Benzinga | Nov 10, 2021 11:02AM EST

UroGen Pharma Unveils New Phase 3 Development Plan For UGN-102 At Spotlight Event

UroGen Pharma Ltd. (NASDAQ:URGN), a biopharmaceutical company dedicated to building and commercializing novel solutions that treat urothelial and specialty cancers, is announcing at its virtual Spotlight Event being held today that, following recent discussions with the U.S. Food and Drug Administration ("FDA"), it plans to conduct a new, single-arm Phase 3 pivotal study of UGN-102 for the treatment of low-grade, intermediate-risk, non-muscle invasive bladder cancer ("NMIBC"). This new study, which is expected to initiate in early 2022, is expected to enroll approximately 220 patients across 90 sites.

"We have worked diligently with the FDA over the past several years to define the unmet need in low-grade NMIBC, with a particular focus on the intermediate risk population that typically experiences multiple recurrences," said Liz Barrett, President and Chief Executive Officer of UroGen. "We are preparing to initiate a single-arm pivotal study of UGN-102 to form the basis for a New Drug Application for UGN-102 in the treatment of low-grade, intermediate-risk NMIBC. We believe this new study increases the probability of regulatory success for UGN-102 given its streamlined design in addition to the encouraging results observed from our Phase 2 OPTIMA II study."

UroGen is grateful to the investigators and patients who are participating in the ATLAS study and believes the data generated will represent an important component of the planned UGN-102 NDA submission, which remains on track for 2024. In light of the new planned Phase 3 trial, UroGen will stop enrollment in the ATLAS trial. Patients already enrolled in ATLAS will have the option to remain in the study until completion.

In addition to the updated clinical plan for UGN-102, the Spotlight Event features presentations on UroGen's earlier-stage, locally administered immunotherapy candidates, UGN-301 and UGN-201, as well as two expert panel discussions on future directions in treating NMIBC, other types of bladder cancer and the potential benefits of UroGen's immunotherapy pipeline products in non-urologic cancers.

Distinguished panelists discussing the current treatment landscape and potential future innovations in NMIBC include:

* Dr. Sandip Prasad, a urologist in community practice with Atlantic Medical Group and the Morristown Medical Center;

* Dr. Gary Steinberg, Professor of Urology and Director of the Bladder Cancer Program at NYU Langone Medical Center; and

* Dr. William Huang, Professor of Urology and Radiology at NYU and Chief of Urology at Tisch Hospital.

The experts reviewing the unmet needs of patients with high-grade NMIBC and the potential for locally applied combination therapy include:

* Dr. Karim Chamie, Associate Professor of Urology at UCLA Medical Center; and

* Dr. Joshua Meeks, Associate Professor of Urology, Biochemistry and Molecular Genetics at Northwestern University Feinberg School of Medicine.

"Immunotherapy for the treatment of high-grade bladder cancer, with primary attention toward our TLR-7 agonist, UGN-201 and our anti-CTLA4 antibody, UGN-301, is a key area of focus of our earlier-stage pipeline," said Dr. Mark Schoenberg, Chief Medical Officer of UroGen. "We have pursued a series of pre-clinical studies to determine whether our RTGel(tm) platform might provide a method for delivering highly potent immunomodulators directly to the bladder surface, avoiding the toxicity associated with systemic administration. Our studies conducted to-date suggest bladder cancer treated with a combination of a TLR-7 agonist and an anti-CTLA4 antibody using our RTGel technology, produces improved survival relative to treatment with other checkpoint inhibitors in RTGel, either alone or in combination with UGN-201."

UroGen is currently conducting non-human primate toxicity studies to facilitate the initiation of a multi-arm Phase 1 study of UGN-301 in early 2022 to be followed by UGN-301 in combination with other agents. This approach leverages UroGen's unique platform for drug delivery and provides an opportunity to evaluate intravesical delivery of its anti-CTLA4 monoclonal antibody in combination with other immuno-modulators, chemotherapies, gene therapy and innate immune stimulators, including UGN-201.