- Approval Based on Phase 3 Trial Results Showing a Complete Response Rate of 58%
- Median Duration of Response Has Not Been Reached
- Therapy Provides an Effective, Kidney-Sparing Option for Patients With This Rare and Difficult-To-Treat Cancer
- First-in-Class Approval Validates UroGen’s Innovative Technology and Future Opportunity Across its Specialty Cancers and Urologic Diseases Portfolio
PRINCETON, NJ, USA I April 15, 2020 I UroGen Pharma Ltd. (Nasdaq: URGN) today announced the U.S. Food and Drug Administration (FDA) granted expedited approval for Jelmyto™(mitomycin) for pyelocalyceal solution, a first-in-class treatment indicated for adults with low-grade upper tract urothelial cancer (LG UTUC). This landmark approval is based on positive results from the Phase 3 OLYMPUS trial that showed Jelmyto provides an effective, kidney-sparing option for patients with this rare and difficult-to-treat cancer.
Jelmyto consists of mitomycin, an established chemotherapy, and sterile hydrogel, using UroGen’s proprietary sustained release RTGel™ technology. It has been designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means.
“UroGen was founded on the vision to improve lives by challenging the current standard of care. Jelmyto, which leverages our innovative technology and expertise in specialty cancers and urologic diseases, is just the beginning as we build a company focused on bringing novel solutions to patients,” said Liz Barrett, President and Chief Executive Officer, UroGen. “We thank the patients and researchers involved in our OLYMPUS trial for helping us advance a transformative treatment in a disease space that has been historically ignored. We are tremendously proud to have pioneered this first-in-class therapy that improves patient care in a difficult-to-treat cancer.”
LG UTUC is a rare cancer that develops in the lining of the upper urinary tract, ureters and kidneys. In the U.S., there are approximately 6,000 – 7,000 new or recurrent LG UTUC patients annually. It is a challenging condition to treat due to the complex anatomy of the urinary tract system. The current standard of care includes multiple surgeries and most patients require a radical nephroureterectomy, which includes the removal of the renal pelvis, kidney, ureter and bladder cuff.1 Treatment is further complicated by the fact that LG UTUC is most commonly diagnosed in patients over 70 years of age, who may already have compromised kidney functionality and may suffer further complications as a result of major surgery.
“Jelmyto offers a new, non-surgical treatment approach for patients who otherwise may require treatment by radical nephroureterectomy, which is associated with declining kidney function and other complications,” said Dr. Seth Lerner, M.D., FACS, Professor of Urology at Baylor College of Medicine in Houston, TX and Principal Investigator of the OLYMPUS trial. “This novel, minimally invasive, kidney-sparing treatment has the potential to transform the way low-grade upper tract urothelial cancer is treated and help patients avoid long-term complications associated with surgery and the loss of their kidney.”
The FDA approval is based on results from the Phase 3 OLYMPUS trial showing Jelmyto achieved clinically significant disease eradication in adults with LG UTUC.Findings include:
- Complete response (CR) (primary endpoint) of 58% in the intent-to-treat population and in the sub-population of patients who were deemed not capable of surgical removal at diagnosis.
- At the 12-month time point for assessment of durability, 19 patients remained in CR, seven had experienced recurrence of disease and nine patients continued to be followed for the 12-month duration of response.
- Kaplan-Meier analysis estimated 12-month durability at 84%2 (based on interim data).
- The most commonly reported adverse events (≥ 20%) were ureteric obstruction, flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria, and vomiting. Most adverse events were mild to moderate and manageable using well established treatments. No treatment-related deaths occurred.
“There has been little progress for decades in the treatment of low-grade upper tract urothelial cancer, and this new option is paradigm-shifting for patients who often face recurrence and major surgery to remove their kidney,” said Andrea Maddox-Smith, Chief Executive Officer, Bladder Cancer Advocacy Network. “We applaud the FDA approval and the impact it will have for this community of elderly patients, many of whom struggle with comorbidities and have been hoping for a surgery-free treatment option.”
UroGen is committed to helping patients access Jelmyto. UroGen Support may help identify appropriate financial assistance programs for patients with commercial, Medicare or Medicaid coverage, as well as those with no insurance coverage. These programs are for eligible patients who have been prescribed Jelmyto and who need help managing the cost of treatment. The appropriate program will depend on the patient’s coverage. Visit http://www.Jelmyto.com or contact UroGen Support at 855-JELMYTO for additional information.
The FDA evaluated Jelmyto under Priority Review, which is reserved for medicines that may represent significant improvements in safety or efficacy in treating serious conditions. Jelmyto was also granted Breakthrough Therapy designation by the FDA, which was created to expedite the development and review of drugs developed for serious or life-threatening conditions with high unmet need.
Conference Call & Webcast Information
Members of UroGen’s management team will host a live conference call and webcast on April 16 at 8:30 AM Eastern Time to review Jelmyto™ (mitomycin) for pyelocalyceal solution approval details and commercialization plans. The live webcast can be accessed by visiting the Investors section of the Company’s website at http://investors.urogen.com. Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (855) 765-5685 (U.S.) or (615) 247-5916 (International) to listen to the live conference call. The conference ID number for the live call is 7864846. An archive of the webcast will be available for two weeks on the Company’s website.
About the Phase 3 OLYMPUS Trial
OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is an open-label, single-arm Phase 3 clinical trial of Jelmyto™ (mitomycin) for pyelocalyceal solution to evaluate the safety, tolerability and tumor ablative effect of Jelmyto in patients with low-grade upper tract urothelial cancer (UTUC). The trial enrolled 74 patients at clinical sites across the U.S. and Israel. Study participants were treated with six weekly instillations of Jelmyto administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine complete response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer. Patients who achieved a CR at the PDE timepoint were then followed for up to 12 months to determine the durability of response with Jelmyto.
About Jelmyto™ (mitomycin) for pyelocalyceal solution
Jelmyto™ (mitomycin) for pyelocalyceal solution is a drug formulation of mitomycin for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel™ technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, Jelmyto is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is delivered to patients using standard ureteral catheters. The U.S. Food and Drug Administration granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG UTUC. Jelmyto is the first drug approved for the treatment of LG UTUC.
SOURCE: UroGen
Post Views: 101
- Approval Based on Phase 3 Trial Results Showing a Complete Response Rate of 58%
- Median Duration of Response Has Not Been Reached
- Therapy Provides an Effective, Kidney-Sparing Option for Patients With This Rare and Difficult-To-Treat Cancer
- First-in-Class Approval Validates UroGen’s Innovative Technology and Future Opportunity Across its Specialty Cancers and Urologic Diseases Portfolio
PRINCETON, NJ, USA I April 15, 2020 I UroGen Pharma Ltd. (Nasdaq: URGN) today announced the U.S. Food and Drug Administration (FDA) granted expedited approval for Jelmyto™(mitomycin) for pyelocalyceal solution, a first-in-class treatment indicated for adults with low-grade upper tract urothelial cancer (LG UTUC). This landmark approval is based on positive results from the Phase 3 OLYMPUS trial that showed Jelmyto provides an effective, kidney-sparing option for patients with this rare and difficult-to-treat cancer.
Jelmyto consists of mitomycin, an established chemotherapy, and sterile hydrogel, using UroGen’s proprietary sustained release RTGel™ technology. It has been designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means.
“UroGen was founded on the vision to improve lives by challenging the current standard of care. Jelmyto, which leverages our innovative technology and expertise in specialty cancers and urologic diseases, is just the beginning as we build a company focused on bringing novel solutions to patients,” said Liz Barrett, President and Chief Executive Officer, UroGen. “We thank the patients and researchers involved in our OLYMPUS trial for helping us advance a transformative treatment in a disease space that has been historically ignored. We are tremendously proud to have pioneered this first-in-class therapy that improves patient care in a difficult-to-treat cancer.”
LG UTUC is a rare cancer that develops in the lining of the upper urinary tract, ureters and kidneys. In the U.S., there are approximately 6,000 – 7,000 new or recurrent LG UTUC patients annually. It is a challenging condition to treat due to the complex anatomy of the urinary tract system. The current standard of care includes multiple surgeries and most patients require a radical nephroureterectomy, which includes the removal of the renal pelvis, kidney, ureter and bladder cuff.1 Treatment is further complicated by the fact that LG UTUC is most commonly diagnosed in patients over 70 years of age, who may already have compromised kidney functionality and may suffer further complications as a result of major surgery.
“Jelmyto offers a new, non-surgical treatment approach for patients who otherwise may require treatment by radical nephroureterectomy, which is associated with declining kidney function and other complications,” said Dr. Seth Lerner, M.D., FACS, Professor of Urology at Baylor College of Medicine in Houston, TX and Principal Investigator of the OLYMPUS trial. “This novel, minimally invasive, kidney-sparing treatment has the potential to transform the way low-grade upper tract urothelial cancer is treated and help patients avoid long-term complications associated with surgery and the loss of their kidney.”
The FDA approval is based on results from the Phase 3 OLYMPUS trial showing Jelmyto achieved clinically significant disease eradication in adults with LG UTUC.Findings include:
- Complete response (CR) (primary endpoint) of 58% in the intent-to-treat population and in the sub-population of patients who were deemed not capable of surgical removal at diagnosis.
- At the 12-month time point for assessment of durability, 19 patients remained in CR, seven had experienced recurrence of disease and nine patients continued to be followed for the 12-month duration of response.
- Kaplan-Meier analysis estimated 12-month durability at 84%2 (based on interim data).
- The most commonly reported adverse events (≥ 20%) were ureteric obstruction, flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria, and vomiting. Most adverse events were mild to moderate and manageable using well established treatments. No treatment-related deaths occurred.
“There has been little progress for decades in the treatment of low-grade upper tract urothelial cancer, and this new option is paradigm-shifting for patients who often face recurrence and major surgery to remove their kidney,” said Andrea Maddox-Smith, Chief Executive Officer, Bladder Cancer Advocacy Network. “We applaud the FDA approval and the impact it will have for this community of elderly patients, many of whom struggle with comorbidities and have been hoping for a surgery-free treatment option.”
UroGen is committed to helping patients access Jelmyto. UroGen Support may help identify appropriate financial assistance programs for patients with commercial, Medicare or Medicaid coverage, as well as those with no insurance coverage. These programs are for eligible patients who have been prescribed Jelmyto and who need help managing the cost of treatment. The appropriate program will depend on the patient’s coverage. Visit http://www.Jelmyto.com or contact UroGen Support at 855-JELMYTO for additional information.
The FDA evaluated Jelmyto under Priority Review, which is reserved for medicines that may represent significant improvements in safety or efficacy in treating serious conditions. Jelmyto was also granted Breakthrough Therapy designation by the FDA, which was created to expedite the development and review of drugs developed for serious or life-threatening conditions with high unmet need.
Conference Call & Webcast Information
Members of UroGen’s management team will host a live conference call and webcast on April 16 at 8:30 AM Eastern Time to review Jelmyto™ (mitomycin) for pyelocalyceal solution approval details and commercialization plans. The live webcast can be accessed by visiting the Investors section of the Company’s website at http://investors.urogen.com. Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (855) 765-5685 (U.S.) or (615) 247-5916 (International) to listen to the live conference call. The conference ID number for the live call is 7864846. An archive of the webcast will be available for two weeks on the Company’s website.
About the Phase 3 OLYMPUS Trial
OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is an open-label, single-arm Phase 3 clinical trial of Jelmyto™ (mitomycin) for pyelocalyceal solution to evaluate the safety, tolerability and tumor ablative effect of Jelmyto in patients with low-grade upper tract urothelial cancer (UTUC). The trial enrolled 74 patients at clinical sites across the U.S. and Israel. Study participants were treated with six weekly instillations of Jelmyto administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine complete response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer. Patients who achieved a CR at the PDE timepoint were then followed for up to 12 months to determine the durability of response with Jelmyto.
About Jelmyto™ (mitomycin) for pyelocalyceal solution
Jelmyto™ (mitomycin) for pyelocalyceal solution is a drug formulation of mitomycin for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel™ technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, Jelmyto is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is delivered to patients using standard ureteral catheters. The U.S. Food and Drug Administration granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG UTUC. Jelmyto is the first drug approved for the treatment of LG UTUC.
SOURCE: UroGen
Post Views: 101
