On March 18, 2021 Hamlet Pharma reported that new treatment advances in an experimental model of bladder cancer (Press release, HAMLET Pharma, MAR 18, 2021, View Source;utm_medium=rss&utm_campaign=repeated-alpha1h-treatments-creates-long-term-protection-against-bladder-tumor-growth-in-mice [SID1234576937]).

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A potent treatment effect of Alpha1H has previously been reported in mice with bladder cancer. We now show that continued treatment can provide long-term protection as well. Repeated treatment prevented tumor growth and the bladders remained similar to the control group without tumors.

In contrast, mice treated with placebo showed rapid tumor progression and severe disease after 12 days. The first round of Alpha1H treatment prevented tumor development for 4 weeks and long-term protection was achieved when treatment was repeated at 4-week intervals, as evidenced by marked differences in tumor size, pathology score, biomarkers and gene expression.

Cancer is often a chronic condition and several rounds of treatment are usually necessary. The results presented here suggest that repeated Alpha1H treatment regimens may be beneficial to ensure long-term protection against bladder cancer. This needs further validation in future clinical trials.

"Repeated treatment rounds appeared to maintain a high degree of protection against tumor progression in mice", says Catharina Svanborg, CMO and chairman of the board of Hamlet Pharma Ltd.

"This information is important for the design of future clinical trials", says Mats Persson, CEO of Hamlet Pharma Ltd.

This information is insider information that Hamlet Pharma AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication trough the agency of the contact persons set out above, on March 18, 2021 at 08.45 CET.

On March 18, 2021 ONK Therapeutics Ltd, an innovative cell therapy company focused on engineered natural killer (NK) cell therapies, reported it has entered into a license with the US National Institutes for Health (NIH) which provides rights to make and use a clinically-validated GMP-grade feeder cell line that will enable robust natural killer (NK) cell expansion, supporting scale-up of the company’s manufacturing process (Press release, ONK Therapeutics, MAR 18, 2021, View Source [SID1234576849]).

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Engineered NK cell therapies are now emerging as an exciting cell therapy platform, that shows promise in overcoming challenges of earlier CAR-T cell therapies, offering alternative therapeutic options for patients with hematological malignancies and solid tumors. Successful ex-vivo expansion of cord-derived NK cells is key to developing large cell batch sizes required to produce truly off-the-shelf engineered NK cell therapy.

This proprietary, GMP grade Epstein Barr Virus-transformed lymphoblastoid cell line (EBV-LCL) has been proven as a method to expand NK cells as part of a clinical trial being led by Dr. Richard Childs at the NIH. The feeder layer enabled a robust expansion of NK cells with greatly enhanced cytotoxic potential and strong expression of Activating Receptors and Death Receptor ligands. Expansion of up to 1,000 fold over 2-3 weeks was demonstrated with the potential for continued exponential expansion over longer periods. This expansion provides a stable and consistent NK cell population that can be subsequently genetically modified and further expanded on a batch scale supportive of clinical trials of ONK’s NK cell therapy candidates.

Prof. Michael O’Dwyer, ONK Therapeutics’ founder and CSO said: "Extensive experience over many years in Dr. Childs’ group at the NIH indicate that co-culture with EBV-LCL cells enables ex-vivo activation and robust proliferation of highly functional NK cells, which can be safely administered to patients. Applying this technology to our cord-derived, dual-targeted NK cell platform will enable the manufacture of large quantities of optimized NK cells for off-the-shelf administration."

ONK Therapeutics is accelerating the development of its NK cell engineering and manufacturing processes for its unique, proprietary dual-targeted NK cell platform that expresses both a chimeric antigen receptor (CAR) targeting a known tumor antigen and a TNF-related apoptosis-inducing ligand variant (TRAILv) targeting the death receptor pathway (i.e. DR4 or DR5).

This agreement with the NIH follows quickly on the heels of a complementary license that ONK Therapeutics recently announced with Anthony Nolan Cell & Gene Therapy Services. This license provides a consistent supply of scalable and ethically-sourced umbilical cord blood and cord-derived NK cells which, in combination with the EBV-LCL feeder layer, will provide the critical starting material for its off-the-shelf cell therapy manufacturing process, to be used for both ONK Therapeutics’ research activities and continued process development work.

Chris Nowers, ONK Therapeutics’ CEO said: "This important license further enhances our manufacturing capability, taking us another step closer to our goal of creating a next generation of uniquely engineered NK cell therapies, with broad applicability across a wide range of targets and tumor types."

On March 18, 2021 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results, including an update on its CD47 clinical programs, for the year ended December 31, 2020 (Press release, Trillium Therapeutics, MAR 18, 2021, View Source [SID1234576872]). All financial amounts in this news release are in United States dollars, unless otherwise stated.

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"2020 was a critical year in Trillium’s evolution, as we completed a wide-ranging transformation program spanning all aspects of our activities, including strategy, governance, leadership, advisory infrastructure, corporate development, funding, investor base, intellectual property and operations," said Jan Skvarka, Trillium’s President and CEO. "At the same time, we substantially advanced dose escalation studies of TTI-622 and TTI-621, which clearly demonstrated class-leading monotherapy activity. Our mission critical goal for 2021 is to rapidly move to proof of concept studies in a range of hematologic malignancy and solid tumor indications. We are incredibly excited about our prospects going forward based upon the unique monotherapy activity of our molecules, which provides a strong foundation for moving to combination studies."

2020 Transformation Program

In 2020, we completed a wide-ranging transformation program under a new leadership, with the following highlights:

Strategy: Reset corporate strategy by discontinuing a lead intra-tumoral cutaneous T-cell lymphoma (CTCL) program, and shifting focus toward large hematological malignancy and solid tumor indications via intravenous administration.
Clinical development: Substantially advanced TTI-622 and TTI-621 dose escalation studies, while demonstrating unique, highly differentiating monotherapy activity; positioned both programs for moving to phase 2 development in 2021.
Corporate development: Received $25 million equity investment from Pfizer, with Dr. Jeff Settleman, Pfizer Oncology Chief Scientific Officer, joining Trillium’s Scientific Advisory Board (SAB).
Governance: Appointed three new directors, including Mr. Paul Walker (partner at NEA), Dr. Mike Kamarck (CTO at Vir Biotechnology, formerly with Merck, Wyeth and Bayer), and Mr. Paolo Pucci (former CEO of ArQule), as well as Dr. Ali Behbahani (NEA partner) as a Board observer.
Leadership: Appointed new Chief Medical Officer, Ingmar Bruns, MD, PhD, a highly experienced and accomplished hematologist-oncologist and drug developer who previously held leadership roles at Pieris Pharmaceuticals and Bayer.
Advisory infrastructure: Formed a highly qualified SAB consisting of Karen Ferrante, MD; Gordon Freeman, PhD; Tom Reynolds, MD, PhD; Steven Rosen, MD; and Jeff Settleman, PhD.
Intellectual property: Solidified leading CD47 SIRPaFc patent estate by receiving a US patent for TTI-622 as a composition of matter, and (to our Licensor) a US patent for the method of using SIRPaFc fusion protein for treating CD47+ cancer including hematologic and solid tumors.
Finance & reporting: Converted functional and reporting currency from CAD to USD, transitioned from foreign private issuer to domestic filer under SEC rules, and converted reporting from IFRS to US GAAP.
Fundraising: Raised more than $300 million through two public fundraising rounds, an equity investment from Pfizer, and the exercise of warrants.
Investor base: Strengthened shareholder base, now consisting primarily of leading specialist life sciences investors.
TTI-622 (SIRPα-IgG4 Fc)

Substantially advanced TTI-622 single agent dose escalation study in relapsed or refractory lymphoma through dose levels from 4 to 18 mg/kg (currently ongoing).
Per the last data update at ASH (Free ASH Whitepaper) 2020 (data cutoff as of November 3, 2020), we reported the following TTI-622 profile:
No major safety concerns and no maximum tolerated dose (MTD) reached through 12 mg/kg dose level;
35% ORR, with six responses (including one complete response) in 17 response evaluable patients, at dose levels of 0.8-12 mg/kg (one patient assessed in 12 mg/kg cohort as of the data cutoff); and
Dose dependent increases in receptor occupancy and TTI-622 serum exposure.
TTI-621 (SIRPα-IgG1 Fc)

Progressed TTI-621 single agent dose escalation study in advanced relapsed or refractory hematologic malignancies through dose levels from 1.0 to 2.0 mg/kg (currently ongoing), though COVID-19 negatively affected the speed of patient enrollment.
As of our last data update at ASH (Free ASH Whitepaper) 2020 (data cutoff as of November 3, 2020), we reported the following TTI-621 profile:
No DLTs reached through dose level 1.4 mg/kg; transient thrombocytopenia observed, though not clinically relevant; and
Monotherapy activity observed in T-cell and B-cell lymphomas, including 17% ORR in CTCL (N=53), 18% ORR in PTCL (N=22%), and 29% ORR in DLBCL (N=9) across dose levels ranging up to 0.5 mg/kg in PTCL and DLBC, and up to 1.4 mg/kg in CTCL.
R&D Day

On April 28, 2021, we plan to hold an R&D Day, at which we will:

Provide a data update for TTI-622 and TTI-621, including data for the 18 mg/kg and 2 mg/kg dose cohorts, respectively;
Announce key strategic priorities in terms of target indications and drug combinations across hematologic malignancies and solid tumors;
Outline clinical development plan and clinical studies to be initiated in 2021.
Annual 2020 Financial Results:

Trillium began reporting its results in accordance with U.S. GAAP effective for the fiscal year ended December 31, 2020. This transition is a result of the Company no longer being classified as a foreign private issuer as defined under the rules of the SEC. As a domestic filer, the Company now prepares consolidated financial statements in accordance with U.S. GAAP, reports with the SEC on domestic forms, and complies with SEC rules and regulations applicable to domestic issuers.

As of December 31, 2020, Trillium had cash and cash equivalents and marketable securities of $291.2 million, compared to $22.7 million at December 31, 2019. The increase in cash and cash equivalents and marketable securities was due mainly to proceeds from financings completed in January 2020 and September 2020.

Net loss for the year ended December 31, 2020 of $59.3 million was higher than the loss of $38.1 million for the year ended December 31, 2019. The net loss was higher due mainly to a non-cash loss of $22.1 million on the revaluation of the deferred share unit liability (reclassified from a liability to equity effective June 30, 2020 on adoption of the new omnibus incentive plan), non-cash stock-based compensation expenses relating to the revaluation of the Company’s stock option liabilities of $12.5 million, and higher manufacturing costs. This was partially offset by lower clinical trial and salary expenses.

On March 18, 2021 PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing novel cancer therapies and infectious disease vaccines based on the Company’s proprietary Versamune T-cell activating technology, reported its financial results for the year ended December 31, 2020 and provided a business update (Press release, PDS Biotechnology, MAR 18, 2021, View Source [SID1234576850]).

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Recent Business Highlights:

Achieved preliminary efficacy benchmark in the Phase 2 combination trial of PDS0101 led by the National Cancer Institute.
Initiated VERSATILE-002, a Phase 2 trial of lead investigational drug candidate PDS0101, in combination with standard of care KEYTRUDA for first-line treatment of patients with metastatic or recurrent HPV-positive head and neck cancer.
Phase 2 trial of lead investigational drug candidate PDS0101, in combination with standard of care chemoradiotherapy for patients with advanced cervical cancer was initiated by the University of Texas MD Anderson Cancer Center.
Expanded consortium for development of PDS0203, a novel, Versamune-based second-generation COVID-19 vaccine to include Blanver Farmoquímica in addition to Farmacore.
Received award commitment of up to $60 million from Brazil’s Ministry of Science, Technology and Innovation (MCTI) to fund clinical development and commercialization of PDS0203.
Strengthened leadership team with the appointment of Seth Van Voorhees as Chief Financial Officer and addition of preeminent oncologist Otis Brawley, M.D. to the board of directors.
"Despite the challenges of 2020, the PDS Biotech team remained focused on the advancement of our Versamune-based drug pipeline," commented Dr. Frank Bedu-Addo, President and Chief Executive Officer of PDS Biotech. "Through that commitment and the strength of our partnerships with leading institutions in the fields of immuno-oncology and infectious disease, we made significant strides in solidifying the safety profile and establishing the efficacy of our Versamune platform and products as we continue to progress our portfolio towards commercialization."

Full Year 2020 Financial Results

For the year ended December 31, 2020, the net loss was approximately $14.8 million, or $0.89 per basic share and diluted share, compared to a net loss of approximately $7.0 million, or $1.44 per basic share and diluted share for the year ended December 31, 2019.

For the year ended December 31, 2020, research and development expenses increased to $7.9 million compared to $6.1 million during the prior year. The increase was primarily the result of increased expenses related to manufacturing and personnel costs for the ongoing clinical studies.

For the year ended December 31, 2020, general and administrative expenses decreased to $7.0 million compared to $11.0 million during 2019. The $4.0 million decrease was due to decreases in personnel costs of $0.4 million, non-cash stock-based compensation of $2.4 million, D&O insurance costs of $0.5 million, legal fees of $0.5 million and professional fees of $0.2 million.

Total operating expenses for 2020 were $14.9 million, a decrease of approximately 29% compared to $21.0 million during the prior year.

The company’s cash balance as of December 31, 2020 was $28.8 million.

Conference Call and Webcast

The conference call is scheduled to begin at 8:00 am ET on Thursday, March 18, 2021. Participants should dial 877-407-3088 (United States) or 201-389-0927 (International) and mention PDS Biotech. Participants can also access the conference call via webcast on the investor relations page of the Company’s corporate website (link).

The event will be archived in the investor relations section of PDS Biotech’s website for 6 months. In addition, a telephonic replay of the call will be available for 6 months. The replay can be accessed by dialing 877-660-6853 (United States) or 201-612-7415 (International) with confirmation code 13716518.

On March 18, 2021 UroGen Pharma Ltd. (Nasdaq: URGN), a biopharmaceutical company dedicated to building and commercializing novel solutions that treat specialty cancers and urologic diseases, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided an overview of the Company’s recent developments (Press release, UroGen Pharma, MAR 18, 2021, View Source [SID1234576873]).

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"2020 was a pivotal year for UroGen with the launch of our first product marking our transition from a clinical to a commercial stage company. Reflecting on our first two full quarters of our Jelmyto launch, we observed strong interest and early uptake from physicians, both in the community and hospital setting," said Liz Barrett, President and Chief Executive Officer of UroGen. "Beyond Jelmyto, we are pleased to have initiated the Phase 3 ATLAS trial for UGN-102 and expect to provide enrollment updates later this year. Additionally, we are looking forward to further realizing the potential of the RTGel platform through strategic collaborations, such as our recent immuno-oncology research agreements with leading academic centers."

Business Highlights and Upcoming Milestones:

Jelmyto (mitomycin) for pyelocalyceal solution:

Achieved net product revenue of $8.0 million for the fourth quarter of 2020. Since the June 1, 2020 launch of Jelmyto, the first and only FDA approved non-surgical treatment option for adult patients with low-grade upper tract urothelial cancer (LG-UTUC), the Company reported $11.8 million in net product revenue.
As of March 1, 2021, 250 sites have been activated, which means they have completed their internal processes and have treated or are ready to treat patients; 31 sites have treated more than one patient.
Effective January 1, 2021, the Company received a permanent and product-specific J-code for Jelmyto from the Centers for Medicare and Medicaid Services (CMS), replacing the previously issued temporary C-code and standardizing and facilitating reimbursement in the hospital outpatient, ambulatory surgery center and physician office settings of care.
UroGen presented final durability data from the Phase 3 OLYMPUS pivotal trial evaluating Jelmyto in LG-UTUC at the Annual Meeting of the Society of Urologic Oncology. Of the 58% (41/71) of patients who achieved a complete response (CR), 12-month durability of response was estimated at 81.8% by Kaplan-Meier analysis.
UGN-102 (mitomycin) for intravesical solution:

Initiated ATLAS, the Phase 3 trial of UGN-102 in the fourth quarter of 2020. The ATLAS trial expects to enroll approximately 630 patients and is exploring UGN-102 as a primary non-surgical treatment compared to standard of care – transurethral resection of bladder tumor (TURBT) – in patients diagnosed with low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
Reported top-line final data from the OPTIMA II Phase 2b trial evaluating UGN-102 in patients with LG-IR-NMIBC, showing that 65% of patients receiving UGN-102 (41/63) achieved a CR three months after the start of therapy. In this subset of patients, duration of response at 12 months following treatment initiation (nine months post CR) was estimated by Kaplan-Meier analysis to be 72.5%. The Company expects to present the full data at an upcoming medical meeting and publish in a peer-reviewed journal.
UGN-302 [UGN-201 (imiquimod) TLR 7/8 + UGN-301 (zalifrelimab) CTLA-4]:

Announced a strategic three-year collaboration agreement with The University of Texas MD Anderson Cancer Center to advance UGN-302, a combination intravesical immunotherapy, which is delivered directly into the bladder, for the treatment of high-grade non-muscle invasive bladder cancer (HG-NMIBC).
UroGen and MD Anderson expect to progress the UGN-302 program in the first half of 2021, which includes potential non-clinical studies for UGN-301 and UGN-201, as well as potential clinical studies for UGN-201.
UroGen continues to pursue additional collaborations and partnerships with leading academic institutions, biotech and pharma.
Strategic Funding:

UroGen and RTW announced a transaction totaling $75 million in funding for UroGen to support the launch of Jelmyto and the development of UGN-102.
RTW will provide UroGen with an upfront cash payment of $75 million and will receive tiered future payments based on global annual net product sales of Jelmyto and UGN-102, if approved.
Fourth Quarter and Full Year 2020 Financial Results:

Jelmyto Revenue: UroGen reported net product sales of Jelmyto for the fourth quarter ended December 31, 2020 of $8.0 million. Net product sales of Jelmyto for the year ended December 31, 2020 were $11.8 million. Jelmyto was launched on June 1, 2020.

R&D Expense: Research and development expenses for the fourth quarter ended December 31, 2020 were $12.4 million, including non-cash share-based compensation expense of $1.4 million. This compares to $20.1 million, including non-cash share-based compensation expense of $1.9 million, for the same period in 2019. Research and development expenses for the year ended December 31, 2020 were $47.3 million, including non-cash share-based compensation expense of $6.4 million. This compares to $49.3 million, including non-cash share-based compensation expense of $8.3 million, for the full year 2019.

SG&A Expense: Selling, general and administrative expenses for the fourth quarter ended December 31, 2020 were $22.2 million, including non-cash share-based compensation expense of $5.1 million. This compares to $19.7 million, including non-cash share-based compensation expense of $6.2 million, for the same period in 2019. Selling, general and administrative expenses for the year ended December 31, 2020 were $90.2 million, including non-cash share-based compensation expense of $21.6 million. This compares to $60.2 million, including non-cash share-based compensation expense of $21.7 million, for the full year 2019.

Net Loss: UroGen reported a net loss of $30.5 million, or basic and diluted net loss per ordinary share of $1.38, for the fourth quarter ended December 31, 2020. This compares to $39.0 million, or basic and diluted net loss per ordinary share of $1.86, for the same period in 2019. UroGen reported a net loss of $128.5 million, or basic and diluted net loss per ordinary share of $5.90, for the year ended December 31, 2020. This compares to $105.1 million, or basic and diluted net loss per ordinary share of $5.12, for the full year 2019.

Cash & Cash Equivalents: As of December 31, 2020, cash, cash equivalents and marketable securities totaled $103.9 million.

2021 Operating Expense Guidance: The Company anticipates operating expenses in the range of $155 to $170 million, including non-cash share-based compensation expense of $24 to $28 million, subject to market conditions.

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 View Source 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 (U.S.) or (615) 247-5916 (International) to listen to the live conference call. The conference ID number for the live call will be 9043018. An archive of the webcast will be available for two weeks on the Company’s website.

About Jelmyto

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 U.S. FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC. On April 15, 2020, the FDA approved Jelmyto, making it the first drug approved for the treatment of LG-UTUC in adult patients.

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.

JELMYTO and UroGen are registered trademarks of UroGen Pharma, Ltd.

©2021 UroGen Pharma, Inc. All rights reserved.

JEL-PT-ISI-002

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 investigational drug formulation of mitomycin in Phase 2b development for the treatment of low-grade intermediate risk non-muscle invasive bladder cancer. Utilizing the RTGel Technology Platform, UroGen’s proprietary 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. The Company reported topline interim results from the Phase 2b OPTIMA II trial in May 2020 and initiated a Phase 3 study to further investigate UGN-102 in the treatment of this condition in December 2020.

About the Phase 3 ATLAS Trial

ATLAS is an global, open-label, randomized controlled Phase 3 trial designed to assess the efficacy and safety of UGN-102, with or without transurethral resection of bladder tumor (TURBT), versus TURBT alone in patients diagnosed with low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC), defined as 1 or 2 of the following: new or recurrent multifocal bladder tumors, a solitary new or recurrent tumor >3 cm, or LG-IR-NMIBC recurrence in less than 12 months following a prior tumor diagnosis requiring endoscopic surgical resection or ablation. The trial is anticipated to enroll approximately 630 patients in over 100 clinical sites in the U.S., Europe and Israel.

Patients will be randomized 1:1 to either UGN-102 or TURBT. Patients in the UGN-102 arm will be treated with six weekly intravesical instillations of UGN-102. At the 3-month time point, patients will be assessed for response. Patients who have demonstrated a complete response to either UGN-102 or TURBT, will continue for long-term follow-up for evidence of recurrence. Patients who demonstrate presence of persistent disease at 3-months, in either arm, will undergo a TURBT and then will also continue for long-term follow up for evidence of recurrence. The primary endpoint of the study is disease free survival.