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Kura Oncology Showcases Preclinical Data for Next-Generation FTI KO-2806 in Combination Therapies

Targeted Therapy
  • Kura Oncology presented preclinical data demonstrating KO-2806's potential to overcome drug resistance when combined with PI3Kα inhibitors, KRAS inhibitors, and antiangiogenic TKIs across multiple tumor types.
  • The company's next-generation farnesyl transferase inhibitor showed ability to re-sensitize tumors to KRAS inhibitors in preclinical NSCLC and colorectal cancer models of relapsed settings.
  • KO-2806 combinations could potentially address more than 200,000 incident patients annually in the U.S., with clinical data to be presented at ESMO Congress 2025 in October.
  • Three Phase 1 clinical trials are ongoing, including combination studies with cabozantinib in renal cell carcinoma and monotherapy evaluation in advanced solid tumors.

BostonGene's AI Model Identifies Five Novel Subtypes to Predict Immunotherapy Response in Kidney Cancer

AITargeted TherapyPrecision MedicineOncology
  • BostonGene published a study in Cell Reports Medicine introducing the largest harmonized transcriptomic and clinical dataset in kidney cancer, analyzing over 3,600 patient samples.
  • Researchers developed a multimodal AI foundation model that identified five novel Harmonized Immune Tumor Microenvironment (HiTME) subtypes in clear cell renal cell carcinoma.
  • The AI model generated clinically interpretable responder scores that correlated with survival outcomes for both immune checkpoint inhibitors and VEGF inhibitors across independent cohorts.
  • The framework revealed a therapy-resistant subgroup characterized by immune-desert phenotypes and angiogenic signaling, highlighting new therapeutic opportunities.

Step Pharma Initiates Phase 1b Trial of Dencatistat for Essential Thrombocythaemia

Rare DiseaseTargeted Therapy
  • Step Pharma has dosed the first participant in a phase 1b clinical trial evaluating dencatistat for essential thrombocythaemia, marking the third indication for this first-in-class CTPS1 inhibitor.
  • The open-label trial will assess safety, tolerability, and preliminary activity in approximately 20 adults with high-risk essential thrombocythaemia who are resistant to or intolerant of hydroxycarbamide therapy.
  • Previous lymphoma trials demonstrated that dencatistat produces dose-dependent and reversible platelet count reduction, providing rationale for its investigation in essential thrombocythaemia treatment.
  • The study expands Step Pharma's clinical program beyond lymphoma and solid tumours, demonstrating the versatility of their "pipeline in a product" strategy.

A Phase 1 Study to Assess STP938 as a Monotherapy in Adults With High Risk Essential Thrombocythaemia

NCT06786234RecruitingPhase 1
Step Pharma, SAS
Posted 6/18/2025

Hengrui Medicine Receives China's First EZH2 Inhibitor Approval for Hematological Malignancies

Drug ApprovalTargeted Therapy
  • Hengrui Medicine's Zemetostat received conditional approval from China's NMPA, becoming the country's first EZH2 inhibitor for treating relapsed or refractory peripheral T-cell lymphoma.
  • The drug demonstrated a 34% objective response rate in Phase II trials for patients with relapsed/refractory follicular lymphoma and EZH2 mutations, addressing a critical unmet medical need.
  • Hengrui's regulatory milestone positions the company to capture significant market share in the global EZH2 inhibitors market, projected to reach $9.59 billion by 2034.
  • The approval represents a strategic breakthrough for Hengrui's transition from generic drugs to innovative therapies, with innovative drug revenue accounting for 61% of total income.

Tazemetostat for the Treatment of Relapsed/Refractory Follicular Lymphoma

NCT05467943CompletedPhase 2
Hutchmed
Posted 7/29/2022

Bicycle Toxin Conjugates Emerge as Novel Cancer Drug Class with Promising Clinical Results

Targeted TherapyOncology
  • Bicycle toxin conjugates (BTCs) represent a rapidly evolving field in targeted oncology that combines the binding affinity of bicyclic peptides with cytotoxic chemotherapy activity.
  • Zelenectide pevedotin (BT8009) demonstrated striking efficacy in Phase 1/2 trials when combined with immune checkpoint inhibitors, showcasing the potential of upfront combination regimens.
  • The technology offers advantages over conventional antibody-drug conjugates through deep tissue penetration and decreased systemic exposure while maintaining therapeutic activity.
  • Regulatory bodies including the US FDA are showing increasing confidence in BTCs through ramp-up programs, indicating growing institutional acceptance of this novel therapeutic approach.

First-in-Class STAT3 Inhibitor Shows Promise in Feline Head and Neck Cancer Trial with Human Applications

Targeted Therapy
  • A novel STAT3-targeted therapy achieved disease control in 35% of cats with head and neck squamous cell carcinoma, marking the first successful targeting of this previously "undruggable" transcription factor.
  • The clinical trial enrolled 20 cats with advanced HNSCC, with seven showing partial response or stable disease and an average survival of 161 days post-treatment.
  • Researchers demonstrated remarkable clinical and molecular similarities between feline and human HNSCC, positioning companion animals as superior disease models compared to traditional mouse studies.
  • The compound works through dual mechanisms, blocking STAT3 activity while elevating PD-1 levels to enhance immune response against tumors.

Abdera Therapeutics Reports Promising Early Data for DLL3-Targeted Radiopharmaceutical ABD-147 in Small Cell Lung Cancer

Targeted Therapy
  • Abdera Therapeutics presented initial Phase 1 clinical data for ABD-147, a novel DLL3-targeting radiopharmaceutical delivering Actinium-225 to small cell lung cancer tumors.
  • The first-in-human trial demonstrated selective tumor uptake, rapid clearance from blood and kidneys, and favorable safety profile with disease stabilization observed in advanced patients.
  • ABD-147 has received FDA Fast Track designation for extensive stage small cell lung cancer and targets DLL3, which is expressed in approximately 85% of SCLC patients.
  • The independent safety review committee recommended continued dose escalation based on promising pharmacokinetic, dosimetry, and preliminary efficacy data from six patients in the initial cohort.

Akiram's Targeted Radiotherapy AKIR001 Advances to Next Phase After Completing First Patient Cohort Without Safety Concerns

Targeted TherapyOncologyOrphan Drug
  • Swedish biotech Akiram Therapeutics completed the first patient cohort of its Phase I trial for AKIR001, a targeted radiopharmaceutical combining an anti-CD44v6 antibody with lutetium-177 for aggressive solid tumors.
  • No dose-limiting toxicities or safety concerns were observed in the initial cohort, allowing the trial to proceed to the next stage as planned at Karolinska University Hospital.
  • The study targets patients with difficult-to-treat cancers including anaplastic thyroid, head and neck, gynecological, and non-small cell lung cancers.
  • AKIR001 delivers radiation directly to tumor cells while minimizing damage to surrounding healthy tissue through its selective targeting of the CD44v6 cancer marker.

[177Lu]Lu-AKIR001 First-in-human Study

NCT06639191Not Yet RecruitingEarly Phase 1
Karolinska University Hospital
Posted 11/1/2024

Larkspur Biosciences Unveils First-in-Class PIP4K2C Degrader LRK-4189 for Microsatellite Stable Colorectal Cancer

Targeted Therapy
  • Larkspur Biosciences announced the discovery of LRK-4189, a first-in-class degrader targeting the lipid kinase PIP4K2C for treating microsatellite stable colorectal cancer.
  • The orally bioavailable drug demonstrated superior efficacy compared to cetuximab in patient-derived spheroids, with 50% response rate versus 35% for the benchmark therapy.
  • LRK-4189 has completed IND-enabling studies and is expected to enter Phase 1 clinical trials in the fourth quarter of 2025.
  • The drug targets cancer cell fitness by degrading PIP4K2C, triggering intrinsic apoptosis and activating immune-mediated tumor clearance mechanisms.

BlossomHill Therapeutics Advances EGFR-Mutant NSCLC Treatment with First-in-Class OMNI-EGFR Inhibitor BH-30643

Targeted TherapyOncology
  • BlossomHill Therapeutics has dosed the first patient in expansion cohorts of the SOLARA Phase 1/2 trial evaluating BH-30643, a first-in-class OMNI-EGFR inhibitor for advanced EGFR-mutant non-small cell lung cancer.
  • BH-30643 demonstrates sub-nanomolar potency against classical and atypical EGFR mutations while maintaining activity against T790M and C797S resistance mutations that commonly limit current therapies.
  • The expansion cohorts will assess objective response rates across diverse EGFR mutation subtypes, including treatment-naive patients, following successful dose escalation that showed favorable pharmacokinetics and preliminary anti-tumor activity.
  • The drug's broad-spectrum approach aims to address multiple EGFR mutations with a single agent while sparing wildtype EGFR and HER2 inhibition for improved tolerability.

A Study of BH-30643 in Subjects With Locally Advanced or Metastatic NSCLC Harboring EGFR and/or HER2 Mutations

NCT06706076RecruitingPhase 1
BlossomHill Therapeutics
Posted 1/9/2025

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