemorrhage, proximal DVT, PE, or major hemorrhage were not observedi.
About Special Protocol Assessments
An SPA agreement indicates concurrence by the FDA with the adequacy and acceptability of specific critical elements of overall protocol design (e.g. entry criteria, dose selection, endpoints, and planned analyses) for a study intended to support a future marketing application. These elements are critical to ensuring that the trial conducted under the protocol can be considered an adequate and well-controlled study that can support marketing approval.ii For more information on Special Protocol Assessments, please visit: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm080571.pdf.
About Quercis Pharma AG
Quercis Pharma AG is a private, biopharmaceutical company based in Zug, Switzerland. Quercis Pharma is advancing a development pipeline of late-stage clinical studies that focus on the prevention of VTE in cancer patients. In addition, Quercis targets other diseases associated with thrombotic events, such as sickle cell disease (SCD), Ebola and COVID-19. The Company's lead drug candidate acts as an antithrombotic with significantly lower risk of adverse events than existing treatments. Quercis plans to initiate two Phase 3 studies for the prevention of VTE in pancreatic cancer and glioblastoma patients and will pursue U.S. FDA approval for the prevention of VTE in all cancer types based on these studies. In addition, the Company is preparing to conduct a number of studies for the treatment of SCD (Phase 2) and COVID-19 (Phase 2/3).
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Anne Marie Fields
Rx Communications Group
i Zwicker JI, Schlechter BL, Stopa JD, et al. Targeting protein disulfide isomerase with the flavonoid isoquercetin to improve hypercoagulability in advanced cancer. JCI insight 2019;4.
SOURCE Quercis Pharma AG
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