ViVerita Therapeutics Announces Research Collaboration with Boehringer Ingelheim to Accelerate Discovery of Novel Cancer Targets

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.

Geneseeq Unveils Groundbreaking Blood Test for Early Detection of Pancreatic Cancer

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.

Penetrium™ Breaks Oncology Barriers: The Solution to Cold Tumors and Metastatic Cancer Unveiled at AACR 2025

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.

argenx Reports First Quarter 2025 Financial Results and Provides Business Update

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).

Merus Announces Financial Results for the First Quarter 2025 and Provides Business Update

On May 7, 2025 Merus N.V. (Nasdaq: MRUS) (Merus, the Company, we, or our), an oncology company developing innovative, full-length multispecific antibodies and antibody drug conjugates (Biclonics, Triclonics and ADClonics), reported financial results for the first quarter and provided a business update (Press release, Merus, MAY 7, 2025, View Source [SID1234652662]).

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"We are very much looking forward to sharing the robust updated interim phase 2 data, on the entire 45 patient data set at 2025 ASCO (Free ASCO Whitepaper). We believe based on these interim data that petosemtamab continues to demonstrate substantial clinical activity superior to historical controls based on the magnitude and consistency of efficacy across ORR, PFS and OS in the overall population and within important subgroups of HPV disease and PD-L1 expression levels," said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. "Additionally, I’m thrilled by the team’s execution in our two phase 3 trials and expect both trials to be substantially enrolled by year end 2025."

Petosemtamab (MCLA-158: EGFR x LGR5 Biclonics): Solid Tumors
LiGeR-HN1 phase 3 trial in 1L recurrent/metastatic (r/m) head and neck squamous cell carcinoma (HNSCC) and LiGeR-HN2 phase 3 trial in 2/3L r/m HNSCC enrolling – with both trials expected to be substantially enrolled by YE25; clinical update on phase 2 trial in combination with pembrolizumab in 1L PD-L1+ HNSCC at 2025 ASCO (Free ASCO Whitepaper); phase 2 trial in 1L, 2L and 3L+ metastatic colorectal cancer (mCRC) enrolling; mCRC initial clinical data planned for 2H25

An updated analysis of the interim clinical data from the phase 2 trial of petosemtamab with pembrolizumab as 1L treatment of PD-L1+ r/m HNSCC will be presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO) (Free ASCO Whitepaper) as detailed in our press release, Merus Announces Abstract Accepted for Presentation at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting. The presentation will include data on the entire 45 patient dataset and follows the early clinical efficacy and encouraging safety data previously presented at 2024 ASCO (Free ASCO Whitepaper), which was detailed in our 2024 press release, Merus’ Petosemtamab in Combination with Pembrolizumab Interim Data Demonstrates Robust Response Rate and Favorable Safety Profile in 1L r/m HNSCC (May 28, 2024).

Merus will hold a conference call and webcast for investors on Thursday, May 22, 2025 at 5:30 p.m. ET. A replay will be available after the completion of the call in the Investors and Media section of our website for a limited time.

Date & Time: May 22, 2025 at 5:30 p.m. ET
Webcast link: Available on our website
Dial-in: Toll Free: (800) 715-9871 / International: (646) 307-1963
Conference ID: 7517301 or Merus NV call

In February 2025, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation (BTD) to petosemtamab in combination with pembrolizumab for the first-line treatment of adult patients with recurrent or metastatic programmed death-ligand 1 (PD-L1) positive HNSCC with combined positive score (CPS) ≥ 1. This designation was detailed in our press release, Petosemtamab granted Breakthrough Therapy designation by the U.S. FDA for 1L PD-L1 positive head and neck squamous cell carcinoma (February 18, 2025). BTD was also granted for petosemtamab monotherapy for the treatment of patients with recurrent or metastatic HNSCC whose disease has progressed following treatment with platinum based chemotherapy and an anti-programmed cell death receptor-1 (PD-1) or anti-programmed death ligand 1 (PD-L1) antibody, detailed in our press release, Petosemtamab granted Breakthrough Therapy Designation by the U.S. FDA (May 13, 2024).

Merus provided updated interim clinical data on petosemtamab in 2L+ r/m HNSCC at the European Society for Medical Oncology Asia Congress, demonstrating a 36% response rate among 75 evaluable patients. The oral presentation was detailed in our press release, Merus’ Petosemtamab Monotherapy Interim Data Continues to Demonstrate Clinically Meaningful Activity in 2L+ r/m HNSCC (Dec. 7, 2024).

LiGeR-HN1, a phase 3 trial evaluating the efficacy and safety of petosemtamab in combination with pembrolizumab in 1L PD-L1+ (CPS≥1) r/m HNSCC compared to pembrolizumab, and LiGeR-HN2, a phase 3 trial evaluating the efficacy and safety of petosemtamab in 2/3L HNSCC compared to standard of care, are enrolling and we expect both trials to be substantially enrolled by YE25. Further, the Company manufactures both drug substance and drug product in the United States and Europe, with a plan to focus on potential commercial manufacturing in the United States.

Merus believes a randomized registration trial in HNSCC with an overall response rate endpoint could potentially support accelerated approval and the overall survival results from the same study could potentially verify its clinical benefit to support regular approval for the Company’s phase 3 trial in 1L, and in phase 3 trial in 2/3L HNSCC.

A phase 2 trial evaluating petosemtamab in combination with standard chemotherapy in 1L and 2L mCRC, and as monotherapy in heavily pretreated (3L+) mCRC, is enrolling. We expect to provide initial clinical data for petosemtamab in mCRC in 2H25.

BIZENGRI (zenocutuzumab-zbco: HER2 x HER3 Biclonics)
Approved by FDA for adults with pancreatic adenocarcinoma or non–small cell lung cancer (NSCLC) that are advanced unresectable or metastatic and harbor a neuregulin 1 (NRG1) gene fusion who have disease progression on or after prior systemic therapy

Merus has exclusively licensed to Partner Therapeutics, Inc. (PTx) the right to commercialize BIZENGRI for the treatment of NRG1+ cancer in the U.S. This was detailed in our press release, Merus and Partner Therapeutics Announce License Agreement for the U.S. Commercialization of Zenocutuzumab in NRG1 Fusion-Positive Cancer (December 2, 2024).

MCLA-129 (EGFR x c-MET Biclonics): Solid Tumors
Investigation of MCLA-129 is ongoing in METex14 NSCLC; phase 2 trial in combination with chemotherapy in 2L+ EGFR mutant (EGFRm) NSCLC enrolling

MCLA-129 is subject to a collaboration and license agreement with Betta Pharmaceuticals Co. Ltd. (Betta), which permits Betta to develop MCLA-129, and potentially commercialize exclusively in China, while Merus retains global rights outside of China.

Collaborations

Incyte Corporation
Since 2017, Merus has been working with Incyte Corporation (Incyte) under a global collaboration and license agreement focused on the research, discovery and development of bispecific antibodies utilizing Merus’ proprietary Biclonics technology platform. For each program under the collaboration, Merus receives reimbursement for research activities and is eligible to receive potential development, regulatory and commercial milestones and sales royalties for any products, if approved.

Eli Lilly and Company
In January 2021, Merus and Eli Lilly and Company (Lilly) announced a research collaboration and exclusive license agreement to develop up to three CD3-engaging T-cell re-directing bispecific antibody therapies utilizing Merus’ Biclonics platform and proprietary CD3 panel along with the scientific and rational drug design expertise of Lilly. The collaboration is progressing well with three programs advancing through preclinical development.

Gilead Sciences
In March 2024, Merus and Gilead Sciences announced a collaboration to discover novel antibody based trispecific T-cell engagers using Merus’ patented Triclonics platform. Under the terms of the agreement, Merus will lead early-stage research activities for two programs, with an option to pursue a third. Gilead will have the right to exclusively license programs developed under the collaboration after the completion of select research activities. If Gilead exercises its option to license any such program from the collaboration, Gilead will be responsible for additional research, development and commercialization activities for such program.

Ono Pharmaceutical
In 2018, the Company granted Ono Pharmaceutical Co., Ltd. (Ono) an exclusive, worldwide, royalty-bearing license, with the right to sublicense, research, test, make, use and market a limited number of bispecific antibody candidates based on Merus’ Biclonics technology platform directed to an undisclosed target combination.

Biohaven
In January 2025, Merus and Biohaven announced a research collaboration and license agreement to co-develop three novel bispecific antibody drug conjugates (ADCs), leveraging Merus’ leading Biclonics technology platform, and Biohaven’s next-generation ADC conjugation and payload platform technologies. Under the terms of the agreement, Biohaven is responsible for the preclinical ADC generation of three Merus bispecific antibodies under mutually agreed research plans. The agreement includes two Merus bispecific programs generated using the Biclonics platform, and one program under preclinical research by Merus. Each program is subject to mutual agreement for advancement to further development, with the parties then sharing subsequent external development costs and commercialization, if advanced.

Cash Runway, existing cash, cash equivalents and marketable securities expected to fund Merus’ operations into 2028

As of March 31, 2025, Merus had $638 million cash, cash equivalents and marketable securities. Based on the Company’s current operating plan, the existing cash, cash equivalents and marketable securities are expected to fund Merus’ operations into 2028.

First Quarter 2025 Financial Results
Collaboration revenue for the three months ended March 31, 2025 increased by $18.6 million as compared to the three months ended March 31, 2024, primarily as a result of commercial material revenue this quarter and higher deferred revenue amortization.

Research and development expense for the three months ended March 31, 2025 increased by $41.5 million as compared to the three months ended March 31, 2024. The increase is primarily driven by an increase of $35.6 million in clinical trial support provided by contract manufacturing and development organizations and contract research organizations, most of which is related to the petosemtamab clinical trials.

General and administrative expense for the three months ended March 31, 2025 increased by $6.0 million as compared to the three months ended March 31, 2024, primarily as a result of increases in personnel related expenses including share-based compensation of $5.3 million; increases in consulting expenses, facilities and depreciation expense also contributed.

Other income (loss), net consists of interest earned and fees paid on our cash and cash equivalents held on account, accretion of investment earnings and net foreign exchange (losses) gains on our foreign denominated cash, cash equivalents and marketable securities. Other gains or losses relate to the issuance and settlement of financial instruments.

MERUS N.V.
CONDENSED CONSOLIDATED BALANCE SHEETS
(UNAUDITED)
(Amounts in thousands, except share and per share data)

March 31,
2025 December 31,
2024
ASSETS
Current assets:
Cash and cash equivalents $ 197,199 $ 293,294
Marketable securities 261,126 243,733
Accounts receivable 14,203 1,261
Prepaid expenses and other current assets 49,744 30,784
Total current assets 522,272 569,072
Marketable securities 179,886 187,008
Property and equipment, net 10,937 10,770
Operating lease right-of-use assets 9,199 9,254
Intangible assets, net 1,703 1,679
Equity Investment 3,449 —
Deferred tax assets 364 1,520
Other assets 3,112 3,390
Total assets $ 730,922 $ 782,693
LIABILITIES AND SHAREHOLDERS’ EQUITY
Current liabilities:
Accounts payable $ 8,984 $ 4,164
Accrued expenses and other liabilities 42,799 43,957
Income taxes payable 8,015 7,317
Current portion of lease obligation 1,772 1,704
Current portion of deferred revenue 27,560 29,934
Total current liabilities 89,130 87,076
Lease obligation 8,084 8,208
Deferred revenue, net of current portion 37,589 39,482
Total liabilities 134,803 134,766
Commitments and contingencies – Note 6
Shareholders’ equity:
Common shares, €0.09 par value; 105,000,000 shares authorized at March 31, 2025 and December 31, 2024; 69,175,766 and 68,828,749 shares issued and outstanding as at March 31, 2025 and December 31, 2024, respectively 6,990 6,957
Additional paid-in capital 1,686,350 1,664,822
Accumulated other comprehensive income (32,360 ) (55,465 )
Accumulated deficit (1,064,861 ) (968,387 )
Total shareholders’ equity 596,119 647,927
Total liabilities and shareholders’ equity $ 730,922 $ 782,693

MERUS N.V.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(UNAUDITED)
(Amounts in thousands, except share and per share data)

Three Months Ended
March 31,
2025 2024
Commercial material revenue $ 13,331 $ —
Collaboration revenue 13,148 7,889
Royalty revenue 9 —
Total revenue 26,488 7,889
Operating expenses:
Research and development 80,116 38,584
General and administrative 22,112 16,114
Total operating expenses 102,228 54,698
Operating loss (75,740 ) (46,809 )
Other income, net:
Interest income, net 7,203 4,917
Foreign exchange gains (loss) (24,316 ) 8,534
Other expense (1,766 ) —
Total other income (loss), net (18,879 ) 13,451

Net loss before income taxes (94,619 ) (33,358 )
Income tax expense 1,855 1,098
Net loss $ (96,474 ) $ (34,456 )
Other comprehensive loss:
Currency translation adjustment 23,105 (7,388 )
Comprehensive loss $ (73,369 ) $ (41,844 )
Net loss per share attributable to common shareholders:
Basic and diluted $ (1.40 ) $ (0.59 )
Weighted-average common shares outstanding:
Basic and diluted 69,017,576 58,085,416