On June 30, 2025 Kazia Therapeutics reported its half yearly results (Filing, 3 mnth, DEC 31, Kazia Therapeutics, 2024, JUN 30, 2025, View Source [SID1234654231]).

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On June 30, 2025 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:​HCM; HKEX:​13) reported that the New Drug Application ("NDA") for the combination of ORPATHYS (savolitinib) and TAGRISSO (osimertinib) has been granted approval by the China National Medical Products Administration ("NMPA") for the treatment of patients with locally advanced or metastatic epidermal growth factor receptor ("EGFR") mutation-positive non-squamous non-small cell lung cancer ("NSCLC") with MET amplification after disease progression on EGFR tyrosine kinase inhibitor ("TKI") therapy (Press release, Hutchison China MediTech, JUN 30, 2025, View Source [SID1234654217]). ORPATHYS is an oral, potent and highly selective MET TKI. TAGRISSO is a third-generation, irreversible EGFR TKI. This approval also triggers a US$11 million milestone payment from AstraZeneca, which markets both ORPATHYS and TAGRISSO in China.

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ORPATHYS was the first selective MET inhibitor approved in China, indicated for adult patients with locally advanced or metastatic NSCLC with MET exon 14 skipping alteration. This new approval by the NMPA was based on data from the SACHI Phase III trial of the ORPATHYS and TAGRISSO combination (NCT05015608), having met the pre-defined primary endpoint of progression-free survival ("PFS") in a pre-planned interim analysis. Primary results were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting in June 2025. In 2024 the NMPA designated the combination as a Breakthrough Therapy, and in 2025 it granted the NDA Priority Review.

Professor Shun Lu, Chief of the Shanghai Lung Cancer Center at Shanghai Chest Hospital, School of Medicine, Shanghai Jiaotong University, and Principal Investigator of the SACHI trial, said, "The approval of the ORPATHYS and TAGRISSO combination is a significant milestone in addressing the complex challenges of lung cancer treatment in China, where the EGFR mutation is common amongst NSCLC patients. For patients who develop MET amplification after progressing on EGFR inhibitors, the combination offers a continued all-oral, chemotherapy-free approach to tackle a critical resistance mechanism. As a researcher and clinician, I am excited about the opportunity to offer this targeted therapy to patients, improving their treatment outcomes and quality of life through innovative research."

"The NMPA approval marks an important step forward in our mission to address MET-driven progression following first-line EGFR-inhibitor therapy in NSCLC patients." said Dr Weiguo Su, Chief Executive Officer and Chief Scientific Officer of HUTCHMED. "Our collaboration with AstraZeneca, built on a shared vision to transform oncology care, has been crucial in reaching this achievement. We are committed to advancing this partnership, continuing our research into further treatment settings, and bringing this innovative combination to patients in China and beyond."

Ms Mary Guan, General Manager of AstraZeneca China Oncology Business, said: "This milestone marks the third indication of ORPATHYS approved in China, bringing a new treatment option to lung cancer patients who develop MET amplification after progressing on EGFR inhibitor therapy. Through our partnership with HUTCHMED, we are committed to expanding the reach of the ORPATHYS and TAGRISSO combination to address progression on first-line therapy and help even more patients with this form of lung cancer."

In the intention to treat (ITT) population of the SACHI trial, the ORPATHYS and TAGRISSO combination reduced the risk of disease progression by 66% with a median PFS of 8.2 months, compared to 4.5 months for chemotherapy, as assessed by investigators. The independent review committee (IRC) also reported a 60% risk reduction in disease progression, with a median PFS of 7.2 months versus 4.2 months, respectively. The safety profile of the ORPATHYS and TAGRISSO combination was tolerable and no new safety signals were observed. Treatment-emergent adverse events of Grade 3 or above occurred in 57% of patients in both the ORPATHYS plus TAGRISSO group and the chemotherapy group, suggesting a favorable safety profile.

About NSCLC and MET aberrations

Lung cancer is the leading cause of cancer death, accounting for about one-fifth of all cancer deaths.[1] Lung cancer is broadly split into NSCLC and small cell lung cancer, with 80-85% classified as NSCLC.[2] The majority of NSCLC patients (approximately 75%) are diagnosed with advanced disease, and approximately 10-15% of NSCLC patients in the US and Europe and 30-40% of patients in Asia have EGFR-mutated ("EGFRm") NSCLC.[3],[4],[5],[6]

MET is a tyrosine kinase receptor that has an essential role in normal cell development. MET overexpression and/or amplification can lead to tumor growth and the metastatic progression of cancer cells, and is one of the mechanisms of acquired resistance to EGFR TKI for metastatic EGFRm NSCLC.

About ORPATHYS

ORPATHYS (savolitinib) is an oral, potent and highly selective MET TKI that has demonstrated clinical activity in advanced solid tumors. It blocks atypical activation of the MET receptor tyrosine kinase pathway that occurs because of mutations (such as exon 14 skipping alterations or other point mutations), gene amplification or protein overexpression.

ORPATHYS is approved in China and is marketed by AstraZeneca for the treatment of adult patients with locally advanced or metastatic NSCLC with MET exon 14 skipping alteration, representing the first selective MET inhibitor approved in China. It is currently under clinical development for multiple tumor types, including lung, kidney, and gastric cancers as a single treatment and in combination with other medicines.

About TAGRISSO

TAGRISSO (osimertinib) is a third-generation, irreversible EGFR-TKI with proven clinical activity in NSCLC, including against central nervous system (CNS) metastases. TAGRISSO (40mg and 80mg once-daily oral tablets) has been used to treat nearly 800,000 patients across its indications worldwide and AstraZeneca continues to explore TAGRISSO as a treatment for patients across multiple stages of EGFRm NSCLC.

There is an extensive body of evidence supporting the use of TAGRISSO as standard of care in EGFRm NSCLC. TAGRISSO improved patient outcomes in early-stage disease in the ADAURA Phase III trial, locally advanced disease in the LAURA Phase III trial, late-stage disease in the FLAURA Phase III trial, and with chemotherapy in the FLAURA2 Phase III trial.

About ORPATHYS and TAGRISSO Combination Development in EGFR-mutated NSCLC

Among patients who experience disease progression following treatment with a third-generation EGFR TKI, approximately 15-50% present with MET aberration, depending on the sample type, detection method and assay cut-off used. TAGRISSO is a third-generation, irreversible EGFR-TKI with proven clinical activity in NSCLC, including against central nervous system metastases. Treatment with ORPATHYS in combination with TAGRISSO has been studied extensively in these patients in the TATTON (NCT02143466) and SAVANNAH (NCT03778229) studies. The encouraging results led to the initiation of several Phase III trials in this setting including the SACHI trial in China (NCT05015608) and the global SAFFRON trial (NCT05261399), as well as the SANOVO trial in China (NCT05009836).

This combination represents a promising chemotherapy-free oral treatment strategy to address mechanisms of resistance in this advanced setting. Positive data from the SACHI randomized Phase III trial led to the filing of a second NDA in China. Strong data from the SAVANNAH single-arm Phase II study was recently presented at the European Lung Cancer Congress (ELCC) in March 2025 demonstrated high, clinically meaningful and durable objective response rate (ORR), with consistent safety results. The SAFFRON randomized Phase III trial is progressing. Following AstraZeneca’s consultation with the US Food and Drug Administration ("FDA"), we look forward to completing the SAFFRON trial as soon as possible to support potential US and other global registration filings.

SACHI: The SACHI China Phase III trial met the primary endpoint of PFS during its interim analysis towards the end of 2024 and a NDA was accepted and granted Breakthrough Therapy Designation and Priority Review status in China in December 2024. SACHI evaluated the combination of ORPATHYS and TAGRISSO for the treatment of patients with EGFRm, MET-amplified locally advanced or metastatic NSCLC after progression on EGFR TKI compared to platinum-based doublet chemotherapy. Results were presented at the ASCO (Free ASCO Whitepaper) Annual Meeting in June 2025.

SAFFRON: In 2023, ORPATHYS and TAGRISSO received Fast Track Designation from the US FDA in this setting. The global SAFFRON Phase III trial is currently ongoing to assess the ORPATHYS plus TAGRISSO combination versus platinum-based doublet chemotherapy in patients with EGFRm, MET-overexpressed and/or amplified, locally advanced or metastatic NSCLC following progression on treatment with TAGRISSO. Patients are being prospectively selected using the high MET level cut-off identified in SAVANNAH.

On June 30, 2025 Iksuda Therapeutics (Iksuda), the developer of class-leading antibody drug conjugates (ADCs) with clinically validated tumour-selective payload release formats, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for IKS014, a human epidermal growth factor receptor 2 (HER2) ADC targeting patients with advanced HER2+ solid tumours, enabling the expansion of ongoing clinical trials (Press release, Iksuda Therapeutics, JUN 30, 2025, View Source [SID1234654184]).

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IKS014 is currently being investigated in an open-label phase 1 dose-escalation study (View Source) designed to evaluate the safety and tolerability of increasing dose levels of IKS014 and establish a recommended phase 2 dose. Preliminary data from this study has demonstrated promising clinical activity across multiple tumour types, including breast, ovarian, gallbladder, and oesophageal cancers, and including patients who have relapsed after prior treatment with Enhertu. Gaining access to U.S. centres for the dose expansion stage of this phase 1 trial will enable efficient confirmation of the role of IKS014 in this important patient sub-set.

Dr Dave Simpson, Chief Executive Officer, Iksuda Therapeutics, said:

"The FDA clearance of our IND application for IKS014 represents a significant milestone in our mission to address the critical unmet needs of patients with HER2-positive cancers, particularly those who have exhausted current standard-of-care options. Early clinical data has been extremely encouraging, and we are excited to expand our program to reach more patients.

"The early efficacy signals that have been observed, particularly in patients that have relapsed after receiving prior treatments of Kadcyla and Enhertu, suggest IKS014 could potentially offer a new treatment option for patients who currently have limited alternatives. We look forward to working with our clinical partners to further evaluate the potential of IKS014."

The first stage of this phase 1 trial, to determine the recommended phase 2 dose for dose expansion, is nearing completion. The expansion phase will assess several patient cohorts including those with HER2-positive breast cancer who are refractory to or cannot tolerate Enhertu, patients with HER2-low breast cancer and patients with HER2-positive positive gastric cancer. The IND clearance will allow Iksuda to access patients across sites in the United States, alongside sites in Australia, New Zealand, and Singapore. The phase 1 trial is expected to complete in 2H 2026.

About IKS014

IKS014 is a potential best-in-class antibody drug conjugate, benefiting from tumour selective activation and release of the cytotoxic agent monomethyl auristatin F (MMAF). In preclinical trials, it displayed impressive activity in high- and low-HER2 expressing tumours with a favourable Therapeutic Index vs other HER2-directed drugs. Iksuda gained exclusive world-wide rights (excluding Greater China and South Korea) to IKS014 from LigaChem Biosciences (View Source).

On June 30, 2025 OS Therapies (NYSE-A: OSTX) ("OS Therapies" or "the Company"), a clinical-stage immunotherapy and Antibody Drug Conjugate (ADC) biopharmaceutical company, reported positive 1-year event free survival (EFS), overall survival and safety clinical trial data updates from the 40 patient treatment arm of its Phase 2b clinical trial of immunotherapy candidate OST-HER2 in the prevention or delay of recurrence in fully resected, lung metastatic osteosarcoma (Press release, OS Therapies, JUN 30, 2025, View Source [SID1234654183]). In data presented by Principal Investigator Dr. Damon Reed at the MIB Factor Osteosarcoma Conference held in Salt Lake City, Utah on Saturday June 28, 2025 the following data updates were released:

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35% (14 out of 40) of OST-HER2-treated patients achieved 1-year event free survival (EFS) compared with 20% of patients from peer-reviewed publication selected by Children’s Oncology Group (COG)1 as equivalent to osteosarcoma patient subpopulation enrolled (p = 0.0194);
A total of 13 out of 40 patients were reported to have experienced severe adverse events (SAEs) during the trial, of which 7 patients’ adverse events were treatment-associated adverse events (TSAEs). All of the 7 patients’ TSAEs were grade 3 TSAEs; 0 patients experienced grade 4 or grade 5 TSAEs. None of the patients for whom TSAEs were reported discontinued the study.
"The updated OST-HER2 data presented at MIB Factor that showed EFS data statistically significantly favoring OST-HER2 treated patients when compared with the leading peer-reviewed publications on historical event free survival outcomes in this subset of the pulmonary metastatic osteosarcoma patient population," said Dr. Robert Petit, Chief Medical & Scientific Officer of OS Therapies. "The favorable safety profile of OST-HER2 compared with standard of care is also an important quality of life factor when assessing potential new treatment options for this difficult to treat patient population."

Additionally, the Company reported a regulatory update regarding the submission of preliminary external control data and comprehensive plans for the finalization of this data package to the US Food & Drug Administration’s ("FDA"). In accordance with the provisions of prevailing Guidance to Industry – including ICH E10 Choice of Control Group in Clinical Trials; Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products 2019; and Rare Diseases – Natural History Studies for Drug Development 2019 – to support a Biologics Licensing Application (BLA) marketing authorization under the US Food & Drug Administration’s ("FDA") Accelerated Approval Program ("Accelerated Approval").

Based on guidance from FDA from its recent Type D Meeting and the submission of subsequent responses to FDA arising from the Meeting, the Company is providing updated superiority peer-reviewed historical external control data, matched external control data and/or real-world external control data. This data, individually or collectively at FDA’s discretion, may be sufficient for FDA to support a marketing authorization in a setting where randomization may not be feasible. Such an approach is common in rare diseases – such as Prevention or Delay of Recurrence in Fully Resected, Lung Metastatic Osteosarcoma – for which the company holds US FDA Orphan Disease Designation (ODD), Fast Track and Rare Pediatric Disease Designation (RPDD).

The Company has submitted clarifying information to FDA Type D Meeting feedback requests regarding proposed statistical methods to be used in FDA’s assessment of external control arm(s) suitable to support Accelerated Approval. The Company is awaiting response from FDA to its End of Phase 2 meeting request where, if granted, the Company will seek full alignment on the individual and/or collective data sets necessary to support Accelerated Approval. The Company has collected multiple sources of potential case matched external control and real-world data potentially suitable, individually or together with historical external control data at FDA’s discretion, to support Accelerated Approval.

"The feedback received from FDA regarding the use of external control comparators in settings where placebo-controlled randomization trials are not feasible increases the avenues available for OST-HER to gain Accelerated Approval," said Paul Romness, Chairman & CEO of OS Therapies. "We have now responded to the follow-up questions from our recent positive Type D Meeting with FDA positioning us to soon be granted an End of Phase 2 meeting. We were also very pleased with the reception the presentation received from the osteosarcoma community."

Mr. Romness continued: "Recent interactions we have had with FDA are consistent with public statements from FDA leadership prioritizing the safety profile of potential new products under consideration for Accelerated Approval that are intended to treat deadly rare diseases where randomized trials may not be feasible, especially in pediatric cancer where no alternative treatment options are approved."

Concurrent with this announcement, the United Kingdom’s (UK) Medicines and Healthcare products Regulatory Agency (MHRA) has agreed to support the Company in utilizing its Clinical Practice Research Datalink (CRPD) to assist the Company in developing data necessary to support potential worldwide marketing authorizations – including in the UK, US and EU – for OST-HER2. The CPRD collects anonymized patient data from a network of healthcare practitioners and institutions across the UK. Primary care data are linked to a range of other health related data to provide a longitudinal, representative population health dataset. The data encompass 60 million patients, including 18 million currently registered patients. Follow-up from the Company’s July 31st, 2025 Scientific Advice Meeting with MHRA is expected by mid-August 2025. (View Source)

OST-HER2 has received Rare Pediatric Disease Designation (RPDD) for osteosarcoma from the US FDA. 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 movie is available via streaming on PBS’ website.

The most recent data updated regarding the OST-HER2 canine osteosarcoma program is available at this link.

On June 30, 2025 NeoGenomics, Inc. (NASDAQ: NEO), a leading provider of oncology diagnostic solutions that enable precision medicine, reported the launch of PanTracer Tissue, a next-generation solid tumor profiling assay, including the option to add testing for homologous recombination deficiency (HRD) (Press release, NeoGenomics Laboratories, JUN 30, 2025, View Source [SID1234654182]). These new options can provide faster, actionable insights to help physicians navigate complex treatment decisions more confidently.

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PanTracer Tissue evaluates over 500 cancer-related genes and aligns with clinical guidelines, covering key biomarkers recommended for therapy selection and additional genomic insights for clinical trial enrollment. Results may be delivered in as little as 8 days, enabling physicians to rapidly initiate treatment strategies. Minimal specimen requirements make it suitable for a wide range of tumor types and practice settings. The assay builds on the company’s tissue-based CGP platform, previously known as NeoComprehensive Solid Tumor.

The addition of PanTracer Tissue + HRD offers enhanced tumor profiling by incorporating homologous recombination deficiency analysis into a single, guideline-aligned test for ovarian cancer. The PanTracer Tissue + HRD offering includes BRCA mutation status and a genomic instability score—critical biomarkers that can help guide the use of PARP inhibitors and other therapies targeting DNA repair pathways. The combined approach streamlines ordering and can shorten time to actionable results by capturing a broader range of clinically relevant genomic alterations, gene fusions, and DNA repair deficiencies.

"With PanTracer Tissue and PanTracer Tissue + HRD we’re unlocking a more complete genomic view from a single sample, giving physicians clearer answers, sooner," said Warren Stone, President and Chief Operating Officer at NeoGenomics. "With the addition of PanTracer Tissue + HRD, we are expanding our portfolio to address the unmet need for ovarian cancer therapy selection with the objective of improving patient care."

PanTracer Tissue and PanTracer Tissue + HRD are part of NeoGenomics’ broader portfolio of precision oncology solutions designed to improve patient outcomes through high-quality, guideline-aligned testing. Revealed at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, the continually expanding PanTracer portfolio reflects the company’s dedicated focus on advancing diagnostic tools that support more informed, personalized care.