On July 8, 2025 Norgine, a leading European specialty pharmaceutical company, reported that Swissmedic has approved the registration of IFINWIL (eflornithine) as monotherapy for the treatment of paediatric patients from one year with high-risk neuroblastoma (HRNB) (Press release, Norgine, JUL 8, 2025, View Source [SID1234654290]).

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"This milestone marks a further regulatory approval of IFINWIL for high-risk neuroblastoma, following approvals in the US, Israel and Australia," said Jörg Plessl, VP and Head of Global Regulatory Affairs at Norgine. "We deeply appreciate Swissmedic and the other Project Orbis partners for their keen awareness of the distinct challenges associated with paediatric oncology. Their agility in utilizing adaptive regulatory pathways is crucial because the stakes are incredibly high when it comes to children’s lives. In these situations, speed and flexibility are not just advantageous, they can make all the difference."

"By collaborating with international regulatory partners through Project Orbis, we are advancing our strategy to broaden access to innovative therapies for paediatric patients in Europe and ANZ." said Dr David Gillen, Chief Medical Officer at Norgine. "At Norgine, we’re proud to take on some of the most complex disease areas, driven by our mission to deliver meaningful treatments where they’re needed most."

HRNB is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life.2 Globally, neuroblastoma affects approximately 10.2 children per million annually under age 15, with around 50% of cases classified as high-risk at diagnosis.3,4 Neuroblastoma originates in the body’s nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands.5 It is considered an aggressive tumour because it often spreads to other parts of the body (metastasises). In most cases, it has spread by the time it is diagnosed.5

Despite intensive multimodal therapy, the 5-year survival rate for HRNB remains below 50%, compared to up over 95% for infants with low-risk disease.2,6 Neuroblastoma accounts for approximately 15% of all paediatric cancer-related deaths, underscoring the urgent need for more effective treatment options.7

About IFINWIL
IFINWIL has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy.8 IFINWIL is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development.9

High-Risk Neuroblastoma (HRNB) Treatment Background
Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death.10 Although there have been some improvements in survival, the prognosis for children with high risk neuroblastoma is still poor with around 80% of high risk relapses occuring within 2 years from diagnosis, and long-term survival remains low, with only about 15% surviving more than 5 years post-relapse.11,12,13 Avoiding relapse is key to long-term survival, and until now, there have been no approved therapies for the post maintenance treatment period in major markets outside of the United States.14

About Project Orbis
Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health).

In April 2024, Norgine submitted an application for approval of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. In April 2025, the Australian Therapeutic Goods Administration (TGA) approved the registration of IFINWIL for the treatment of adults and paediatric patients with HRNB, who have responded to prior multiagent, multimodality therapy.

These regulatory milestones support Norgine’s efforts to deliver patient access to IFINWIL and bring a further treatment option in the field of paediatric oncology.

On July 8, 2025 Caris Life Sciences (NASDAQ: CAI), a leading, patient-centric, next-generation AI TechBio company and precision medicine pioneer, reported a landmark study published in Scientific Reports, a Nature journal, demonstrating the accuracy and clinical utility of the Caris Assure blood-based biopsy assay across the cancer continuum (Press release, Caris Life Sciences, JUL 8, 2025, View Source [SID1234654291]).

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The Caris Assure platform is a breakthrough single assay that combines comprehensive, highly sophisticated molecular profiling of all 23,000+ genes across DNA and RNA in plasma, setting a new blood-based testing standard and revolutionizing management of the cancer care journey. Additionally, Caris sequences DNA in the "buffy coat," the layer of centrifuged blood between the red blood cells and plasma, which contains white blood cells and platelets. Caris Assure combines Whole Exome Sequencing (WES) and Whole Transcriptome Sequencing (WTS) with advanced machine learning on a single platform. Caris Assure is currently used for therapy selection and is capable of enabling accurate early detection and highly sensitive disease monitoring.

"We designed Caris Assure to be more than a liquid biopsy test. This assay takes advantage of advanced sequencing and computing technology to capture genetic information available from the whole exome and whole transcriptome from the tumor material circulating in the blood and DNA from the patient’s white blood cells," said David Spetzler, MS, PhD, MBA, President of Caris. "This enables us to provide a very comprehensive individualized picture of a patient’s specific disease state. This is the first time so much molecular information has been available from a single blood test, allowing for increased accuracy and additional insights to inform physicians in helping to make cancer care decisions."

The Caris Assure liquid biopsy platform, powered by the Assure Blood-based Cancer Detection AI (ABCDai), was trained on over 376,000 whole exome and whole transcriptome tissue profiles and over 7,000 matched blood and tissue samples. In addition to therapy selection, the study validates the performance of Caris Assure in:

Multi-Cancer Early Detection (MCED): Achieved sensitivities of 83.1% to 95.7% across cancer stages I–IV at 99.6% specificity.
Diagnostic Pathway Predictor: Accurately identified the diagnostic pathway for MCED-positive cancers using the ABCDai-GPS model.
MRD and Recurrence Monitoring: Demonstrated significant predictive power for recurrence, with hazard ratios of 33.4 (p < 0.005) for MRD and 4.39 (p = 0.008) for therapeutic monitoring.
Caris Assure does not require a prior tissue biopsy. Instead, its AI model identifies tumor-derived signals based on patterns learned from hundreds of thousands of tumors. This enables a tissue-agnostic, highly sensitive approach to cancer detection and monitoring.

While Caris Assure is currently commercially available for therapy selection in advanced cancers, this study lays the groundwork for expanding into early detection, MRD and therapeutic monitoring. Caris is actively pursuing reimbursement and regulatory pathways to bring these applications to market.

"This is more than a validation study; it is a blueprint for the future of cancer diagnostics," added Spetzler. "Our goal is to create a seamless ecosystem where patients and clinicians can rely on one platform throughout the entire cancer journey."

On July 7, 2025 ImmunityBio, Inc. (NASDAQ: IBRX) reported that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for ANKTIVA (nogapendekin alfa inbakicept-pmln) in combination with Bacillus Calmette-Guérin (BCG) for the treatment of certain bladder cancer patients (Press release, ImmunityBio, JUL 7, 2025, View Source [SID1234654259]). This is the first marketing approval outside the U.S. for this novel lymphocyte-stimulating agent.

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"With the MHRA’s authorization of ANKTIVA plus BCG, we can now offer our immunotherapy outside the U.S. to help patients with a disease that, if not effectively treated, can lead to bladder removal," said Dr. Patrick Soon-Shiong, Founder, Executive Chairman and Global Chief Scientific and Medical Officer of ImmunityBio. "This immune-boosting, lymphocyte-stimulating agent, the first of its kind, is central to our Cancer BioShield platform, which is designed to restore immune function and support long-term disease control."

"ImmunityBio is honored to have received this important authorization from the UK MHRA. In light of the United States Most-Favored-Nation Prescription Drug Pricing policy implemented on May 12, 2025, we are actively evaluating our go-to-market strategy for the UK," said Richard Adcock, CEO and President of ImmunityBio.

ANKTIVA is a first-in-class IL-15 agonist that activates and proliferates natural killer (NK) cells and CD4+ and CD8+ T cells. It is designed to restore immune competence by reversing lymphopenia—a condition in which cancer and conventional therapies, such as chemotherapy, radiation and checkpoint inhibitors, reduce the number and function of immune cells. Restoring immune function is essential for immunosurveillance, immunogenic cell death, and sustained tumor control. The BioShield platform’s effectiveness can be monitored using a routine complete blood count (CBC).

ANKTIVA was designated a Breakthrough Therapy by the FDA and received approval from both the FDA and MHRA based on its safety and efficacy outcomes of complete response (CR) and duration of response (DOR). In a single-arm, multicenter trial, 77 evaluable patients received ANKTIVA with BCG for up to 37 months.

As of the November 2023 data cutoff, the duration of complete response for some patients exceeded 47 months and remains ongoing. These extended duration of complete responses beyond 24 months with ANKTIVA and BCG surpasses the benchmark for meaningful clinical results set by experts from the International Bladder Cancer Group.

ImmunityBio has also submitted regulatory applications to the European Medicines Agency (EMA) to expand availability of ANKTIVA across the 27 European Union (EU) member states, as well as Iceland, Norway and Liechtenstein.

About NMIBC CIS

Bladder cancer is the 10th most commonly-diagnosed cancer globally,2 and in the UK, the Action Bladder Cancer UK estimates approximately 23,000 patients are diagnosed annually.1 At the time of diagnosis, about 80% of cases are non-muscle invasive bladder cancer (NMIBC), wherein the cancer is found only on the inner layer of the bladder wall.3 The standard therapy for NMIBC is intravesical instillation (delivery to the bladder via a catheter) of bacillus Calmette-Guerin (BCG).4,5 BCG is a benign bacteria that induces an immune response in the bladder in proximity to the cancer cells, leading to clearance of the cancer in many patients. In ~30-40% of patients, however, BCG will fail, and in ~50% that initially respond, cancer will recur.6

About ANKTIVA

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response.

ANKTIVA is a first-in-class IL-15 agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo.

ANKTIVA was approved by the FDA in 2024 for BCG-unresponsive non-muscle invasive bladder cancer CIS with or without papillary tumors. For more information, visit Anktiva.com.

Indication and Important Safety Information from the FDA Label

INDICATION AND USAGE: ANKTIVA is an interleukin-15 (IL-15) receptor agonist indicated with Bacillus Calmette-Guerin (BCG) for the treatment of adult patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

WARNINGS AND PRECAUTIONS: Risk of Metastatic Bladder Cancer with Delayed Cystectomy. Delaying cystectomy can lead to the development of muscle invasive or metastatic bladder cancer, which can be lethal. If patient with CIS do not have a complete response to treatment after a second induction course of ANKTIVA with BCG, reconsider cystectomy.

DOSAGE AND ADMINISTRATION: For lntravesical Use Only. Do not administer by subcutaneous or intravenous routes. Instill intravesically only after dilution. Total time from vial puncture to the completion of the intravesical instillation should not exceed 2 hours.

USE IN SPECIFIC POPULATIONS: Pregnancy: May cause fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception.

ADVERSE REACTIONS: The most common (≥15%) adverse reactions, including laboratory test abnormalities, are increased creatinine, dysuria, hematuria, urinary frequency, micturition urgency, urinary tract infection, increased potassium, musculoskeletal pain, chills and pyrexia.

For more information about ANKTIVA, please see the Full Prescribing Information at www.anktiva.com.

You are encouraged to report negative side effects of prescription drugs to FDA.

Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact lmmunityBio at 1-877-ANKTIVA (1-877-265-8482)

On July 7, 2025 Inventiva (Euronext Paris and Nasdaq: IVA) ("Inventiva" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of oral therapies for the treatment of metabolic dysfunction-associated steatohepatitis ("MASH"), reported the receipt of a $10 million milestone payment from Chia Tai-Tianqing Pharmaceutical Group Co., Ltd ("CTTQ"), a subsidiary of Sino Biopharm (Press release, Inventiva Pharma, JUL 7, 2025, View Source [SID1234654260]).

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This milestone payment follows the successful settlement of the second tranche of €115.6 million1 in gross proceeds (net proceeds of €108.5 million) of the previously announced structured financing of up to €348 million2 (the "Structured Financing").

In September 2022, Inventiva entered into a licensing and collaboration agreement with CTTQ (as amended on October 11, 2024, the "CTTQ License Agreement") to develop and commercialize lanifibranor, Inventiva’s proprietary compound, for the treatment of MASH and potentially other metabolic diseases in Mainland China, Hong Kong Special Administrative Region, Macau Special Administrative Region and Taiwan. Under the CTTQ License Agreement, Inventiva is eligible to receive up to an additional $265 million of clinical, regulatory and commercial milestone payments, as well as royalties in the low single digits on annual net sales of lanifibranor, if approved.

Based on the results from the Phase 2b NATIVE clinical trial, lanifibranor was granted Breakthrough Therapy Designation for MASH by the U.S. Food and Drug Administration in October 2020 and by the Chinese National Medical Products Administration (NMPA) in December 2023. This designation could potentially accelerate the development and regulatory review of drug candidates for serious or life-threatening conditions. CTTQ joined Inventiva’s ongoing NATiV3 pivotal Phase 3 clinical trial, which includes over 60 sites across mainland China. Furthermore, CTTQ has completed a Phase I bridging study and confirmed no significant ethnic differences, thereby paving the way to seek regulatory approval in China based on the results of the NATiV3 trial.

On July 7, 2025 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-stage clinical biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported two important developments that aim to reinforce the global positioning of potential first-in-class radioenhancer JNJ-1900 (NBTXR3), which is licensed by Janssen Pharmaceutica NV, a Johnson & Johnson company (Press release, Nanobiotix, JUL 7, 2025, View Source [SID1234654360]).

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Health authorities in major European countries have agreed to formally reclassify JNJ-1900 (NBTXR3) from a medical device to a drug, completing a process initiated by Johnson & Johnson to harmonize the product candidate’s regulatory status with classifications already in place in the United States and other major markets. The reclassification follows updated insights into the product candidate’s mechanism of action.

In parallel, Nanobiotix has filed a new composition of matter patent for JNJ-1900 (NBTXR3) that aims to reinforce the intellectual property foundation supporting the product candidate.

"These updates reflect our continued commitment to the JNJ-1900 (NBTXR3) program," said Laurent Levy, co-founder and chairman of the executive board at Nanobiotix. "We are pleased with the progress achieved with health authorities and proud to continue advancing this potential first-in-class product candidate toward patients in need."

JNJ-1900 (NBTXR3) is currently the subject of a comprehensive global clinical development program across multiple tumor types and therapeutic combinations, including a pivotal Phase 3 trial in head and neck cancer.

About JNJ-1900 (NBTXR3)

JNJ-1900 (NBTXR3) is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. Its proof-of-concept was achieved in soft tissue sarcomas through a successful randomized Phase 2/3 study in 2018. The product candidate’s mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that JNJ-1900 (NBTXR3) could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

Radiotherapy-activated JNJ-1900 (NBTXR3) is being evaluated across multiple solid tumor indications as a single agent or combination therapy. The program is led by NANORAY-312—a global, randomized Phase 3 study in locally advanced head and neck squamous cell cancers. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of JNJ-1900 (NBTXR3) activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the Phase 3 study.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a collaboration strategy to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several Phase 1 and Phase 2 studies evaluating JNJ-1900 (NBTXR3) across tumor types and therapeutic combinations. In 2023, Nanobiotix announced a license agreement for the global co-development and commercialization of JNJ-1900 (NBTXR3) with Janssen Pharmaceutica NV, a Johnson & Johnson company.