On May 20, 2025 4SC AG ("4SC") (Frankfurt Stock Exchange, Prime Standard: VSC; ISIN: DE000A3E5C40) reported to have received a Negative Opinion on the Company’s Market Authorization Application for Resminostat (Kinselby) for the treatment of patients with advanced stage cutaneous T-cell lymphoma (CTCL) from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) (Press release, 4SC, MAY 20, 2025, https://www.pressetext.com/news/20250523025 [SID1234653364]).

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Following an Oral Explanation on 20 May 2025, and the CHMP’s review of the data on quality, safety and efficacy for Resminostat (Kinselby) in the treatment of patients with advanced stage mycosis fungoides (MF) and Sézary syndrome (SS) that have achieved at least stable disease with at least one prior systemic therapy or Total Skin Electron Beam therapy (TSEB) – the CHMP considers by consensus that the efficacy of the above-mentioned medicinal product is not sufficiently demonstrated, and therefore, recommends the refusal of the granting of the marketing authorization for Resminostat (Kinselby).

As already communicated on 20 May 2025, 4SC will thus cease all efforts to develop and commercialize Resminostat (Kinselby). Currently, 4SC has at least a 12-month cash runway to finance the Company’s projected expenses as the Management and Supervisory Boards consider all options for the Company, including liquidation.

Jason Loveridge, Ph.D., CEO of 4SC, commented: "We had hoped that the long wait for a maintenance treatment to help manage CTCL would soon be over. We are therefore deeply disappointed with this outcome as it is a further setback for people living with this rare and incurable disease."

On May 20, 2025 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported the launch of the Guardant Hereditary Cancer test, a germline test that identifies genetic variants associated with cancer risk to help cancer care teams provide optimal patient care (Press release, Guardant Health, MAY 20, 2025, View Source [SID1234653266]).

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Germline genetic testing is recommended by medical practice guidelines for patients diagnosed with cancer to enable genetically targeted treatment and identify relatives who may benefit from personalized cancer screening and prevention.1 The testing analyzes inherited genetic variants, typically present in all the cells of the body, that may predispose an individual to certain risks or diagnoses, including hereditary cancers and other genetic conditions. The new Guardant Hereditary Cancer test is a blood-based germline panel test that identifies guideline-recommended genetic variants across 82 genes associated with an increased risk for more than 12 tumor types, including breast, colorectal, prostate, endometrial, renal and others.

"Introducing a best-in-class hereditary cancer test is another important step in achieving our mission to conquer cancer with data," said Helmy Eltoukhy, Guardant Health chairman and co-CEO. "It expands the portfolio we offer to support healthcare providers across the entire continuum of cancer care and allows them to access an even broader set of precision oncology tools through a single source. The germline testing will help them develop more informed personalized treatment plans for patients, reduce risk and improve outcomes."

Physicians may order the Guardant Hereditary Cancer test for patients with a personal or family history of hereditary cancer for several reasons, such as guiding treatment or care decisions, assessing risk for secondary cancer development, and providing information that can help identify other family members who may be at increased risk for certain cancers. Discussion of risk-reducing strategies, enhanced screening, and referral to a specialist or genetic counseling is recommended.

The Guardant Hereditary Cancer test requires only a simple blood draw and can be ordered as a standalone test or added to Guardant360 liquid biopsy tests with no additional sample required. Results are available in two to three weeks.

On May 20, 2025 PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported it will present results from the Phase 3 SURPASS-ET clinical trial in an oral session at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 30-June 3 in Chicago (Press release, PharmaEssentia, MAY 20, 2025, View Source [SID1234653265]).

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SURPASS-ET (NCT04285086) is evaluating ropeginterferon alfa-2b-njft as a second-line treatment for patients with essential thrombocythemia (ET). Earlier this year, PharmaEssentia announced positive topline Phase 3 results, with ropeginterferon alfa-2b-njft demonstrating a significantly higher durable clinical response rate compared to anagrelide (42.9% vs. 6.0%; p=0.0001), along with a favorable safety profile and a greater reduction in JAK2 V617F allelic burden over 12 months.

Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi for the treatment of adults with polycythemia vera (PV). BESREMi has been recognized by the National Comprehensive Cancer Network (NCCN) as a preferred first-line cytoreductive therapy for adults with symptomatic, low-risk PV and the only preferred therapeutic option for both high-risk and low-risk (symptomatic) patients, regardless of treatment history.

"Current options for patients with ET are limited. Standard therapies like hydroxyurea have notable drawbacks and do not target the underlying biology of the disease, while anagrelide has been associated with toxicity concerns and limited efficacy," said Ruben Mesa, M.D., co-principal investigator, presenting author, and President of Atrium Health Levine Cancer Institute, the largest cancer program in the Carolinas which includes the Comprehensive Cancer Center at Wake Forest Baptist. "This marks the first registrational Phase 3 trial of a long-acting interferon in ET, demonstrating not only well-tolerated blood count control but also a measurable reduction in JAK2 mutation allele burden. These findings support further investigation of ropeginterferon as a second-line option for patients with ET who are seeking additional treatment approaches."

"The ASCO (Free ASCO Whitepaper) meeting is an important opportunity to share detailed findings of our positive data from the SURPASS-ET study with the medical community," said Albert Qin, M.D., Ph.D., Chief Medical Officer of PharmaEssentia USA. "These data highlight a significant advance in the treatment of essential thrombocythemia and reinforce our commitment to delivering innovative, non-chemotherapy options for patients living with myeloproliferative neoplasms."

Presentation Details

Title: Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial
Abstract Number: 6500
Presenter: Dr. Ruben Mesa
Session: Hematologic Malignancies — Leukemia, Myelodysplastic Syndromes, and Allotransplant
Date: Monday, June 2, 2025
Time: 3:00 p.m. – 6:00 p.m. CDT

About Essential Thrombocythemia

Essential thrombocythemia is a chronic, rare blood disorder that is the most common type of myeloproliferative neoplasm. Essential thrombocythemia is most often caused by genetic mutations that cause the bone marrow to produce too many platelets, which can obstruct blood flow and cause a stroke, heart attack or pulmonary embolism.

About BESREMi (ropeginterferon alfa-2b-njft) in polycythemia vera (PV)

BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients.

BESREMi has orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. BESREMi has been approved in more than 40 countries, with approval from the European Medicines Agency (EMA) in 2019, by the US Food and Drug Administration (FDA) in 2021, and by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023. It was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications.

BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.

Please see full Prescribing Information, including Boxed Warning.

On May 20, 2025 KaliVir Immunotherapeutics, Inc., a clinical-stage biotechnology company developing cutting-edge, multi-mechanistic oncolytic immunotherapy programs, reported the successful completion of the first cohort in its STEALTH-001 study, a Phase 1/1b clinical trial of VET3-TGI for patients with incurable, advanced solid tumors (Press release, KaliVir Immunotherapeutics, MAY 20, 2025, View Source [SID1234653264]).

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VET3-TGI is a novel oncolytic immunotherapy designed to target and selectively kill tumor cells while also expressing an immuno-stimulatory transgene payload consisting of interleukin-12 and a TGFbeta inhibitor. The Data Safety Committee, charged with monitoring the safety and overall risk-benefit of treatment on the STEALTH-001 (NCT06444815) clinical study, convened following dosing of the first cohort in the dose-escalation portion of the study, and approved continuation of dosing for the next intratumoral (IT) and intravenous (IV) infusion cohorts.

"The safety profile demonstrated in our initial first-in- human cohort is critical as it opens up expanded dosing in both the study’s IV infusion and IT injection arms which will continue in parallel on study STEALTH-001. We are excited to assess not only the safety of VET3-TGI but further investigate both proof of concept and anti-tumor activity moving forward," said James Burke, M.D., Chief Medical Officer of KaliVir Immunotherapeutics.

The STEALTH-001 trial is a dose escalation and expansion study evaluating VET3-TGI administered through direct IT injection and IV infusion. The trial is evaluating VET3-TGI as a monotherapy and in combination with a checkpoint inhibitor in patients with pathologically confirmed, advanced, unresectable or metastatic solid tumors. The study continues to progress as planned through its dose escalation phase.

"Completing this first cohort reinforces our commitment to advancing our VET platform and its potential to address significant unmet needs in oncology," said Helena Chaye, Ph.D., CEO of KaliVir Immunotherapeutics. "We remain focused on our mission to develop novel oncolytic virus candidates with the potential to transform the treatment landscape for patients with advanced cancer."

On May 20, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported a collaboration to develop a companion diagnostic (CDx) test with Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers (Press release, Tempus, MAY 20, 2025, View Source [SID1234653263]). Tempus completed confirmatory testing in Verastem’s Phase 2 RAMP-201 clinical trial, which evaluated the combination of avutometinib and defactinib to treat recurrent low-grade serous ovarian cancer (LGSOC) and was the basis of the recent U.S. Food and Drug Administration’s (FDA) accelerated approval of the combination in KRAS-mutated recurrent LGSOC.

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LGSOC is a rare form of ovarian cancer that disproportionately affects younger women, is highly recurrent and has a poor response rate to chemotherapy. It accounts for approximately 6% to 10% of serous ovarian cancers1. Tempus’ FDA-approved xT CDx assay is being leveraged as an investigational assay in Verastem’s global Phase 3 RAMP-301 clinical trial. The investigational assay prospectively assesses KRAS status in patients with recurrent LGSOC to group patients into KRAS-mutation or KRAS-wild type cohorts for analysis in the primary and secondary endpoints of the study.

"We look forward to continuing to work with Verastem to pursue an unmet need for patients with LGSOC, who, until now, had very few treatment options," said Mike Yasiejko, Executive Vice President and General Manager, Genomics, at Tempus. "Our xT CDx assay is uniquely positioned to support this work."

"Collaborating with Tempus to evaluate KRAS mutation status using the xT assay was an important component of the RAMP-201 clinical trial. Continuing our collaboration to fully develop a CDx assay is part of our post-marketing commitment to the FDA for our recent accelerated approval of avutometinib plus defactinib and is a critical step in bringing targeted therapies to patients with recurrent KRAS-mutant LGSOC," said John Hayslip, MD, Chief Medical Officer of Verastem Oncology.

xT CDx is a qualitative Next Generation Sequencing (NGS)-based in vitro diagnostic device intended for use in the detection of substitutions (single nucleotide variants (SNVs) and multi-nucleotide variants (MNVs)) and insertion and deletion alterations (INDELs) in 648 genes, as well as microsatellite instability (MSI) status, using DNA isolated from Formalin-Fixed Paraffin Embedded (FFPE) tumor tissue specimens, and DNA isolated from matched normal blood or saliva specimens, from previously diagnosed cancer patients with solid malignant neoplasms. The test is intended as a CDx to identify patients who may benefit from treatment with the targeted therapies listed in the Companion Diagnostic Indications table in accordance with the approved therapeutic product labeling. Additionally, xT CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with previously diagnosed solid malignant neoplasms. Genomic findings other than those listed in the Companion Diagnostic Indications table are not prescriptive or conclusive for labeled use of any specific therapeutic product. xT CDx is a single-site assay performed at Tempus AI, Inc., Chicago, IL. For the complete xT CDx label, including companion diagnostic indications and important risk information, please visit Tempus’ document library here.