1stOncology™: Cancer Intelligence Service – Page 110 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Geneseeq’s Breakthrough Cancer Detection Blood Test Published in Nature Medicine

On May 28, 2025 Geneseeq reported the publication of results from its large-scale multi-cancer early detection (MCED) study in Nature Medicine, one of the world’s leading peer-reviewed medical journals (Press release, Geneseeq, MAY 28, 2025, View Source [SID1234653457]). The publication presents findings from the DECIPHE-Omnia Study (Detecting Early Cancer by Inspecting ctDNA Features), a landmark effort evaluating CanScan, Geneseeq’s advanced blood-based test for early cancer detection.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

CanScan is a non-invasive blood test powered by AI-driven whole-genome sequencing. It analyzes subtle cancer-specific changes in circulating cell-free DNA (cfDNA) using Geneseeq’s proprietary MERCURYTM Technology. By integrating fragmentomics, genomic, and epigenomic features, the test identifies whether a cancer signal is present and accurately predicts the tissue of origin (TOO). In 2023, CanScan received Breakthrough Device Designation from the U.S. FDA.

A New Way to Catch Cancer Early

Unlike traditional cancer screening tools that are limited to one type of cancer and often involve invasive procedures, CanScan offers a convenient, comprehensive solution: a single blood test that screens for more than a dozen cancers at once.

In the study:

CanScan detected early-stage cancers with high accuracy
Identified cases missed by routine physical exams
Produced a low false-positive rate, helping reduce unnecessary follow-ups
"This study brings us closer to making routine multi-cancer screening a reality," said Dr. Yang Shao, CEO of Geneseeq. "Our vision is a future where a simple blood test can help save lives through earlier diagnosis."

The Landmark DECIPHER-Omnia Study

The DECIPHER-Omnia Study is a multi-phase clinical research program involving more than 8,000 participants to date across three stages:

Test development and training using samples from cancer patients and healthy donors
Independent clinical validation in a separate cohort
A large-scale ongoing screening study in asymptomatic individuals aged 45–75 (the JINLING cohort)
The newly published Nature Medicine paper presents interim findings from over 3,700 participants in the JINLING cohort.

Key Interim Results:

Specificity: 98.1%
Sensitivity: 53.5% across all detected cancers; 62.1% for targeted cancer types
Early-stage detection: 93% of confirmed cases were Stage 0, I, or II
Positive Predictive Value (PPV): 25% — 10 times higher than standard screening (2.2%)
Low false positives: Reduced unnecessary follow-up procedures
Detection of missed cancers: CanScan identified 53.3% of cancers overlooked by standard screening
Looking Ahead

The JINLING cohort study has now successfully completed enrollment of its target 15,000 participants and is currently in the follow-up and data analysis phase. The complete dataset, along with ongoing monitoring, is expected to provide deeper insights into the real-world utility of CanScan for population-level cancer screening.

Bexion Pharmaceuticals, Inc. Announces Acceptance of Abstracts at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting

On May 28, 2025 Bexion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing a novel class of biologic therapy to treat solid tumor cancers and chemotherapy-induced peripheral neuropathy (CIPN), reported that two abstracts were accepted at the upcoming 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 30, 2025 – June 3, 2025, in Chicago, Illinois (Press release, Bexion, MAY 28, 2025, View Source [SID1234653456]). Details of the abstracts are included below.

Abstract Details:

Title: Effect of BXQ-350, a novel sphingolipid metabolism modulator, on neuronal environments through modulation of gene pathways
Abstract Number: e24058
Author: Michael Gazda, Tariq Arshad, Jim Beach, Nikhil Wilkins, Adam Creighbaum, Timothy Stephens
Session Title: Publication Only: Symptom Science and Palliative Care

Title: A phase 1 study to evaluate the safety and tolerability of BXQ-350, a novel sphingolipid metabolism modulator, in pediatric diffuse intrinsic pontine glioma and diffuse midline glioma
Abstract Number: e14044
Author: Tariq Arshad, Michael Gazda, Jim Beach, Kathleen Dorris, Margot Lazow, Trent Hummel, Richard Curry III, Adam Creighbaum
Session Title: Publication Only: Central Nervous System Tumors

The full abstracts are currently available in the ASCO (Free ASCO Whitepaper) digital program.

About BXQ-350
Bexion’s lead drug candidate is BXQ-350, a first-in-class biologic containing the multifunctional sphingolipid activator protein, Saposin C, and a phospholipid. Multiple Phase 1 clinical trials in adult and pediatric patients have demonstrated a robust safety profile for BXQ-350 with evidence of single agent activity across multiple solid tumor types. Additionally, other clinical and non-clinical data suggest BXQ-350 has activity in chemotherapy-induced peripheral neuropathy, an area of high unmet medical need in patients treated with oxaliplatin and other chemotoxic agents.

Halozyme Announces Bristol Myers Squibb Received European Commission Approval for Subcutaneous Opdivo® (nivolumab) Co-Formulated with ENHANZE® Across Multiple Solid Tumor Indications

On May 28, 2025 Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) reported that Bristol Myers Squibb received European Commission (EC) approval of a new Opdivo (nivolumab) subcutaneous formulation developed with ENHANZE, Halozyme’s proprietary recombinant human hyaluronidase enzyme (rHuPH20), for use across multiple adult solid tumors as monotherapy, monotherapy maintenance following completion of intravenous nivolumab plus Yervoy (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib (Press release, Halozyme, MAY 28, 2025, View Source [SID1234653455]).

"The European approval of Opdivo SC represents an advancement for certain cancer patients, who now have the option to receive their Opdivo as a 3-to-5-minute SC injection," said Dr. Helen Torley, president and chief executive officer of Halozyme. "This approval is just one of the 11 growth catalysts for our commercialized SC products expected this year."

The positive EC decision is supported by positive results from the Phase 3 CheckMate -67T trial. For more information on the study and its findings, please view Bristol Myers Squibb’s press release issued on May 28, 2025.

The approval by the EC is valid in all 27 member states of the European Union (EU), as well as Iceland, Liechtenstein and Norway.

On December 27, 2024, subcutaneous nivolumab and hyaluronidase-nvhy, marketed under the brand name Opdivo Qvantig, was approved by the U.S. Food and Drug Administration.

Biohaven Presents Oncology Program Updates and Preliminary Clinical Data Showcasing Innovative Trop2 and FGFR3 Antibody Drug Conjugates (ADCs) Incorporating Novel TopoIx Payload with Potential to Treat a Wide Variety of Tumors

On May 28, 2025 Biohaven Ltd. (NYSE: BHVN) (Biohaven), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported an update and preliminary clinical data from its oncology development programs at Biohaven’s 2025 R&D Day, held concurrently with the Yale Innovation Summit in New Haven, Connecticut (Press release, Biohaven Pharmaceutical, MAY 28, 2025, View Source [SID1234653454]). The presentation slides from Biohaven’s R&D day for Oncology and its other platforms will be available on the Events and Presentations page of the Biohaven website just prior to their presentations.

Biohaven reported that its novel next-generation trophoblast cell surface antigen 2 (Trop2) directed antibody drug conjugate (ADC), BHV-1510, demonstrated encouraging preliminary clinical activity both as a monotherapy and in combination with Regeneron’s anti-PD-1 antibody cemiplimab. Early clinical data is consistent with BHV-1510’s preclinical profile showing high ADC stability, differentiated safety and efficacy, immunogenic cell death, and anti-PD-1 synergism. Monotherapy tumor reductions including partial responses have been seen in patients failing standard of care therapies. The combination of BHV-1510 and cemiplimab in the ongoing Phase 1 study shows encouraging anti-tumor activity, with tumor shrinkage in the first 6 out of 6 patients treated, including confirmed partial responses and in patients with brain metastasis (Figure 1). The majority of patients treated with the combination had failed prior anti–PD-1/PD-L1 therapies. BHV-1510 showed a favorable pharmacokinetic (PK) profile, with very low levels of free payload. As monotherapy, the main toxicity observed thus far in the Phase 1 study has been stomatitis, an expected on-target Trop2 class toxicity that has been manageable. Importantly, there were no cases of payload-associated interstitial lung disease (ILD), and low rates of gastrointestinal (e.g., diarrhea) and hematologic toxicities observed. The combination with cemiplimab was well tolerated with no dose limiting toxicity to date in initial cohorts.

Nushmia Khokhar, M.D., Chief Medical Officer of Oncology at Biohaven, commented, "The early clinical data with BHV-1510 dosed in patients who failed standard of care treatment are highly encouraging, particularly the observed potential synergy with anti-PD-1 therapy. These findings, combined with the promising efficacy and tolerability profile of our novel TopoIx payload and stable linker technology, support the potential of BHV-1510 to advance into earlier lines of therapy for challenging tumor types."

Biohaven also announced the first patient has been dosed in the Phase 1 study of BHV-1530, a potential first-in-class fibroblast growth factor receptor 3 (FGFR3)-directed ADC which utilizes the proprietary Topolx payload. BHV-1530 has potential in indications of cancers driven by FGFR3 alterations and/or upregulated FGFR3 protein expression, including urothelial cancers and other solid tumors (Figure 2). FGFR3 is a clinically validated target in oncology, with one small molecule inhibitor (erdafitinib) approved. There are no FGFR3 ADCs beyond BHV-1530 advanced in clinical testing.

Michael Song, M.D., Ph.D., Principal Investigator and leading medical oncologist and hematologist at NEXT Oncology with over 22 years of experience in cancer patient care and cancer research, stated, "We are excited to partner with Biohaven and dose the first patient on this important study. This is an exciting, validated target with potential to extend therapeutic benefit to several FGFR3 driven tumors."

Biohaven is also advancing a portfolio of innovative technologies to modernize next-generation ADCs through strategic collaborations with Merus and GeneQuantum (Figure 3). The preclinical programs leverage Biohaven’s differentiated ADC platform directed against undisclosed novel validated and emerging high-value targets, and incorporating the TopoIx payload that preclinically demonstrated immunogenic cell death and synergistic efficacy with PD-1/PD-L1 checkpoint inhibitors.

Brian Lestini, M.D., Ph.D., President of Oncology at Biohaven, commented, ‘We are excited to be in the clinic with two innovative ADCs and to share the early clinical experience with the first of our two programs, demonstrating the potential of our oncology portfolio to deliver a wide range of optimized, next-generation ADCs. The early clinical data from the Trop2 ADC, BHV-1510, shows the predicted profile and potential of the proprietary TopoIx payload as seen preclinically, and supports broad investigation of ADCs incorporating TopoIx and highly stable linker technologies. Similarly, initiation of the first-in-human study of BHV-1530, an FGFR3 directed ADC, demonstrates the versatility of our approach to generate novel drugs with the potential to address a wide variety of unmet needs in oncology. Together with our collaborations with Merus and GeneQuantum as well as licensed conjugation technology from Yale University, Biohaven’s platform has the potential to generate multiple differentiated mono- and bispecific ADC therapies with greater potency and selectivity over currently available ADC approaches."

Pimicotinib Demonstrates Best-in-Class Potential with Significant Efficacy and Clinically Meaningful Improvements in Patients with Tenosynovial Giant Cell Tumor

On May 28, 2025 Merck, a leading science and technology company, reported the presentation of detailed positive results from Part 1 of the global Phase 3 MANEUVER trial evaluating pimicotinib, a potentially best-in-class investigational colony stimulating factor-1 receptor (CSF-1R) inhibitor in development by Abbisko Therapeutics Co., Ltd., in the treatment of patients with tenosynovial giant cell tumor (TGCT) (Press release, Merck KGaA, MAY 28, 2025, View Source [SID1234653453]). Once-daily pimicotinib demonstrated a statistically significant improvement in the primary endpoint of objective response rate (ORR) assessed by blinded independent review committee (BIRC) compared with placebo at week 25 (54.0% vs. 3.2% for placebo (p<0.0001). The study also demonstrated statistically significant and clinically meaningful improvements in all secondary endpoints related to key patient-reported outcomes in TGCT. These findings will be presented Sunday, June 1 in an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Abstract #11500).

"The impact that TGCT has on patients goes far beyond the physical presence of the tumor. It affects their ability to work, to move freely, and to engage in everyday activities," said Prof. Niu Xiaohui, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital. "In MANEUVER, we observed the highest ORR seen to date with a systemic therapy, together with statistically significant improvements in measures of pain, stiffness, and range of motion. These improvements in outcomes that matter to patients with TGCT and the physicians who care for them show the potential of pimicotinib to allow patients to go about their daily lives with fewer negative effects of their disease."

In MANEUVER, which enrolled patients from China, Europe and North America, the effect of pimicotinib had an early onset, with 41.3 % (26 of 63) of patients experiencing objective response to therapy after 13 weeks. By the data cutoff for primary analysis, nearly all patients in the pimicotinib group (58 of 63 patients; 92.1%) had a decrease in tumor size per BIRC based on RECIST v1.1; one patient achieved a complete response and 33 patients achieved a partial response. The median duration of response was not reached by the data cutoff. The analysis of tumor volume score (TVS, an endpoint designed specifically for TGCT) showed that nearly two-thirds of patients treated with pimicotinib experienced a reduction in tumor volume of at least 50% (61.9% vs. 3.2% for placebo, p<0.0001).

Pimicotinib also demonstrated statistically significant and clinically meaningful improvement across all additional secondary endpoints relevant to patients’ daily lives, and these improvements were seen regardless of achieving objective tumor response to pimicotinib. Pimicotinib improved active range of motion (p=0.0003) and physical function measured by PROMIS-PF scale (p=0.0074). Pimicotinib also reduced worst stiffness (p<0.0001) and worst pain (p<0.0001).

"TGCT, although rare, has a significant impact on the daily lives of the primarily working-age adults who live with the disease, due to swelling, pain, stiffness, and limited mobility caused by the growth of these tumors in and around the joints," said Danny Bar-Zohar, appointed CEO Healthcare and current Global Head of R&D and Chief Medical Officer. "The landmark global Phase 3 MANEUVER study data will help redefine how TGCT is treated, and we plan regulatory submissions to start this year."

Pimicotinib was well-tolerated, and the safety profile was consistent with previously reported data, with no evidence of cholestatic hepatotoxicity or hair/skin hypopigmentation. Treatment-emergent adverse events (TEAEs) leading to treatment discontinuation occurred in one patient (1.6%) treated with pimicotinib; TEAEs leading to dose reduction occurred in 7.9% (n=5) of pimicotinib-treated patients.

About MANEUVER

The pivotal Phase 3 MANEUVER study is a three-part, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of pimicotinib in patients with TGCT who require systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy. The study is being conducted by Abbisko Therapeutics in China (n=45), Europe (n=28), and the US and Canada (n=21).

In the double-blind Part 1, 94 patients were randomized 2:1 to receive either 50 mg QD of pimicotinib (n=63) or placebo (n=31) for 24 weeks. The primary endpoint is objective response rate (ORR) at week 25, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 by blinded independent central review in the intent-to-treat (ITT) population. Secondary endpoints include tumor volume score, active range of motion, stiffness by Numeric Rating Scale (NRS), pain by Brief Pain Inventory (BPI), and physical function measured by Patient-Reported Outcomes Measurement Information System (PROMIS).

After the double-blind Part 1, eligible patients could continue to the open-label Part 2 for up to 24 weeks of dosing, results of which are expected in mid-2025. Patients who complete Part 2 may then enter the open-label extension phase (Part 3) for extended treatment and safety follow-up.

About Pimicotinib (ABSK021)

Pimicotinib (ABSK021), which is being developed by Abbisko Therapeutics, is a novel, orally administered, highly selective and potent small-molecule inhibitor of CSF-1R. Pimicotinib was recently granted Priority Review by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for the treatment of patients with tenosynovial giant cell tumor (TGCT) who require systemic therapy. Pimicotinib has been granted breakthrough therapy designation (BTD) for the treatment of unresectable TGCT by China National Medical Products Administration (NMPA) and the US Food and Drug Administration (FDA), and priority medicine (PRIME) designation from the European Medicines Agency (EMA). Merck holds worldwide commercialization rights for pimicotinib.