UroGen Pharma Reports First Quarter 2020 Financial Results and Recent Corporate Developments
Received
Jelmyto Launch on Track for
Reported UGN-102 Phase 2b Trial Interim Data with 65% Complete Response at Three Months and 85% Durability at Nine Months in Low-Grade Non-Muscle Invasive Bladder Cancer
Conference Call and Webcast to be held Today at
“The first quarter of 2020 was marked by flawless execution by our team as we prepared for the landmark FDA approval of Jelmyto, the first and only non-surgical treatment for patients with low-grade upper tract urothelial cancer. It has been our mission to pioneer novel treatments for patients in areas of unmet need, and our team has developed innovative solutions to engage key stakeholders so we can bring this effective, kidney-sparing therapy to patients who have been waiting for new treatment options,” said
Recent Highlights and Upcoming Milestones
Jelmyto (mitomycin) for pyelocalyceal solution, formerly known as UGN-101, for adult patients with low-grade upper tract urothelial cancer (LG-UTUC)
-
UroGen announced the
U.S. FDA granted approval of Jelmyto for the treatment of patients with LG-UTUC onApril 15, 2020 . Jelmyto is the first and only FDA approved non-surgical treatment option for patients with LG-UTUC. - 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. No treatment-related deaths occurred.
- The Lancet Oncology published pivotal results from the Phase 3 OLYMPUS trial, reporting that 59% of LG-UTUC patients treated with Jelmyto achieved a Complete Response (CR). Durability at 12-months (based on interim data) was estimated to be 84% by Kaplan-Meier analysis.
- At the 12-month time point for assessment of durability, 19 patients remained in CR, seven had experienced recurrence of disease, nine patients continued to be followed for the 12-month duration of response, and median duration of response was not reached as of the FDA-approval date.
Jelmyto Commercial Readiness
-
The Company expects to commence the official commercial launch of Jelmyto on
June 1, 2020 . - To maximize engagement with health care professionals and key stakeholders in this current environment, the Company has developed innovative solutions, including a virtual platform, to allow for an effective launch.
- The team has completed the application for submission of the C-code, and submission of the J-code application to CMS is in process. The company continues to expect a C-code will be secured by October and a J-code by the end of the year.
UGN-102 (mitomycin) for intravesical solution for patients with low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC)
-
UroGen announced a positive interim data analysis of UGN-102 in patients with LG-IR-NMIBC, featured in a late-breaking abstract published in the April Supplement to
The Journal of Urology . - The Phase 2b OPTIMA II trial demonstrated a CR rate at three months following onset of treatment of 65% (41/63 patients). In this subset of patients, 31/32 patients (97%) and 17/20 patients (85%) remained free of disease at six- and nine-months follow-up, respectively.
- The most commonly reported adverse events seen to date (≥ 10%) were reported as mild to moderate and include dysuria, hematuria, urinary frequency, fatigue, urgency and urinary tract infection.
-
The detailed results presentation from the Phase 2b OPTIMA II trial will be available online via the
American Urological Association (AUA) inmid-May 2020 . - The Company remains on track to initiate a pivotal Phase 3 trial in 2H 2020.
-
There are no drugs currently approved by the FDA as first-line treatment for LG-IR-NMIBC. UGN-102 has the potential to provide a non-surgical treatment alternative for approximately 80,000 patients diagnosed with LG-IR-NMIBC in
the United States alone.
First Quarter 2020 Financial Results; 2020 Guidance
-
As of
March 31, 2020 , cash, cash equivalents and marketable securities totaled$159.2 million , excluding restricted cash. -
Research and development expenses for the three months ended
March 31, 2020 were$16.6 million , including non-cash share-based compensation expense of$1.9 million , and a one-time payment of$6.6 million to unwind our obligation to the IIA. -
Selling, general and administrative expenses for the three months ended
March 31, 2020 were$22.0 million , including non-cash share-based compensation expense of$5.7 million . -
UroGen reported a net loss of
$37.8 million , or basic and diluted net loss per ordinary share of$1.79 , for the three months endedMarch 31, 2020 . -
The 2020 financial guidance set forth during the Company’s full year and fourth quarter 2019 quarterly call on
March 2, 2020 remains the same based on current business goals and anticipated activities.
Conference Call & Webcast Information
Members of UroGen’s management team will host a live conference call and webcast today at
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 (
CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||
( |
||||||
(Unaudited) | ||||||
Assets | ||||||
CURRENT ASSETS: | ||||||
Cash and cash equivalents |
$ |
26,468 |
$ |
49,688 |
||
|
92,623 |
|
97,389 |
|||
Restricted cash |
|
1,223 |
|
523 |
||
Prepaid expenses and other current assets |
|
1,552 |
|
1,034 |
||
TOTAL CURRENT ASSETS |
|
121,866 |
|
148,634 |
||
NON-CURRENT ASSETS: | ||||||
Property and equipment, net |
|
991 |
|
977 |
||
Restricted deposit |
|
223 |
|
223 |
||
Right of use asset |
|
3,433 |
|
3,735 |
||
|
40,137 |
|
48,555 |
|||
Other non-current assets |
|
279 |
|
264 |
||
TOTAL ASSETS |
$ |
166,929 |
$ |
202,388 |
||
Liabilities and Shareholders' equity | ||||||
CURRENT LIABILITIES: | ||||||
Accounts payable and accrued expenses |
$ |
8,500 |
$ |
11,186 |
||
Employee related accrued expenses |
|
4,075 |
|
6,711 |
||
Other current liabilities |
|
1,632 |
|
1,585 |
||
TOTAL CURRENT LIABILITIES |
|
14,207 |
|
19,482 |
||
NON-CURRENT LIABILITIES: | ||||||
Long-term lease liability |
|
2,268 |
|
2,604 |
||
TOTAL LIABILITIES |
|
16,475 |
|
22,086 |
||
TOTAL SHAREHOLDERS’ EQUITY |
|
150,454 |
|
180,302 |
||
TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY |
$ |
166,929 |
$ |
202,388 |
||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||
( |
||||||||
(Unaudited) | ||||||||
Three months ended |
||||||||
2020 |
2019 |
|||||||
REVENUES | $ |
— |
$ |
— |
||||
COST OF REVENUES | — | — | ||||||
GROSS PROFIT | — | — | ||||||
OPERATING EXPENSES: | ||||||||
Research and development expenses |
|
16,588 |
|
|
9,726 |
|
||
Selling, general and administrative expenses |
|
21,973 |
|
|
12,707 |
|
||
OPERATING LOSS |
|
(38,561 |
) |
|
(22,433 |
) |
||
INTEREST AND OTHER INCOME, NET |
|
768 |
|
|
989 |
|
||
NET LOSS |
$ |
(37,793 |
) |
$ |
(21,444 |
) |
||
STATEMENT OF COMPREHENSIVE LOSS | ||||||||
NET LOSS |
$ |
(37,793 |
) |
$ |
(21,444 |
) |
||
OTHER COMPREHENSIVE INCOME: | ||||||||
UNREALIZED GAIN ON MARKETABLE SECURITIES |
|
35 |
|
— | ||||
COMPREHENSIVE LOSS |
$ |
(37,758 |
) |
$ |
(21,444 |
) |
||
NET LOSS PER ORDINARY SHARE, BASIC AND DILUTED |
$ |
1.79 |
|
$ |
1.11 |
|
||
WEIGHTED AVERAGE SHARES OUTSTANDING, BASIC AND DILUTED |
|
21,158,161 |
|
|
19,340,082 |
|
||
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 UGN-101, Jelmyto (mitomycin) for pyelocalyceal solution, to evaluate the safety, tolerability and tumor ablative effect of Jelmyto in patients with low-grade UTUC. Seventy-one patients were treated at clinical sites across
About JelmytoTM
Jelmyto (mitomycin) for pyelocalyceal solution, is a drug formulation of mitomycin indicated for the treatment of adult patients with 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 or nephrostomy tube. The
About Upper Tract Urothelial Cancer (UTUC)
Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the
IMPORTANT SAFETY INFORMATION
You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.
Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:
- are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose.
Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
- are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
- Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?
- Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
- You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
- JELMYTO is given to your kidney through a tube called a catheter.
- During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:
- JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
- To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
- Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:
- Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
- Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: side pain, urinary tract infection, blood in your urine, kidney problems, tiredness, nausea, stomach pain, trouble with urination, vomiting, low red blood cell count, frequent urination, itching, chills, and fever.
Please see JELMYTO Full Prescribing Information, including the Patient Information at www.jelmyto.com.
About
UroGen is a biopharmaceutical company dedicated to building novel solutions that treat specialty cancers and urologic diseases because patients deserve better options. UroGen has developed RTGelTM reverse-thermal hydrogel, a proprietary sustained release, hydrogel-based platform technology that has the potential to improve 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. UroGen’s approved JelmytoTM (mitomycin) for pyelocalyceal solution, and pipeline treatment 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 and low-grade non-muscle invasive bladder cancer, respectively. UroGen is headquartered in
COVID-19 Pandemic Potential Impact
UroGen continues to gather information in this very fluid and rapidly-evolving environment regarding the potential impact of the COVID-19 pandemic on our Company, however, we are not currently able to quantify or predict with any certainty the overall scope of impact on UroGen, or any resulting delays in the availability of Jelmyto™ (mitomycin) for pyelocalyceal solution. Our primary focus is on the health and well-being of patients, caregivers, and UroGen employees at this critical juncture.
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: the potential of Jelmyto™ to transform the treatment of LG-UTUC; the potential of UroGen’s proprietary RTGel™ technology platform to improve therapeutic profiles of existing drugs; the planned commercial launch of Jelmyto on
View source version on businesswire.com: https://www.businesswire.com/news/home/20200507005185/en/
INVESTOR CONTACT:
Senior Director, Investor Relations
Kate.Bechtold@urogen.com
914-552-0456
MEDIA CONTACT:
Senior Director, Communications
Eric.VanZanten@urogen.com
610-529-6219
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