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Genexine’s First-in-Class SOX2 Degrader GX-BP1 Demonstrates Up to 96% TGI and Eliminates Tumor Regrowth in Preclinical Models

On April 28, 2026 Genexine, Inc. (KOSDAQ: 095700), a clinical-stage biotechnology company, reported new preclinical data for its SOX2-targeting bioPROTAC candidate, GX-BP1, presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2026 in San Diego.

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GX-BP1 is a first-in-class bioPROTAC drug candidate designed to selectively degrade SOX2, a transcription factor widely recognized as a key driver of tumor progression, cancer stemness, and therapeutic resistance, yet historically considered undruggable. By directly eliminating SOX2 via the ubiquitin-proteasome system, GX-BP1 targets a central driver of tumor relapse, metastasis, and resistance to standard therapies.

Preclinical data demonstrated strong anti-tumor activity across multiple models. GX-BP1 monotherapy achieved approximately 70% tumor growth inhibition (TGI), confirming meaningful standalone efficacy.

In combination settings, GX-BP1 restored sensitivity in chemotherapy-resistant models and enhanced the efficacy of EGFR-targeted therapies, including osimertinib, by suppressing SOX2-driven resistance mechanisms.

Notably, the combination of GX-BP1 with carboplatin and paclitaxel showed a clear dose-dependent response, achieving 87% to 96% TGI, with near-complete tumor growth suppression at higher doses. In addition, while tumor regrowth was observed in the osimertinib monotherapy group, the combination of GX-BP1 with osimertinib completely prevented tumor relapse, accompanied by effective elimination of cancer stem cell populations.

These findings position GX-BP1 as a potential backbone therapy for combination strategies, with broad applicability across chemotherapy, targeted therapy, and immunotherapy settings, particularly in resistant or refractory disease.

Genexine has also established a clinically relevant delivery approach using a lung-targeted lipid nanoparticle (LNP) system, enabling efficient systemic delivery of over 70% GX-BP1 mRNA to lung tissues within 24 hours.

GX-BP1 has demonstrated a comprehensive preclinical profile, including in vivo efficacy, pharmacokinetics, biodistribution, and safety, supporting advancement into IND-enabling studies. Genexine is actively pursuing global licensing and strategic partnership opportunities for GX-BP1 and its bioPROTAC platform-based pipelines.

"GX-BP1 represents a differentiated therapeutic approach that directly targets a central driver of cancer resistance," said Jaehyun Choi, Ph.D., Chief Executive Officer and Head of R&D at Genexine. "We believe its strong combination potential, and GX-BP1’s robust preclinical profile position it as a promising next-generation therapeutic candidate, and we are advancing discussions with global partners."

(Press release, Genexine, APR 28, 2026, View Source [SID1234664875])

U.S. Food and Drug Administration Accepts New Drug Application for Zipalertinib for the Treatment of Locally Advanced or Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations

On April 28, 2026 Taiho Oncology, Inc., Taiho Pharmaceutical Co., Ltd., and Cullinan Therapeutics, Inc. (Nasdaq: CGEM) reported that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for zipalertinib for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion (ex20ins) mutations whose disease has progressed on or after platinum-based chemotherapy, with or without amivantamab. The Prescription Drug User Fee Act (PDUFA) target action date is February 27, 2027.

The NDA is supported by data from the Phase 2b part of the REZILIENT1 clinical trial of zipalertinib monotherapy in patients with NSCLC harboring EGFR ex20ins mutations who have received prior therapy. The study met its primary endpoint of objective response rate. Study results from REZILIENT1 were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and simultaneously published in the Journal of Clinical Oncology.

"Zipalertinib was discovered at Taiho Pharmaceutical Co., Ltd., and has been developed with a focus on addressing the unmet needs of patients with EGFR exon 20 insertion-mutated non-small cell lung cancer," said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. "The FDA’s acceptance of the NDA for zipalertinib is an important milestone for this program, and we look forward to working with FDA during the review process."

"Zipalertinib is a compound created using Taiho Pharmaceutical’s proprietary drug discovery and development technologies, Cysteinomix, with the aim of delivering a new treatment option to address high unmet medical needs," said Takeshi Sagara, PhD, Executive Director, Board Member, Medical Affairs, Translational Development, Clinical Development, Discovery and Preclinical Research at Taiho Pharmaceutical. "The FDA’s acceptance of the NDA represents an important milestone, reflecting the scientific and clinical data accumulated to date. We will continue to work closely with Taiho Oncology, Cullinan Therapeutics and the FDA throughout the review process, with the shared goal of ultimately delivering a new treatment option to patients with non-small cell lung cancer EGFR exon 20 insertion mutations."

"FDA acceptance of the zipalertinib NDA is an important step toward making zipalertinib available for people living with non-small cell lung cancer with EGFR exon 20 insertion mutations, who continue to face limited treatment options," said Jeffrey Jones, MD, MBA, Chief Medical Officer, Cullinan Therapeutics. "We are deeply grateful to the patients and families who have participated in the REZILIENT program, and to the investigators, study teams, and advocates whose collaboration made achievement of this milestone possible. We believe zipalertinib has the potential to help address a significant unmet need, and we look forward to working with our partners at Taiho with the goal of bringing zipalertinib to patients waiting for new treatment options."

Summary of Primary Study Results:

Zipalertinib demonstrated clinically meaningful efficacy in the primary efficacy population (n=176), including 51 patients who had received prior amivantamab.
The confirmed objective response rate (ORR) was 35%. Median duration of response (mDOR) was 8.8 months.
In patients treated after prior platinum-based chemotherapy only (n=125), ORR was 40% with a mDOR of 8.8 months.
In exploratory subgroup analyses:
Patients who had received prior amivantamab without other ex20ins-targeted therapy (n=30) showed a confirmed ORR of 30% and mDOR of 14.7 months.

Patients with brain metastases (n=68) showed a confirmed ORR of 31% and a mDOR of 8.3 months.
The safety profile of zipalertinib was manageable and consistent with previously reported data.¹ The most common treatment-emergent adverse events were paronychia, rash, anemia, dermatitis acneiform, diarrhea, dry skin, nausea and stomatitis. Most treatment-emergent adverse events were grade 1 or 2 per NCI-Common Terminology Criteria for Adverse Events (CTCAE v5.0).
Zipalertinib is an oral EGFR tyrosine kinase inhibitor. Zipalertinib received Breakthrough Therapy Designation in 2021 for the treatment of patients with locally advanced or metastatic NSCLC harboring EGFR ex20ins mutations who have previously received platinum‑based systemic chemotherapy.

About REZILIENT1

REZILIENT1 (Researching Zipalertinib in EGFR Non-Small Cell Lung Cancer Tumors) is a Phase 1/2 clinical trial (NCT04036682) to evaluate efficacy and safety of zipalertinib in adult patients with locally advanced or metastatic NSCLC harboring EGFR ex20ins mutations who have received prior therapy. Patients were treated with oral zipalertinib 100 mg twice daily. The primary endpoints were objective response rate (ORR) and duration of response (DOR) as assessed by blinded independent central review (ICR) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Adverse events were characterized and graded according to the NCI-Common Terminology Criteria for Adverse Events (CTCAE v5.0).

About Zipalertinib

Zipalertinib (development code: CLN-081/TAS6417) is an orally available small molecule designed to target activating mutations in EGFR. The molecule was selected because of its ability to inhibit EGFR variants with ex20ins mutations, while sparing wild-type EGFR. Zipalertinib is designed as a next generation, irreversible EGFR inhibitor for the treatment of a genetically defined subset of patients with non-small cell lung cancer. Zipalertinib has received Breakthrough Therapy Designation from the FDA for the treatment of patients with locally advanced or metastatic NSCLC harboring epidermal growth factor EGFR ex20ins mutations who have previously received platinum-based systemic chemotherapy. Zipalertinib is investigational and has not been approved by any health authority.

Zipalertinib is being developed by Taiho Oncology, Inc., and its parent company, Taiho Pharmaceutical Co., Ltd. worldwide, and in collaboration with Cullinan Therapeutics, Inc. in the U.S.

About EGFR Exon 20 Insertion Mutations

NSCLC is a common form of lung cancer and up to 4% of all cases globally have EGFR ex20ins, which makes them the third most common EGFR mutation subtype.2 In the United States, approximately 16% of patients with NSCLC harbor EGFR mutations,2 with insertions at exon 20 accounting for up to 12% of these mutations.

(Press release, Taiho, APR 28, 2026, View Source [SID1234664874])

Sapient Launches Tumor Protein Mapping Platform to Characterize Functional Biology Across Critical Dimensions in Human Tumors

On April 28, 2026 Sapient, a leader in multi-omics data generation for biomarker discovery and clinical insight delivery, reported the launch of its Tumor Protein Mapping Platform as a suite of mass spectrometry-based discovery proteomics workflows designed to map functional tumor biology across four critical dimensions: the druggable cell surface proteome, phosphorylation-driven signaling pathways, the tumor immune microenvironment, and therapeutic resistance mechanisms. The platform is now available to biopharma sponsors and comprises four purpose-built workflows – SurfaceSeek, SignalingSeek, ImmuneSeek, and ResistanceSeek – each optimized for both fresh-frozen and FFPE human tumor samples.

Central to the platform is SurfaceSeek, which directly measures proteins that are functionally deployed on the tumor cell surface to enable confident identification and validation of druggable targets for ADC, T-cell engager, and radioligand therapies. The workflow combines Sapient’s mass spectrometry-based discovery proteomics with selective enrichment of mature N-linked glycoproteins to preferentially identify proteins that have completed intracellular trafficking and are exposed on the extracellular surface. This enables direct characterization of surface target accessibility, density, and tumor selectivity, and brings peptide-level resolution to identify protein isoforms and post-translationally modified proteoforms bearing extracellular domains compatible with therapeutic binding.

The platform’s additional workflows complement and extend SurfaceSeek findings by mapping functional tumor biology that determines therapeutic outcome. SignalingSeek quantifies tumor signaling pathway activation via the measurement of phosphorylation events across critical oncogenic pathways, enabling direct assessment of on-target pathway modulation as well as identification of adaptive signaling and resistance pathways. ImmuneSeek measures functionally active tumor immune cells and the immune pathways that are driving therapeutic response, immune evasion, or suppression, while ResistanceSeek identifies the coordinated protein networks through which tumors adapt to therapeutic pressure across modalities.

"Building upon our next-generation FFPE Proteomics offering, we have developed specialized workflows that enable precise, multi-dimensional characterization of tumor biology at the protein level – and directly in human tumor tissue," said Jeramie Watrous, PhD, Co-Founder and Head of Analytical R&D at Sapient. "The key technical innovation is that these workflows – including SurfaceSeek’s selective glycoprotein enrichment and SignalingSeek’s deep phosphoproteomics – perform with exceptional concordance in both fresh-frozen and FFPE samples. This means we can apply them retrospectively to the vast biorepositories of archived tissue already collected, unlocking dimensions of functional tumor biology that were previously inaccessible in these samples."

"Tumors are not defined by a single biological dimension. They are complex and changing systems where surface target accessibility, signaling pathway activation, immune function, and resistance mechanisms all interact to determine therapeutic outcome," said Mo Jain, MD, PhD, Founder and Chief Scientific Officer at Sapient. "By mapping each of these dimensions directly at the protein level, we give drug development teams a comprehensive, unified view of what is actually governing drug response – moving oncology development beyond genomic inference alone, adding new layers of insight derived from the direct measurement of dynamic human tumor biology."

"This platform was shaped by many conversations with oncology leaders where we kept hearing the same thing: they were navigating some of the highest-stakes decisions in drug development, such as which targets to pursue, without the ability to directly measure those targets or the mechanisms that influence therapeutic outcome," added Jonathan Usuka, PhD, MBA, Chief Executive Officer at Sapient. "We designed the workflows to interrogate key dimensions of tumor biology that determine whether a drug will succeed, so our clients can advance their programs with direct evidence rather than inference."

All four workflows are available as standalone services or can be delivered in combination to provide integrated, multi-dimensional tumor characterization, and may be supported by Sapient’s DynamiQ Tumor-Tissue virtual biobank which offers streamlined access to annotated FFPE tumors and tissues.

(Press release, Sapient Discovery, APR 28, 2026, View Source [SID1234664873])

Remepy Announces Collaboration with Merck KGaA, Darmstadt Germany to Advance Hybrid Drugs Across Therapeutic Areas

On April 28, 2026 Remepy, the pioneer of Hybrid Drugs, reported a collaboration with Merck KGaA, Darmstadt Germany, a leading science and technology company, to explore the development of Hybrid Drugs across multiple therapeutic areas.

The collaboration will initially focus on some programs in the US in a rare tumor area, while potentially establishing a broader framework to explore additional Hybrid Drug opportunities across the therapeutic portfolio of Merck KGaA, Darmstadt, Germany, in the future.

Hybrid Drugs are an emerging therapeutic category that combine pharmacological treatments with personalized digital therapeutic protocols delivered through a mobile app. Research increasingly shows that the most effective care is multidisciplinary and integrative, combining medication with behavioral and therapeutic interventions. Hybrid Drugs integrate these evidence-based motor, physical, and cognitive interventions alongside medication to improve clinical outcomes1.

"We are excited about our collaboration with Merck KGaA, Darmstadt, Germany, that is a leading science and technology company," said Dr. Michal Tsur, Co-founder and Co-CEO at Remepy. "Remepy’s Hybrid Drug platform combines traditional drugs with evidence-based AI enabled digital interventions delivering personalized, adaptive integrative treatment. The new advances in regulatory frameworks, supporting the integration of software with drugs, enable the pharma industry to use the power of the digital world to differentiate drugs, enhance their efficacy and amplify their label. We are looking forward to accelerating the delivery of innovative and effective therapies to patients who need them most."

Recent developments, including the introduction of FDA Prescription Drug Use-Related Software (PDURS) guidance, and evolving SaMD-Drug combination pathways are creating clearer regulatory routes for integrating digital therapeutic components directly into pharmaceutical products. Advances in FDA digital health initiatives and guidance on AI for medical devices are opening the door to a new generation of innovative therapeutic solutions.

Hybrid Drugs introduce a new economic model for digital health. By combining a drug and a therapeutic application into a single prescription product, hybrid drugs shift digital health innovation into the established economics of pharmaceutical development, commercialization, and reimbursement.

(Press release, Merck KGaA, APR 28, 2026, View Source [SID1234664872])

Ivonescimab Receives Major Recommendations Across Multiple Therapies in the 2026 CSCO NSCLC Guideline

On April 28, 2026 Akeso, Inc. (9926.HK) ("Akeso" or the "Company") reported that ivonescimab, the company’s first-in-class PD-1/VEGF bispecific antibody, has secured multiple authoritative updates and upgrades across first-line and later-line settings in the officially updated 2026 Chinese Society of Clinical Oncology (CSCO) Guideline for the Diagnosis and Treatment of Non-Small Cell Lung Cancer (NSCLC). These strong recommendations further solidify ivonescimab’s breakthrough clinical value and its position as a new standard of care (SOC), highlighting its leadership as a next-generation immunotherapy driving the IO 2.0 era.

Key Highlights

In the Phase III HARMONi-2 study, ivonescimab monotherapy demonstrated superior progression-free survival compared with pembrolizumab in first-line PD-L1-positive (TPS ≥1%) NSCLC. Based on these results, ivonescimab has been upgraded to a Class I recommendation for first-line treatment of both squamous and non-squamous NSCLC with PD-L1 TPS ≥1%. This indication was previously approved in China and included in the National Reimbursement Drug List (NRDL). The upgrade strengthens its position as a preferred first-line option for PD-L1-positive advanced NSCLC.

In the Phase III HARMONi-6 study, ivonescimab plus chemotherapy showed positive results versus PD-1 inhibitor plus chemotherapy in first-line squamous NSCLC. This combination has received a new Class II recommendation for first-line treatment of squamous NSCLC. The supplemental application for this indication is currently under regulatory review.

Latest CSCO Guideline Recommendations for Ivonescimab

Post-resistance treatment in EGFR-mutant NSCLC: Ivonescimab plus chemotherapy maintains a Class I recommendation.
First-line treatment for advanced driver gene-negative squamous NSCLC with PD-L1 TPS ≥1%: Ivonescimab monotherapy upgraded to Class I recommendation.
First-line treatment for advanced driver gene-negative non-squamous NSCLC with PD-L1 TPS ≥1%: Ivonescimab monotherapy upgraded to Class I recommendation.
First-line treatment for advanced driver gene-negative squamous NSCLC: Ivonescimab plus chemotherapy newly added as Class II recommendation.
To date, the breakthrough clinical value of ivonescimab has been demonstrated in dozens of clinical trials and real-world experience involving more than 70,000 patients. It has gained wide acceptance among oncologists and patients, contributing to the ongoing advancement of immuno-oncology treatment paradigms globally.

(Press release, Akeso Biopharma, APR 28, 2026, View Source [SID1234664871])