Full Press Release Details
UroGen Pharma Ahead of Schedule to Complete UGN-102 NDA Submission and Reports 2024 Second Quarter Financial
Results and Business Highlights
PRINCETON, N.J. August 13, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and
commercializing innovative solutions that treat urothelial and specialty cancers, today announced financial results for the second quarter ended June 30, 2024, and provided an overview of recent developments.
Our immediate priority is completing the submission of a New Drug Application in the very near term for UGN-102,
which we believe has the potential to be a practice-changing therapy for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer,
said Liz Barrett, President and Chief Executive Officer of UroGen. The compelling body of clinical data, including the ENVISION trial, which demonstrated an unprecedented 82.3% 12-month duration of
response by Kaplan-Meier analysis in patients who had previously achieved a complete response at three months, reinforces the opportunity for UGN-102 to be the first
FDA-approved medicine for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer.
Ms. Barrett continued, We estimate that approximately 82,000 patients suffering from this highly recurrent disease each year may benefit from an
innovative treatment, creating an estimated five-billion-dollar market opportunity. Our immediate commercial focus is preparing for UGN-102 s potential approval and launch with the goal to establish our
leadership in urothelial cancers.
Q2 2024 and Recent Business Highlights:
UGN-102 (mitomycin) for intravesical solution:
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC):
Next-generation novel mitomycin-based formulation for urothelial cancers
Public offering of ordinary shares and pre-funded warrants
Second quarter 2024 financial results
Revenue: JELMYTO net product revenues were $21.8 million and $21.1 million for the three months ended June 30, 2024, and 2023, respectively.
R&D Expense: Research and development expenses for the second quarter of 2024 were $15.4 million, including non-cash share-based compensation expense of $0.6 million as compared to $11.6 million, including non-cash share-based compensation expense
of $0.5 million, for the same period in 2023.
SG&A Expense: Selling, general and administrative expenses for the second quarter of
2024 were $30.1 million, including non-cash share-based compensation expense of $3.0 million. This compares to $22.5 million, including
non-cash share-based compensation expense of $1.7 million, for the same period in 2023.
Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $5.8 million in the second quarter of 2024, compared to
$5.3 million in the same period in 2023.
Interest Expense on Long-Term Debt: Interest expense related to the up to $200 million term
loan facility with funds managed by Pharmakon Advisors was $3.5 million in the second quarter of 2024, compared to $3.8 million in the same period in 2023.
Net Loss: UroGen reported a net loss of $33.4 million or ($0.91) per basic and diluted share in the second quarter of 2024 compared with a net
loss of $24.1 million or ($1.03) per basic and diluted share in the same period in 2023.
Cash & Cash Equivalents:
As of June 30, 2024, cash, cash equivalents and marketable securities totaled $241.3 million.
2024 Revenue, Operating Expense, and RTW Expense Guidance: With respect to the Company s
previously provided full-year 2024 JELMYTO revenue guidance, the Company sees a path toward the lower end of the guidance range. With respect to the Company s previously provided full-year 2024 operating expense guidance, the Company expects to
be toward the higher end of the guidance range, with a revised non-cash share-based compensation expense of $9 to $13 million, subject to market conditions. The anticipated full year 2024 non-cash financing expense related to the prepaid obligation to RTW Investments is unchanged and expected to be in the range of $21 to $26 million. The rate for the cash component of the RTW obligation will be
13% of global net product sales of JELMYTO in 2024.
Conference Call & Webcast Information: Members of UroGen s
management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review UroGen s financial results and provide a general business update.
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.
CONSOLIDATED BALANCE SHEETS
(U.S. dollars in thousands)
| June 30, 2024 | December 31, 2023 | |||||||
| Cash and cash equivalents and marketable securities | $ | 241,280 | $ | 141,470 | ||||
| Total assets | $ | 281,849 | $ | 178,311 | ||||
| Total liabilities | $ | 251,535 | $ | 243,523 | ||||
| Total shareholders equity (deficit) | $ | 30,314 | $ | (65,212 | ) |
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(U.S. dollars in thousands, except share and per share data)
| Three months ended June 30, | Six months ended June 30, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Revenue | $ | 21,848 | $ | 21,139 | $ | 40,629 | $ | 38,331 | ||||||||
| Cost of revenue | 2,229 | 2,443 | 3,957 | 4,708 | ||||||||||||
| Gross profit | 19,619 | 18,696 | 36,672 | 33,623 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development expenses | 15,402 | 11,584 | 30,896 | 24,082 | ||||||||||||
| Selling, general and administrative expenses | 30,056 | 22,494 | 57,355 | 46,968 | ||||||||||||
| Total operating expenses | 45,458 | 34,078 | 88,251 | 71,050 | ||||||||||||
| Operating loss | (25,839 | ) | (15,382 | ) | (51,579 | ) | (37,427 | ) | ||||||||
| Financing on prepaid forward obligation | (5,773 | ) | (5,344 | ) | (11,433 | ) | (10,568 | ) | ||||||||
| Interest expense on long-term debt | (3,461 | ) | (3,761 | ) | (5,908 | ) | (7,314 | ) | ||||||||
| Interest and other income, net | 1,708 | 405 | 3,323 | 1,035 | ||||||||||||
| Loss before income taxes | $ | (33,365 | ) | $ | (24,082 | ) | $ | (65,597 | ) | $ | (54,274 | ) | ||||
| Income tax expense | (38 | ) | (54 | ) | (92 | ) | (75 | ) | ||||||||
| Net loss | $ | (33,403 | ) | $ | (24,136 | ) | $ | (65,689 | ) | $ | (54,349 | ) | ||||
| Net loss per ordinary share basic and diluted | $ | (0.91 | ) | $ | (1.03 | ) | $ | (1.87 | ) | $ | (2.33 | ) | ||||
| Weighted average shares outstanding, basic and diluted | 36,821,915 | 23,462,016 | 35,106,524 | 23,371,878 |
JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for the treatment
of adult patients with LG-UTUC. It is recommended for primary treatment of biopsy-proven LG-UTUC in patients deemed appropriate candidates for renal-sparing therapy.
JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for
administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for
chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.
APPROVED USE FOR JELMYTO
JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the
upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).
IMPORTANT SAFETY INFORMATION
receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.
receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:
How will I receive JELMYTO?
After receiving JELMYTO:
JELMYTO may cause serious side effects, including:
You are encouraged to report
negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You
may also report side effects to UroGen Pharma at 1-855-987-6436.
Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.
About Upper Tract Urothelial Cancer (UTUC)
cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively
referred to as upper tract urothelial cancers (UTUC). In the U.S., there are approximately 6,000 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients
over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can
lead to a high rate of recurrence and relapse.
UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3
development for the treatment of LG-IR-NMIBC. Utilizing UroGen s proprietary RTGel technology, a
sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by
non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen anticipates
completing its NDA submission for UGN-102 in the very near term with a potential FDA decision as early as the first quarter of 2025, assuming priority review.
About UroGen Pharma Ltd.
company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary
sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen s sustained release technology is designed to enable longer exposure of the urinary tract tissue to
medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and investigational treatment
Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without
limitation, statements regarding: expected timing for completing the NDA submission for UGN-102 and potential FDA approval; the potential of UGN-102, including to be a
practice-changing therapy and the first FDA approved medicine for LG-IR-NMIBC; the estimated patient population and market opportunity for
UGN-102; the expectation that the ENVISION DOR data will support an NDA for UGN-102; the potential and advantages of UGN-103 and UGN-104, including to potentially offer manufacturing efficiencies and additional intellectual property protection for UroGen s low-grade urothelial cancer franchise; the
ongoing and planned clinical trials for UGN-103 and UGN-104; UroGen s goals and commercial focus; 2024 financial guidance; the estimated patient population and
demographics for UTUC; the potential of UroGen s proprietary RTGel technology to improve therapeutic profiles of existing drugs; and the potential of UroGen s sustained release technology to make local delivery more effective as compared
to other treatment options. Words such as anticipate, assume, could, plan, potential, will, or other words that convey uncertainty of future events or outcomes to identify these
forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: preliminary results may not be indicative of results that may be observed in the future; the timing and
success of clinical trials and potential safety and other complications thereof; unforeseen delays that may impact the timing of progressing clinical trials and reporting data; results from the ENVISION Phase 3 trial may not be sufficient to support
an NDA submission for UGN-102; even if an NDA for UGN-102 is accepted by the FDA, there is no guarantee that such NDA will be given priority review or that such NDA will
be sufficient to support approval of UGN-102 on the timeframe expected, or at all; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to obtain and maintain
adequate intellectual property rights and adequately protect and enforce such rights; the ability to maintain regulatory approval; complications associated with commercialization activities; the labeling for any approved product; competition in
UroGen s industry; the scope, progress and expansion of developing and commercializing UroGen s product candidates; the size and growth of the market(s) for UroGen s product and product candidates and the rate and degree of market
acceptance thereof vis- -vis alternative therapies; UroGen s ability to attract or retain key management, members of the board of directors and personnel;
UroGen s RTGel technology may not perform as expected; UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology; and UroGen s financial condition and need for additional
capital in the future. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen s Quarterly Report on Form 10-Q, filed with
the SEC on May 13, 2024, as well as in the Risk Factors section of UroGen s Quarterly Report on Form 10-Q being filed with the SEC later today (which are available
at www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and UroGen s actual results could differ materially and adversely from those anticipated or implied thereby. Any
forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.
Senior Director, Investor Relations