UroGen Pharma Reports First Quarter 2019 Financial Results and Recent Corporate Developments

Recent Data Presentation Highlights Consistent Complete Response Rate
and Strong Durability at Six-Months in Evaluable Patients with Low-Grade
Upper Tract Urothelial Cancer (LG UTUC)

Completion of UGN-101 Rolling NDA Submission to the FDA On Track for
2H 2019

Pre-Commercial Activities and Infrastructure Build-out Accelerate
Ahead of Planned U.S. Approval and Launch of UGN-101 in 1H 2020

Conference Call and Webcast to be Held Today at 8:30 AM ET

NEW YORK–(BUSINESS WIRE)–UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical
company developing treatments to address unmet needs in uro-oncology,
today announced financial results for the first quarter ended March 31,
2019 and provided an overview of the Company’s recent developments.

“The accomplishments and performance of UroGen during the first quarter
have set the stage for a pivotal and exciting year ahead as we prepare
for the potential approval and commercialization of UGN-101, our first
investigational candidate. Our top priority remains completion of our
rolling New Drug Application (NDA) for UGN-101 for the treatment of
patients with low-grade upper tract urothelial cancer (LG UTUC) and,
with our commercial preparations well underway, we are confident in our
readiness to deliver on a strong launch in the first half of 2020,” said
Liz Barrett, President and Chief Executive Officer of UroGen. “Our
company sees great possibilities for the RTGel platform within
uro-oncology, especially around new therapeutic modalities. Our goal is
to leverage that and continue to build a company with sustainable growth
via a robust pipeline as well as assessing opportunities via external
partnerships.”

Recent Highlights

• UGN-101 Clinical Development:
  • At the 114^th American Urological Association (AUA)
    Annual Meeting in Chicago, Seth Lerner, M.D. delivered a
    presentation during the plenary session that highlighted the unmet
    need and potential for UGN-101 to change the treatment paradigm
    for patients with LG UTUC.
    • The updated analysis demonstrated that in the OLYMPUS
      intent-to-treat population, 71 patients had undergone primary
      disease evaluation (PDE) at the time of the analysis and 42 of
      the 71 patients (59 percent) achieved a complete response
      (CR). Forty-one patients entered follow-up. At the time of the
      analysis, 27 patients underwent a six-month evaluation, and 24
      out of 27 patients (89 percent) have remained disease free at
      six months. The most common adverse events observed were
      urinary tract infection, ureteral narrowing and stricture
      formation. The majority of ureteral events were reported as
      mild to moderate and have resolved. Full Phase 3 data from the
      OLYMPUS trial is anticipated for 2H 2019.
  • The Company remains on track to complete its rolling NDA for
    UGN-101 in 2H 2019 and is planning for approval in 1H 2020. If
    approved, UGN-101 would be the first drug approved for the
    non-surgical treatment of LG UTUC. Full data is planned for 2H
    2019.
• Pipeline Advancement:
  • UGN-102:
    • The Company continues to enroll patients in its Phase 2b
      OPTIMA II clinical trial of UGN-102 (mitomycin gel) for
      intravesical instillation as a first-line chemoablation agent
      in the treatment of patients with intermediate risk low-grade
      non-muscle invasive bladder cancer (LG NMIBC), a form of
      disease associated with a high risk of recurrence.
    • Initial data from the OPTIMA II trial of UGN-102 is expected
      in 2H 2019.
    • There are currently no drugs approved by the FDA as first-line
      treatment for LG NMIBC. UGN-102 represents a substantial
      opportunity in UroGen’s pipeline and has the potential to be a
      treatment option for up to approximately 80,000 patients for
      whom repetitive surgical resection via Transurethral Resection
      of Bladder Tumor (TURBT) remains the standard of care.
  • UGN-201:
    • UroGen is currently evaluating the optimal pathway to advance
      UGN-201, a TLR7/8 immunomodulatory agent for the treatment of
      high-grade bladder disease.
• Commercial Preparations:
  • The Company continues to accelerate its pre-commercial activities
    and infrastructure build-out to support the anticipated U.S.
    approval and launch of UGN-101 targeted for 1H 2020. The focus is
    on building awareness of our RTGel technology and unmet needs in
    UTUC to support rapid adoption and seamless integration of UGN-101
    into the urologist practice following regulatory approval.
  • A strong team of seven medical science liaisons (MSLs) have been
    strategically deployed across the U.S. to engage in scientific
    exchange and clinical support.
  • The Company launched www.UTUC.com,
    the first resource designed to address a void in the urology space
    by educating patients about UTUC and available treatment options.
• Business Development:
  • UroGen recently entered into an agreement with Janssen Research &
    Development, LLC (Janssen) to conduct an early-stage feasibility
    evaluation in a therapeutic area of mutual interest. UroGen and
    Janssen will each conduct certain activities under the terms of
    the agreement.

First Quarter 2019 Financial Results; 2019 Guidance

• As of March 31, 2019, cash and cash equivalents totaled $246.7
  million. This includes net proceeds of approximately $161.4 million
  from a public offering of ordinary shares in January 2019.
• Research and development expenses for the three months ended March 31,
  2019 were $9.7 million, including non-cash share-based compensation
  expense of $2.3 million.
• General and administrative expenses for the three months ended March
  31, 2019 were $12.7 million, including non-cash share-based
  compensation expense of $5.1 million.
• The Company reported a net loss of $21.4 million, or basic and diluted
  net loss per ordinary share of $1.11, for the three months ended March
  31, 2019.
• The 2019 financial guidance set forth during the Company’s year-end
  earnings call on February 28^th 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 8:30 am Eastern Time to review the Company’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. Alternatively, please call (888) 771-4371 (U.S.) or (847)
585-4405 (International) to listen to the live conference call. The
conference ID number for the live call will be 48486174. An archive of
the webcast will be available for two weeks on the Company’s website.

About UroGen Pharma Ltd.

UroGen Pharma Ltd. (Nasdaq:URGN) is a clinical-stage biopharmaceutical
company developing advanced non-surgical treatments to address unmet
needs in the field of urology, with a focus on uro-oncology. UroGen has
developed RTGel™ 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 lead investigational candidates,
UGN-101 (mitomycin gel) for instillation, and UGN-102 (mitomycin gel)
for intravesical instillation, are designed to potentially 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 New York, NY with operations in Los Angeles,
CA and Israel.

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: plans to conduct an early stage
feasibility evaluation; the potential of UGN-101 for LG UTUC; the timing
for completion of the rolling NDA for UGN-101; the anticipated timing
for full Phase 3 data from the OLYMPUS trial; the potential approval of
UGN-101 and the timing thereof; the expectation that UGN-101, if
approved, will be the first drug approved for the non-surgical treatment
of LG UTUC; the timing for completion of pre-commercial activities and
infrastructure build-out in anticipation of a potential commercial
launch of UGN-101; the expected readiness of UroGen for a potential
commercial launch of UGN-101 and the strength and timing of the
potential commercial launch of UGN-101; the potential of UroGen’s
proprietary RTGel™ technology platform to improve therapeutic profiles
of existing drugs; the anticipated timing for initial data from the
OPTIMA II trial; the opportunity and potential of UGN-102 for LG NMIBC;
and UroGen’s 2019 guidance. These statements are subject to a number of
risks, uncertainties and assumptions, including, but not limited to: the
timing and success of clinical trials, including the OLYMPUS Phase 3
trial and the OPTIMA II Phase 2b trial and potential safety and other
complications thereof; the ability to obtain regulatory approval within
the timeframe expected, or at all; the ability to maintain regulatory
approval; complications associated with achieving commercial readiness
for the launch of a new product; the labeling for any approved product;
the scope, progress and expansion of developing and commercializing
UroGen’s product candidates; the size and growth of the market(s)
therefor and the rate and degree of market acceptance thereof vis-à-vis
alternative therapies; risks that UroGen’s net loss for 2019 may differ
materially from the anticipated range previously provided by UroGen and
affirmed in this press release due to changes in UroGen’s operating
plans and/or due to estimates that may prove to be incorrect; and
UroGen’s ability to attract or retain key management, members of the
board of directors and personnel. In light of these risks and
uncertainties, and other risks and uncertainties that are described in
the Risk Factors section of UroGen’s Form 10-Q filed with the SEC on the
date hereof and other filings that UroGen makes with the SEC from time
to time (which are available at http://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.

Contacts

UROGEN CONTACT:
Kate Bechtold
Director, Corporate
Communications & Investor Relations
[email protected]
914-552-0456