Jazz Pharmaceuticals Announces Statistically Significant Overall Survival and Progression-Free Survival Results for Zepzelca® (lurbinectedin) and Atezolizumab Combination in First-Line Maintenance Therapy for Extensive-Stage Small Cell Lung Cancer

On October 15, 2024 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported positive top-line results from the Phase 3 clinical trial evaluating Zepzelca (lurbinectedin) in combination with the PD-L1 inhibitor atezolizumab (Tecentriq) compared to atezolizumab alone when administered as a maintenance treatment for adults with extensive-stage small cell lung cancer (ES-SCLC) following induction therapy with carboplatin, etoposide and atezolizumab (Press release, Jazz Pharmaceuticals, OCT 15, 2024, View Source [SID1234647211]). The combination of Zepzelca and atezolizumab demonstrated a statistically significant improvement in the primary endpoints of overall survival (OS) and progression-free survival (PFS), as assessed by an independent review facility (IRF), compared to treatment with atezolizumab alone.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Each year, approximately 30,000 new cases of small cell lung cancer (SCLC) are reported in the U.S. A majority of these patients are diagnosed with extensive stage disease, which is aggressive and often difficult to treat, with poor prognosis,i,ii,iii" said Luis Paz-Ares, M.D., Ph.D., head of medical oncology at the Hospital Universitario 12 de Octubre in Madrid, Spain, and IMforte trial principal investigator. "These trial results demonstrate the efficacy of lurbinectedin, the most widely used agent in second-line SCLC in the United States, in combination with standard-of-care atezolizumab for patients in first-line maintenance treatment, a much-needed advancement for patients with extensive disease."

"The results of the Phase 3 IMforte trial are highly encouraging and showed a statistically significant benefit for the Zepzelca and atezolizumab combination for extensive-stage small cell lung cancer patients receiving this treatment in the first-line maintenance setting. These results demonstrate the potential of this regimen to delay disease progression and extend survival for patients with this aggressive disease," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "We are pleased with these clinically meaningful results and plan to submit an sNDA in the first half of 2025 to support this combination in the first-line maintenance setting. We thank the investigators and patients who are involved in this trial, along with our partners at Roche."

The combination was generally well-tolerated. The preliminary safety data in the ongoing trial was consistent with the known safety profiles of Zepzelca and atezolizumab with no new safety signals observed in the combination arm.

Jazz and Roche plan to submit these data for presentation at a future medical meeting.

About the IMforte Phase 3 Trial
IMforte (NCT05091567) is an ongoing Phase 3, randomized, multicenter maintenance trial evaluating the efficacy, safety and pharmacokinetics of Zepzelca plus atezolizumab, compared with standard-of-care first-line maintenance with atezolizumab alone, in adults (≥18 years) with ES-SCLC, following induction therapy with carboplatin, etoposide and atezolizumab. The primary endpoints for this study are OS and IRF-assessed PFS.

The trial consists of two phases: an induction phase and a maintenance phase. Participants were required to have an ongoing response or stable disease per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 after the induction phase of four cycles of carboplatin, etoposide, and atezolizumab to be considered for eligibility screening for the maintenance phase. Eligible participants were randomized in a 1:1 ratio to receive either lurbinectedin plus atezolizumab or atezolizumab in the maintenance phase.

The trial is sponsored by Roche and co-funded by Jazz Pharmaceuticals. Additional information about the trial, including eligibility criteria and a list of clinical trial sites, can be found at: View Source (ClinicalTrials.gov Identifier: NCT05091567).

About Small Cell Lung Cancer
In the U.S., approximately 13 percent of lung cancers are small cell.ii Approximately 30,000 new cases of small cell lung cancer (SCLC) are reported in the U.S. each year.ii,iii The risk for developing SCLC is much higher among current or former tobacco smokers; however, SCLC can also be caused by exposure to secondhand smoke, asbestos, some inhaled chemicals, radiation and air pollution. People with a family history of lung cancer may also be at a higher risk, too.iv SCLC is the most aggressive form of lung cancer and it tends to spread quickly to other parts of the body including the brain, liver and bone.v,vi A large percentage of SCLC patients on treatment briefly achieve a response, although the cancer often returns and is usually more aggressive and resistant to regimens that were previously effective.ii

About Zepzelca (lurbinectedin)
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and eventual cell death.iv

The FDA approved Zepzelca under accelerated approval in June 2020 for the treatment of adult patients with metastatic SCLC with disease progression on or after platinum-based chemotherapy. The approval is based on overall response rate (ORR) and duration of response demonstrated in an open-label, monotherapy clinical study. In December 2021, Jazz and PharmaMar announced the initiation of LAGOON, a confirmatory Phase 3 clinical trial of Zepzelca for the treatment of patients with relapsed small cell lung cancer. If positive, LAGOON could confirm the benefit of Zepzelca in the treatment of small cell lung cancer (SCLC) when patients progress following 1L treatment with a platinum-based regimen and support full approval in the U.S.

Zepzelca is a prescription medicine used to treat adults with SCLC that has spread to other parts of the body (metastatic) and who have received treatment with chemotherapy that contains platinum, and it did not work or is no longer working. Zepzelca is approved based on response rate and how long the response lasted.

Important Safety Information for ZEPZELCA

Before receiving ZEPZELCA, tell your healthcare provider about all of your medical conditions, including if you:

have liver or kidney problems.
are pregnant or plan to become pregnant. ZEPZELCA can harm your unborn baby.
Females who are able to become pregnant:

Your healthcare provider should do a pregnancy test before you start treatment with ZEPZELCA.
You should use effective birth control (contraception) during treatment with and for 6 months after your last dose of ZEPZELCA.
Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with ZEPZELCA.
Males with female partners who are able to become pregnant should use effective birth control during treatment with and for 4 months after their last dose of ZEPZELCA.

are breastfeeding or plan to breastfeed. It is not known if ZEPZELCA passes into your breastmilk. Do not breastfeed during treatment with ZEPZELCA and for 2 weeks after your last dose of ZEPZELCA. Talk to your healthcare provider about the best way to feed your baby during treatment with ZEPZELCA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Certain other medicines may affect how ZEPZELCA works.
What should I avoid while using ZEPZELCA?
Avoid eating or drinking grapefruit, Seville oranges, or products that contain grapefruit juice and Seville oranges during treatment with ZEPZELCA.
ZEPZELCA can cause serious side effects, including:

Low blood cell counts. Low blood counts including low neutrophil counts (neutropenia) and low platelet counts (thrombocytopenia) are common with ZEPZELCA, and can also be severe. Some people with low white blood cell counts may get fever, or an infection throughout the body (sepsis), that can cause death. Your healthcare provider should do blood tests before you receive each treatment with ZEPZELCA to check your blood cell counts.
Tell your healthcare provider right away if you develop:
fever or any other signs of infection
unusual bruising or bleeding
tiredness
pale colored skin
Liver problems. Increased liver function tests are common with ZEPZELCA and can also be severe. Your healthcare provider should do blood tests to check your liver function before you start and during treatment with ZEPZELCA.
Tell your healthcare provider right away if you develop symptoms of liver problems including:
loss of appetite
nausea or vomiting
pain on the right side of your stomach area (abdomen)
Leakage of ZEPZELCA out of your vein during the infusion. If ZEPZELCA leaks into the tissues around your infusion site, it can cause damage and death of tissue cells around the infusion site. You may need to have surgery to remove any dead tissue. Tell your healthcare provider right away if you see any ZEPZELCA leaking out of your vein or around the catheter during your infusion, or if you notice any redness, swelling, itching or discomfort at the infusion site at any time.
Severe muscle problems (rhabdomyolysis). Tell your healthcare provider if you have severe muscle pain or weakness.
Your healthcare provider may temporarily stop treatment, lower your dose, or permanently stop ZEPZELCA if you develop serious side effects during treatment with ZEPZELCA.

The most common side effects of ZEPZELCA include:

tiredness
low white and red blood cell counts
increased kidney function blood test (creatinine)
increased liver function blood tests
increased blood sugar (glucose)
nausea
decreased appetite
muscle and joint (musculoskeletal) pain
low level of albumin in the blood
constipation
trouble breathing
low levels of sodium and magnesium in the blood
vomiting
cough
diarrhea
These are not all of the possible side effects of ZEPZELCA.

Call your doctor for medical advice about side effects. 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 Jazz Pharmaceuticals at 1-800-520-5568.

Please see full Prescribing Information including Patient Information, and discuss with your doctor.

ZEPZELCA is a trademark of Pharma Mar, S.A. used by Jazz Pharmaceuticals under license.

Tecentriq (atezolizumab) is a registered trademark of Genentech, a member of the Roche Group.

What is atezolizumab?
Atezolizumab is a prescription medicine used to treat:

Adults with a type of lung cancer called small cell lung cancer (SCLC). Atezolizumab may be used with the chemotherapy medicines carboplatin and etoposide as your first treatment when your lung cancer:

is a type called "extensive-stage small cell lung cancer," which means that it has spread or grown.
It is not known if atezolizumab is safe and effective when used in children for the treatment of SCLC.

Important Safety Information

What is the most important information about atezolizumab?
Atezolizumab can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. You can have more than one of these problems at the same time. These problems may happen anytime during your treatment or even after your treatment has ended.

Call or see your healthcare provider right away if you develop any new or worse signs or symptoms, including:

Lung problems

cough
shortness of breath
chest pain
Intestinal problems

diarrhea (loose stools) or more frequent bowel movements than usual
stools that are black, tarry, sticky, or have blood or mucus
severe stomach-area (abdomen) pain or tenderness
Liver problems

yellowing of your skin or the whites of your eyes
severe nausea or vomiting
pain on the right side of your stomach area (abdomen)
dark urine (tea colored)
bleeding or bruising more easily than normal
Hormone gland problems

headaches that will not go away or unusual headaches
eye sensitivity to light
eye problems
rapid heartbeat
increased sweating
extreme tiredness
weight gain or weight loss
feeling more hungry or thirsty than usual
urinating more often than usual
hair loss
feeling cold
constipation
your voice gets deeper
dizziness or fainting
changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness
Kidney problems

decrease in your amount of urine
blood in your urine
swelling of your ankles
loss of appetite
Skin problems

rash
itching
skin blistering or peeling
painful sores or ulcers in mouth or nose, throat, or genital area
fever or flu-like symptoms
swollen lymph nodes
Problems can also happen in other organs.
These are not all of the signs and symptoms of immune system problems that can happen with atezolizumab. Call or see your healthcare provider right away for any new or worse signs or symptoms, including:

Chest pain, irregular heartbeat, shortness of breath, or swelling of ankles
Confusion, sleepiness, memory problems, changes in mood or behavior, stiff neck, balance problems, tingling or numbness of the arms or legs
Double vision, blurry vision, sensitivity to light, eye pain, changes in eyesight
Persistent or severe muscle pain or weakness, muscle cramps
Low red blood cells, bruising
Infusion reactions that can sometimes be severe or life-threatening. Signs and symptoms of infusion reactions may include:

chills or shaking
itching or rash
flushing
shortness of breath or wheezing
dizziness
feeling like passing out
fever
back or neck pain
Complications, including graft-versus-host disease (GVHD), in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if you underwent transplantation either before or after being treated with atezolizumab. Your healthcare provider will monitor you for these complications.

Getting medical treatment right away may help keep these problems from becoming more serious. Your healthcare provider will check you for these problems during your treatment with atezolizumab. Your healthcare provider may treat you with corticosteroid or hormone replacement medicines. Your healthcare provider may also need to delay or completely stop treatment with atezolizumab if you have severe side effects.

Before you receive atezolizumab, tell your healthcare provider about all of your medical conditions, including if you:

have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus
have received an organ transplant
have received or plan to receive a stem cell transplant that uses donor stem cells (allogeneic)
have received radiation treatment to your chest area
have a condition that affects your nervous system, such as myasthenia gravis or Guillain-Barré syndrome
are pregnant or plan to become pregnant. Atezolizumab can harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with atezolizumab. Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with atezolizumab.
You should use an effective method of birth control during your treatment and for at least 5 months after the last dose of atezolizumab.
are breastfeeding or plan to breastfeed. It is not known if atezolizumab passes into your breast milk. Do not breastfeed during treatment and for at least 5 months after the last dose of atezolizumab.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of atezolizumab when used in lung cancer with other anti-cancer medicines include:

feeling tired or weak
nausea
hair loss
constipation
diarrhea
decreased appetite
Atezolizumab may cause fertility problems in females, which may affect the ability to have children. Talk to your healthcare provider if you have concerns about fertility.

These are not all the possible side effects of atezolizumab. Ask your healthcare provider or pharmacist for more information about the benefits and side effects of atezolizumab.

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

Anixa Biosciences Announces Second Dose Administered to Patient in Ovarian Cancer CAR-T Clinical Trial

On October 15, 2024 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that it has administered the second dose of its CAR-T therapy to an individual patient (Press release, Anixa Biosciences, OCT 15, 2024, View Source [SID1234647210]). This follows a positive response after the initial treatment in the ongoing Phase 1 clinical trial of its chimeric antigen receptor T-cell (CAR-T) therapy for ovarian cancer. The study is being conducted in collaboration with Moffitt Cancer Center.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Previously, Anixa and Moffitt received approval for a single-patient IND application, allowing the administration of a second dose to a patient whose tumor biopsy revealed cellular infiltration and necrosis, indicating biological activity of the CAR-T therapy.

After the first infusion, the patient remained stable, did not require alternative treatment and her quality of life was good, leading to the decision to administer a second dose to further enhance these positive results. These findings offer promising indications that the CAR-T therapy may serve as a successful long-term treatment option, highlighting its potential effectiveness in the fight against ovarian cancer.

"While this is a single patient, the positive clinical activity observed, including necrosis and T cell infiltration, is an encouraging sign of the therapy’s potential effectiveness," stated Dr. Robert Wenham, Chair of the Department of Gynecologic Oncology at Moffitt and the principal investigator of the trial. "Based on these results, we recently submitted an amendment to the current trial protocol to allow patients who may benefit to receive a second dose. We are excited to continue evaluating this treatment in our ongoing trial, and we are optimistic about the potential long-term benefits it may offer to patients with ovarian cancer."

Dr. Monica Avila, the patient’s treating oncologist, stated, "My patient received her first infusion in May 2023. We are now nearly 18 months from that date, and she is doing well and is now receiving this second dose. I am thrilled with her status and look forward to observing further progress."

Dr. Amit Kumar, CEO of Anixa Biosciences, stated, "We are proud to have an outstanding team, including Dr. Wenham and Dr. Avila, and we are encouraged by the positive response seen in this patient following the initial dose of our CAR-T therapy. The continued clinical improvements reinforce our confidence in the potential of this groundbreaking treatment to provide hope for patients battling ovarian cancer. We remain committed to advancing this innovative therapy and bringing new, effective treatment options to the forefront."

The Phase 1 clinical trial at Moffitt is treating recurrent ovarian cancer patients who have failed standard-of-care therapies. To date, six patients have been treated in the dose escalation trial, three in the first cohort and three in the second cohort. Dose escalation will continue after confirming the previous dosages are safe.

AtomVie Global Radiopharma and Radiopharm Theranostics Partner to Develop and Manufacture 177Lu-BetaBart Radioantibody for Treatment of Multiple Solid Tumors

On October 15, 2024 AtomVie Global Radiopharma (AtomVie), a leading radiopharmaceutical Contract Development and Manufacturing Organization (CDMO), reported to have entered into an agreement with Radiopharm Ventures (RV), a Joint Venture between Radiopharm Theranostics (RAD) and MD Anderson Cancer Center (MDACC), to develop and manufacture 177Lu-BetaBart, a 177Lutetium-conjugated B7-H3 targeting radioantibody (Press release, Radiopharm Theranostics, OCT 15, 2024, View Source [SID1234647209]). This partnership leverages both companies’ expertise to advance novel radiotherapeutic solutions in areas of high unmet medical needs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

B7-H3 is an immune checkpoint molecule that is overexpressed in several tumor types and represents a highly attractive target for antibody-based cancer immunotherapy. Deregulated B7-H3 expression is linked with tumor aggressiveness and poor outcomes. 177Lu-BetaBart is the first targeted radiopharmaceutical in development against the 4Ig subtype of B7-H3, which is the most common subtype expressed on human tumors. The monoclonal antibody, invented at MDACC, has been specifically engineered with a shorter blood circulation time and reduced affinity for on-target off-tissue toxicity, leading to a final molecule that is highly promising for human use in clinical settings. Phase I/II First-In-Human therapeutic trial with 177Lu-BetaBart in multiple tumor types in the US, is expected for mid-2025

This collaboration brings together RAD’s world-class platform of radiotherapeutic products and AtomVie’s leading in manufacturing and distributing radiopharmaceuticals globally. 177Lu-BetaBart is part of a broader pipeline of distinct, highly differentiated technologies developed by RAD and RV. These span peptides, small molecules, and monoclonal antibodies for use in cancer, sourced from top-tier universities and institutes globally. The pipeline is designed with the strong potential to be either first-to-market or best-in-class.

AtomVie’s extensive expertise in clinical development, including technology transfer, process and method development, clinical supply and global distribution, provides a reliable foundation for advancing 177Lu-BetaBart from the clinic towards commercialization. With its new state-of-the-art, scalable 72,300 sq ft facility, set to open in early 2025, AtomVie is the partner of choice to support the growing global pipeline of radiotherapeutics.

"Our collaboration with AtomVie is a significant step forward in our mission to bring innovative radiopharmaceutical therapies to patients," said Riccardo Canevari, Managing Director & CEO of RAD. "Their proven track record in manufacturing and global distribution assures us that we are in capable hands as we progress through the clinical stages and prepare for potential commercialization."

Bruno Paquin, CEO of AtomVie, commented, "We are thrilled to partner with RAD on such an important project. With our expertise in radiopharmaceutical manufacturing, we are confident that we can support RAD in advancing their innovative pipeline. This partnership reinforces our commitment to transforming patients’ lives with high-quality radiopharmaceuticals, as we continue to expand our capabilities in our new facility and empower novel radiotherapeutics to market."

TRIANA Biomedicines and Pfizer Enter Into A Research Collaboration to Discover Novel Molecular Glue Degraders for Multiple Disease Areas

On October 15, 2024 TRIANA Biomedicines, Inc. (TRIANA), a leading biopharmaceutical company focused on building a target-first molecular glue discovery pipeline for inactivating difficult to address disease targets, reported that it has entered into a strategic collaboration and licensing agreement with Pfizer to discover novel molecular glue degraders for multiple targets in several disease areas, including oncology (Press release, Triana Biomedicines, OCT 15, 2024, View Source [SID1234647208]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are thrilled to partner with Pfizer to create potentially transformative medicines for disease targets with critical unmet needs, leveraging our molecular glue and E3-ligase pairing platform" said Patrick Trojer, Ph.D., President and Chief Executive Officer of TRIANA Biomedicines. "This collaboration agreement signifies an important milestone in the evolution of TRIANA, as the company advances towards delivering on its product focused strategy."

"This collaboration with TRIANA Biomedicines on molecular glue discovery reflects our commitment to exploring cutting-edge technologies to drive the next wave of potential breakthroughs," said Jeff Settleman, Ph.D., Chief Scientific Officer of Pfizer Oncology. "We look forward to working together to advance scientific innovation for patients living with cancer."

Under the terms of the collaboration agreement, TRIANA will receive an upfront payment of $49 million, and is eligible to receive potential future milestone payments exceeding $1.5 billion as well as tiered royalties. TRIANA will leverage its target-first and proximity-first molecular glue discovery platform to identify novel molecular glue degraders against multiple targets across various disease areas including oncology.

TRIANA will lead the discovery and identification of potential development candidates. Pfizer has the exclusive option for an exclusive license to pursue further preclinical and clinical development.

UroGen Announces FDA Acceptance of its New Drug Application for UGN-102

On October 15, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported U.S. Food and Drug Administration (FDA) acceptance of the New Drug Application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution (Press release, UroGen Pharma, OCT 15, 2024, View Source [SID1234647207]). UGN-102 could become the first FDA-approved medicine for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The FDA acceptance of our NDA is a pivotal moment in our journey to bring UGN-102 to patients," said Liz Barrett, President and Chief Executive Officer of UroGen. "UGN-102 could be the first FDA-approved medicine for LG-IR-NMIBC, offering a novel approach that could expand treatment options and address unmet needs. There is an urgent need for innovative solutions in this space, and we are dedicated to collaborating with the FDA as we prepare for a potential launch of UGN-102 in 2025."

Dr. Mark Schoenberg, Chief Medical Officer of UroGen, stated, "The NDA for UGN-102 is backed by a robust data set demonstrating impressive durability of response across three clinical trials and a favorable safety profile. Notably, the ENVISION trial successfully met its primary endpoint, showing a 79.6% complete response rate at three months after the first instillation of UGN-102. Additionally, the latest results from that trial revealed an 82.3% 12-month duration of response by Kaplan-Meier estimate in patients who achieved a complete response at 3 months. The most common treatment-emergent adverse events in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. Additionally, the safety profile observed in the ENVISION trial was consistent with that seen in other studies of UGN-102. We believe that, if approved, UGN-102’s ability to achieve durable complete responses and potentially reduce recurrence rates while extending treatment-free intervals will represent a significant advance in managing LG-IR-NMIBC."

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently under regulatory review for approval in 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. An NDA for UGN-102 is currently under review by the FDA with a potential decision expected by June 13, 2025. The U.S. market for LG-IR-NMIBC that UGN-102 can address, if approved, is valued at approximately $5 billion.

About ENVISION

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as primary chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

About Non-Muscle Invasive Bladder Cancer (NMIBC)

In the U.S. bladder cancer is the second most common urologic cancer in men. LG-IR-NMIBC represents approximately 22,000 newly diagnosed bladder cancer patients each year and an estimated 60,000 recurrences annually among patients diagnosed from previous years. Bladder cancer primarily affects older populations with the median age of diagnosis 73 years and an increased risk of comorbidities. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.