Pd20-02 Efficacy and Safety of Padeliporfin Vascular Targeted Photodynamic Therapy (Vtp) for Treatment of Low-Grade Upper Tract Urothelial Cancer (Lg Utuc): Phase 3 Preliminary Results

You have accessJournal of UrologyBladder Cancer: Upper Tract Transitional Cell Carcinoma I (PD20)1 May 2024PD20-02 EFFICACY AND SAFETY OF PADELIPORFIN VASCULAR TARGETED PHOTODYNAMIC THERAPY (VTP) FOR TREATMENT OF LOW-GRADE UPPER TRACT UROTHELIAL CANCER (LG UTUC): PHASE 3 PRELIMINARY RESULTS Jonathan Coleman, Neal Shore, Gautier Marcq, Marc Colombel, Sarah Psutka, Jay Raman, Vitaly Margulis, Nirmish Singla, Hooman Djaladat, Reinhard Aigner, Carles Xavier Raventos Busquets, Gianni Amato, Yaniv Cohen, Axelle Mroz, Natalia Koudinova, Inna Krasnopolskaya, Genia Alpert, Avigdor Scherz, UCM 301 Investigators, and Ronald Kaufman Jonathan ColemanJonathan Coleman , Neal ShoreNeal Shore , Gautier MarcqGautier Marcq , Marc ColombelMarc Colombel , Sarah PsutkaSarah Psutka , Jay RamanJay Raman , Vitaly MargulisVitaly Margulis , Nirmish SinglaNirmish Singla , Hooman DjaladatHooman Djaladat , Reinhard AignerReinhard Aigner , Carles Xavier Raventos BusquetsCarles Xavier Raventos Busquets , Gianni AmatoGianni Amato , Yaniv CohenYaniv Cohen , Axelle MrozAxelle Mroz , Natalia KoudinovaNatalia Koudinova , Inna KrasnopolskayaInna Krasnopolskaya , Genia AlpertGenia Alpert , Avigdor ScherzAvigdor Scherz , UCM 301 Investigators , and Ronald KaufmanRonald Kaufman View All Author Informationhttps://doi.org/10.1097/01.JU.0001009556.54476.eb.02AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: Padeliporfin VTP has demonstrated safety and efficacy for UTUC treatment in a Phase 1 study (NCT03617003). We report the preliminary efficacy and safety outcomes of Padeliporfin VTP for Treatment of LG UTUC in ENLIGHTED, a Phase 3 trial (NCT04620239). METHODS: This is an open-label phase 3 study from USA, EU and Israel. Inclusion criteria are up to 2 biopsy-proven LG UTUC with index tumor≤15 mm in the kidney and≤20 mm in the ureter with an absence of high-grade cells on cytology. VTP is performed via retrograde upper tract endoscopy under anesthetic and low light conditions, Padeliporfin is injected IV and a laser light diffuser fiber 20-40 mm is positioned in proximity of the tumor through the scope. After Padeliporfin injection, the laser fiber is illuminated for 10 min. The fiber can be repositioned to provide up to 3 treatments within the upper tract. Pts are treated in two phases: Induction (ITP) and Maintenance Treatment Phases (MTP). ITP consists of 1-3 VTPs provided at 4-week intervals until achieving complete response (CR) or treatment failure Patients achieving CR will proceed to the MTP and be followed with endoscopic evaluation every 3 mos with VTP provided for recurrent tumors only up to 12 mos. Pts with high-grade recurrences will be deemed treatment failures and removed from the study to standard of care treatment. Pts completing the MTP, will be followed for an additional 48 mos for long-term outcomes. Primary outcome is CR on endoscopic evaluation and selective cytology at the time of primary response evaluation (28+/-3 days post last treatment) during padeliporfin VTP ITP. A total of 100 pts are to be enrolled. RESULTS: 12 pts have been treated as of 31 Oct 2023. 9 pts completed ITP and had CR 6/9 (67%) and PR 3/9 (33%). The most frequent treatment related adverse events were flank pain 36%, vomiting 15%, nausea 9%, hematuria 9%, all Grade 1-2, resolved within few days. Grade 3 serious adverse events 6% (flank pain, hypertension, renal colic) were resolved within 2 days. CONCLUSIONS: Padeliporfin VTP has shown evidence of safety and efficacy with preliminary data that is consistent with prior experience. Recruitment for the ENLIGHTED trial is ongoing with results expected to provide basis for approval of a new therapy that clinically benefits pts. Source of Funding: Steba/Impact biotech © 2024 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 211Issue 5SMay 2024Page: e446 Advertisement Copyright & Permissions© 2024 by American Urological Association Education and Research, Inc.Metrics Author Information Jonathan Coleman More articles by this author Neal Shore More articles by this author Gautier Marcq More articles by this author Marc Colombel More articles by this author Sarah Psutka More articles by this author Jay Raman More articles by this author Vitaly Margulis More articles by this author Nirmish Singla More articles by this author Hooman Djaladat More articles by this author Reinhard Aigner More articles by this author Carles Xavier Raventos Busquets More articles by this author Gianni Amato More articles by this author Yaniv Cohen More articles by this author Axelle Mroz More articles by this author Natalia Koudinova More articles by this author Inna Krasnopolskaya More articles by this author Genia Alpert More articles by this author Avigdor Scherz More articles by this author UCM 301 Investigators More articles by this author Ronald Kaufman More articles by this author Expand All Advertisement PDF downloadLoading ...