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Imfinzi approved in the EU as first and only perioperative immunotherapy for muscle-invasive bladder cancer

On July 4, 2025 AstraZeneca reported that Imfinzi (durvalumab) has been approved in the European Union (EU) for the treatment of adult patients with resectable muscle-invasive bladder cancer (MIBC) in combination with gemcitabine and cisplatin as neoadjuvant treatment, followed by Imfinzi as monotherapy adjuvant treatment after radical cystectomy (surgery to remove the bladder) (Press release, AstraZeneca, JUL 4, 2025, View Source [SID1234654238]).

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The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the NIAGARA Phase III trial, which were published in The New England Journal of Medicine.

In a planned interim analysis, the Imfinzi-based perioperative regimen demonstrated a statistically significant 32% reduction in the risk of disease progression, recurrence, not undergoing surgery, or death versus neoadjuvant chemotherapy with radical cystectomy alone (based on event-free survival [EFS] hazard ratio [HR] of 0.68; 95% confidence interval [CI] 0.56-0.82; p<0.0001). Estimated median EFS was not yet reached for the Imfinzi arm versus 46.1 months for the comparator arm. An estimated 67.8% of patients treated with the regimen were event free at two years compared to 59.8% in the comparator arm.

Results from the key secondary endpoint of overall survival (OS) showed that the Imfinzi-based perioperative regimen reduced the risk of death by 25% versus the comparator arm (based on OS HR of 0.75; 95% CI 0.59-0.93; p=0.0106). Median survival was not yet reached for either arm. An estimated 82.2% of patients treated with the regimen were alive at two years compared to 75.2% in the comparator arm.

In 2024, over 35,000 people in the five major European countries were treated for MIBC.1 Despite this representing a curative-intent setting, many patients experience disease recurrence after surgery with current standard-of-care neoadjuvant chemotherapy.2

Dr Michiel Van der Heijden, medical oncologist and Group Leader at the Netherlands Cancer Institute, and investigator in the NIAGARA trial, said: "The durvalumab-based perioperative regimen is an important new treatment option for patients in Europe with muscle-invasive bladder cancer, as currently nearly half experience disease recurrence despite treatment with neoadjuvant chemotherapy and surgery to remove the bladder. The NIAGARA results showed how this regimen reduced the risk of recurrence by nearly a third and significantly extended survival, underscoring its potential to transform clinical practice in this curative-intent setting."

Dave Fredrickson, Executive Vice President, Oncology Haematology Business Unit, AstraZeneca, said: "Imfinzi is poised to transform the standard of care for muscle-invasive bladder cancer in Europe as the first and only perioperative immunotherapy for these patients. In the NIAGARA Phase III trial, more than 80 per cent of patients were still alive two years after treatment with the Imfinzi regimen, setting a new survival benchmark for a disease that has seen few treatment advances in decades."

Imfinzi was generally well tolerated, and no new safety signals were observed in the neoadjuvant and adjuvant settings. Further, adding Imfinzi to neoadjuvant chemotherapy was consistent with the known profile for this combination and did not compromise patients’ ability to complete surgery compared to neoadjuvant chemotherapy alone. Immune-mediated adverse events were consistent with the known profile of Imfinzi, manageable and mostly low-grade.

The European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) has published its assessment of the NIAGARA regimen against the Magnitude of Clinical Benefit Scale (MCBS), awarding it the highest possible grade of "A" in the curative setting.3 The ESMO (Free ESMO Whitepaper)-MCBS facilitates improved decision-making regarding the value of anti-cancer therapies and is used in several ways, including in clinical guidelines and in health technology assessments in a growing number of countries.4

Imfinzi is approved in the US and other countries in this setting based on the NIAGARA results. Regulatory applications for this indication are currently under review in Japan and several other countries. Imfinzi is also approved in other curative-intent settings based on the PACIFIC and AEGEAN Phase III trials in non-small cell lung cancer (NSCLC), and in limited-stage small cell lung cancer (SCLC) based on the ADRIATIC Phase III trial.

Notes

Muscle-invasive bladder cancer
Bladder cancer is the 9th most common cancer in the world, with more than 614,000 patients diagnosed each year.5 The most common type of bladder cancer is urothelial carcinoma, which begins in the urothelial cells of the urinary tract.6 Bladder cancer is considered muscle-invasive when there is evidence of the tumour invading the muscle wall of the bladder but no distant metastases.6 In MIBC, approximately 50% of patients who undergo bladder removal surgery experience disease recurrence.2 Treatment options that prevent disease recurrence after surgery are critically needed in this curative-intent setting.

NIAGARA
NIAGARA is a randomised, open-label, multi-centre, global Phase III trial evaluating perioperative Imfinzi as treatment for patients with MIBC before and after radical cystectomy. In the trial, 1,063 patients were randomised to receive four cycles of Imfinzi plus neoadjuvant chemotherapy prior to cystectomy followed by eight cycles of Imfinzi monotherapy, or neoadjuvant chemotherapy alone prior to cystectomy with no further treatment after surgery. NIAGARA is the largest global Phase III trial in this setting.

The trial is being conducted at 192 centres in 22 countries across North America, South America, Europe, Australia and Asia. Its dual primary endpoints are EFS and pathologic complete response at the time of cystectomy. Key secondary endpoints are OS and safety.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

In May 2025, Imfinzi plus standard-of-care Bacillus Calmette-Guérin induction and maintenance therapy met the primary endpoint of disease-free survival for patients with high-risk non-muscle-invasive bladder cancer in the POTOMAC Phase III trial.

In lung cancer, Imfinzi is the global standard of care based on OS in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy (CRT). Additionally, Imfinzi is approved as a perioperative treatment in combination with neoadjuvant chemotherapy in resectable NSCLC, and in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved for limited-stage SCLC in patients whose disease has not progressed following concurrent platinum-based CRT; and in combination with chemotherapy for the treatment of extensive-stage SCLC.

Imfinzi is also approved in combination with chemotherapy in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU.

In March 2025, perioperative Imfinzi added to standard-of-care chemotherapy met the primary endpoint of EFS in the MATTERHORN Phase III trial in resectable gastric and gastroesophageal junction cancers.

Imfinzi in combination with chemotherapy followed by Imfinzi monotherapy is approved as a 1st-line treatment for primary advanced or recurrent endometrial cancer (mismatch repair deficient disease only in the US and EU). Imfinzi in combination with chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient advanced or recurrent endometrial cancer in the EU and Japan.

Since the first approval in May 2017, more than 414,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with NSCLC, bladder cancer, breast cancer, ovarian cancer and several gastrointestinal cancers.

OS Therapies Granted End of Phase 2 Meeting by US FDA for OST-HER2 Program in the Prevention or Delay of Recurrent, Fully Resected, Pulmonary Metastatic Osteosarcoma

On July 3, 2025 OS Therapies (NYSE-A: OSTX) ("OS Therapies" or "the Company"), a clinical-stage immunotherapy and Antibody Drug Conjugate (ADC) biopharmaceutical company, reported it was granted an End of Phase 2 Meeting by the United States Food & Drug Administration ("FDA") to review the OST-HER2 program in the prevention or delay of recurrent, fully resected, pulmonary metastatic osteosarcoma (Press release, OS Therapies, JUL 3, 2025, View Source [SID1234654242]). The Company expects the meeting to occur in the third quarter of 2025. The End of Phase 2 Meeting marks a pivotal point in the drug development process, and a significant milestone towards market access.

The Company intends to seek alignment with FDA to begin a Rolling Review process for the forthcoming Biologics Licensing Application ("BLA") submission for OST-HER2. The Rolling Review process means that a company can submit completed sections of its Biologic License Application (BLA) for review by FDA, rather than waiting until every section of the BLA is completed before the entire application can be reviewed. This can contribute to augmented interactions with FDA and potentially an expedited BLA approval timeline.

OST-HER2 has received FDA Orphan Disease Designation (ODD), Fast Track and Rare Pediatric Disease Designation (RPDD) from FDA. Under the RPDD program, if the Company receives Accelerated Approval prior to September 30, 2026, it will become eligible to receive a Priority Review Voucher (PRV) that it intends to sell. The most recent PRV sale, valued at $160 million, occurred in June 2025.

OST-HER2, an immunotherapy for osteosarcoma that uses a HER2-bioengineered form of the bacterium Listeria monocytogenes to trigger a strong immune response against HER2-expressing cancer cells, is featured in the movie Shelter Me: The Cancer Pioneers. The movie offers a look into canine comparative oncology, a field that compares treatment of cancers in dogs to those in people and covers developing treatments for rare forms of cancer. The trailer is available here and the movie is available via streaming on PBS’ website.

The most recent data regarding the OST-HER2 canine osteosarcoma program is available at this link. The Company has formed subsidiary OS Animal Health to advance the canine osteosarcoma program.

Thermo Fisher’s NGS Assay Receives FDA Approval as a Companion Diagnostic for ZEGFROVY and for Tumor Profiling

On July 3, 2025 Thermo Fisher Scientific, the world leader in serving science, reported the U.S. Food and Drug Administration (FDA) has approved the Oncomine Dx Express Test on the Ion Torrent Genexus Dx Integrated Sequencer as an in vitro diagnostic (IVD) assay for use as a companion diagnostic (CDx) for Dizal’s ZEGFROVY (sunvozertinib) and in tumor profiling (Press release, Thermo Fisher Scientific, JUL 3, 2025, View Source [SID1234654241]). This approval brings rapid next-generation sequencing (NGS) to decentralized clinical settings closer to where patients receive care. With the ability to deliver essential genomic insights in as little as 24 hours*, this approval helps advance the accessibility of precision oncology tools and enables more timely decision making.

A patient’s tumor profile has the potential to guide precision oncology care. However, delays in obtaining results can hinder clinicians’ ability to make informed decisions, potentially causing patients to miss out on targeted therapies, which can impact treatment efficacy and patient outcomes. Furthermore, a significant number of patients miss out on targeted therapies due to inefficiencies or lack of access to testing, highlighting the critical role of timely genomic profiling. The Oncomine Dx Express Test was designed to simplify the NGS workflow and connect patients everywhere to precision oncology.

Now with the Oncomine Dx Express Test on the Ion Torrent Genexus Dx Integrated Sequencer, laboratories across a variety of clinical settings can deliver rapid genomic profiling with exceptional accuracy and ease, helping to ensure patients can benefit from the latest advancements in precision oncology.

The Oncomine Dx Express Test is intended for use as a companion diagnostic for ZEGFROVY (sunvozertinib), a targeted therapy from Dizal, to identify patients with non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations. In addition, the test has been approved for tumor profiling in solid tumors and detects cancer mutations with evidence of clinical significance and potential clinical significance across 46 genes.

Accelerating informed precision oncology care

The Genexus Dx Integrated Sequencer automates the NGS workflow — from sample preparation to data analysis and reporting — making the technology accessible to more laboratories, including those that are smaller and without previous NGS expertise. It also enables laboratories to generate timely CDx results as well as tumor profiling reports in as little as 24 hours, helping accelerate insights and access to precision oncology.

"NGS has been instrumental in advancing precision oncology, but these insights often aren’t available early enough to inform real-world care," said Kathy Davy, president of Clinical Next-Generation Sequencing at Thermo Fisher Scientific. "With our rapid NGS solutions, we aim to deliver timely results to clinicians and their patients prior to the initiation of treatment. We’ve been leading companion diagnostics in collaboration with our pharma partners for a decade, and this approval signifies the next step in our journey bringing rapid, decentralized NGS CDx to drug development. We are proud of our collaboration with Dizal to bring ZEGFROVY to the US market."

Biodesix, one of Thermo Fisher’s collaborators in the validation of Oncomine Dx Express Test on the Genexus Dx Integrated Sequencer, will be the first lab to launch testing services.

"The Biodesix team is excited at the prospect of using the newly FDA approved Genexus Dx Integrated Sequencer for clinical testing using the Oncomine Dx Express test and for supporting our biopharma client projects," said Gary Pestano, Ph.D., chief development officer at Biodesix. "Automation of the systems for nucleic acid purification, library preparation, sequencing and report generation has the potential to significantly improve our NGS laboratory workflows by reducing hands-on time and by delivering rapid turnaround times – from sample receipt to results."

ZEGFROVY (sunvozertinib) CDx approval

ZEGFROVY, developed by Dizal, was granted FDA accelerated approval following Breakthrough Therapy designation and Priority Review for the treatment of NSCLC patients with EGFR exon 20 insertion mutations. With the approval of the Oncomine Dx Express Test on the Genexus Dx Integrated Sequencer as a companion diagnostic for this therapy, clinicians can now quickly identify eligible patients – supporting earlier intervention and expanding access to targeted therapy.

"ZEGFROVY is a highly effective and currently the only approved EGFR TKI targeting EGFR exon 20 insertion mutations. The approval of ZEGFROVY will have a significant impact on platinum-pretreated patients with NSCLC harboring EGFR exon 20 insertion mutations, a difficult-to-treat lung cancer subtype," said Susan Chen, senior vice president, head of clinical operation at Dizal. "Today’s FDA approval of the Oncomine Dx Express Test offers access to ZEGFROVY through decentralized companion diagnostic testing. The ability to match patients to our therapy quickly and accurately, regardless of mutation location, can improve outcomes for this patient population."

InnoCare Announces Approval of Clinical Trial of a Novel ADC ICP-B794 in China

On July 3, 2025 InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, reported the approval of the Investigational New Drug (IND) by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) to conduct the clinical trial of a novel B7-H3 targeted ADC ICP-B794 (Press release, InnoCare Pharma, JUL 3, 2025, View Source [SID1234654240]).

ICP-B794 is a novel ADC comprising a humanized anti-B7-H3 monoclonal antibody conjugated to potent in-house developed payload via a protease-cleavable linker. This combination ensures precise targeting of tumor cells while minimizing off-target effects, offering a promising treatment for solid tumors such as lung cancer, esophageal cancer, nasopharyngeal cancer, head and neck squamous cell carcinomas, prostate cancer, and others.

Currently, there are no B7-H3 targeted therapies approved for marketing globally. B7-H3 is a type I transmembrane protein that is highly expressed in a variety of solid tumors. Due to its specific expression in tumor cells, it is considered a highly promising anti-tumor target.

Dr. Jasmine Cui, the Co-founder, Chairwoman, and CEO of InnoCare, said, "ICP-B794 is developed from the Company’s proprietary ADC platform. The platform is designed to deliver ADCs with strong tumor-killing efficacy and an adequate therapeutic window, thereby broadening treatment options for cancer patients and improving their clinical outcomes. As the platform continues to evolve, the Company is poised to expand its portfolio with multiple differentiated ADC candidates, further advancing precision medicine in oncology."

With ongoing efforts to address the growing needs in solid tumors, InnoCare is committed to building a competitive drug portfolio aimed at treating a broad range of solid tumor indications. The Company is expanding the scope of its pipeline through a combination of targeted therapies, immune-oncology approaches, and cutting-edge ADC technology. The R&D team is focused on discovering and developing novel platforms that target various solid tumors, utilizing innovative technologies to identify and advance potential drug candidates that offer significant clinical benefits. InnoCare’s proprietary ADC technology platform, alongside promising precision medicine candidates like TRK inhibitor zurletrectinib (ICP-723), positions the Company to establish a strong presence in the field of solid tumor treatment.

Akeso’s First Bispecific ADC (Trop2/Nectin4 ADC) Enters Clinical Trials, Strengthening Leadership in ‘IO+ADC’ 2.0 Strategy

On July 3, 2025 Akeso, Inc. (9926.HK) ("Akeso" or the "Company") reported the successful enrollment of the first patient in its Phase Ia clinical trial for AK146D1, a bispecific Antibody-Drug Conjugate (ADC) targeting Trop2 and Nectin4. AK146D1 is Akeso’s first bispecific ADC to enter clinical trials (Press release, Akeso Biopharma, JUL 3, 2025, View Source [SID1234654239]).

AK146D1 has recently received approval from the U.S. FDA, Australia’s TGA, and China’s NMPA to begin clinical trials.

Currently, Akeso’s globally first-in-class bispecific antibodies, cadonilimab (PD-1/CTLA-4) and ivonescimab (PD-1/VEGF), have both been approved in China. The company has also rolled out a series of leading therapeutic strategies worldwide. Akeso is the only company globally with two approved cancer immunotherapy bispecific antibodies, positioning it with a strong global leadership advantage in the IO+ADC space.

Building on this, ADC therapeutics have become a key component of Akeso’s "IO+ADC" 2.0 strategy, reshaping the global treatment landscape and setting new standards for cancer care.

Dr. Yu Xia, Founder, Chairwoman, President, and CEO of Akeso, said:
"Leveraging our extensive experience in the development of multispecific antibody drugs, the advancement of innovative bispecific ADCs like AK146D1 enables the company to unlock the global clinical potential of its innovative product portfolio, expanding opportunities for the ongoing evolution of cancer therapies."

"Our globally first-in-class bispecific antibodies cadonilimab and ivonescimab have been successfully approved in China and have shown groundbreaking clinical value across various cancer treatments. This has established a strong global advantage for the company in the IO 2.0 space. Additionally, the efficient development of ADCs, particularly a series of innovative bispecific ADCs, will further strengthen our leadership in the ADC 2.0 field. As a result, after the evolution of IO + chemotherapy, IO + ADC, and IO 2.0 + ADC, Akeso has once again created a global competitive edge in "IO+ADC" 2.0. We are excited about the potential of these next-generation treatments to benefit patients worldwide."

Trop2 and Nectin4 are highly expressed in various tumor types but exhibit relatively low expression in normal tissues, making them ideal targets for ADC therapy. By targeting both Trop2 and Nectin4, AK146D1 aims to provide a broader therapeutic window and potentially overcome resistance seen with single-target ADCs, thus improving treatment efficacy.

While there are approved monoclonal ADCs targeting Trop2 and Nectin4, AK146D1 is the first bispecific ADC targeting both markers. Preclinical studies of AK146D1 have shown promising results, demonstrating strong anti-tumor activity and favorable safety profiles in tumors expressing Trop2 or Nectin4. The development of AK146D1 holds significant potential for improving treatment outcomes for cancer patients, and Akeso is excited about the potential benefits it could offer to clinicians and patients worldwide.