On March 24, 2025 Artiva Biotherapeutics, Inc. (Nasdaq: ARTV) (Artiva), a clinical-stage biotechnology company whose mission is to develop effective, safe, and accessible cell therapies for patients with devastating autoimmune diseases and cancers, reported financial results for the full year ended December 31, 2024, and highlighted recent progress (Press release, Artiva Biotherapeutics, MAR 24, 2025, View Source [SID1234651394]).

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"2024 was a transformational year for Artiva including initiating dosing of AlloNK in patients with autoimmune disease across our trials, a successful initial public offering strengthening our balance sheet, and expanding key leadership across the organization with expertise in cell therapy and autoimmune disease," said Fred Aslan, M.D., CEO of Artiva. "We look forward to sharing initial data from our AlloNK program in autoimmune disease this year. We also look forward to sharing updated clinical data from our non-Hodgkin’s lymphoma (NHL) trial with AlloNK which continues to mature as one of the strongest data sets for the allogeneic field, demonstrating deep B-cell depletion, continued durability of response, and the compatibility of our treatment regimen with outpatient administration."

Recent Business Highlights

Corporate and Financial Updates

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Expanded Board of Directors: In January 2025, Artiva appointed Dan Baker, M.D., as an independent member of its Board of Directors. Dr. Baker brings over two decades of drug development experience in the pharmaceutical industry. He is currently the interim Chief Development Officer of Cue Biopharma, Inc., and previously held a 19-year tenure at Johnson & Johnson (Janssen/Centocor) most recently as the Vice President of Immunology R&D.
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Bolstered Key Development Leadership: Artiva appointed key leadership with cell therapy and autoimmune expertise across the development organization, including Benjamin Dewees as Senior Vice President (SVP), Regulatory Affairs, David Moriarty, Ph.D., as SVP, Clinical Operations, and Feng Xu as SVP, Biometrics. Collectively, the leadership team brings experience developing therapies targeting autoimmune indications including systemic lupus erythematosus (SLE), lupus nephritis (LN), rheumatoid arthritis (RA) and Sjogren’s disease from their tenures at companies such as Kyverna Therapeutics, Inc., Horizon Therapeutics plc and IGM Biosciences, Inc.
Upcoming Milestones

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Initial data for AlloNK (also known as AB-101) on autoimmune indications from at least one of the following trials expected in H1 2025:
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Artiva Sponsored Trial in SLE/ LN: Ongoing Phase 1/1b trial evaluating AlloNK in combination with rituximab or obinutuzumab in patients with SLE with or without LN.
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Ongoing IIT Basket Trial: Investigator-initiated basket trial (IIT) assessing the safety, tolerability, and clinical activity of AlloNK plus rituximab in patients with RA, pemphigus vulgaris, granulomatosis with polyangiitis/microscopic polyangiitis, and SLE. The trial is being conducted by Integral Rheumatology & Immunology Specialists, a community rheumatology clinic.
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Updated clinical data from the Phase 1/2 trial exploring AlloNK + rituximab in patients with relapsed/refractory B-cell NHL showing continued durability of response to be presented at a medical conference in 2025

Full Year 2024 Financial Results

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Cash, Cash Equivalents and Investments. As of December 31, 2024, Artiva had cash, cash equivalents, and investments of $185.4 million. This includes $179.0 million in gross proceeds from Artiva’s completed initial public offering in July 2024 in which it sold 14,920,000 shares of its common stock, including partial exercise of the overallotment option. Existing cash, cash equivalents, and investments as of December 31, 2024, are expected to fund operations at least through the end of 2026.
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Collaboration Revenue. Collaboration revenue was zero for the year ended December 31, 2024, compared to $32.9 million for the year ended December 31, 2023.
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Research and Development Expenses. Research and development expenses were $50.3 million for each of the years ended December 31, 2024 and 2023.
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General and Administrative Expenses. General and administrative expenses were $17.2 million for the year ended December 31, 2024, compared to $13.9 million for the year ended December 31, 2023.
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Other Income, net. Other income, net, was $1.9 million for the year ended December 31, 2024, compared to other income, net, of $2.0 million for the year ended December 31, 2023.
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Net Loss. Net loss totaled $65.4 million for the year ended December 31, 2024, as compared to net income of $28.7 million for the year ended December 31, 2023, with non-cash stock-based compensation expense of $7.0 million and $7.1 million for the years ended December 31, 2024 and 2023, respectively.

On March 24, 2025 Agilent Technologies Inc. (NYSE: A) reported its role in supporting Autolus Therapeutics’ FDA approval for AUCATZYL, a recently approved CAR T therapy. Agilent’s advanced cell analysis instrumentation, specifically the xCELLigence Real-Time Cell Analysis (RTCA) technology, was applied in developing and validating AUCATZYL. The xCELLigence RTCA technology supported the development and implementation of the potency assay (Press release, Agilent, MAR 24, 2025, View Source [SID1234651384]). By providing precise and reliable cell analysis solutions, Agilent assisted Autolus in achieving the high standards required for FDA approval.

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Dr. Xiaobo Wang, Vice President and General Manager of the Cell Function and Phenotyping Business at Agilent stated, "Agilent is honored to have contributed to this significant milestone in cancer treatment. The collaboration between Agilent and Autolus highlights the importance of innovative technologies and cooperative efforts in advancing cancer therapies."

David Brochu, Chief Technical Officer at Autolus Therapeutics added, "Agilent’s support was invaluable in our journey to FDA approval. Agilent’s state-of-the-art xCELLigence RTCA technology facilitated the accuracy, reliability and precision of our potency analytical procedure."

The xCELLigence Real-Time Cell Analysis (RTCA) technology is a cutting-edge platform that continuously monitors cell behavior in real-time without using labels or dyes. This technology measures electrical impedance to provide dynamic information on cell numbers, viability, and morphology. Unlike traditional endpoint assays, xCELLigence RTCA offers real-time insights, enabling more accurate and timely decision-making in drug development. Its ability to deliver continuous, real-time data sets it apart in the market, providing researchers with a deeper understanding of cellular responses and enhancing the development of effective therapies.

Agilent and Autolus will attend the 2025 Hybrid US Bioassay Conference from March 24-25 in Tucson, Arizona. Mike Merges, Vice President of Analytical Development, QC Control Operations, Automation, and CMC (Chemistry, Manufacturing, and Controls) at Autolus, along with Jakub Dragun, Quality Control Consultant at Autolus, and Xiaobo Wang, VP/GM of the Cell Function and Phenotyping Business at Agilent, will present a Main Session Podium Talk titled: ‘Lessons Learned in CAR T Cell Product Potency Measurement: A Cross-Functional Investigation and the Importance of Partnership.’ The talk will be held Monday, March 24, from 1:30-2:00 PM in the Sabino Room at the Marriott Tucson University Park Hotel and is open to all conference attendees.

Agilent is committed to advancing scientific innovation and supporting groundbreaking therapies that enhance health outcomes. To learn more about Agilent’s real-time cell analysis solutions, visit Agilent Cell Analysis.

On March 24, 2025 CEL-SCI Corporation (NYSE American: CVM) reported new data has been published from its prior Phase 3 study of Multikine* (Leukocyte Interleukin, Injection) in newly diagnosed, treatment naïve, resectable, locally advanced head and neck cancer patients in the highly regarded peer reviewed journal Pathology and Oncology Research (POR) (Press release, Cel-Sci, MAR 24, 2025, View Source [SID1234651383]). The article titled "Neoadjuvant Leukocyte Interleukin Injection Immunotherapy Improves Overall Survival in Low-risk Locally Advanced Head and Neck Squamous Cell Carcinoma -The IT-MATTERS Study" included a comprehensive presentation of results from CEL-SCI’s Phase 3 trial, the largest study ever conducted for newly diagnosed locally advanced head and neck cancer.

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"We are pleased that the wealth of data resulting from our completed Phase 3 study is now published in this international oncology journal," stated CEL-SCI’s Chief Scientific Officer, Dr. Eyal Talor. "We believe the marked improvement in quality of life offered by Multikine neoadjuvant treatment appeared to have had a positive impact on patients’ quality of life, and in addition to its favorable safety profile, tolerability and efficacy, it is likely to improve adoption rates for Multikine following regulatory approval."

The new, previously unpublished findings included the following patient quality of life data:

Quality of life (QoL) was assessed and validated through use of two instruments, EORTC QLQ-C30 and EORTC QLQ-H&N 35 across all clinical sites.
EORTC QLQ-C30 is a 30-item questionnaire developed by the European Organisation for Research and Treatment of Cancer (EORTC) to assess the health-related QoL of cancer patients.
EORTC QLQ-H&N 35 is a questionnaire designed to assess the QoL of head and neck cancer patients in conjunction with the general cancer-specific EORTC QLQ-C30.
QoL variables were assessed at baseline, before and after the Multikine treatment, and periodically during long-term follow up. These assessments included questions regarding pain in the mouth, jaw, and throat, problems swallowing, sense of smell and taste, ability for selfcare and mobility including walking, using the toilet, shortness of breath, emotional wellbeing including irritability and depression, and many other daily health assessment factors.
As a neoadjuvant therapy, patients were treated with Multikine before surgery. Pre-surgery objective early response to treatment with Multikine was confirmed by pathology at surgery. There were 45 objective early responders (which included 5 complete responders following 3-weeks of Multikine treatment) in the Multikine treated + standard of care group and zero (none reported by investigators) in the control group, which received the standard of care treatments only (i.e., surgery plus radiotherapy or surgery plus chemoradiotherapy; with cisplatin as the chemotherapeutic agent per the study protocol and NCCN Guidelines).
95.1% of complete responders to Multikine reported improved QoL
Complete responders reported a 100% (wherein all respondents scored the highest possible improvement from baseline) on 60% (39/65) of the QoL measures assessed including sleep, appetite, pain, emotional state, condition of mouth, sense of smell and taste, and social, family and public interactions.
QoL results for complete responders were measured and sustained for over 3 years following treatment with Multikine.
89.4% of partial responders to Multikine (those exhibiting greater than 30% reduction in tumor – confirmed by pathology at surgery) also reported improved assessed QoL measures from baseline.
About the Completed Phase 3 and Upcoming Confirmatory Registration Study

Based on the exceptional efficacy results and favorable safety profile for Multikine in a cohort of patients in the Phase 3 study, the U.S. Food and Drug Administration has given CEL-SCI the go ahead to initiate a confirmatory Registration Study of Multikine in newly diagnosed, previously untreated resectable stage 3 and 4 head and neck cancer patients who had no lymph node involvement and low PD-L1 tumor expression. There is a high unmet need in this patient population, for which no advancement in overall survival has been forthcoming in decades, despite many previous attempts by others.

Upon the Registration Study achieving full enrollment, CEL-SCI plans to seek early approval based on early tumor responses which were shown to correspond with survival rates. During the completed Phase 3 clinical trial, the 5-year overall survival rate of the target patient population (disease stage 3 and 4 patients who had no lymph node involvement and low PD-L1 tumor expression), which is the same population that will participate in the confirmatory study, increased to 73% when patients were treated with Multikine vs 45% for control patients who received only standard of care treatments.

About Pathology and Oncology Research (POR)

A Switzerland-based highly regarded international journal, POR is dedicated to keeping scientists informed of developments in its focused biomedical fields which span the gap between basic research and clinical medicine.

On March 24, 2025 Oncodesign Precision Medicine (OPM) (ISIN: FR001400CM63; Ticker: ALOPM), a clinical-stage company developing innovative therapies to overcome immune evasion and drug resistance, reported the submission to Swiss authorities of the study protocol for its Phase 1b/2a clinical trial REVERT (RIPK2 for rEsistant and adVanced mElanoma tReatmenT) with OPM-101, its RIPK2 inhibitor in advanced melanoma patients with resistance to anti-PD-1 (Press release, Oncodesign Precision Medicine, MAR 24, 2025, https://www.businesswire.com/news/home/20250324831272/en/Oncodesign-Precision-Medicine-OPM-Announces-Protocol-Submission-for-its-Phase-1b2a-Study-REVERT-for-OPM-101-in-Combination-with-Pembrolizumab-in-Patients-with-Advanced-Melanoma-Resistant-to-Anti-PD1 [SID1234651382]). This submission of the study protocol follows the announcement of positive results from the Phase 1 study in October 2024.

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OPM-101 is a first-in-class RIPK2 inhibitor administered orally, with the potential to treat several cancers. This milestone underscores OPM’s commitment to addressing significant unmet medical needs in oncology.

Globally, melanoma accounts for approximately 325,000 new cases and more than 57,000 deaths annually, with the highest incidences observed in regions such as Europe, North America, and Oceania. In Europe, the incidence of melanoma is rising, and there is an increasing focus on improving treatments for patients who develop resistance to immune checkpoint inhibitors (ICIs) like anti-CTLA4 and anti-PD1/PD-L1 therapies. Although ICIs have become a cornerstone of advanced melanoma treatment for several years, approximately 40-60% of patients experience resistance or progression, creating a pressing need for innovative therapeutic options.

In experimental cancer models, the addition of OPM-101 to anti-PD-1 antibody therapy has been shown to significantly enhance anti-tumor efficacy and promote greater sensitivity to PD-1 blockade, delaying or reducing loss of response to ICI therapy and demonstrating durable responses in preclinical models studied to date. The OPM team has shown that the underlying mechanisms of OPM-101 involve enhanced tumor antigen presentation and recognition, associated with remodelling of CD8+ T cell infiltration into the tumor stroma. Moreover, OPM-101 alone has also demonstrated significant antitumor activity based on its immunomodulatory properties.

REVERT Study Objectives and Design

This Phase 1b/2a clinical trial is an international, multicenter, open-label study designed to assess the safety and preliminary efficacy of OPM-101 in combination with pembrolizumab in metastatic melanoma patients treated with anti-PD-1 monotherapy or combination therapy (except with anti-LAG-3), and who have developed resistance to this treatment.

The trial will include around 45 patients at 10-13 sites in Switzerland, France, Italy and Spain, and will be conducted through two main phases:

Phase 1b: to assess the safety of OPM-101 in combination with pembrolizumab in patients who have developed resistance to this treatment by anti-PD1, and to select the recommended dose to be used in the second part of the study. This phase will involve a minimum of 6 patients.
Phase 2a: to assess the Disease Control Rate (DCR), safety and key biomarkers associated with immune response with the selected dose of OPM-101 in combination with pembrolizumab in patients who have developed resistance to anti-PD-1. This phase will be conducted on an expanded sample of around 35 patients.
The Phase 1b/2a study builds on the safety data from the Phase 1 study conducted in healthy volunteers (VS) in 2023-2024, which demonstrated a favorable safety profile for OPM-101 and robust pharmacokinetic and pharmacodynamic properties.

The results of Phase 1b will be used to determine the recommended dose for Phase 2a and to gather preliminary efficacy data on a limited number of patients. All data collected during this first phase will be reviewed and analyzed by an independent panel of experts forming the Data and Safety Monitoring Board (DSMB). During this open-label Phase 2a, efficacy data will be generated and reviewed on an ongoing basis, enabling trends in treatment efficacy to be identified before the interim analysis on the first 10 patients receiving the recommended dose and the final analysis on all patients. These efficacy data will be based on the analysis of images obtained for patient follow-up after 12 and 24 weeks of treatment, making it possible to define the Disease Control Rate (DCR) and the Objective Response Rate (ORR). In addition, circulating and tumor biomarkers of antitumor activity will be measured to document OPM-101’s mechanism of action.

The study is coordinated by Pr. Olivier Michielin, Head of the Precision Medicine Oncology Service and Head of the Department of Oncology at Geneva University Hospitals in Switzerland.

Phase 1b of the study will start at the same time in several hospitals in Switzerland. Submission of the application for authorization of the clinical study to the Swiss regulatory authorities (Swissmedic) and to the Ethics Committees of the centers involved in phase 1b of the study, was completed on March 24, 2025. The study is scheduled to start by July 2025.

For phase 2b, patients will be enrolled in additional centers in France, Italy and Spain. Submission of the application for clinical trial authorization in these selected European Union countries will take place in the 4th quarter of 2025, in order to obtain authorization to conduct the extension phase of the study, which could start in the first quarter of 2026.

Completion of this Phase 1b/2a clinical trial is expected by the end of 2027.

"In parallel with phase 1 VS, which enabled us to build a solid foundation around the PK/PD relationship of OPM-101 in humans, we have explored the antitumor activity of our compound alone and in combination with an anti-PD1 in mice. These preclinical results, reinforced by many recent international publications, have convinced us of its major potential in oncology, which is in fact the company’s primary mission.", said Philippe Genne, Chairman and CEO of Oncodesign Precision Medicine. "We are continuing to work on OPM-101’s innovative mechanism of action. In the current financial climate, this study is a major choice for OPM. We are also looking for a pharmaceutical partner to explore its potential in the treatment of IBD – Chronic Inflammatory Bowel Disease."

"Immunotherapy represents a real revolution for many patients suffering from many different types of cancer. This therapeutic approach is fundamentally changing the way we treat cancers by stimulating our immune system to attack and kill cancer cells. These approaches are the result of over a century of investigation and therapeutic advances. Immune checkpoint inhibitors (ICIs) such as anti-PD1s have been used for more than 10 years in several types of cancer, including melanoma and lung cancer. These therapies have proven highly effective with long-lasting responses, marking dramatic progress for many patients," added Jan Hoflack, Scientific Director of Oncodesign Precision Medicine. "Nevertheless, they come with a greater risk of toxicity including immune-related side effects, and a significant number of patients are or become resistant to these approaches. OPM-101, our RIPK2 inhibitor, has the potential to increase the response rate and reduce the side effects of these ICIs. This is what we will be evaluating in this Phase 1b/2a study in melanoma."

Prof. Olivier Michielin, Head of the Precision Oncology Service and Head of the Department of Oncology at the University Hospitals of Geneva, concluded: "Checkpoint inhibitors have revolutionized the management of melanoma. Despite high response rates in the advanced setting and clearly demonstrated long-term benefit, most patients progress under these treatments. Strategies to counteract the mechanisms of resistance to checkpoint inhibitors are therefore essential. OPM’s clinical trial is therefore extremely important, as it addresses exactly this population for whom we lack therapeutic options. What’s more, the prospect of being able to increase the efficacy of checkpoint inhibitors, while reducing toxicity, is of course extremely attractive and will be one of the hypotheses tested with OPM-101, as part of the study."

About OPM-101 in Oncology

OPM-101 is a first-in-class, potent, selective, orally available RIPK2 kinase inhibitor in oncology that has demonstrated the ability to maximize the immunogenicity of tumor cell death and activate a systemic anti-tumor immune response. Although initially linked to chronic inflammatory diseases, the RIPK2 pathway has been increasingly recognized since 2022 as a critical player in tumor progression. In preclinical studies, OPM-101 exhibited synergistic effects with anti-PD1 therapies, enhancing anti-tumor immunity and overcoming resistance in models treated with checkpoint inhibitors alone.

On March 24, 2025 IceCure Medical Ltd. (Nasdaq: ICCM) ("IceCure", "IceCure Medical" or the "Company"), developer of minimally-invasive cryoablation technology that destroys tumors by freezing as an alternative to surgical tumor removal, reported that interim results from the Company’s ICESECRET study of cryoablation for patients with small renal masses ("SRM") who cannot be offered kidney preserving surgery were presented at the European Association of Urology Conference in Madrid, Spain which took place March 21-24, 2025 (Press release, IceCure Medical, MAR 24, 2025, View Source [SID1234651381]). The oral presentation titled "Safety and efficacy of cryoablation in small renal masses, using liquid nitrogen-based cryoablation system: ICESECRET Study Interim analysis" was delivered by Dr. Nasir Said of Bnai Zion Medical Center, Israel.

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"We are very pleased to see these impressive data shared at a major urology conference in Europe, where ProSense is approved for renal tumors" stated Eyal Shamir, IceCure’s Chief Executive Officer. "These data support the adoption of cryoablation as a safe and effective option for patients who are otherwise ineligible for kidney preserving surgery, a large unmet need." Conclusions and data in the presentation included the following:

Cryoablation is a viable alternative for SRMs, especially for tumors ≤3 cm;
111 patients were evaluated at a mean follow-up of approximately 3 years;
Recurrence free rate was 88.7% in patients with tumors ≤3 cm, low risk and a successful procedure at a mean follow up of 3.4 years;
Recurrence free rate was 87.8% in patients with tumors ≤3 cm at low risk with at a mean follow up of 3.4 years;
Recurrence free rate was 87.2% in patients with tumor size ≤3 cm at a mean follow up of 3.5 years;
Safety results include 17 mild adverse events, 3 moderate events, and 1 severe complication observed; and
The mean age of the patients was 69 and 84.2% had comorbidities, the most common of which were hypertension (77%) and diabetes (47%).
ProSense is approved for benign and malignant kidney tumors in the U.S., Europe, and numerous other countries.

About ICESECRET
ICESECRET, a prospective, multicenter, single-arm clinical trial is being performed at Bnai Zion Medical Center in Haifa, Israel and Shamir Medical Center in Be’er Ya’akov, Israel and is being led by Principal Investigator Prof. Halahmi Sarel. The trial includes 114 patients (138 lesions) with localized SRM of ≤5 cm that were ablated with ProSense cryoablation under CT guidance. Follow-up visits are performed six weeks, six months, one year, and then annually up to five years after the procedure. During the follow-up visits, data related to local recurrence, based on CT imaging, is collected. Safety was determined by monitoring procedure-related adverse events throughout the study.

About ProSense
The ProSense Cryoablation System is a minimally invasive cryosurgical tool that provides the option to destroy tumors by freezing them. The system uniquely harnesses the power of liquid nitrogen to create large lethal zones for maximum efficacy in tumor destruction in benign and cancerous lesions, including breast, kidney, lung, and liver.

ProSense enhances patient and provider value by accelerating recovery, reducing pain, surgical risks, and complications. With its easy, transportable design and liquid nitrogen utilization, ProSense opens that door to fast and convenient office-based procedures for breast tumors.