On May 7, 2025 Accuray Incorporated (NASDAQ: ARAY) reported that new data presented at the European Society for Radiotherapy and Oncology (ESTRO) meeting reinforces the benefits of the company’s CyberKnife System in the treatment of prostate cancer at multiple stages of the cancer journey (Press release, Accuray, MAY 7, 2025, View Source [SID1234652668]). The studies, shared at the annual congress held in Vienna, Austria, indicate the system’s accuracy and precision enable treatment of high-risk disease, as well as recurrent prostate cancer following prostatectomy, with stereotactic body radiation therapy (SBRT), expanding access to a non-invasive, short course of care to more men.

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"At this year’s ESTRO meeting important analyses of real-world evidence (RWE) underscored the benefits of our unique robotic and helical platforms, reaffirming their use as patients’ primary care option or along with other modalities such as surgery, chemotherapy or immunotherapy. Stand out studies focused on the company’s CyberKnife System for the treatment of prostate cancer, building on a robust body of clinical data supporting its use and confirming the durability and quality of life after 10 years post-treatment. We’re grateful to the clinicians who continue to evaluate our technologies and advance personalized and precise care of patients through the work that they do," said Suzanne Winter, president and CEO of Accuray.

During the meeting, Accuray hosted a symposium titled, "Integrating Advanced Techniques in Genitourinary Radiotherapy: Harmonizing Precision, Personalization, and Efficacy," attended by 350 healthcare professionals. The event featured global thought leaders who spoke on advancements in the treatment of genitourinary indications – prostate, kidney, and bladder cancers – with radiotherapy. Patient care in these areas is dynamic and evolving rapidly, with SBRT now recognized as a safe and effective alternative to conventional treatments for localized prostate cancer and evidence continuing to build for the use of SBRT in salvage prostate treatments.

CyberKnife Platform: Empowering Advances in Prostate Cancer Patient Care
A retrospective analysis titled, "Long-term outcomes and treatment efficacy in high-risk prostate cancer patients treated with stereotactic extreme hypofractionated radiotherapy," reports on 262 men over the age of 70 or with severe comorbidities who were diagnosed with either non-metastatic high-risk or very high-risk prostate cancer and received SBRT delivered with the CyberKnife System in five sessions. With a median follow-up of 39 months, investigators found that treatment with the system offers a promising option with favorable outcomes and low rates of acute and late urogenital (UG) and gastrointestinal (GI) toxicities.

"Early results from a trial on stereotactic salvage radiotherapy for macroscopic prostate bed recurrence after prostatectomy: STARR (NCT05455736)" is a prospective study evaluating the use of stereotactic salvage radiotherapy (SSRT) delivered with the CyberKnife System in five sessions. Androgen deprivation therapy was prohibited during SSRT. An early analysis of 51 patients with a median follow up of 16 months found that the CyberKnife System provides an effective and convenient option with mild toxicity for the treatment of recurrent cancer in the prostate bed following prostatectomy. The study investigators concluded, "Only mild toxicity was reported, underlining the safety of the treatment. Moreover, SSRT may be considered a convenient approach considering the shorter treatment duration if compared to standard approach."

On May 7, 2025 ViVerita Therapeutics ("ViVerita"), a US-based next-generation precision oncology company, reported a strategic research collaboration with Boehringer Ingelheim aimed at accelerating the discovery and validation of novel therapeutic targets (Press release, ViVerita Therapeutics, MAY 7, 2025, View Source [SID1234652667]). Under the terms of this collaboration, ViVerita will leverage its industry-leading in vivo CRISPR-based discovery platform to evaluate a curated set of putative targets identified by Boehringer Ingelheim, assessing their functions under physiologically relevant conditions.

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Cancer continues to be one of the leading challenges in medicine responsible for one in six deaths globally and treatment options for many cancers remain limited. One of the reasons is that drug discovery efforts are focusing on a limited number of known drug targets. In the collaboration with ViVerita, Boehringer Ingelheim aims to change this and pave the way to novel treatments for people living with cancer.

Cancer target identification has traditionally relied on studying cell lines grown in vitro. While this approach is widely adopted and has contributed to numerous important discoveries, it also has relevant shortcomings, primarily due to the inability of the in vitro systems to faithfully model key physiological conditions present in the tumor microenvironment. Conversely, many putative targets identified in vitro do not validate in vivo.

"The ViVerita platform uniquely combines transformative in vivo high-throughput genetic screening technologies with faithful disease models. It will empower the discovery of cancer driver pathway-specific targets that have so-far been challenging to address. It also enables high-throughput validation of putative targets discovered using other approaches under physiological conditions," said Xuewen Pan, Co-Founder, President and CEO of ViVerita Therapeutics. "We are very excited to work with Boehringer Ingelheim, a leader in innovative oncology research and drug development, to discover new therapies to benefit cancer patients."

The collaboration with ViVerita aligns with Boehringer Ingelheim’s strategy to identify and validate novel, clinically relevant drivers of tumors to develop innovative first-in-class treatment options for patients in need.

On May 7, 2025 Geneseeq Technology Inc., in collaboration with leading clinical institutions, reported it has developed a cutting-edge blood-based screening test that could transform early detection of pancreatic cancer-potentially saving by identifying the disease at more treatable stages (Press release, Geneseeq, MAY 7, 2025, View Source [SID1234652666]). Published in the Journal of Clinical Oncology (Impact Factor: 50.7), this study represents the most comprehensive assessment to date of using cell-free DNA (cfDNA) fragmentomics and artificial intelligence (AI) for early pancreatic cancer detection.

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Pancreatic ductal adenocarcinoma (PDAC) is one of the most lethal malignancies, largely because it is rarely caught early and diagnosed too late for curative treatment. The five-year survival rate remains around 12%, and currently tools-such as imaging and CA19-9 blood test-often miss early-stage cases. There is currently no recommended population-wide screening method for PDAC.

The new test model from Geneseeq analyzes cfDNA fragmentomics-specific patterns of DNA fragments shed into the bloodstream by cancer cells. By applying advanced machine learning algorithm to shallow whole-genome sequencing data, the test can detect subtle genomic and epigenetic changes associated with early-stage PDAC.

Key clinical results:

Achieved 93.4% sensitivity and 95.2% specificity in the training cohort
Reached 90.91-97.3% sensitivity and 92.8-94.5% specificity in multiple validation cohorts
Demonstrated strong performance even in early-state cancers
Outperformed CA19-9, especially in individuals with normal bilirubin levels
"Our cfDNA fragmentomics model offers a practical, highly accurate, and non-invasive option for detecting pancreatic cancer early," said Dr. Hua Bao, VP of R&D at Geneseeq. "It could support earlier identification of at-risk individuals, allowing timely clinical follow-up and potentially improving outcomes."

What makes this approach especially promising is its clinical feasibility. The test uses low-coverage sequencing (as little as 0.5×), making it cost-effective and suitable for broader population screening. The test also showed high stability, even with lower DNA sequencing data, and could be used to monitor high-risk patients or suspicious pancreatic lesions. The researchers also estimated that applying this test at the population level could reduce pancreatic cancer mortality by up to 27%, by catching more cancers at a treatable stage.

Further research is underway to refine the model’s application in screening programs and to validate its effectiveness in more diverse populations. Clinicians may soon have a powerful new tool to help combat one of the hardest-to-detect cancers.

On May 7, 2025 Hyundai Bioscience reported the results of its Penetrium combination preclinical studies at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held in Chicago (Press release, Hyundai Bioscience, MAY 7, 2025, View Source [SID1234652665]).

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The studies established that the repeated failures of immunotherapy and antibody treatments in cold tumors are not primarily caused by genetic resistance, as traditionally believed, but by pseudo-resistance**—a physical failure of infiltration resulting from extracellular matrix (ECM) stiffening. For the first time, Penetrium has been shown to structurally overcome this barrier.

Correcting 80 Years of Misconception: The Real Problem Is Not Genetic Mutations—It’s Pseudo-Resistance

For decades, the dominant theory behind cancer treatment failure centered around genetic mutations.

However, Hyundai Bioscience’s latest findings show that the actual obstacle lies in stiffened ECM that prevents immune cells and antibodies from reaching the tumor core.

Penetrium remodels the ECM, restoring infiltration pathways and enabling effective drug and immune cell access—providing a fundamental solution to the cold tumor problem.

Penetrium + Immunotherapy: Exceptional Results in Triple-Negative Breast Cancer (TNBC) Models

① In a TNBC mouse model, Penetrium combined with anti-PD-1 therapy led to:

A 48.3% reduction in tumor burden compared to anti-PD-1 monotherapy,
Complete elimination of metastasis observed in the combination group, which remained present in the monotherapy group.
Significantly, necrosis was induced within 3 days of Penetrium administration, and necrotic areas continued to grow with repeated dosing**—a trend not observed in the control group.

② Penetrium + Antibody Therapy: Complete Suppression of Lung Metastasis in a Metastatic Lung Cancer Model

In a metastatic lung cancer model:

Bevacizumab monotherapy showed only 33% metastasis suppression,
The Penetrium combination group recorded 0% lung metastasis at 100 mg/kg dosage.
Additionally, a marked decrease in MMP-9 and VEGF expression confirmed ECM normalization and the restoration of drug penetration routes at the molecular level.

③ Penetrium + Chemotherapy: Overcoming the Limitations of Paclitaxel

Paclitaxel monotherapy was found to paradoxically promote lung metastasis.

However, when combined with Penetrium, metastatic lesion areas were reduced by over 70–80% compared to controls.

Furthermore, suppression of MMP-9 and restoration of E-cadherin demonstrated that Penetrium not only enhances chemotherapy efficacy but also structurally blocks metastasis—without additional toxicity.

Validated in Naturally Occurring Canine Mammary Cancer Model: Stronger Responses Observed in Metastatic Tumors

In a naturally occurring canine mammary cancer model:

The Penetrium + POLYTAXEL combination reduced primary tumor volume by up to 38.7%, compared to 21.1% with monotherapy,
Metastatic lymph node lesions showed up to 78.99% tumor volume reduction.
Notably, metastatic lesions responded more strongly than primary tumors, challenging the long-standing notion that metastatic cancer is untreatable.

Proven Safety and Clinical Readiness

Penetrium was administered at less than 9% of the NOAEL dose established in a 13-week GLP-compliant toxicity study.

Its safety has also been verified in humans during a Phase 2 COVID-19 trial using the same API, niclosamide.

These results confirm Penetrium is fully prepared for human clinical application.

Official Statement from Dr. Soo-Jung Kim, Head of Research

"Penetrium is the world’s first platform to structurally solve the infiltration failure that has caused repeated failure of immunotherapy and antibody therapy in cold tumors.

Our combination study with immunotherapy exceeded expectations, and Hyundai Bioscience will soon initiate clinical trials of Penetrium combinations for both TNBC and metastatic lung cancer."

— Dr. Soo-Jung Kim, Head of Research, Hyundai Bioscience

Clinical Expansion Underway

Hyundai Bioscience is currently:

Conducting a Phase 1 trial for prostate cancer in South Korea,
Preparing an investigator-initiated trial for acute myeloid leukemia (AML) in France.
Penetrium: Launching a Tumor-Agnostic Cancer Treatment Platform

Penetrium ECM Remodeling Therapy is evolving into a tumor-agnostic universal cancer treatment platform with applicability not only to TNBC and metastatic lung cancer, but also to pancreatic, gallbladder, ovarian cancers, and hematological malignancies such as AML.

This breakthrough research is the first to structurally resolve infiltration failure in cold tumors, while also proving that metastatic cancers can be effectively treated**—a historic achievement in oncology.

On May 7, 2025 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported its first quarter 2025 financial results and provided a business update (Press release, argenx, MAY 7, 2025, View Source [SID1234652663]).

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"We continue to execute on our bold innovation agenda, guided by our ‘Vision 2030’ to reach 50,000 patients across 10 labeled indications," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "We remain committed to delivering meaningful outcomes with VYVGART by setting a new benchmark for sustained efficacy and safety, and generating data that matter most to improving the lives of patients. This strategy has driven strong launch fundamentals to date, and we see consistent patient and prescriber expansion in both gMG and CIDP. Looking forward, we have several reasons to be confident in our growth trajectory. We are thrilled to bring even more optionality to gMG and CIDP patients with the recent approval of our pre-filled syringe for self-injection in the United States, receiving an optimal label that supports our ability to reach patients earlier in the treatment paradigm. In line with our ‘Vision 2030’, we are advancing 10 Phase 2 and 10 Phase 3 studies across efgartigimod, empasiprubart and ARGX-119, creating significant opportunity to expand into new therapeutic areas and reach broader patient populations. By year end, we expect key insights from proof-of-concept and registrational studies across many of these programs, while continuing to progress four IND candidates that reflect the depth and diversity of our pipeline."

Advancing Vision 2030

argenx has established its strategic priorities to advance "Vision 2030", aiming to treat 50,000 patients globally with its medicines, secure 10 labeled indications across all approved medicines, and advance five pipeline candidates into Phase 3 development by 2030.

Expand the global VYVGART opportunity and launch VYVGART SC as a pre-filled syringe

VYVGART (IV: efgartigimod alfa-fcab and SC: efgartigimod alfa and hyaluronidase-qvfc) is a first-and-only targeted IgG Fc-antibody fragment approved in three indications, including generalized myasthenia gravis (gMG) globally, primary immune thrombocytopenia (ITP) in Japan, and chronic inflammatory demyelinating polyneuropathy (CIDP) in the U.S., Japan and China. The VYVGART-SC pre-filled syringe (PFS) is now approved for use in the U.S. and EU. argenx is well-positioned to sustain commercial growth through 2025, driven by global expansion, earlier treatment adoption, and the launch of the PFS to support growth momentum in both gMG and CIDP. In addition to bringing VYVGART to more patients early in the treatment paradigm, argenx is working to reach broader MG populations with ongoing studies in seronegative, ocular, and pediatric MG

• Generated global product net sales (inclusive of both VYVGART and VYVGART SC) of $790 million in the first quarter of 2025

◦ Strong underlying fundamentals across key patient and prescriber metrics with 99% product net sales growth year-over-year from first quarter 2024, and 7% product net sales growth from fourth quarter 2024

• Multiple regulatory decisions on approval for PFS completed or underway:

◦ First patients treated with VYVGART-SC PFS for self-injection in the U.S. and Germany following regulatory approval

◦ Received positive recommendation from Committee for Medicinal Products for Human Use (CHMP) of European Medicines Agency (EMA) for VYVGART-SC (PFS and vial) for CIDP

◦ PFS decision on approval for gMG and CIDP expected in Japan and Canada by end of year

• Evidence generation through Phase 4 and label-enabling studies in MG, CIDP and ITP:

◦ Topline results expected in second half of 2025 for seronegative gMG (ADAPT-SERON) and first half of 2026 for ocular and pediatric MG (ADAPT-OCULUS, JR)

◦ Topline results from Phase 4 switch study to inform treatment decisions when switching patients on IVIg to VYVGART SC in CIDP expected in second half of 2025 and to be presented at an upcoming medical meeting

◦ ADVANCE-NEXT topline results expected in second half of 2026 to support FDA submission of VYVGART IV for primary ITP

Execute 10 registrational and 10 proof-of-concept studies across efgartigimod, empasiprubart and ARGX-119 to advance the next wave of launches

argenx continues to demonstrate breadth and depth within its immunology pipeline, advancing multiple first-in-class product candidates with potential across high-need indications. argenx is solidifying its leadership in FcRn biology with efgartigimod, complement inhibition with empasiprubart and in the role of MuSK at the neuromuscular junction with ARGX-119.

Efgartigimod Development

Efgartigimod is being evaluated in 15 severe autoimmune diseases (including MG, CIDP, and ITP), exploring the significance of FcRn biology across neurology and rheumatology indications, as well as new therapeutic areas.

• Registrational studies are currently ongoing in three subsets of myositis, thyroid eye disease (TED), and Sjögren’s disease.

◦ Topline results from ALKIVIA study evaluating three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and dermatomyositis (DM)) expected in second half of 2026

◦ Topline results from two registrational UplighTED studies (TED) expected in second half of 2026

◦ Topline results from registrational UNITY study (Sjögren’s disease) expected in 2027

• Proof-of-concept studies ongoing in lupus nephritis (LN), systemic sclerosis (SSc) and antibody mediated rejection (AMR); topline results expected for LN in fourth quarter of 2025, SSc in second half of 2026, and AMR in 2027

Empasiprubart Development

Empasiprubart is currently being evaluated in four indications, including two registrational studies in multifocal motor neuropathy (MMN) and CIDP, and proof-of-concept studies in delayed graft function (DGF) and DM.

• Topline results from registrational EMPASSION study (MMN) evaluating empasiprubart head-to-head versus IVIg expected in second half of 2026

• Registrational EMVIGORATE study in CIDP evaluating empasiprubart head-to-head versus IVIg expected to start in first half of 2025

• Topline results expected for DGF in second half of 2025 and for DM in first half of 2026

ARGX-119 Development

ARGX-119 is being evaluated in congenital myasthenic syndromes (CMS), amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA).

• Phase 1b proof-of-concept study ongoing in CMS; topline results expected in second half of 2025

• Phase 2a proof-of-concept study ongoing in ALS; topline results expected in first half of 2026

• SMA proof-of-concept study on track to start in 2025

Advance four new pipeline molecules and generate sustainable value through continued investment in Immunology Innovation Program

argenx continues to invest in its Immunology Innovation Program (IIP) to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213, targeting FcRn and further solidifying argenx’s leadership in this biology; ARGX-121, a first-in-class molecule targeting IgA; ARGX-109, targeting IL-6, which plays an important role in inflammation, and a fourth pipeline candidate, a first-in-class sweeping antibody for which the target has not yet been disclosed. Phase 1 results from ongoing ARGX-109 study expected in second half of 2025, and from ongoing ARGX-213 study and ARGX-121 study expected in first half of 2026.

FIRST QUARTER 2025 FINANCIAL RESULTS

argenx SE

UNAUDITED CONDENSED CONSOLIDATED INTERIM STATEMENTS OF PROFIT OR LOSS

DETAILS OF THE FINANCIAL RESULTS

Total operating income for the three months ended March 31, 2025, was $807 million compared to $413 million for the same period in 2024, and consists of:

• Product net sales of VYVGART and VYVGART SC for the three months ended March 31, 2025, were $790 million compared to $398 million for the same period in 2024.

• Other operating income for the three months ended March 31, 2025, was $17 million compared to $12 million for the same period in 2024. The other operating income primarily relates to research and development tax incentives and payroll tax rebates.

Total operating expenses for the three months ended March 31, 2025, were $668 million compared to $506 million for the same period in 2024, and mainly consists of:

• Cost of sales for the three months ended March 31, 2025, was $81 million compared to $43 million for the same period in 2024. The cost of sales was recognized with respect to the sale of VYVGART and VYVGART SC.

• Research and development expenses for the three months ended March 31, 2025, were $309 million compared to $225 million for the same period in 2024. The expenses mainly relate to:

◦ the clinical development and expansion of efgartigimod in 15 severe autoimmune diseases;

◦ the ramp-up of studies for our development of empasiprubart into MMN, DGF, DM and CIDP;

◦ the investments for ARGX-119 in proof-of-concept studies ongoing in ALS and CMS; and

◦ other discovery and preclinical pipeline candidates.

• Selling, general and administrative expenses for the three months ended March 31, 2025, were $276 million compared to $236 million for the same period in 2024. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to global commercialization of the VYVGART franchise, and personnel expenses.

Financial income for the three months ended March 31, 2025, was $37 million compared to $39 million for the same period in 2024.

Exchange gains for the three months ended March 31, 2025, were $27 million compared to exchange losses of $19 million for the same period in 2024. Exchange gains and losses are mainly attributable to unrealized exchange rate gains or losses on the cash, cash equivalents and current financial assets denominated in Euro.

Income tax for the three months ended March 31, 2025, consisted of $33 million of income tax expense compared to income tax benefit of $13 million for the same period in 2024. Income tax expense for the three months ended March 31, 2025, consists of $29 million of current income tax expense and $4 million of deferred tax expense, compared to $6 million of current income tax expense and $19 million of deferred tax benefit for the comparable prior period.

Profit for the period of three months ended March 31, 2025, was $169 million compared to a loss for the period of $62 million in 2024. The profit per share was $2.78 compared to a loss per share of $1.04 for the three months ended March 31, 2025 and 2024, respectively.

FINANCIAL GUIDANCE

The financial guidance on the combined selling, general and administrative expenses and research and development expenses remains unchanged at approximately $2.5 billion.

EXPECTED 2025 FINANCIAL CALENDAR

• May 27, 2025: Annual General Meeting of Shareholders in Amsterdam, the Netherlands

• July 31, 2025: Half Year and Second Quarter 2025 Financial Results and Business Update

• October 30, 2025: Q3 2025 Financial Results and Business Update

CONFERENCE CALL DETAILS

The first quarter 2025 financial results and business update will be discussed during a conference call and webcast presentation today at 2:30 pm CET/8:30 am ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website.

Dial-in numbers:

Please dial in 15 minutes prior to the live call.

Belgium 32 800 50 201
France 33 800 943355
Netherlands 31 20 795 1090
United Kingdom 44 800 358 0970
United States 1 888 415 4250
Japan 81 3 4578 9081
Switzerland 41 43 210 11 32

This press release contains inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation (Regulation 596/2014).