On June 25, 2025 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion recommending approval of a new film-coated tablet formulation of BRUKINSA (zanubrutinib) for all approved indications (Press release, BeOne Medicines, JUN 25, 2025, View Source [SID1234654125]). The CHMP positive opinion will now be reviewed by the European Commission, which will grant the marketing authorization for the tablet formulation in the European Union and in the European Economic Area countries Norway and Iceland.

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"The CHMP’s positive opinion of our new tablet formulation of BRUKINSA is an important step toward bringing this thoughtful, patient-centered innovation to people facing certain B-cell cancers across Europe," said Giancarlo Benelli, Senior Vice President and Head of Europe, BeOne. "We look forward to a potential approval later this year and remain committed to delivering our impactful medicines to more patients in the region."

The BRUKINSA tablets have been shown to be bioequivalent to the BRUKINSA capsules based on the results of two single-dose, open-label, randomized Phase 1 crossover studies in healthy subjects. The recommended dose of BRUKINSA remains – 320 mg daily. The BRUKINSA tablets are 160 mg each, allowing patients to halve their daily pill intake and take two tablets daily. The new tablet formulation maintains BRUKINSA’s dosing flexibility by providing patients and prescribers with the option of once- or twice-daily dosing and is designed to simplify management of dose reductions as per label recommendation. Additionally, the BRUKINSA tablets are smaller than the capsules and have a film coat, which makes them easier to swallow.

BeOne Medicines will begin to convert BRUKINSA from capsules to tablets in regions outside China in 2025 as part of our commitment to sustainable business practices, including reducing our impact on the environment. This adjustment will decrease the bottle size by ~70% while also enabling the shipment of this medication with reduced temperature controls, which we expect to reduce energy needs, greenhouse gas emissions, and global transport costs.

Today’s announcement follows the U.S. Food and Drug Administration (FDA) approval of the new tablet formulation of BRUKINSA for all five approved indications earlier this month. In the U.S., BRUKINSA is the leader in new patient starts for chronic lymphocytic leukemia (CLL) across all lines of therapy, and for the first time, has become the overall BTK inhibitor market share leader.1

Important Safety Information
The current European Summary of Product Characteristics (SmPC) of BRUKINSA is available from the website of the European Medicines Agency.

This information is intended for a global audience. Product indications vary by region.

On June 16, 2025 PDS Biotechnology presented its corporate presentation (Presentation, PDS Biotechnology, JUN 25, 2025, View Source [SID1234654111]).

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On June 25, 2025 enGene Holdings Inc. (Nasdaq: ENGN, or "enGene" or the "Company"), a clinical-stage, non-viral gene-based immunotherapy company, reported that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to detalimogene voraplasmid (also known as detalimogene, and previously EG-70), the Company’s lead investigational therapy for the treatment of high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) (Press release, enGene, JUN 25, 2025, View Source [SID1234654126]).

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The RMAT program is intended to expedite the development and review of regenerative medicine therapies for serious or life-threatening conditions, where preliminary clinical evidence suggests potential to address unmet medical needs. This designation provides enGene with several regulatory advantages, including early and frequent engagement with the FDA, potential for rolling review and priority review, and other benefits like Fast Track and Breakthrough Therapy designations.

"Receiving the RMAT designation highlights the promising profile of detalimogene and its potential to address the high unmet need in NMIBC," stated Ron Cooper, Chief Executive Officer of enGene. "Bladder cancer patients with limited options cannot wait, and we are enthusiastic about potentially expediting the regulatory process to bring a first-in-class treatment to patients."

The designation was based on previously disclosed preliminary results from the ongoing pivotal LEGEND study, which demonstrated compelling clinical activity and a generally favorable tolerability profile in patients with BCG-unresponsive NMIBC with CIS.

Detalimogene is designed for streamlined administration in urology clinics — including community practices, where approximately 70% of urologists provide care. The therapy is being evaluated for its ability to treat NMIBC though the non-viral stimulation of a local immune response in the bladder, presenting a potentially transformative option for patients who otherwise face limited choices beyond radical cystectomy.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on our healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can take the form of papillary outgrowths from the bladder wall, which are typically resected, or carcinoma in situ (CIS), flat, multifocal lesions that are unable to be resected, and the two can co-occur. About 75-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50-70%) and are subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.

About Detalimogene
Detalimogene is a novel, investigational, non-viral gene-based immunotherapy for patients with high-risk NMIBC, including Bacillus Calmette-Guérin (BCG)-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response.

Detalimogene was developed using the Company’s Dually Derivatized Oligochitosan (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, simplify safe handling and cold storage complexities, and streamline both manufacturing processes and administration paradigms.

Detalimogene has received Fast Track designation from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive CIS NMIBC, with or without resected papillary tumors, who are unable to undergo cystectomy. Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

About the Pivotal LEGEND Trial
Detalimogene is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk NMIBC. LEGEND’s pivotal cohort (Cohort 1) consists of approximately 100 patients with high-risk, BCG-unresponsive NMIBC with CIS (with or without papillary disease) and is designed to serve as the basis of the Company’s planned Biologics License Application (BLA) filing. In addition to this pivotal cohort, LEGEND includes three additional cohorts, including NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3). The LEGEND trial is actively enrolling patients with sites participating in the USA, Canada, Europe, and the Asia-Pacific region.

On June 25, 2025 Oncotelic Therapeutics, Inc. (OTCQB: OTLC) ("Oncotelic" or the "Company"), a clinical-stage biopharmaceutical company focused on RNA-based therapeutics, reported the publication of a peer-reviewed research article highlighting TGFB2 gene methylation as a positive prognostic biomarker for pancreatic ductal adenocarcinoma (PDAC) (Press release, Oncotelic, JUN 25, 2025, View Source [SID1234654112]). The paper, published in collaboration with Sapu Biosciences, LLC ("Sapu"), a wholly owned subsidiary of GMP Biotechnology Limited ("GMP Bio"), in which Oncotelic owns a 45% stake, appears in the journal International Journal of Molecular Sciences and is entitled: "TGFB2 Gene Methylation in Tumors with Low CD8+ T-Cell Infiltration Drives Positive Prognostic Overall Survival Responses in Pancreatic Ductal Adenocarcinoma."

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Access the publication online at: View Source

The study was co-authored by Dr. Sanjive Qazi, Dr. Michael Potts, Scott Myers, Dr. Stephen Richardson, and Dr. Vuong Trieu.

Key Findings

PDAC remains one of the most lethal malignancies with limited treatment options, typically restricted to cytotoxic regimens like FOLFIRINOX. This study identifies DNA methylation signatures of the TGFB2 gene as a novel biomarker for improved overall survival, particularly in immunosuppressed tumor microenvironments characterized by low CD8+ T-cell infiltration.

Notably, patients exhibiting high TGFB2 methylation along with low expression of immune markers such as CD3D, LCK, and HLA-DRA demonstrated a highly significant median overall survival exceeding 50 months. The data suggest that TGFB2 methylation is a favorable prognostic indicator and may inform patient stratification for therapies targeting TGFB2 mRNA-such as OT-101, Oncotelic’s investigational antisense oligonucleotide.

In addition, the study underscores the importance of profiling TGFB1, TGFB2, and TGFB3 methylation to better characterize tumor immune status and select candidates for immunotherapy in otherwise resistant "cold" tumors.

Leadership Commentary

"Our latest discovery significantly enhances our understanding of the TGFB gene complex in PDAC, particularly in immunologically cold tumors," said Dr. Sanjive Qazi, lead author. "These results support further clinical development of OT-101 in PDAC, especially among patients with low T-cell infiltration and high TGFB2 methylation."

"PDAOAI, our AI-powered chatbot platform, played a pivotal role in the literature mining and analysis for this paper," added Scott Myers, Product Manager. "The integration of AI into the scientific process is accelerating discovery."

"Large language models like PDAOAI are transforming how we identify, extract, and interpret biomedical insights," said Dr. Michael Potts, VP of Data Science at Oncotelic.

The underlying source data and referenced literature used in the manuscript are accessible via Oncotelic’s proprietary AI platform, PDAOAI. Engage with the research on the public PDAOAI Discord community.

On June 25, 2025 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application (No. 168178) for REYOBIQTM (Rhenium Re186 Obisbemeda) for the treatment of pediatric patients with supratentorial recurrent, refractory, or progressive high-grade glioma (HGG) and ependymoma (Press release, Plus Therapeutics, JUN 25, 2025, View Source [SID1234654113]). The trial will be referred to as the ReSPECT-PBC trial and is funded by a $3.0M research grant from the U.S. Department of Defense.

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"We are very excited for the opportunity to study the novel radiotherapeutic, REYOBIQ, in difficult-to-treat pediatric brain tumors. Surgery and external beam radiation have been the mainstays of treatment for pediatric high-grade glioma and, sadly, outcomes have not improved for many decades. We believe this novel therapy that may allow higher doses of radiation while limiting the exposure to normal developing brain and delivering this agent via convection enhanced delivery (CED) directly into the tumor has the potential to make a big difference in our patients’ lives and improve outcomes," said Dr. Ashley S. Plant, M.D., Principal Investigator for the trial, attending physician in Hematology, Oncology, Neuro-oncology, and Stem Cell Transplantation at Ann & Robert H. Lurie Children’s Hospital of Chicago, A.M. Khokhar Research Scholar and Associate Professor of Pediatrics at Feinberg School of Medicine Northwestern University. "I am extremely passionate about bringing this therapy into the pediatric space and hopeful about the benefits to my patients."

Phase 1/2a Trial Design Highlights:

The Phase 1/2a trial is a two-part, single-arm, prospective study aimed at determining the maximum tolerated dose (MTD), safety, and tolerability of REYOBIQ in pediatric patients aged 6 to 21 years (with consideration for patients up to 25 years on a case-by-case basis).

Key elements of the trial design include:

Phase 1a/b (Dose Escalation): This phase will enroll an estimated 24 patients using a modified 3+3 dose escalation scheme to establish the MTD and recommended Phase 2 dose (RP2D). Safety assessment and alignment with the FDA will occur at defined intervals.

Phase 2a: This phase will enroll approximately 32 patients (12 with ependymoma and 20 with HGG) at the RP2D to assess efficacy.

Pediatric HGG including ependymoma are rare (approximately 3.3 cases per 100,000 persons) but aggressive brain tumors with limited treatment options and poor prognosis, particularly in recurrent settings. Standard treatments, including surgical resection and external beam radiation therapy, often fail to prevent recurrence, with 5-year survival rates as low as 22% for HGG, depending on tumor grade and resection extent. REYOBIQ’s targeted delivery via CED bypasses the blood-brain barrier, offering a novel approach to potentially improve outcomes for these patients.

"This pediatric FDA clearance builds on our successful track record in the safe administration and promising efficacy signals observed in adult patients with more common central nervous cancers," said Marc Hedrick, M.D., Plus Therapeutics President and Chief Executive Officer. "We expect REYOBIQ will offer much needed hope to children with aggressive and difficult-to-treat brain cancers and their families."

REYOBIQ is a novel radiotherapeutic designed to deliver high doses of beta radiation directly to brain tumors while minimizing damage to surrounding healthy tissue. The ReSPECT-PBC (pediatric brain cancer) trial builds on promising preclinical data and clinical results from the Company’s adult recurrent glioblastoma trial (ReSPECT-GBM). As recently published in Nature Communications, ReSPECT-GBM demonstrated favorable safety and clinical response with a doubling of overall survival for those patients receiving a therapeutic dose of radiation defined as > 100 Gy.

For more information, please contact @plustherapeutics.com or visit www.clinicaltrials.gov (identifier to be assigned upon registration).

About REYOBIQ (rhenium Re186 obisbemeda)
REYOBIQ (rhenium Re186 obisbemeda) is a novel injectable radiotherapy specifically formulated to deliver direct targeted high dose radiation in CNS tumors in a safe, effective, and convenient manner to optimize patient outcomes. REYOBIQ has the potential to reduce off target risks and improve outcomes for CNS cancer patients, versus currently approved therapies, with a more targeted and potent radiation dose. Rhenium-186 is an ideal radioisotope for CNS therapeutic applications due to its short half-life, beta energy for destroying cancerous tissue, and gamma energy for real-time imaging. REYOBIQ is being evaluated for the treatment of recurrent glioblastoma and leptomeningeal metastases in the ReSPECT-GBM and ReSPECT-LM clinical trials. ReSPECT-GBM is supported by an award from the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), and ReSPECT-LM is funded by a three-year $17.6M grant by the Cancer Prevention & Research Institute of Texas (CPRIT). The Company’s clinical trial for pediatric brain cancer is supported by a $3 million grant from the U.S. Department of Defense’s Peer Reviewed Cancer Research Program.