Personalis to Participate at the 24th Annual Needham Virtual Healthcare Conference

On March 26, 2025 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for precision oncology, reported that its management team will be attending the 24th Annual Needham Virtual Healthcare Conference on Wednesday, April 9, 2025 (Press release, Personalis, MAR 26, 2025, View Source [SID1234651494]).

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GV20 Therapeutics Announces Collaboration Agreement with Mitsubishi Tanabe Pharma Corporation to Develop Novel Antibody-Drug Conjugates

On March 26, 2025 GV20 Therapeutics (GV20), a clinical-stage AI-powered biotherapeutics company, reported to have entered into a strategic research collaboration with Mitsubishi Tanabe Pharma Corporation (MTPC) (Press release, GV20 Therapeutics, MAR 26, 2025, View Source [SID1234651493]). Under this collaboration, MTPC will leverage GV20’s antibodies, which are specifically directed against novel tumor antigen targets discovered through GV20’s proprietary STEAD AI platform, to generate potentially first-in-class antibody-drug conjugates.

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"This agreement underlines the strength of GV20’s STEAD platform in discovering novel tumor targets and human-derived antibodies." said Ying Gong, Ph.D., Chief Business Officer of GV20. "We are excited to partner with MTPC, leveraging their deep expertise in linker-payload technology and therapeutics development, to advance innovative antibody-drug conjugate (ADC) therapies against these novel targets."

Under the terms of the agreement, GV20 will receive an upfront payment and is eligible for milestone payments. MTPC will receive an exclusive right to negotiate a license to these antibodies during the collaboration term.

Sapience Therapeutics to Present Multiple Posters at the American Association for Cancer Research (AACR) Annual Meeting 2025

On March 26, 2025 Sapience Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery and development of peptide therapeutics to address oncogenic and immune dysregulation that drive cancer, reported four poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, taking place April 25-30, 2025, in Chicago, Illinois (Press release, Sapience Therapeutics, MAR 26, 2025, View Source [SID1234651492]). Sapience will present non-clinical results from both of its clinical programs, lucicebtide (formerly known as ST101), a first-in-class antagonist of C/EBPβ, and ST316, a first-in-class antagonist of β-catenin and its co-activator, BCL9, as well as results from its preclinical Fra1 antagonist peptide (FraAP) program, a first-in-class antagonist of the activator protein 1 (AP-1) complex.

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"We are thrilled to be presenting a compendium of non-clinical data supportive of our clinical and preclinical SPEARs programs, including lucicebtide, ST316, and our FraAP program, at the upcoming AACR (Free AACR Whitepaper) Annual Meeting," said Jim Rotolo, Ph.D., Sapience’s Chief Scientific Officer. "Results from our lucicebtide and ST316 studies further elucidate their respective mechanisms of action and highlight their potential for combination strategies. Additionally, preclinical data demonstrate the potential of our FraAP program to induce anti-cancer activity by targeting the AP-1 complex, particularly in head and neck squamous cell carcinoma."

Poster Presentation Details and Abstract Highlights:

Title: "The clinical C/EBPβ antagonist peptide lucicebtide synergizes with molecularly targeted therapies in GBM"
Session Title: Targeted Therapies and Combinations 1
Location: Poster Section 34
Abstract Number: 839
Date and Time: Sunday, April 27, 2025, 2:00 pm – 5:00 pm CDT
Abstract Summary:

In non-clinical studies, the mechanism of lucicebtide, which has previously demonstrated antitumor activity via direct cell death and immune activation within the tumor microenvironment, enabled a synthetic lethal effect with molecularly targeted therapeutics, such as EGFR inhibitors, that are typically not effective when used as monotherapies for the treatment of glioblastoma (GBM). Transcriptional analysis utilizing GBM genetics and signatures of C/EBPβ activity were performed to identify potential target populations likely to benefit from lucicebtide combinations.
Title: "FraAP, a peptide antagonist against the activator protein 1 transcription factor complex, demonstrates cancer cell cytotoxicity and reduced invasion in vitro and tumor regression in vivo in HNSCC models"
Session Title: Gene Regulation and Transcription Factors 2
Location: Poster Section 10
Abstract Number: 1415
Date and Time: Monday, April 28, 2025, 9:00 am – 12:00 pm CDT
Abstract Summary:

FraAP selectively binds to and inhibits key oncogenic drivers of the AP-1 transcription factor complex, resulting in the suppression of AP-1 driven pathways required for cancer cell cycle progression, proliferation, and invasion and promotes apoptosis in preclinical studies. Further, in a head and neck squamous cell carcinoma (HNSCC) xenograft model, administration of FraAP results in significant tumor growth inhibition, further demonstrating the anti-cancer potential of targeting the AP-1 complex.
Title: "An ex vivo organoid study to identify biomarkers of sensitivity to ST316, a clinical-stage β-catenin antagonist peptide"
Session Title: Biomarkers and Molecular Targets for Prevention
Location: Poster Section 43
Abstract Number: 2361
Date and Time: Monday, April 28, 2025, 9:00 am – 12:00 pm CDT
Abstract Summary:

Analysis of 60 patient-derived tumor organoids revealed that tumors with TP53 WT and mutations in the MAPK pathway significantly correlate with sensitivity to ST316, a β-catenin antagonist peptide. Further, in non-clinical models, combining ST316 with encorafenib, a BRAF inhibitor, showed synergistic effects in BRAFV600E-mutant colorectal cancer (CRC) organoids, demonstrating a promising combination approach in this poor prognosis and underserved patient population. Across studies, findings support rational combinations of ST316 with clinically relevant MAPK inhibitors.
Title: "Reprogramming of MDSCs by ST316, a clinical peptide antagonist of β-Catenin/BCL9, enhances anti-tumor immuno- and chemotherapy"
Session Title: Modifiers of Tumor Microenvironment
Location: Poster Section 30
Abstract Number: 3275
Date and Time: Monday, April 28, 2025, 2:00 pm – 5:00 pm CDT
Abstract Summary:

Results reveal a novel role for Wnt/β-catenin signaling in myeloid-derived suppressor cell (MDSC) biology, a key mediator of immune-suppression, whereby antagonism of β-catenin with ST316 leads to suppression of MDSC expansion. In combination studies, ST316 significantly enhances the anti-tumor efficacy of anti-PD-1 immunotherapy and standard-of-care chemotherapies for advanced CRC such as FOLFIRI. While anti-PD-1 and FOLFIRI alone each resulted in expansion of immunosuppressive MDSCs, combination with ST316 prevented this expansion. These data suggest that therapeutically targeting the β-catenin/BCL9 interaction may be an effective strategy to enhance both chemotherapy and immunotherapy in Wnt-driven tumors that respond poorly to monotherapy.
More information can be found on the AACR (Free AACR Whitepaper) Annual Meeting 2025 website.

About Lucicebtide (formerly known as ST101)
Lucicebtide, a first-in-class antagonist of C/EBPβ, has completed the main portion of a Phase 2 dose expansion study in recurrent GBM (rGBM) (NCT04478279). An ongoing window-of-opportunity sub-study is evaluating lucicebtide in combination with radiation and temozolomide in patients with newly diagnosed GBM (ndGBM) and as a monotherapy in patients with rGBM, with patients receiving ST101 before and after surgical resection in both cohorts. ST101 has been granted Fast Track designation for rGBM from the U.S. Food and Drug Administration (U.S. FDA) and orphan designations for glioma from the U.S. FDA and the European Commission.

About ST316
ST316 is a first-in-class antagonist of the interaction between β-catenin and its co-activator, BCL9, a complex responsible for driving oncogene expression and immune exclusion in multiple cancers where aberrant Wnt/β-catenin pathway signaling is observed. ST316 exposure in cancer cells prevents BCL9-driven nuclear localization of β-catenin and formation of the enhanceosome protein complex. Disruption of this interaction selectively suppresses the transcription of oncogenic Wnt target genes that regulate proliferation, migration, invasion and the metastatic potential of tumor cells, as well as genes that regulate the immunosuppression of the tumor microenvironment. ST316 creates a pro-immune tumor microenvironment and in preclinical models has shown to be synergistic with checkpoint inhibition. Due to its selectivity and downstream modulation of the Wnt/β-catenin pathway, ST316 presents an opportunity to safely and effectively target Wnt/β-catenin driven pathologies without the toxicities previously seen with other Wnt pathway agents.

ST316-101 (NCT05848739) is a first-in-human, open-label, Phase 1-2 Dose Escalation and Expansion study designed to determine the safety, tolerability, PK, PD and early efficacy of ST316. The Phase 1 Dose Escalation portion of the study tested various dose levels of ST316 in patients with select advanced solid tumors that are known to harbor abnormalities of the Wnt/β-catenin signaling pathway, including colorectal cancer (CRC). ST316 is currently being tested in the Phase 2 portion of the study in CRC patients in combination with relevant standards of care and in multiple lines of treatment. The U.S. Food and Drug Administration (U.S. FDA) has granted Orphan Drug Designation to ST316 for the treatment of familial adenomatous polyposis (FAP).

Hoth Therapeutics Advances Cancer Fighting HT-KIT Program with New Patent Filing and Preclinical Study Initiation

On March 26, 2025 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a biopharmaceutical company focused on developing first-in-class therapies for allergic and inflammatory diseases, reported two major developments in the ongoing advancement of its proprietary antisense oligonucleotide (ASO) cancer fighting drug candidate, HT-KIT (Press release, Hoth Therapeutics, MAR 26, 2025, View Source [SID1234651491]).

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The Company has filed amended claims with the U.S. Patent and Trademark Office for its lead ASO technology targeting MS4A6A and FcεRIβ—genes implicated in allergic inflammation and mast cell-related diseases. These refined claims enhance intellectual property protection for HT-KIT and further position it as a novel therapeutic platform with broad clinical potential, including use in treating conditions such as anaphylaxis, mastocytosis, and allergic asthma.

In parallel, Hoth has initiated a GLP-compliant 4-week intravenous toxicity study with a 14-day recovery period in C57BL/6 mice. This preclinical study, conducted in partnership with OnTargetx R&D Inc. and ITR Laboratories, is designed to evaluate the safety profile of HT-KIT in support of upcoming regulatory filings. The study will include multiple dose groups, detailed pathology assessments, and pharmacokinetic profiling.

"These strategic milestones mark significant momentum for HT-KIT as we move toward clinical readiness, "said Robb Knie, CEO of Hoth Therapeutics. "The strengthened patent position and robust preclinical package will be instrumental as we explore partnerships and advance our discussions with regulators."

Next Steps in Development

Hoth Therapeutics is committed to advancing HT-KIT toward clinical evaluation. The company is currently conducting additional preclinical studies to further validate HT-KIT’s efficacy and safety profile, with plans to initiate regulatory discussions for first-in-human trials.

Antengene to Present Results From Four Cutting Edge Preclinical Studies at AACR 2025 with Focuses Including AnTenGagerTM TCEs and Synthetic Lethality

On March 26, 2025 Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for hematologic malignancies and solid tumors, reported that it will release results from four preclinical studies in poster presentations at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (AACR 2025), taking place from April 25th to 30th at the McCormick Place Convention Center, Chicago, the United States (Press release, Antengene, MAR 26, 2025, View Source [SID1234651490]). These four posters will feature Antengene’s four highly differentiated and high-potential programs, including ATG-201 (CD19 x CD3 TCE) and ATG-042 (MTAPnull-selective PRMT5 Inhibitor), which are poised to enter clinical development in the second half of 2025; ATG-110 (LY6G6D x CD3 TCE), which was developed on the AnTenGagerTM TCE 2.0 platform for the treatment of microsatellite stable colorectal cancer; and a companion diagnostic antibody developed to assess CD24 expression and guide clinical studies of CD24-targeted therapies.

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Details of the Poster Presentation:
ATG-201 (CD19 x CD3 T cell engager)
Title: ATG-201, a novel T-cell engager (TCE) effectively depletes B cells with reduced risk of CRS for the treatment of B cell malignancies and B cell related autoimmune diseases
Abstract Number: 7326
Session Category: Immunology
Session Title: T Cell Engagers and Novel Antibody-Based Therapies
Date: April 30, 2025
Time: 9:00 AM – 12:00 PM (Central Time)
10:00 PM, April 30, 2025 – 1:00 AM, May 1, 2025 (Beijing Time)
Location: Poster Section 40

Introduction: By depleting autoreactive B cells, CD19-targeted CAR-T have shown early yet promising efficacy in treating patients with B cell-driven autoimmune diseases. However, the clinical application of TCE continues to be greatly hindered by the unfavorable pharmacokinetics and toxicity associated with cytokine release syndrome. To overcome these limitations, Antengene developed ATG-201, a "2+1" CD19 x CD3 TCE, which was evaluated in a series of in vitro studies for binding affinity, T cell dependent cytotoxicity (TDCC) cytokine release, and drug developability. The in vivo anti-lymphoma efficacy and pharmacodynamic effect were evaluated in Raji xenograft model. The tissue resident B cell depletion was assessed in CD34+ hematopoietic stem cells humanized mice. Pharmacokinetic parameters of ATG-201 were evaluated in normal Balb/c mice.
Results: Compared to benchmark TCEs, ATG-201 demonstrated stronger naïve B cell depletion activity with much lower cytokine release in vitro. ATG-201 showed potent anti-lymphoma activity in PBMC-humanized subcutaneous Raji xenograft model with significant augment of infiltrated CD8+ T cells in tumor microenvironment and limited proinflammatory cytokines detected in plasma. Single dose ATG-201 completely and deeply depleted B cells in blood, bone marrow and spleen of CD34+ cells humanized mice. ATG-201 demonstrated potent efficacy in mouse systemic lupus erythematosus model, inhibiting the lymph node swelling and auto-antibody producing.
Conclusions: ATG-201 demonstrated CD19-dependent CD3 binding and activation, inducing effective B cell depletion in vitro and in vivo with low cytokine release, which provides potential for the treatment of B cell malignancies and B cell related autoimmune diseases. ATG-201 is poised to enter clinical development in the second half of 2025.
ATG-042 (MTAPnull-selective PRMT5 Inhibitor)
Title: Preclinical characterization of ATG-042, a novel MTAPnull-selective PRMT5 inhibitor
Abstract Number: 4230
Session Category: Experimental and Molecular Therapeutics
Session Title: HDAC and Methyltransferase Inhibitors
Date: April 29, 2025
Time: 9:00 AM – 12:00 PM (Central Time)
10:00 PM, April 29, 2025 – 1:00 AM, April 30, 2025 (Beijing Time)
Location: Poster Section 16

Introduction: Targeting the PRMT5-MTA complex has become a promising strategy for treating MTAPnull cancer in a synthetically lethal manner, avoiding on-target off-tumor hematological toxicity when using first-generation, non-selective PRMT5 inhibitors. Herein, Antengene developed ATG-042, a novel MTAPnull-selective PRMT5 small molecule inhibitor with good brain penetration. In this study, the in vitro activity and MTAP selectivity of ATG-042 were profiled using HCT116 MTAP wild type (wt) cells, HCT116 MTAP knock out (ko) cells and multiple endogenous MTAPnull cell lines. The in vivo efficacy was tested in cell derived xenograft (CDX) mouse models with HCT116 MTAP wt cells, HCT116 MTAP ko cells, LU99 cells (MTAPnull) and U87MG-luc (MTAPnull). The pharmacokinetic and toxicological properties were assessed with corresponding assay methods.
Results: ATG-042 showed excellent anti-proliferative activities on multiple endogenous MTAPnull cell lines with IC50 values between 10nM and 100nM. ATG-042 demonstrated high permeability, good metabolic stability, and low risk of drug-drug interaction. In vivo PK study shows that ATG-042 is well absorbed, with a dose-dependent increase in plasma distribution and high oral bioavailability in mice, SD rats and beagle dogs. Furthermore, ATG-042 is brain-penetrable (B/P ratio=51% in mice; KPuubrain=0.73 in rats). ATG-042 showed robust in vivo efficacy in both subcutaneous CDX models (HCT116 -MTAP ko, LU99) and orthotopic CDX model (U87MG-luc) as a single agent. In addition, ATG-042 also exhibited potential synergy in combination with other drugs for antitumor therapy.
Conclusions: ATG-042 is an oral MTAPnull-selective PRMT5 inhibitor with potent efficacy against MTAPnull tumor. It also demonstrated good tolerability and brain penetrability. ATG-042 is poised to enter clinical development in the second half of 2025.
ATG-110 (LY6G6D x CD3 T cell engager)
Title: ATG-110, a novel "2+1" LY6G6D-targeted T-cell engager (TCE) for the treatment of MSS colorectal cancer
Abstract Number: 3509
Session Category: Immunology
Session Title: T Cell Engagers
Date: April 28, 2025
Time: 2:00 PM – 5:00 PM (Central Time)
3:00 AM – 6:00 AM, April 29, 2025 (Beijing Time)
Location: Poster Section 38

Introduction: Colorectal cancer (CRC) is one of the most common cancers worldwide and requires more effective and safer therapies to improve the poor survival outcome, particularly in patients with microsatellite stable (MSS) colorectal cancer, who exhibit primary resistance to immune checkpoint inhibitors and lack effective treatment options. T cell engagers have shown encouraging efficacy in treating hematological malignancies, while exhibiting suboptimal clinical efficacies in solid tumors. The risk of cytokine release syndrome (CRS) remains as a significant challenge clinically. ATG-110 is a novel "2+1" LY6G6D x CD3 TCE developed by Antengene. In this study, ATG-110 was evaluated in a series of preclinical in vitro studies for binding affinity, T cell activation and cytokine release, T cell dependent cytotoxicity (TDCC), and drug developability. The in vivo anti-tumor efficacy was evaluated in PBMC-humanized immunodeficient NDG mice engrafted with LY6G6Dmedium-expression HT55 or LY6G6Dvery low-expression SW480 MSS CRC cells.
Results: ATG-110 binds to LY6G6D-positive cell lines, including LY6G6D-overexpression 293T, HT55, LS1034, with the nanomolar grade EC50. The CD3 binding site of ATG-110 is concealed by the LY6G6D Fab arm before binding to LY6G6D, due to the steric hindrance. Therefore, ATG-110 demonstrated limited binding capability to CD3+ cells before LY6G6D crosslinking. It activates T cells and induces cytokine release only in the presence of LY6G6D+ cells. In vitro, ATG-110 resulted in potent T cell dependent cytotoxicity with single-digit pM IC50 values on HT55 and SW480 cells. ATG-110 also showed potent anti-tumor activity in PBMC-humanized SW480 xenograft model and exhibited complete response (CR) in PBMC-humanized HT55 xenograft model. Furthermore, ATG-110 also demonstrated good drug developability.
Conclusions: ATG-110 demonstrated LY6G6D-dependent CD3 binding and activation with low risk of CRS. It showed powerful in vitro and in vivo anti-tumor efficacy against colorectal cancer, which warrants further clinical evaluation.
ATG1144 (CD24 CDx Antibody)
Title: Development of a diagnostic antibody for CD24 targeted therapy
Abstract Number: 671
Session Category: Clinical Research
Session Title: Diagnostic Biomarkers 2
Date: April 27, 2025
Time: 2:00 PM – 5:00 PM (Central Time)
3:00 AM – 6:00 AM, April 28, 2025 (Beijing Time)
Location: Poster Section 29

Introduction: CD24 has emerged as a promising therapeutic target for anti-cancer treatment. Several clinical trials are being conducted to evaluate the safety and efficacy of CD24-targeted therapies. Here, Antengene developed and characterized an anti-CD24 diagnostic antibody to enhance the screening and selection of patients based on CD24 expression. In this study, the authors described the discovery of the diagnostic antibody, and the evaluation of accuracy, sensitivity (selectivity), specificity, and assay precision of the antibody.
Results: Monoclonal antibody clone ATG1144 binds to the hCD24 core peptide in ELISA with an EC50 of 0.06 nM. Distinct membrane staining on human normal esophageal tissue FFPE specimens can also be observed with IHC staining using ATG1144. For accuracy assessment, six CDX and twenty human specimens, comprising both positive and negative specimens (including solid tumors and B-cell non-Hodgkin lymphomas), were validated. Samples exhibiting high, medium, and low CD24 expression levels were evaluated for sensitivity and specificity, and the interpreted results aligned with the reference outcomes. FFPE tissues from three distinct patients were evaluated for assay precision assessment. The TMA IHC staining result revealed that 50-80% of patients with lung, breast, bladder, ovarian, or liver cancer have CD24 expression on tumor cell surface with low expression in the para-cancerous normal tissue.
Conclusions: ATG1144 specifically binds to human CD24 with high sensitivity as demonstrated by IHC staining. The development and validation of the method have been finalized using Leica Bond III platforms. These data suggest a potential diagnostic use of ATG1144 for identifying CD24+ patients.