UroGen Pharma Ltd

Previous wording

We are a biotechnology company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers. We have developedRTGel reverse-thermal hydrogel, a proprietary sustained release, hydrogel-based technology that has the potential to improve therapeutic profiles of existing drugs. Our technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our approved productJelmyto (mitomycin) for pyelocalyceal solution, and our investigational candidate, UGN-102 (mitomycin) for intravesical solution, are designed to ablate tumors by non-surgical means and to treat several forms of non-muscle invasive urothelial cancer, including low-grade upper tract urothelial cancer (“low-grade UTUC”) and low-grade intermediate risk non-muscle invasive bladder cancer (“low-grade intermediate risk NMIBC”), respectively. In addition, our immuno-uro-oncology pipeline includes UGN-301 (zalifrelimab), an anti-CTLA-4 antibody, which we intend to study as both monotherapy and combination therapy.

Current wording

We are a biotechnology company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers. We have developedRTGel reverse-thermal hydrogel, a proprietary sustained release, hydrogel-based technology that has the potential to improve therapeutic profiles of existing drugs. Our technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our approved productJelmyto (mitomycin) for pyelocalyceal solution, and our investigational candidate, UGN-102 (mitomycin) for intravesical solution, are designed to ablate tumors by non-surgical means and to treat several forms of non-muscle invasive urothelial cancer, including low-grade upper tract urothelial cancer (“low-grade UTUC”) and low-grade intermediate risk non-muscle invasive bladder cancer (“low-grade intermediate risk NMIBC”), respectively. In addition, our immuno-uro-oncology pipeline includes UGN-301 (zalifrelimab), an anti-CTLA-4 antibody, which we intend to study as a combination therapy with multiple agents.

Previous wording

If approved, UGN-102 may become the first FDA-approved medicine for low-grade intermediate risk NMIBC.** **We estimate that the annual treatable patient population of low-grade UTUC in the United States is approximately 6,000 to 7,000 and the annual treatable population of low-grade intermediate risk NMIBC is approximately 82,000, of which approximately 23,000 are estimated to be newly diagnosed and 59,000 are estimated to be recurrent patients. We estimate that the total addressable market opportunity for UGN-102 in low-grade intermediate risk NMIBC is potentially over $5.0 billion, assuming an expected pricing range of $16,000 to $19,000 per dose.

Current wording

If approved, UGN-102 would become the first U.S. Food and Drug Administration ("FDA") approved medicine for low-grade intermediate risk NMIBC.** **We estimate that the annual treatable population of low-grade intermediate risk NMIBC is approximately 82,000, of which approximately 23,000 are estimated to be newly diagnosed and 59,000 are estimated to be recurrent patients. We estimate that the total addressable market opportunity for UGN-102 in low-grade intermediate risk NMIBC is potentially over $5.0 billion, assuming an expected pricing range of $16,000 to $19,000 per dose.

Previous wording

RTGel (or hydrogel) is a novel proprietary polymeric biocompatible, reverse thermal gelation hydrogel technology, which, unlike the general characteristics of most forms of matter, is liquid at lower temperatures and converts into gel form when warmed to body temperature. We believe that these characteristics promote ease of delivery into and retention of drugs in body cavities, including the bladder and the upper urinary tract, forming a transient reservoir of drug that dissolves over time while preventing rapid excretion, providing for increased dwell time.RTGel leverages the physiologic flow of urine to provide a natural exit from the body.

Current wording

RTGel is a novel proprietary polymeric biocompatible, reverse thermal gelation hydrogel technology, which, unlike the general characteristics of most forms of matter, is liquid at lower temperatures and converts into gel form when warmed to body temperature. We believe that these characteristics promote ease of delivery into and retention of drugs in body cavities, including the bladder and the upper urinary tract, forming a transient reservoir of drug that dissolves over time while preventing rapid excretion, providing for increased dwell time.RTGel leverages the physiologic flow of urine to provide a natural exit from the body.

Previous wording

On April 15, 2020, the U.S. Food and Drug Administration ("FDA") approved our NDA forJelmyto (mitomycin) for pyelocalyceal solution, formerly known as UGN-101, for the treatment of adult patients with low-grade UTUC.Jelmyto consists of mitomycin, an established chemotherapy, and sterile hydrogel, using our proprietary sustained releaseRTGel technology. It has been designed to prolong exposure of upper urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. New product exclusivity forJelmyto expired on April 15, 2023, however, Orphan Drug exclusivity extends until April 15, 2027. Additionally, the main patents that protectJelmyto in the United States are set to expire in January 2031. These patents were listed in the FDA's Orange Book (Approved Drug Products with Therapeutic Equivalence Evaluations).

Current wording

On April 15, 2020, the FDA approved our new drug application (“NDA") forJelmyto (mitomycin) for pyelocalyceal solution, formerly known as UGN-101, for the treatment of adult patients with low-grade UTUC.Jelmyto consists of mitomycin, an established chemotherapy, and sterile hydrogel, using our proprietary sustained releaseRTGel technology. It has been designed to prolong exposure of upper urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. New product exclusivity forJelmyto expired on April 15, 2023, however, Orphan Drug exclusivity extends until April 15, 2027. Additionally, the main patents that protectJelmyto in the United States are set to expire in January 2031. These patents are listed in the FDA's Orange Book (Approved Drug Products with Therapeutic Equivalence Evaluations).

Previous wording

In June 2020, we initiated our commercial launch ofJelmyto in the United States. We have staffed, trained and prepared a customer-facing team that includes territory business managers with deep experience in both urology and oncology. These territory business manager positions are led by seven regional business director positions, who are in turn supported by seven regional operations manager positions. Each region is additionally supported by one to two clinical nurse educators to provide education and training around instillation, as well as a field reimbursement manager to help ensure access and reimbursement for appropriate patients and key account directors who engage with C-suite individuals to introduce aJelmytoservice line. In addition, our organization currently includes several medical science liaisons who appropriately engage with physicians interested in learning more about UroGen,Jelmyto and our technology, both in person and virtually. In total, our customer-facing team comprises approximately 80 colleagues.

Current wording

In June 2020, we initiated our commercial launch ofJelmyto in the United States. We have staffed, trained and prepared a customer-facing team that includes territory business managers with deep experience in both urology and oncology. These territory business manager positions are led by seven regional business director positions, who are in turn supported by seven regional operations manager positions. Each region is additionally supported by one to two clinical nurse educators to provide education and training around instillation, as well as a field reimbursement manager to help ensure access and reimbursement for appropriate patients and key account directors who engage with C-suite individuals to introduce aJelmytoservice line. In addition, our organization currently includes several medical science liaisons who appropriately engage with physicians interested in learning more about UroGen,Jelmyto and our technology, both in person and virtually. In total, our customer-facing team comprises approximately 90 colleagues.

Previous wording

In each of the first three fiscal years beginning after the initiation of our commercial launch ofJelmytoin June 2020, we have experienced a moderate decline in revenue during the third quarter from the preceding quarter. We believe this result is primarily attributable to the nature of low-grade disease, which does not require immediate treatment and therefore we believe there is an impact in the summer months. However, it is too early to say with confidence whether this seasonality trend will continue in future periods. Moreover, our futureJelmytorevenue will be impacted by various factors and we expect ourJelmyto revenue to fluctuate quarter-to-quarter for the foreseeable future.

Current wording

In each of the first three fiscal years beginning after the initiation of our commercial launch ofJelmytoin June 2020, we experienced a moderate decline in revenue during the third quarter from the preceding quarter. We believe this result was primarily attributable to the nature of low-grade disease, which does not require immediate treatment and therefore we believe there could be an impact in the summer months. However, we did not observe this trend in 2024 and therefore cannot say with confidence whether this seasonality trend will continue in future periods. Moreover, our futureJelmytorevenue will be impacted by various factors and we expect ourJelmyto revenue to fluctuate quarter-to-quarter for the foreseeable future.

Previous wording

UGN-102 is administered locally using the standard practice of intravesical instillation directly into the bladder via a urethral catheter. The instillation into the bladder is expected to take place in a physician’s office as a non-operative outpatient treatment, in comparison with trans-urethral resection of bladder tumor ("TURBT") or similar surgical procedures, which are operations often conducted under general anesthesia in an operating room and may require an overnight stay. Complete surgical tumor removal often has limited success due to the inability to properly identify, reach and resect all tumors. We believe that an effective chemoablation agent can potentially provide better eradication of tumors irrespective of the detectability and location of the tumors. In addition, by reducing the need for surgery, patients may avoid potential complications associated with surgery and general anesthesia.

Current wording

UGN-102 is administered locally using the standard practice of intravesical instillation directly into the bladder via a urethral catheter. The instillation into the bladder is expected to take place in a physician’s office as a non-operative outpatient treatment, in comparison with TURBT or similar surgical procedures, which are operations often conducted under general anesthesia in an operating room and may require an overnight stay. Complete surgical tumor removal often has limited success due to the inability to properly identify, reach and resect all tumors. We believe that an effective chemoablation agent can potentially provide better eradication of tumors irrespective of the detectability and location of the tumors. In addition, by reducing the need for surgery, patients may avoid potential complications associated with surgery and general anesthesia.

Previous wording

On July 27, 2023, we announced topline data from our Phase 3 trials, ATLAS and ENVISION. In the ATLAS trial, UGN-102 with or without TURBT met its primary endpoint of disease-free survival, reducing risk of recurrence, progression, or death by 55% compared to TURBT alone. Results of the ATLAS trial also showed a 64.8% CR rate at three months for patients who only received UGN-102, compared to a 63.6% CR rate at three months for patients who only received a TURBT. The ENVISION trial met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% rate of CR at three-months following the initial instillation. In both trials, the safety profile of UGN-102 was acceptable, and comparable to that observed in previous clinical trials of UGN-102.

Current wording

In February 2022, we announced the initiation of the Phase 3 ENVISION trial, targeting enrollment of 220 patients across 90 sites. In December 2022, we completed our target enrollment of the Phase 3 ENVISION trial. As a result of the FDA's acceptance of a single arm approach, we stopped enrollment of the Phase 3 ATLAS trial without knowledge of the data. However, at the time enrollment was stopped, patients who had signed an informed consent were able to complete screening, and if eligible were randomized into the trial. ATLAS continued until the last ongoing patient completed the month 15 visit. On July 27, 2023, we announced topline data from our Phase 3 trials, ATLAS and ENVISION. In the ATLAS trial, UGN-102 with or without TURBT met its primary endpoint of disease-free survival, reducing risk of recurrence, progression, or death by 55% compared to TURBT alone. Results of the ATLAS trial also showed a 64.8% CR rate at three months for patients who only received UGN-102, compared to a 63.6% CR rate at three months for patients who only received a TURBT. The ENVISION trial met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% rate of CR at three-months following the initial instillation. In both trials, the safety profile of UGN-102 was acceptable, and comparable to that observed in previous clinical trials of UGN-102.

Previous wording

In October 2023 we announced our agreement with the FDA on plans for submission of an NDA for UGN-102 (mitomycin) for intravesical solution. The FDA indicated that the current clinical development plan for UGN-102, which includes evaluation of duration of response 12 months after 3-month CR from the pivotal ENVISION trial, will support submission of an NDA for the treatment of low-grade intermediate risk NMIBC. The FDA indicated that it may seek the advice of the Oncologic Drugs Advisory Committee as part of the NDA review process. The FDA also agreed that the UGN-102 NDA can utilize a rolling review, allowing for early submission of the Chemistry, Manufacturing and Controls ("CMC") sections of the NDA, which we submitted in January 2024. Based on our agreement with the FDA, we expect to complete the submission of the rolling NDA for UGN-102 in the third quarter of 2024, with a potential FDA acceptance in the fourth quarter of 2024 and potential FDA approval in the first quarter of 2025 (assuming priority review) or the second quarter of 2025 (assuming standard review).

Current wording

In October 2023 we announced our agreement with the FDA on plans for submission of an NDA for UGN-102 (mitomycin) for intravesical solution. The FDA indicated that the current clinical development plan for UGN-102, which includes evaluation of duration of response 12 months after 3-month CR from the pivotal ENVISION trial, will support submission of an NDA for the treatment of low-grade intermediate risk NMIBC. The FDA indicated that it may seek the advice of the Oncologic Drugs Advisory Committee as part of the NDA review process. The FDA also agreed that the UGN-102 NDA can utilize a rolling review, allowing for early submission of the Chemistry, Manufacturing and Controls ("CMC") sections of the NDA, which we submitted in January 2024.

Previous wording

In the ENVISION trial, the 12-month DOR data by Kaplan-Meier estimate for patients who achieved a CR at three months after the first instillation of UGN-102 was 82.3% (95% CI, 75.9%, 87.1%). The ENVISION trial met its primary endpoint with patients having a 79.6% (73.9%, 84.5%) CR rate at three months after the first instillation of UGN-102. Among the patients in the ENVISION trial who achieved a CR at three months, 76.4% (69.8%, 82.3%) maintained a CR at 12 months. Among all 240 patients enrolled in the ENVISION trial, 60.8% (54.3%, 67.0%) were in CR at 12 months. In the ENVISION trial, DOR Kaplan-Meier estimates at 15 (n=43) and 18 (n=9) months were both 80.9% (95% CI, 73.9%, 86.2%). The ENVISION trial demonstrated a similar safety profile to that observed in the OPTIMA II and ATLAS trials, with treatment-emergent adverse events typically mild-to-moderate in severity.

Current wording

On June 13, 2024, we announced positive secondary endpoint DOR data from the Phase 3 ENVISION trial investigating UGN-102 for intravesical solution in patients with low-grade intermediate risk NMIBC. In the ENVISION trial, the 12-month DOR data by Kaplan-Meier estimate for patients who achieved a CR at three months after the first instillation of UGN-102 was 82.3% (95% CI, 75.9%, 87.1%). The ENVISION trial met its primary endpoint with patients having a 79.6% (73.9%, 84.5%) CR rate at three months after the first instillation of UGN-102. Among the patients in the ENVISION trial who achieved a CR at three months, 76.4% (69.8%, 82.3%) maintained a CR at 12 months. Among all 240 patients enrolled in the ENVISION trial, 60.8% (54.3%, 67.0%) were in CR at 12 months. In the ENVISION trial, DOR Kaplan-Meier estimates at 15 (n=43) and 18 (n=9) months were both 80.9% (95% CI, 73.9%, 86.2%). The ENVISION trial demonstrated a similar safety profile to that observed in the OPTIMA II and ATLAS trials, with treatment-emergent adverse events typically mild-to-moderate in severity.

Previous wording

In January 2024 we entered into a licensing and supply agreement with medac Gesellschaft für klinische Spezialpräparate m.b.H. (“medac”) to develop UGN-103 and UGN-104 which are intended to be a next-generation formulation of UGN-102 andJelmyto, respectively, that combine medac’s proprietary 80 mg mitomycin formulation with ourRTGel technology, which we believe will provide advantages related to production, cost, supply and product convenience. In April, we announced that the FDA has accepted our Investigational New Drug Application ("IND") for UGN-103. We have initiated a Phase 3 clinical study to support the NDA for UGN-103 in low-grade intermediate risk NMIBC, and plan to enroll approximately 87 patients. The first patients are expected to be dosed in the third quarter of 2024, with full enrollment anticipated by the first half of 2025. An NDA filing is projected for the first half of 2026, followed by a standard review period and potential approval and, if approved, the commercial launch in the first half of 2027. We also plan to initiate a Phase 3 trial of UGN-104 in low-grade UTUC early next year.

Current wording

In January 2024 we entered into a licensing and supply agreement with medac Gesellschaft für klinische Spezialpräparate m.b.H. (“medac”) to develop UGN-103 and UGN-104 which are intended to be a next-generation formulation of UGN-102 andJelmyto, respectively, that combine medac’s proprietary 80 mg mitomycin formulation with ourRTGel technology, which we believe will provide advantages related to production, cost, supply and product convenience. In April 2024, we announced that the FDA accepted our Investigational New Drug Application ("IND") for UGN-103 and we initiated our Phase 3 UTOPIA trial, a single-arm, multicenter study that will evaluate the efficacy and safety of UGN-103 in low-grade intermediate risk NMIBC. We plan to enroll 87 patients in the UTOPIA trial, with patients receiving 75 mg of UGN-103 via intravesical instillation once a week for six weeks. Efficacy will be assessed by the complete response rate at the three-month visit. Patients who have a complete response at the three-month visit, defined as having no detectable disease in the bladder, will enter the follow-up period of the study. Patients will remain on study until disease recurrence, disease progression, death, or the last patient completes 12 months of follow-up (i.e., 15 months after the first instillation), whichever occurs first. In October 2024, we announced the first patient dosed in the UTOPIA trial. An NDA submission is projected for the first half of 2026, followed by a standard review period and potential approval and, if approved, the commercial launch in the first half of 2027. We also plan to initiate a Phase 3 trial of UGN-104 in low-grade UTUC in early 2025.

Previous wording

Our immuno-uro-oncology pipeline includes UGN-301, an anti-CTLA-4 monoclonal antibody, which we intend to study as a standalone agent and as a combination therapy. UGN-301 is delivered using our proprietaryRTGel technology, which has been designed to significantly improve the effectiveness of certain intravesical therapies.

Current wording

Our immuno-uro-oncology pipeline includes UGN-301, an anti-CTLA-4 monoclonal antibody, which we intend to study as a combination therapy with multiple agents. UGN-301 is delivered using our proprietaryRTGel technology, which has been designed to significantly improve the effectiveness of certain intravesical therapies.

Previous wording

The first combination we are investigating clinically involves the sequential use of UGN-201 (imiquimod), a toll like receptor 7 ("TLR 7") agonist, and UGN-301 in high-grade NMIBC. UGN-201 is a liquid formulation of imiquimod for intravesical administration that has been optimized for delivery in the urinary tract. The second combination we are investigating clinically involves the sequential administration of gemcitabine and UGN-301 to the bladder in high-grade NMIBC. Gemcitabine is a chemotherapy that is used intravesically to treat high grade NMIBC where it is administered as a liquid formulation. We believe these two combinations could elicit both an innate and adaptive immune response, which may translate into a long-lasting acquired immune response, and potentially represent a valid post-TURBT adjuvant treatment of high-grade NMIBC. We believe that these combinations make local therapy a potentially more effective treatment option while minimizing systemic exposure and potential side effects.

Current wording

The first combination we are investigating clinically involves the sequential use of UGN-201 (imiquimod), a toll like receptor 7 ("TLR 7") agonist, and UGN-301 in high-grade NMIBC. UGN-201 is a liquid formulation of imiquimod for intravesical administration that has been optimized for delivery in the urinary tract. The second combination we are investigating clinically involves the sequential administration of gemcitabine and UGN-301 to the bladder in high-grade NMIBC. Enrollment of patients in this arm investigating the combination of UGN-301 and gemcitabine is complete. Gemcitabine is a chemotherapy that is used intravesically to treat high grade NMIBC where it is administered as a liquid formulation. We believe these two combinations could elicit both an innate and adaptive immune response, which may translate into a long-lasting acquired immune response, and potentially represent a valid post-TURBT adjuvant treatment of high-grade NMIBC. We are investigating these combinations to determine if they may make local therapy a potentially more effective treatment option while minimizing systemic exposure and potential side effects.

Previous wording

In March 2022, we announced FDA clearance of our IND to begin a novel Phase 1 clinical study of UGN-301 in patients with recurrent NMIBC. The novel study design utilizes a Master Protocol that we believe is a more efficient and streamlined approach to development. It will provide more flexibility to add study arms as the trial progresses and is expected to increase efficiency and potentially reduce costs. We expect the Master Protocol will allow us to more quickly evaluate safety, tolerability and dosing of UGN-301 in combination with additional immunomodulators and chemotherapies, with the goal of developing optimized treatment regimens for patients. The multi-arm Phase 1 study, which is expected to support the development of UGN-301 in high-grade NMIBC, was initiated in April 2022 and is actively enrolling. Safety and dosing data from the first arm evaluating UGN-301 as monotherapy are expected in late 2024.

Current wording

In March 2022, we announced FDA clearance of our IND to begin a novel Phase 1 clinical study of UGN-301 in patients with recurrent NMIBC. The novel study design utilizes a Master Protocol that we believe is a more efficient and streamlined approach to development. It will provide more flexibility to add study arms as the trial progresses and is expected to increase efficiency and potentially reduce costs. We expect the Master Protocol will allow us to more quickly evaluate safety, tolerability and dosing of UGN-301 in combination with additional immunomodulators and chemotherapies, with the goal of developing optimized treatment regimens for patients. The multi-arm Phase 1 study, which is expected to support the development of UGN-301 in high-grade NMIBC, was initiated in April 2022 and enrollment is nearing completion. Safety and dosing data from the first arm evaluating UGN-301 as monotherapy will be presented in late 2024.

Previous wording

During the three and six months ended June 30, 2024, we recognized $21.8 million and $40.6 million of revenue, respectively from sales of our product,Jelmyto.

Current wording

During the three and nine months ended September 30, 2024, we recognized $25.2 million and $65.8 million of revenue, respectively, from sales of our product,Jelmyto.

Previous wording

Other thanJelmyto, which was approved by the FDA in April 2020, we have not received approval of any of our product candidates. UGN-102 and UGN-301 are still in clinical development. As such, we cannot estimate the actual amounts necessary to successfully complete the development and commercialization of our product candidates or whether, or when, we may achieve profitability. Until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needs through a combination of equity or debt financings and collaboration arrangements.

Current wording

Other thanJelmyto, which was approved by the FDA in April 2020, we have not received approval of any of our product candidates. UGN-102, UGN-103, UGN-104 and UGN-301 are still in clinical development. As such, we cannot estimate the actual amounts necessary to successfully complete the development and commercialization of our product candidates or whether, or when, we may achieve profitability. Until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needs through a combination of equity or debt financings and collaboration arrangements.

Previous wording

Revenue was $21.8 million and $21.1 million for the three months ended June 30, 2024 and 2023, respectively. The increase in revenue of $0.7 million primarily reflects the increased volume of sales ofJelmyto.

Current wording

Revenue was $25.2 million and $20.9 million for the three months ended September 30, 2024 and 2023, respectively. The increase in revenue of $4.3 million primarily reflects the increased volume of sales ofJelmyto,including CREATES Act sales of $2.6 million in the third quarter of 2024, compared to $1.1 million of CREATES Act sales in the third quarter of 2023. The increase in volume of sales ofJelmyto was partially offset by higher revenue reserves primarily driven by estimated Medicare refunds for discarded drugs.

Previous wording

Cost of revenue was $2.2 million and $2.4 million for the three months ended June 30, 2024 and 2023, respectively. The decrease of $0.2 million is primarily attributable to certain nonrecurring payments made in connection with our supply arrangement in the prior year.

Current wording

Cost of revenue was $2.5 million and $2.4 million for the three months ended September 30, 2024 and 2023, respectively. The increase of $0.1 million is primarily attributable to the increased volume of sales of*Jelmyto,*partially offset by certain nonrecurring payments made in connection with our supply arrangement in the prior year.

Previous wording

Research and development expenses were $15.4 million and $11.6 million for the three months ended June 30, 2024 and 2023, respectively. The increase of $3.8 million is primarily attributable to higher costs related to manufacturing of UGN-102 which are recognized within research and development expenses prior to our product candidates receiving FDA approval as well as research and development expenses in connection with our initiation of a Phase 3 study for UGN-103, partially offset by lower UGN-102 clinical trial costs and costs related to the research into ingredient scale-up and production efficiency forJelmyto.

Current wording

Research and development expenses were $11.4 million and $10.2 million for the three months ended September 30, 2024 and 2023, respectively. The increase of $1.2 million is primarily attributable to higher costs related to the initiation of the Phase 3 UTOPIA trial for UGN-103, partially offset by lower UGN-102 clinical trial costs and costs related to the research into ingredient scale-up and production efficiency forJelmyto.

Previous wording

Selling and marketing expenses were $18.9 million and $13.9 million for the three months ended June 30, 2024 and 2023, respectively. The increase in selling and marketing expenses of $5.0 million is primarily attributable to UGN-102 brand marketing costs as well as an increase in overall commercial operation costs including meetings, conferences, and trainings.

Current wording

Selling and marketing expenses were $17.8 million and $12.6 million for the three months ended September 30, 2024 and 2023, respectively. The increase in selling and marketing expenses of $5.2 million is primarily attributable to UGN-102 brand marketing costs as well as an increase in overall commercial operation costs related to the planned expansion of the sales force and incentive compensation, meetings, conferences, and trainings.

Previous wording

General andadministrative expenses were $11.2 million and $8.6 million for the three months ended June 30, 2024 and 2023, respectively. The increase in general and administrative expenses of $2.6 million is primarily attributable tohigher compensation expenses, legal and compliance activities, pre-commercialization marketing communication expenses related to UGN-102, third-party advisory services, and ongoing managed services.

Current wording

General andadministrative expenses were $11.2 million and $9.2 million for the three months ended September 30, 2024 and 2023, respectively. The increase in general and administrative expenses of $2.0 million is primarily attributable tohigher compensation expenses, expenses related pre-commercialization readiness support related to UGN-102, general third-party advisory services, and ongoing managed services.

Previous wording

Financing on prepaid forward obligation was $5.8 million and $5.3 million for the three months ended June 30, 2024 and 2023, respectively. The measurement of financing on prepaid forward obligation is an accounting estimate under the "imputed interest method" of accounting (see Note 3 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report) which is affected by estimated future payments to RTW, which are based on a percentage of revenues. The increase in financing on prepaid forward obligation of $0.5 million was driven primarily by changes in underlying assumptions for remeasuring the effective rate.

Current wording

Financing on prepaid forward obligation was $5.9 million and $5.5 million for the three months ended September 30, 2024 and 2023, respectively. The measurement of financing on prepaid forward obligation is an accounting estimate under the "imputed interest method" of accounting (see Note 3 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report) which is affected by estimated future payments to RTW Investments (“RTW"), which are based on a percentage of revenues. The increase in financing on prepaid forward obligation of $0.4 million was driven primarily by changes in underlying assumptions for remeasuring the effective rate.

Previous wording

Interest expense was $3.5 million and $3.8 million for the three months ended June 30, 2024 and 2023, respectively. The decrease of $0.3 million was primarily attributable to the decrease in the margin interest rate and the related impact to amortization of the discount on the Pharmakon loan as a result of the amended and restated loan agreement in March 2024.

Current wording

Interest expense was $2.7 million and $3.8 million for the three months ended September 30, 2024 and 2023, respectively. The decrease of $1.1 million was primarily attributable to the decrease in the margin interest rate and the related impact to amortization of the discount on the Pharmakon loan as a result of the amended and restated loan agreement in March 2024.

Previous wording

Interest and other income, net was $1.7 million and $0.4 million for the three months ended June 30, 2024 and 2023, respectively. The increase of $1.3 million in interest and other income, net was primarily due to higher cash and investment balances.

Current wording

Interest and other income, net was $2.6 million and $0.9 million for the three months ended September 30, 2024 and 2023, respectively. The increase of $1.7 million in interest and other income, net was primarily due to higher cash and investment balances.

Previous wording

The following table sets forth our results of operations for the six months ended June 30, 2024 and 2023.

Current wording

The following table sets forth our results of operations for the nine months ended September 30, 2024 and 2023.

Previous wording

Cost of revenue was $4.0 million and $4.7 million for the six months ended June 30, 2024 and 2023, respectively. The decrease of $0.7 million is primarily attributable to certain nonrecurring payments made in connection with our supply arrangement in the prior year, lower shipping and warehousing costs, and decrease in theJelmytounit cost.

Current wording

Cost of revenue was $6.4 million and $7.1 million for the nine months ended September 30, 2024 and 2023, respectively. The decrease of $0.7 million is primarily attributable to certain nonrecurring payments made in connection with our supply arrangement in the prior year, lower shipping and warehousing costs, and a decrease in theJelmyto unit cost.

Previous wording

Research and development expenses were $30.9 million and $24.1 million for the six months ended June 30, 2024 and 2023, respectively. The increase of $6.8 million is primarily attributable to higher manufacturing costs, which are recognized as research and development expense prior to our product candidates receiving FDA approval, and regulatory related expense in connection with UGN-102 as well as research and development expenses, in connection with our initiation of a Phase 3 study for UGN-103, partially offset by lower UGN-102 clinical trial costs and costs related to the research into ingredient scale-up and production efficiency forJelmyto.

Current wording

Research and development expenses were $42.3 million and $34.3 million for the nine months ended September 30, 2024 and 2023, respectively. The increase of $8.0 million is primarily attributable to higher manufacturing costs, which are recognized as research and development expense prior to our product candidates receiving FDA approval, and regulatory related expense in connection with UGN-102 as well as research and development expenses, in connection with the Phase 3 UTOPIA trial for UGN-103, partially offset by lower UGN-102 clinical trial costs and costs related to the research into ingredient scale-up and production efficiency forJelmyto.

Previous wording

Selling and marketing expenses were $36.0 million and $28.1 million for thesix months ended June 30, 2024 and 2023, respectively. The increase in selling and marketing expenses of $7.9 million is primarily attributable to UGN-102 brand marketing costs as well as an increase in overall commercial operation costs including meetings, conferences, trainings and software costs.

Current wording

Selling and marketing expenses were $53.8 million and $40.7 million for thenine months ended September 30, 2024 and 2023, respectively. The increase in selling and marketing expenses of $13.1 million is primarily attributable to UGN-102 brand marketing costs as well as an increase in overall commercial operation costs including compensation, advisory, meetings, conferences, trainings and software costs.

Previous wording

General andadministrative expenses were $21.4 million and $18.9 million for thesix months ended June 30, 2024 and 2023, respectively. The increase in general and administrative expenses of $2.5 million is primarily attributable to higher compensation expenses, legal and compliance activities, marketing communication expenses related to UGN-102, third-party advisory services, and ongoing managed services, partially offset by lower professional services.

Current wording

General andadministrative expenses were $32.5 million and $28.1 million for thenine months ended September 30, 2024 and 2023, respectively. The increase in general and administrative expenses of $4.4 million is primarily attributable to higher compensation expenses, communication expenses related to UGN-102, third-party advisory services, and ongoing managed services.

Previous wording

Financing on prepaid forward obligation was $11.4 million and $10.6 million for the six months ended June 30, 2024 and 2023, respectively. The measurement of financing on prepaid forward obligation is an accounting estimate under the "imputed interest method" of accounting (see Note 3 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report) which is affected by estimated future payments to RTW, which are based on a percentage of revenues. The increase in financing on prepaid forward obligation of $0.8 million was driven primarily by changes in underlying assumptions for remeasuring the effective rate.

Current wording

Financing on prepaid forward obligation was $17.3 million and $16.0 million for the nine months ended September 30, 2024 and 2023, respectively. The measurement of financing on prepaid forward obligation is an accounting estimate under the "imputed interest method" of accounting (see Note 3 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report) which is affected by estimated future payments to RTW, which are based on a percentage of revenues. The increase in financing on prepaid forward obligation of $1.3 million was driven primarily by changes in underlying assumptions for remeasuring the effective rate.

Previous wording

Interest expense was $5.9 million and $7.3 million for the six months ended June 30, 2024 and 2023, respectively. The decrease of $1.4 million was primarily attributed to the decrease in the margin interest rate and the related impact to amortization of the discount on the Pharmakon loan as a result of the amended and restated loan agreement in March 2024.

Current wording

Interest expense was $8.6 million and $11.1 million for the nine months ended September 30, 2024 and 2023, respectively. The decrease of $2.5 million was primarily attributed to the decrease in the margin interest rate and the related impact to amortization of the discount on the Pharmakon loan as a result of the amended and restated loan agreement in March 2024.

Previous wording

Interest and other income, net was $3.3 million and $1.0 million for the six months ended June 30, 2024 and 2023, respectively. The increase of $2.3 million in interest and other income, net was primarily due to higher cash and investment balances.

Current wording

Interest and other income, net was $5.9 million and $1.9 million for the nine months ended September 30, 2024 and 2023, respectively. The increase of $4.0 million in interest and other income, net was primarily due to higher cash and investment balances.

Previous wording

As of June 30, 2024, we had $241.3 million in cash and cash equivalents and marketable securities. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation, and is held primarily in U.S. dollars.

Current wording

As of September 30, 2024, we had $254.2 million in cash and cash equivalents and marketable securities. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation, and is held primarily in U.S. dollars.

Previous wording

In December 2019, we entered into the ATM Sales Agreement with TD Cowen pursuant to which we may from time to time offer and sell our ordinary shares having an aggregate offering price of up to $100.0 million.

Current wording

In December 2019, we entered into the ATM Sales Agreement with TD Securities (USA) LLC (f/k/a Cowen and Company, LLC) (“TD Cowen”) pursuant to which we may from time to time offer and sell our ordinary shares having an aggregate offering price of up to $100.0 million.

Previous wording

In the first quarter of 2024, we sold 3,400,468 ordinary shares under the ATM Sales Agreement, for gross proceeds of approximately $56.1 million. The net proceeds to us after deducting sales commissions to TD Cowen were approximately $54.7 million. The remaining capacity under the ATM Sales Agreement was approximately $27.3 million as of June 30, 2024. The shares will be offered and sold pursuant to our shelf registration statement on Form S-3 filed with the SEC on November 15, 2022, which was declared effective on November 29, 2022.

Current wording

In the first quarter of 2024, we sold 3,400,468 ordinary shares under the ATM Sales Agreement, for gross proceeds of approximately $56.1 million. The net proceeds to us after deducting sales commissions to TD Cowen were approximately $54.7 million. We did not sell any ordinary shares under the ATM Sales Agreement in the three months ended September 30, 2024. The remaining capacity under the ATM Sales Agreement was approximately $27.3 million as of September 30, 2024. The shares will be offered and sold pursuant to our shelf registration statement on Form S-3 filed with the SEC on November 15, 2022, which was declared effective on November 29, 2022.

Previous wording

In March 2021, we entered into a prepaid forward agreement with RTW, pursuant to which RTW agreed to provide us with an upfront cash payment of $75.0 million to support the launch ofJelmyto and the development of UGN-102, and we agreed to provide RTW with tiered future payments based on global annual net product sales ofJelmyto and UGN-102, if approved. In May 2021, following the receipt of necessary regulatory approvals, we received the $75.0 million prepaid forward payment ($72.4 million net of transaction costs) from RTW.

Current wording

In March 2021, we entered into a prepaid forward agreement with RTW, pursuant to which RTW agreed to provide us with an upfront cash payment of $75.0 million to support the launch ofJelmyto and the development of UGN-102, and we agreed to provide RTW with tiered future payments based on global annual net product sales ofJelmyto and, subject to FDA approval, UGN-102, UGN-103 and UGN-104. In May 2021, following the receipt of necessary regulatory approvals, we received the $75.0 million prepaid forward payment ($72.4 million net of transaction costs) from RTW.

Previous wording

On March 13, 2024, we entered into an amended and restated loan agreement with Pharmakon for an additional third and fourth tranche of senior secured loan. The third tranche of $25.0 million is required to be drawn by September 30, 2024, subject to customary bring down conditions and deliverables. The fourth tranche of $75.0 million will become available at our option no later than August 29, 2025, subject to (i) having successfully drawn the immediately preceding $25.0 million tranche, (ii) receiving FDA approval of an NDA for UGN-102 no later than June 30, 2025 and (iii) the satisfaction of customary bring down conditions and deliverables.

Current wording

On March 13, 2024, we entered into an amended and restated loan agreement with Pharmakon for an additional third and fourth tranche of senior secured loan. The third tranche of $25.0 million was funded in September 2024. The fourth tranche of $75.0 million will become available at our option no later than August 29, 2025, subject to (i) receiving FDA approval of an NDA for UGN-102 no later than June 30, 2025 and (ii) the satisfaction of customary bring down conditions and deliverables.

Previous wording

On June 17, 2024, we entered into the Underwriting Agreement with the Underwriters, relating to the issuance and sale in a public offering of 5,000,000 ordinary shares of the Company for $17.50 per share and pre-funded warrants to purchase 1,142,857 ordinary shares of the Company for $17.499 per pre-funded warrant. The offering closed on June 20, 2024. The gross proceeds from this closing of the offering were $107.5 million, before deducting underwriting discounts and commissions and estimated offering expenses of $7.3 million. Each pre-funded warrant has an exercise price of $0.001 per ordinary share, subject to customary adjustments, is exercisable at any time and will not expire until exercised in full. The pre-funded warrants may not be exercised if the aggregate number of ordinary shares beneficially owned by the holder thereof immediately following such exercise would exceed a specified beneficial ownership limitation. In addition, the Underwriters were granted an option exercisable for 30 days, to purchase up to 921,428 additional shares at the public offering price, less the underwriting discounts and commissions. On July 18, 2024, we completed the closing of the sale of 921,428 additional shares in the offering following the exercise in full of the Underwriters’ option to purchase additional shares, which resulted in additional gross proceeds of $16.1 million before deducting underwriting discounts and commissions and estimated offering expenses of $1.0 million.

Current wording

On June 17, 2024, we entered into the Underwriting Agreement with the Underwriters, relating to the issuance and sale in a public offering of 5,000,000 ordinary shares of the Company for $17.50 per share and pre-funded warrants to purchase 1,142,857 ordinary shares of the Company for $17.499 per pre-funded warrant. The offering closed on June 20, 2024. The gross proceeds from this closing of the offering were $107.5 million, before deducting underwriting discounts and commissions and estimated offering expenses of $7.3 million. Each pre-funded warrant has an exercise price of $0.001 per ordinary share, subject to customary adjustments, is exercisable at any time and will not expire until exercised in full. The pre-funded warrants may not be exercised if the aggregate number of ordinary shares beneficially owned by the holder thereof immediately following such exercise would exceed a specified beneficial ownership limitation. In addition, the Underwriters were granted an option exercisable for 30 days, to purchase up to 921,428 additional shares at the public offering price, less the underwriting discounts and commissions. On July 18, 2024, we completed the closing of the sale of 921,428 additional shares in the offering following the exercise in full of the Underwriters’ option to purchase additional shares, which resulted in additional gross proceeds of $16.1 million before deducting underwriting discounts and commissions and offering expenses of $1.0 million.

Previous wording

We have incurred losses since our inception and negative cash flows from our operations, and as of June 30, 2024 we had an accumulated deficit of $744.4 million. We anticipate that we will continue to incur losses for the reasonably foreseeable future. Our primary uses of capital are, and we expect will continue to be, commercialization activities, research and development expense, including third-party clinical research and development services, laboratory and related supplies, clinical costs, including manufacturing costs, legal and other regulatory expense and general and administrative costs, partially offset by proceeds from sales ofJelmyto.

Current wording

We have incurred losses since our inception and negative cash flows from our operations, and as of September 30, 2024 we had an accumulated deficit of $768.7 million. We anticipate that we will continue to incur losses for the reasonably foreseeable future. Our primary uses of capital are, and we expect will continue to be, commercialization activities, research and development expense, including third-party clinical research and development services, laboratory and related supplies, clinical costs, including manufacturing costs, legal and other regulatory expense and general and administrative costs, partially offset by proceeds from sales ofJelmyto.

Previous wording

We routinely evaluate our liquidity needs, including assessment of our current financial condition, sources of liquidity including current cash and cash equivalents and marketable securities and management’s cash flow projections. Our ability to continue as a going concern is expected to be impacted by our ability to raise additional capital to fund our operations, produce cash inflows fromJelmyto product sales and develop UGN-102. Based on our cash, cash equivalents and marketable securities as of June 30, 2024, together with management’s cash flow projections, we believe we have sufficient cash and cash equivalents to fund our operations beyond one year from the issuance of our condensed consolidated financial statements appearing elsewhere in this Quarterly Report. We may need to raise additional capital in the future. There can be no assurances that we will be able to secure such additional financing on terms that are satisfactory to us, in an amount sufficient to meet our needs, or at all. In the event we are not successful in obtaining sufficient funding, this could force us to delay, limit, or reduce our product development, commercialization efforts or other operations.

Current wording

We routinely evaluate our liquidity needs, including assessment of our current financial condition, sources of liquidity including current cash and cash equivalents and marketable securities and management’s cash flow projections. Our ability to continue as a going concern is expected to be impacted by our ability to raise additional capital to fund our operations, produce cash inflows fromJelmyto product sales and advance UGN-102 through regulatory approval. Based on our cash, cash equivalents and marketable securities as of September 30, 2024, together with management’s cash flow projections, we believe we have sufficient cash and cash equivalents to fund our operations beyond one year from the issuance of our condensed consolidated financial statements appearing elsewhere in this Quarterly Report. We may need to raise additional capital in the future. There can be no assurances that we will be able to secure such additional financing on terms that are satisfactory to us, in an amount sufficient to meet our needs, or at all. In the event we are not successful in obtaining sufficient funding, this could force us to delay, limit, or reduce our product development, commercialization efforts or other operations.