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UroGen Announces Outcome of Oncologic Drugs Advisory Committee for UGN-102 for the Treatment of Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (LG-IR-NMIBC)

On May 21, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a leading biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported the outcome of today’s meeting of the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA), which discussed the new drug application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution (Press release, UroGen Pharma, MAY 21, 2025, View Source [SID1234653276]). By a narrow margin, the ODAC voted 4 to 5 that the benefit/risk of UGN-102 (mitomycin) for intravesical solution was favorable for the treatment of recurrent LG-IR-NMIBC.

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"While we are disappointed by today’s outcome, we continue to believe our clinical data support UGN-102 for the treatment of recurrent LG-IR-NMIBC, a disease with no FDA-approved therapies," said Liz Barrett, President and CEO of UroGen. "The FDA carefully considers the independent advice from ODAC, and we look forward to working with the FDA as they complete their review of the application for UGN-102."

The ODAC reviewed the body of clinical data supporting the efficacy and safety of UGN-102, including the results from the Phase 3 ENVISION study. "Low-grade intermediate-risk non-muscle invasive bladder cancer is a highly recurrent disease and often requires patients – many of whom are elderly – to undergo repeat surgeries under general anesthesia. This is a disease with high unmet needs, and we believe patients deserve more options," said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. "UroGen remains committed to developing innovative treatment options to people living with recurrent LG-IR-NIMBC."

The most common treatment-emergent adverse events in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention, which are typically manageable in routine urologic practice. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.

The NDA for UGN-102 is currently under review by the FDA with a Prescription Drug User Fee Act (PDUFA) date of June 13, 2025.

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 recurrent 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. UroGen completed the submission of the rolling NDA for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA target action date of June 13, 2025.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include trans-urethral resection of bladder tumor (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. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.

About ENVISION

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as a chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at three months 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).

Purple Biotech Reports First Quarter 2025 Financial Results

On May 21, 2025 Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that seek to overcome tumor immune evasion and drug resistance, reported financial results for the three months ended March 31, 2025 (Press release, Purple Biotech, MAY 21, 2025, View Source [SID1234653275]).

"We are pleased to report continued progress on our milestones, including additional confirmatory biomarker data from our randomized Phase 2 study of CM24 and the collaboration for our upcoming NT219 plus cetuximab or pembrolizumab Phase 2 trial in head and neck cancer. The latest data presentations for these two important assets at the recent American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting provide further evidence for, and increase our confidence in, patient selection and potential positive outcomes," stated Purple Biotech CEO Gil Efron. "In parallel, our promising CAPTN-3 T-cells and NK cells engagers platform continues to generate compelling pre-clinical data, and we look forward to presenting these data on our tri-body technology at an upcoming scientific conference. We are advancing our pipeline while prudently managing expenses, with a cash runway through mid-2026."

Recent Clinical & Corporate Highlights:

● Final Phase 2 data for CM24 study presented at AACR (Free AACR Whitepaper) Annual Meeting 2025

● Statistically significant efficacy in biomarker subgroup analyses was observed:

● 78% reduction in risk of death and 81% reduction in risk of progression or death in defined pretreatment ranges of serum or tumor CEACAM1 subgroup

● 61% reduction in risk of death and 72% reduction in risk of progression or death in defined pretreatment ranges of serum CEACAM1 or myeloperoxidase (MPO) subgroup

● 90% reduction in risk of death and 81% reduction in risk of progression or death in high tumor CEACAM1 and low PD-L1 combined positive score (CPS) subgroup

● The biomarkers identified in the CM24 Phase 2 study are planned to be used for patient selection in the Phase 2b study, planned to be initiated in the second half of 2025

Purple Biotech reported final results from its randomized Phase 2 study of CM24 in pancreatic ductal adenocarcinoma patients in a poster presentation titled "Final analysis of the randomized Phase 2 cohort of CM24 with nivolumab and chemotherapy in pancreatic cancer & potential serum biomarkers" at the AACR (Free AACR Whitepaper) Annual Meeting 2025 held in April 2025. Statistically significant efficacy in biomarker subgroup analyses was observed. These findings support the patient selection strategy for the Phase 2b study of CM24, which is planned to be initiated in the second half of 2025.

● NT219 Phase 2 Head and Neck Cancer trial to evaluate NT219 with standard-of-care drugs. NT219 to be combined with pembrolizumab (Keytruda) or cetuximab (Erbitux)

● New positive head and neck cancer data presented at AACR (Free AACR Whitepaper)

● NT219’s suppression of brain metastasis in colorectal cancer published in Neuro Oncology

● Patent in U.S. enhances global IP protection for commercialization

A Phase 2 study of NT219 for the treatment of recurrent/metastatic squamous cell carcinoma of the head and neck (R/M SCCHN) will combine NT219 with the standard-of-care anti-PD1 checkpoint inhibitor pembrolizumab (Keytruda) or with the epidermal growth factor receptor (EGFR) blocker cetuximab (Erbitux). The study will also explore potential biomarkers that were previously identified in a prior NT219 study. The investigator-initiated Phase 2 trial is led by Dr. Antonio Jimeno, Professor and Director of the Head and Neck Cancer Program at the University of Colorado Anschutz Medical Campus.

Two posters reporting new positive NT219 data were presented at AACR (Free AACR Whitepaper) Annual Meeting 2025. The poster titled "NT219 overcomes immune evasion mechanisms in head and neck squamous cell carcinoma (HNSCC)" demonstrated that NT219 inhibits major targets and signaling pathways that play a key role in tumor immune evasion, including STAT3 and IRS-to-β-catenin pathways. In a clinical setting, upregulation of pIGF1R and pSTAT3 were correlated with patient response and suggested as potential biomarkers for NT219 treatment. These and other findings demonstrated the potential of NT219 to restore the efficacy of immunotherapies and expand the patient population that can benefit from these drugs. The poster titled "APC-loss as a potential biomarker for NT219 treatment in colorectal cancer" suggested that the response to NT219 is associated with enhanced wnt/β-catenin signaling or loss of function mutation of its negative regulator APC (APC-loss).

An independent study of NT219 titled "IRS2 as a driver of brain metastasis in colorectal cancer: a potential target for novel therapeutic strategies" was published in the peer reviewed journal Neuro Oncology. The findings demonstrate that a combination therapy of NT219 and 5-fluorouracil (5-FU) inhibits colorectal cancer brain metastasis through the IRS2 pathway. The research conducted by Prof. Ido Wolf, Dr. Tami Rubinek, and their team at Tel Aviv University and Sourasky Medical Center found that IRS2, a novel target of NT219, is a driver of brain metastasis in colorectal cancer.

The U.S. Patent and Trademark Office issued a patent for NT219 used in combination with EGFR antibodies for treating cancer patients who have acquired resistance to EGFR therapies. This latest U.S. patent completes the geographic patent protection for NT219 used in combination with cetuximab in major markets, such as the United States, Europe, China and Japan, and we believe this additional patent positions the Company well for the potential future commercialization of NT219.

● Pre-clinical research collaboration with Icahn School of Medicine at Mount Sinai for CAPTN-3 tri-specific antibody platform

Purple Biotech entered into a Research Collaboration Agreement with the Icahn School of Medicine at Mount Sinai in New York to explore the immunoregulation of NK and T cells within the tumor microenvironment by CAPTN-3 multi-specific engagers, designed with the purpose of enhancing tumor-specific immunity against various cancer types. This collaboration offers an opportunity to deepen the understanding of tumor immune evasion mechanisms that CAPTN-3 uniquely addresses, with the goal of paving the way for effective treatments for many challenging tumor indications. Purple Biotech is working with Principal Investigator Amir Horowitz, PhD, and his team at Mount Sinai to validate the unique aspects of the CAPTN-3 design in a wide screen of patient-derived tumors, potentially providing new insights for overcoming resistance to standard frontline immunotherapies.

● Strengthened the management team with the appointment of Shai Lankry as Chief Financial Officer

Financial Results for the Three Months Ended March 31, 2025

Research and Development Expenses were $0.8 million for the three months ended March 31, 2025, reflecting a decrease of $2.6 million, or 76.5%, from $3.4 million in the same period of 2024. The reduction was primarily due to reduced clinical trial related expenses.

General and Administrative Expenses were $0.6 million for the three months ended March 31, 2025, compared to $1 million in the same period of 2024, representing a decrease of $0.4 million, mainly due to a $0.3 million reduction in cash and non-cash salaries and related expenses.

Operating Loss was $1.4 million for the three months ended March 31, 2025, a decrease of $3.1 million, or 68.9%, compared to $4.5 million in the same period of 2024, mainly due to the decrease in research and development expenses.

Adjusted Operating Loss (as reconciled below) was $1.3 million for the three months ended March 31, 2025, a decrease of $2.9 million, compared to $4.2 million in the same period of 2024, primarily due to the decrease in research and development expenses.

Finance Income, net was $1.0 million for the three months ended March 31, 2025, compared to $0.7 million in the same period of 2024, representing an increase of $0.3 million, primarily attributable to a non-cash gain resulting from the revaluation of outstanding warrants.

Net Loss was $0.5 million, or $0.17 per basic and diluted ADS for the three months ended March 31, 2025, compared to a net loss of $3.8 million, or $2.8 per basic and diluted ADS, in the same period of 2024. The decrease in net loss was mainly due to the $3.1 million decrease in operating expenses and $0.3 million increase in finance income, net.

As of March 31, 2025, Purple Biotech had cash and cash equivalents and short-term deposits of $6.7 million. This cash position provides a cash runway into mid 2026.

During the three months ended March 31, 2025, the Company sold, under the Open Market Sale AgreementSM with Jefferies LLC, approximately 63 thousand ADSs, at an average price of $3.5 per ADS. Net proceeds to the Company were approximately $166 thousand, net of issuance expenses.

PharmaMar has submitted EU marketing authorization application for lurbinectedin in combination with atezolizumabin first-line maintenance therapy for extensive-stage small cell lung cancer

On May 21, 2025 PharmaMar (MSE:PHM) reported it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Zepzelca (lurbinectedin) in combination with atezolizumab (Tecentriq) for the maintenance treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC), whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin and etoposide (Press release, PharmaMar, MAY 21, 2025, View Source [SID1234653274]).

The MAA submission is based on the statistically significant and clinically meaningfulresults from the pivotal Phase 3 IMforte trial. The data of this study will be shared as an oral presentation "Lurbinectedin (lurbi) + atezolizumab (atezo) as first-line (1L) maintenance treatment (tx) in patients (pts) with extensive-stage small cell lung cancer (ES-SCLC): Primary results of the Phase 3 IMforte trial" at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), ASCO (Free ASCO Whitepaper) 2025, on June 2nd.

Orionis Biosciences Announces Strategic Partnership with Genentech to Discover and Develop Molecular Glue Class Medicines for Cancer

On May 21, 2025 Orionis Biosciences, a privately held, clinical-stage life sciences company focused on the rational discovery and development of drug modalities with induced proximity mode of action, reported a second multi-year collaboration with Genentech, a member of the Roche Group, to discover small-molecule monovalent glue medicines for novel and challenging targets in oncology (Press release, Orionis Biosciences, MAY 21, 2025, View Source [SID1234653273]). Orionis announced its first collaboration with Genentech, to discover novel small-molecule medicines for challenging targets in major disease areas, including oncology and neurodegeneration, in September 2023.

Orionis’s small-molecule Allo-Glue platform integrates multiple proprietary technologies to advance the
discovery and design of molecular glues for highly challenging disease targets. It combines advanced
chemical biology tools, engineered cellular assay systems, custom-built high-throughput robotic
automation and specialized artificial intelligence infrastructure, and enables the generation and analysis of hundreds of millions of in-cell protein interaction events. The AI-driven chemistry stack integrates
predictive modeling, generative design, and a compound interrogation engine operating across a vast
chemical space. Collectively, these capabilities accelerate the systematic discovery and simultaneous
optimization for potency, selectivity, and synthetic accessibility of monovalent molecular glue modalities.

Under the terms of the agreement, Orionis will be responsible for the discovery and optimization of
molecular glues, while Genentech will be responsible for subsequent later-stage preclinical and clinical
development, regulatory filing, and commercialization of such small molecules. Orionis will receive an
upfront payment of $105 million and is eligible for potential research, development, commercial and net
sales milestone payments, whose total could exceed $2 billion as well as potential tiered royalties upon sale of collaboration products.

"We are thrilled about this second collaboration with Genentech, which expands our collaborative efforts
in applying induced proximity concepts to include molecular glue types beyond targeted protein degraders," said Niko Kley, Co-Founder and CEO of Orionis Biosciences. "The expansion of our existing strategic alliance underlines a strong alignment in vision for molecular glues, and the excellent work by both teams during the past year".

"By integrating advanced cellular sensing technologies, large-scale robotic automation and specialized
artificial intelligence, at Orionis we have built a unique capability for discovery of new drug modalities to
rationally reprogram protein networks," said Riccardo Sabatini, Orionis Chief Data Scientist. "It’s an
exciting time in small-molecule drug development".

"Molecular glues, including protein degraders and non-degraders, are an exciting therapeutic modality,
providing access to disease-related proteins that have proven challenging and elusive to more traditional
treatment modalities," said Boris L. Zaïtra, Head of Roche Corporate Business Development. "This new collaboration with Orionis has the potential to significantly enhance our innovation to bring transformative cancer medicines to patients".

Olema Oncology to Participate in Upcoming Investor Conferences

On May 21, 2025 Olema Pharmaceuticals, Inc. ("Olema" or "Olema Oncology", Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, reported that the Company will participate in the following upcoming investor conferences (Press release, Olema Oncology, MAY 21, 2025, View Source [SID1234653272]):

Jefferies Global Healthcare Conference
Date and Time: Wednesday, June 4, 2025 at 11:40 a.m. ET
Format: Fireside Chat
Location: New York, NY

Goldman Sachs 46th Annual Global Healthcare Conference
Date and Time: Wednesday, June 11, 2025 at 8 a.m. ET
Format: Fireside Chat
Location: Miami, FL

Live webcasts and recordings of these presentations will be available, as permitted by the event host, in the Events and Presentations section of Olema’s investor relations website at ir.olema.com.