On June 2, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported results from the ENVISION and ATLAS clinical studies exploring investigational therapy UGN-102 (mitomycin) for intravesical solution for the treatment for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (Press release, UroGen Pharma, JUN 2, 2025, View Source [SID1234653626]). The data are featured at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL.

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UGN-102 data featured at ASCO (Free ASCO Whitepaper):

Duration of Response (DOR) Following Treatment with UGN-102 in Patients with Recurrent, Low-Grade, Intermediate-Risk, Non-Muscle Invasive, Bladder Cancer: 18-Month DOR Data from the Phase 3 ENVISION Trial: Abstract #4598, Poster #398
Impact of Tumor Burden or Focality in Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer on Response to Treatment with UGN-102: A Substudy of the Phase 3 ENVISION Trial: Abstract #4597, Poster #397
Treatment of Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer with UGN-102: Results of the Phase 3 ATLAS and ENVISION Studies. Published Abstract 501822
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 administered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the submission of the rolling new drug application (NDA) for UGN-102 in August 2024, ahead of schedule. The U.S. Food and Drug Administration (FDA) accepted the NDA for UGN-102 and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025. On May 21, 2025, an Oncologic Drugs Advisory Committee (ODAC) of the FDA 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. See "Forward-Looking Statements" below.

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 an ongoing, single-arm, multinational, multicenter 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 evaluates 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).

On June 2, 2025 Diakonos Oncology Corp., a clinical-stage biotechnology company developing a new generation of immunotherapies to treat challenging and aggressive cancers, reported promising results from its Phase I clinical trial of Dubodencel (DOC1021), a patient-derived dendritic cell (DC) therapy for the treatment of patients with glioblastoma (Press release, Diakonos Oncology, JUN 2, 2025, View Source [SID1234653642]). The trial included patients with particularly challenging disease characteristics and demonstrated a favorable safety profile along with early signs of clinical activity. These results support advancement to a randomized Phase II trial.

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"This milestone represents a significant advance for Diakonos Oncology and, more importantly, for the patients who urgently need better treatment options for glioblastoma and other difficult-to-treat cancers," said Jay Hartenbach, President and Chief Operating Officer of Diakonos Oncology. "The favorable safety profile and encouraging efficacy signals observed in our Phase I trial support the progression to Phase II. We look forward to advancing this next stage to further assess the potential of our dendritic cell therapy platform and deliver new hope to patients."

The study evaluated Dubodencel (DOC1021), a patient-derived immunotherapy prepared from mobilized peripheral blood mononuclear cells (PBMCs), loaded with autologous tumor lysate and amplified tumor mRNA, and administered near deep cervical lymph nodes. Sixteen newly diagnosed patients, with a median age of 61 years (range 47-73), of which 94% were MGMT unmethylated, and 25% with subtotal resection, completed three courses of the injection every two weeks alongside weekly pegylated interferon (peg-IFN), following chemotherapy and radiation. Four dose levels, ranging from 3.5-36 million total cells, were tested. Two additional patients with recurrent glioblastoma were also treated.

Key findings included:

Excellent Safety Profile: The most common adverse events (AEs) were mild flu-like symptoms and injection-site reactions, with no dose-limiting toxicities reported.
Immune Response: Post-vaccination analysis showed significant expansion of CD4+ and CD8+ central memory T-cell compartments in 13 of 13 and 11 of 13 patients, respectively. Spatial transcriptomics in tumor samples from three patients revealed clusters or "immune triads" consisting of activated CD4, CD8 and migratory microglial cells, that were increased in frequency and density post-injection. After dosing, Tregs appeared to be excluded from these inflammatory triads.
Improved Survival: The 12-month overall survival (OS) rate was 88%, significantly higher than the expected ~60% for standard of care and four patients are still alive at 22-33 months of follow-up. Recurrent glioblastoma patients survived 10-12 months.
Insight into Pseudo-Progression: When MRI imaging showed signs of potential progression early after treatment despite lacking signs of clinical progression, those that were observed rather than re-operated had a significantly longer survival. Three of the 8 patients that were observed are still alive and clinically well without tumor on imaging. This suggests an immune-reactive microenvironment may be manifesting as pseudo-progression early after treatment.
"These results are highly encouraging, particularly for a patient population with such challenging prognostic factors, including MGMT unmethylated patients," said Dr. Joseph Georges, Neurosurgeon at Banner Health in Phoenix, Arizona. "The observed survival outcomes combined with robust immunogenicity suggest that DOC1021 has the potential to induce a clinically relevant antitumor response. Furthermore, the identification of pseudo-progression may provide important insights for refining radiographic assessment and patient management in future clinical trials."

Diakonos Oncology recently completed a $20 million SAFE (Simple Agreements for Future Equity) financing round with backing from new investors Baylor College of Medicine, The Brain Tumor Investment Fund, and the Buttonwood Titan QC Fund, and existing shareholders, reflecting strong investor confidence in the company’s pioneering double-loaded dendritic cell immunotherapy platform. This funding will support the accelerated development of the Phase II glioblastoma program and enable the expansion of the platform to explore additional cancer indications.

About Dubodencel

Dubodencel, also known as DOC1021, is a first-in-class, double-loaded autologous dendritic cell therapy that uniquely combines tumor lysate and amplified tumor-derived mRNA. The immunotherapy is made with a patient’s dendritic cells combined with mRNA and proteins prepared from freshly obtained patient tumor specimens.

The unique double loading approach, which mimics a viral infection, unlocks a synergistic and exponentially more powerful tumor killing TH1 response driven by dual protein and RNA antigen sourcing, and it allows targeting of the complete cancer antigen pool. Moreover, the approach does not require any molecular modification of the patient’s immune cells for manufacturing, and does not require preconditioning of bone marrow or high dose IL-2 for administration. Dubodencel allows for a simple administration in the outpatient setting and broad reach via community cancer centers.

In addition to the recently opened Phase 2 GBM study, a clinical trial of Diakonos’ Dubodencel is ongoing for the treatment of pancreatic cancer. Diakonos has received Fast Track designations from the FDA for both the GBM and pancreatic cancer programs, in October 2023 and May 2024, respectively. The company has also received Orphan Drug Designation for the GBM program in January 2024.

On June 2, 2025 Kivu Bioscience, a biotech company developing next-generation antibody-drug conjugates, reported a manufacturing partnership with Sterling Pharma Solutions, a global contract development and manufacturing organisation, to produce cGMP-quality material for Phase 1 clinical trials of its lead oncology antibody-drug conjugate (ADC) candidate, KIVU-107 (Press release, Kivu Bioscience, JUN 2, 2025, View Source [SID1234653658]).

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Under the agreement, Sterling will manufacture cGMP clinical material for KIVU-107 at its dedicated bioconjugation facility in Deeside, UK. The collaboration includes process familiarisation, analytical development, process optimisation, and scale-up activities in preparation for a cGMP manufacturing campaign.

"We chose Sterling as our manufacturing partner based on their deep expertise in ADC development, proven track record of clinical supply, and commitment to quality," said Mohit Trikha, Ph.D., President and Chief Operating Officer, Kivu Bioscience. "This partnership marks an important milestone as we advance KIVU-107 toward first-in-human studies and deliver on our mission to bring kinder, gentler and efficacious next-generation ADC therapies to patients."

KIVU-107 is a potential first-in-class antibody-targeted conjugate which enables site-specific conjugation. The resulting structure positions the linker-payload in a natural cavity in the antibody, providing excellent stability, reduced hydrophobicity, and an increased therapeutic index relative to first-generation ADC therapies.

"Our team at Deeside has extensive experience supporting complex ADC programs from early development through clinical manufacturing," said Chad Telgenhof, Chief Commercial Officer at Sterling Pharma Solutions. "We’re excited to support Kivu to bring this promising new oncology candidate into the clinic, leveraging our expertise and investment in world-class ADC development and clinical manufacturing capabilities."

Sterling’s 6,500-square-metre Deeside facility offers a range of ADC services, from discovery-stage development through to clinical supply. In October 2024, Sterling announced a £10 million investment at the site to double its GMP manufacturing capacity, as the second phase of an ongoing strategy to increase the capabilities that it can offer customers.

On June 2, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported the completion of enrollment in the Phase 1 clinical trial of its breast cancer vaccine (Press release, Anixa Biosciences, JUN 2, 2025, View Source [SID1234653595]). This novel vaccine, invented at Cleveland Clinic, is also being developed in partnership with Cleveland Clinic and the Phase 1 trial is fully funded by a grant from the U.S. Department of Defense (DoD).

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The vaccine is designed to stimulate the immune system to target breast cancer before it can recur or develop. A total of 35 women have received the vaccine in the trial, including 26 in the triple-negative breast cancer ("TNBC") group, four in the prevention group, and five in the pembrolizumab group. These participants represent three distinct patient cohorts:

TNBC Group: Women who have completed treatment for triple-negative breast cancer, are currently cancer-free, and are at risk of recurrence.
Prevention Group: Women who are cancer-free but carry genetic mutations that place them at high risk of developing breast cancer and have elected to undergo preventative mastectomy to lower their risk.
Pembrolizumab (Keytruda) Group: Women who are receiving pembrolizumab in a post-operative setting. These women are receiving the vaccine concurrently with pembrolizumab.
The final patient visits are scheduled for August 2025. Once completed, the final study report will be submitted to the Department of Defense. A Clinical Study Report (CSR) will then be prepared for submission to the U.S. Food and Drug Administration. As part of the continued development process, the Investigational New Drug (IND) application will be transferred from Cleveland Clinic to Anixa.

Anixa and Cleveland Clinic plan to submit for presentation all trial data at a major upcoming scientific meeting.

Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences, commented, "We are very encouraged by the data we are seeing from this trial. Preliminary results show that our breast cancer vaccine is well tolerated, with more than 70% of patients demonstrating protocol-defined immune responses. These encouraging findings are guiding our planning for Phase 2 trials, which will include discussions with the FDA, protocol development, manufacturing, and clinical site selection. While cancer vaccines have traditionally faced significant hurdles, our approach is aimed at a novel target that has not been previously explored in this context. We believe this could represent a new paradigm in immuno-oncology. The breast cancer market, particularly for triple-negative breast cancer and genetically high-risk populations, continues to face a major unmet need. Our vaccine may offer a unique, immunologic pathway for both prevention and treatment."

On June 2, 2025 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta (γδ) T cell therapies for cancer and autoimmune diseases, reported new long-term clinical data from its fully enrolled Phase 1 trial of INB-200 in patients with newly diagnosed glioblastoma multiforme (GBM) (Press release, In8bio, JUN 2, 2025, View Source [SID1234653611]). The data were presented in an oral session on May 30th at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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"Half of the patients who received multiple doses remained progression free for greater than a year and a half, demonstrating functional recoveries, with several patients also having the ability to return to work. No new relapses have been reported since the last clinical update in October 2024" stated Burt Nabors, M.D., Division Director, Neuro-Oncology at the Heersink School of Medicine at the University of Alabama at Birmingham and Principal Investigator of the study. "These early data highlight the potential of repeated intracranial dosing of IN8bio’s gamma-delta T cells to extend mPFS in GBM, including in patients with chemotherapy-resistant tumors."

The Phase 1 results of INB-200 demonstrate that repeated dosing of IN8bio’s proprietary Drug Resistant Immunotherapy (DRI) – which utilizes genetically modified gamma-delta T cells delivered directly into the brain – in combination with SOC maintenance chemotherapy (temozolomide), led to an mPFS of 16.1 months. This represents an improvement of more than double (+9.2 months or +132.6%) the historical mPFS of 6.9 months under the SOC Stupp protocol. These mPFS results have already surpassed the historical mOS of 14.6 months associated with the SOC Stupp protocol alone. By comparison, a 2 to 3 month improvement in mPFS has historically been considered as clinically significant and the bar for approval by the Food and Drug Administration (FDA).

Importantly, no dose-limiting toxicities (DLTs), CRS, or ICANS have been observed among patients treated with INB-200 (n=13). The majority of adverse events were Grade 1-2 and consistent with those typically associated with radiation and temozolomide. No treatment-related deaths have occurred.

Highlights from the Clinical Data as of May 31, 2025:

INB-200

Four patients (40%) who received repeated doses of INB-200 remain alive and progression free for a median of over two years, with three returning to work
No additional relapses were observed since the last data update on October 18, 2024
Among patients who received multiple doses of INB-200, mPFS reached 16.1 months, compared to 6.9 months with SOC and 8.3 months for patients who received only a single dose of INB-200
Repeat dosing demonstrated no additional safety risks, with most side effects being mild and attributable to the SOC therapy
50% of patients receiving repeated doses remained progression-free >18 months versus 0% of patients who received a single dose

INB-400

Data from our Phase 2 clinical trial of INB-400 in patients with newly diagnosed GBM, including three additional clinical sites, also show encouraging preliminary results: current mPFS is at 10.8 months. Additional updates expected late 2025

"Our goal is to achieve deeper responses and eliminate more cancer cells to ultimately extend the time patients can remain progression free and alive," stated William Ho, CEO and cofounder of IN8bio. "The data presented at ASCO (Free ASCO Whitepaper) by Dr. Nabors speaks to the potential of IN8bio’s gamma-delta T cells to provide a game-changing immunotherapy for this dire and life-threatening cancer. We believe that INB-200 represents a novel direction in therapy for the treatment of solid tumor cancers like GBM. The complete data from our Phase 1 trial and supporting data from our Phase 2 trial represent the first time a gamma-delta T cell therapy has shown the potential to extend mPFS beyond historical benchmarks."

IN8bio’s approach delivers gamma-delta T cells directly to the tumor cavity after surgery, applying sustained immune pressure to eliminate residual cancer cells. The Company’s DRI technology is designed to treat newly diagnosed GBM by harnessing the natural tumor-targeting power of gamma-delta T cells and the sensitizing effects of chemotherapy. This approach aims to eliminate the chemo-resistant cancer and stem cells that often survive SOC treatment and can lead to relapse.

Conference Call Details

IN8bio will host a conference call and webcast featuring Dr. Burt Nabors, the Principal Investigator, today, Monday, June 2, 2025 at 8:30 am EDT to review the updated clinical data from the ASCO (Free ASCO Whitepaper) presentation. The webcast can be accessed by clicking this link and can also be accessed on the Events & Presentations page of the Company’s website. To participate in the live call, please register using this link. It is recommended that participants register at least 15 minutes in advance of the call. Once registered, participants will be informed of the dial-in number and will be provided a unique PIN.

(1) Historical reported mPFS and mOS from Stupp Protocol (surgery, radiotherapy plus maintenance temozolomide); doi: 10.1056/NEJMoa043330