- Initiated submission of a rolling NDA to the FDA for UGN-102;
12-month duration of response data from ENVISION expected to
support completion of NDA submission
- Announced next-generation novel mitomycin-based RTGel
formulations for LG-IR-NMIBC and LG-UTUC programs from medac GmbH
licensing agreement with potential IP protection until December
2041
- JELMYTO® achieved net product revenues of $82.7 million in
2023, an increase of ~28% compared with 2022
- Announced signing of a restructured agreement with Pharmakon
Advisors that provides more favorable terms and provides UroGen up
to an additional $100 million credit facility
- Conference call and webcast to be held today at 10:00 AM
ET
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 fourth quarter and full year ended December 31, 2023, and
provided an overview of recent developments.
“In 2023, UroGen achieved important operational and clinical
milestones, setting us up for further success in the coming years,”
said Liz Barrett, President and Chief Executive Officer of UroGen.
“The Phase 3 ATLAS and ENVISION trials both produced meaningful and
unprecedented results underscoring the potential of UGN-102 to
fundamentally change the way patients with low-grade
intermediate-risk non-muscle invasive bladder cancer are treated.
We look forward to reporting the 12-month duration of response data
from ENVISION in the second quarter of 2024 and to completing
submission of an NDA in September of this year. UGN-102 has the
potential to address a more than $3 billion market opportunity and,
if approved, has the potential to be transformative for our
company. JELMYTO continues to show double digit growth with patient
and physician adoption expected to continue to increase.”
2023 and Recent Business Highlights:
UGN-102 (mitomycin) for intravesical solution:
- In January 2024, UroGen initiated submission of a rolling New
Drug Application (NDA) to the U.S. Food and Drug Administration
(FDA) for UGN-102 as a treatment of low-grade intermediate-risk
non-muscle invasive bladder cancer (LG-IR-NMIBC). The first part of
the submission was the Chemistry, Manufacturing and Controls (CMC)
sections. The FDA indicated that evaluation of duration of complete
response at 12-months from the pivotal ENVISION trial will be
sufficient to support submission of the NDA. The company plans to
complete the submission in September 2024 with a potential FDA
decision as early as the first quarter of 2025.
- Positive top-line data from ATLAS Phase 3 clinical trial
highlighting UGN-102 as non-surgical treatment option for
LG-IR-NMIBC published in The Journal of Urology.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade
upper tract urothelial cancer (LG-UTUC):
- Generated annual net product revenue of $82.7 million in 2023,
compared with $64.4 million in 2022 in representing ~28% annual
growth.
- Patient enrollment forms, new patient starts, and total doses
each grew approximately 25% year-over-year. First-time writers,
activated sites and repeat accounts also continue to grow.
- A retrospective study investigated whether patients with
higher-volume low-grade disease could achieve disease-free status
using partial ablation or biopsy before JELMYTO treatment during
initial ureteroscopy. The study found no significant difference in
disease-free rates between complete ablation (78.6%), partial
ablation (57.6%), or biopsy-only (66.7%) groups during initial
ureteroscopy. Tumor size prior to JELMYTO induction also showed no
significant impact on disease-free rates. The study aimed to find
alternatives to nephroureterectomy for preserving kidney function
and to assess JELMYTO's efficacy in managing larger volume disease.
Findings from the study were published online in Urologic Oncology:
Seminars and Original Investigations online.
Next-generation novel mitomycin-based formulation for
urothelial cancers
- In January 2024, UroGen announced a license and supply
agreement with medac GmbH to develop a next-generation novel
mitomycin-based formulation for urothelial cancers. UGN-103 and
UGN-104 combine UroGen’s RTGel® technology with medac’s licensed
mitomycin formulation. The agreement and development program
potentially allow UroGen to extend the patent protection on its
urothelial cancer franchise. medac has intellectual property
protection for its mitomycin formulation expected to last until
June 2035 and UroGen has pending U.S. patent applications which may
provide protection until December 2041.
- UroGen plans to initiate Phase 3 studies to explore the safety
and efficacy of UGN-103 and UGN-104 in LG-IR-NMIBC and LG-UTUC,
respectively, in 2024.
Up to $100 Million Letter of Credit Facility with Funds
Managed by Pharmakon
- Announced signing of a restructured loan agreement with
Pharmakon Advisors providing UroGen additional funding of up to
$100 million. Under the terms of the restructured agreement, the
interest rate on the previously funded $100 million loan is
reduced, and the initiation of the payback period will be delayed
following UGN-102 approval. The restructured agreement also
includes a credit facility of up to $100 million. As part of the
agreement, UroGen is required to draw down on the first tranche of
$25 million of the credit facility by September 30, 2024, and will
have the option to access as much as an additional $75 million
following UGN-102 approval.
Fourth Quarter and Full Year 2023 Financial Results
JELMYTO Revenue: UroGen reported JELMYTO net product
revenues of $23.5 million in the fourth quarter of 2023, compared
to $18.1 million for the same period in 2022. Net JELMYTO product
revenue for the full year 2023 was $82.7 million, compared to $64.4
million in 2022. Despite strong unit growth, full year 2023 net
revenues were impacted by higher than forecast 340B chargebacks and
first time estimated Medicare refunds for discarded drug, offset by
non-patient purchases.
R&D Expense: Research and development expenses for
the fourth quarter of 2023 were $11.3 million, including non-cash
share-based compensation expense of $0.5 million as compared to
$14.5 million, including non-cash share-based compensation expense
of $0.6 million, for the same period in 2022. Research and
development expenses for the full year 2023 were $45.6 million,
including non-cash share-based compensation expense of $1.9 million
as compared to $52.9 million, including non-cash share-based
compensation expense of $2.6 million, in 2022.
SG&A Expense: Selling, general and administrative
expenses for the fourth quarter of 2023 were $24.6 million,
including non-cash share-based compensation expense of $2.1
million. This compares to $21.6 million, including non-cash
share-based compensation expense of $1.8 million, for the same
period in 2022. Selling, general and administrative expenses for
the full year 2023 were $93.3 million, including non-cash
share-based compensation expense of $7.4 million. This compares to
$82.8 million, including non-cash share-based compensation expense
of $8.0 million, in 2022.
Financing on Prepaid Forward Obligation: UroGen reported
non-cash financing expense related to the prepaid forward
obligation to RTW Investments of $5.5 million in the fourth quarter
of 2023, compared to $5.1 million in the same period in 2022.
Non-cash financing expense related to the RTW Investments
obligation was $21.6 million for the full year 2023, compared to
$21.6 million in 2022. The rate applied to cash payments incurred
in 2023 is 13% based on global JELMYTO net product sales of $82.7
million in 2023.
Interest Expense on Long-Term Debt: Interest expense
related to the $100 million term loan facility with funds managed
by Pharmakon Advisors was $3.6 million and $14.7 million,
respectively for the fourth quarter and full year 2023, compared to
$3.2 million and $8.4 million in the same periods in 2022. The
higher amount in 2023 was a result of interest expense for four
full quarters in 2023, whereas the first and second tranches of the
loan were funded in March 2022 and December 2022, respectively.
Net Loss: UroGen reported a net loss of $26.0 million or
($0.72) per basic and diluted share in the fourth quarter of 2023
compared with a net loss of $28.9 million or ($1.25) per basic and
diluted share in the same period in 2022. Net loss was $102.2
million or ($3.55) per basic and diluted share in the full year
2023 compared with a net loss of $109.8 million or ($4.81) per
basic and diluted share in 2022.
Cash & Cash Equivalents: As of December 31, 2023,
cash, cash equivalents and marketable securities totaled $141.5
million.
2024 Revenue, Operating Expense and RTW Expense Guidance:
The Company anticipates full year 2024 net product revenues from
JELMYTO to be in the range of $95 to $102 million. Increased
discounts related to Medicare refunds for discarded drugs and 340B
purchases will further impact net revenues in 2024. The Company
also expects full year 2024 operating expenses in the range of $175
to $185 million, including non-cash share-based compensation
expense of $6 to $11 million, subject to market conditions. The
Company also reiterates anticipated full year 2024 non-cash
financing expense related to the prepaid obligation to RTW
Investments in the range of $21 to $26 million. Of this amount
approximately $12.4 to $13.3 million is expected to be in cash.
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.
UROGEN PHARMA LTD.
SELECTED CONSOLIDATED BALANCE
SHEETS
(U.S. dollars in thousands)
(Unaudited)
December 31, 2023
December 31, 2022
Cash and cash equivalents and marketable
securities
$
141,469
$
99,963
Total assets
$
178,311
$
135,619
Total liabilities
$
243,523
$
224,980
Total shareholders' deficit
$
(65,212
)
$
(89,361
)
UROGEN PHARMA LTD.
CONSOLIDATED STATEMENTS OF
OPERATIONS AND COMPREHENSIVE LOSS
(U.S. dollars in thousands,
except share and per share data)
(Unaudited)
Three months ended December 31,
(Unaudited)
Year ended
December 31,
2023
2022
2023
2022
Revenue1
$
23,530
$
18,092
$
82,713
$
64,357
Cost of revenue
2,286
2,263
9,361
7,654
Gross profit
21,244
15,829
73,352
56,703
Operating expenses:
Research and development expenses
11,302
14,477
45,614
52,906
Selling, general and administrative
expenses
24,551
21,634
93,274
82,838
Total operating expenses
35,853
36,111
138,888
135,744
Operating loss
(14,609
)
(20,282
)
(65,536
)
(79,041
)
Financing on prepaid forward
obligation
(5,505
)
(5,081
)
(21,552
)
(21,559
)
Interest expense on long-term debt
(3,586
)
(3,223
)
(14,715
)
(8,438
)
Interest and other income, net
1,538
406
3,479
1,010
Loss before income taxes
$
(22,162
)
$
(28,180
)
$
(98,324
)
$
(108,028
)
Income tax expense
(3,854
)
(689
)
(3,920
)
(1,775
)
Net loss
$
(26,016
)
$
(28,869
)
$
(102,244
)
$
(109,783
)
Net loss per ordinary share basic and
diluted
$
(0.72
)
$
(1.25
)
$
(3.55
)
$
(4.81
)
Weighted average shares outstanding, basic
and diluted
36,153,634
23,088,891
28,834,303
22,806,812
1.
2023 full-year JELMYTO net revenues
include gross-to-net unfavorability driven by higher 340B
chargebacks and estimated Medicare refunds for discarded drugs,
offset by $4.4 million in CREATES Act sales, of which $2.4 million
was realized in Q4 2023.
About JELMYTO
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
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: urinary
tract infection, blood in your urine, side pain, nausea, trouble
with urination, kidney problems, vomiting, tiredness, stomach
(abdomen) pain.
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)
Urothelial 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.
About UGN-102
UGN-102 (mitomycin) for intravesical solution is an innovative
drug formulation of mitomycin, currently in Phase 3 development for
the treatment of low-grade, intermediate-risk, non-muscle invasive
bladder cancer (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. Assuming positive findings from
the durability of response endpoint from the ENVISION Phase 3
study, UroGen anticipates completing its NDA submission for UGN-102
in September with a potential FDA decision as early as the first
quarter of 2025.
About UroGen Pharma Ltd.
UroGen is a biotech 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 UGN-102 (mitomycin) for intravesical
solution for patients with low-grade non-muscle invasive bladder
cancer are designed to ablate tumors by non-surgical means. UroGen
is headquartered in Princeton, NJ with operations in Israel. Visit
www.UroGen.com to learn more or follow us on X (Twitter),
@UroGenPharma.
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:
operational and clinical milestones achieved in 2023 will set the
Company up for further success in the coming years; the potential
of UGN-102 to fundamentally change the way patients with
LG-IR-NMIBC are treated; the expected timing for the release of
12-month duration of response data from ENVISION in the second
quarter of 2024 and that such data will be sufficient to support
the submission of an NDA for UGN-102; the expected timing for
completing a submission of an NDA to the FDA for UGN-102 and the
potential FDA decision; the potential of UGN-102 to address more
than $3 billion in market opportunity and to be transformative to
the Company; expectations that JELMYTO patient and physician
adoption is expected to continue to increase; the potential of the
license and supply agreement with medac GmbH to allow UroGen to
extend patent protection on its urothelial cancer franchise;
UroGen’s pending U.S. patent applications potentially providing
protection for UGN-103 and UGN-104 until December 2041; UroGen’s
plans to initiate Phase 3 studies to explore the safety and
efficacy of UGN-103 and UGN-104 in 2024; UroGen’s ability to access
an additional $75 million under the amended credit facility with
Pharmakon Advisors, in addition to the required $25 million draw;
UroGen’s 2024 financial guidance; the potential of UroGen’s
proprietary RTGel technology to improve therapeutic profiles of
existing drugs; and UroGen’s sustained release technology making
local delivery potentially more effective as compared to other
treatment options. Words and phrases such as “anticipate,”
“assume,” “believe,” “expected,” “if,” “indicate,” “look forward
to,” “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; the ability to obtain regulatory approval
within the timeframe expected, or at all; the findings from the
durability of response endpoint from the ENVISION Phase 3 study may
not be positive, and in such event, UroGen’s NDA pathway could be
negatively impacted; even if the durability of response endpoint
data from the ENVISION Phase 3 study are positive there is no
guarantee that the current clinical development plan for UGN-102
will ultimately support submission of an NDA, notwithstanding the
current agreement with the FDA; even if an NDA for UGN-102 is
accepted by the FDA, there is no guarantee that such NDA will be
sufficient to support approval of UGN-102 on the timeframe
expected, or at all; UroGen’s ability to access the additional $75
million under the amended credit facility with Pharmakon Advisers
(in addition to the required $25 million draw) is subject to
certain drawdown conditions; the ability to maintain regulatory
approval; the ability to obtain and maintain adequate intellectual
property rights and adequately protect and enforce such rights;
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 our product and product candidates and the rate
and degree of market acceptance thereof vis-à-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 for the quarter ended September 30, 2023, filed with
the SEC on November 14, 2023, and in UroGen’s Annual Report on Form
10-K for the year ended December 31, 2023, being filed with the SEC
today (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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240314112098/en/
INVESTOR CONTACT: Vincent Perrone Senior Director, Investor
Relations vincent.perrone@UroGen.com 609-460-3588 ext. 1093
MEDIA CONTACT: Cindy Romano Director, Corporate Communications
cindy.romano@UroGen.com 609-460-3583 ext. 1083
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