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Novel RAS-PI3K Inhibitor Enters Clinical Trials, Targeting Cancer Growth Without Metabolic Side Effects

Targeted Therapy
  • Researchers at the Francis Crick Institute and Vividion Therapeutics have developed compounds that selectively block the interaction between cancer-driving RAS protein and PI3K enzyme while preserving normal cellular functions.
  • The lead compound demonstrated tumor growth suppression in mouse models of RAS-mutated lung cancer and HER2-driven tumors without causing hyperglycemia, a common side effect of traditional PI3K inhibitors.
  • The investigational drug has entered Phase 1 clinical trials to evaluate safety and efficacy in patients with RAS or HER2 mutations, potentially offering treatment for approximately 20% of all cancers.
  • Combination therapy studies showed enhanced and longer-lasting tumor suppression compared to single-agent treatments, suggesting potential for multi-modal therapeutic approaches.

Telomir-1 Demonstrates Selective Killing of Triple-Negative Breast Cancer Cells Through Iron-Dependent Mechanism

OncologyTargeted Therapy
  • Telomir Pharmaceuticals reports that Telomir-1 significantly reduces viability of aggressive triple-negative breast cancer cells through dose-dependent iron regulation mechanisms.
  • Laboratory studies demonstrate the compound shuts down cellular energy pathways and mitochondrial function, with effects reversible upon iron supplementation.
  • The discovery exploits metabolic vulnerabilities unique to aggressive cancer cells while potentially sparing healthy tissue that manages iron differently.
  • Telomir plans to expand testing to additional cancer types and conduct animal studies in preparation for IND submission.

ITM's Phase 3 COMPETE Trial Dosimetry Data Shows Targeted Tumor Uptake for 177Lu-edotreotide in Neuroendocrine Tumors

Targeted Therapy
  • ITM Isotope Technologies Munich presented dosimetry data from 207 patients in the Phase 3 COMPETE trial, demonstrating that 177Lu-edotreotide delivered targeted radiation to tumors while minimizing healthy tissue exposure.
  • The radiopharmaceutical achieved significantly higher absorbed doses in tumors (110.0 ± 90.8 Gy) compared to normal organs, with kidney and bone marrow doses remaining well below safety thresholds.
  • Grade ≥1 renal adverse events occurred less frequently with 177Lu-edotreotide versus everolimus (14.7% vs. 21.2%), supporting the favorable safety profile previously demonstrated in the trial.
  • The research received the prestigious EANM Marie Curie Award and represents the first large-scale dosimetry analysis in a randomized Phase 3 radiopharmaceutical trial for gastroenteropancreatic neuroendocrine tumors.

Efficacy and Safety of 177Lu-edotreotide PRRT in GEP-NET Patients

NCT03049189Active, Not RecruitingPhase 3
ITM Solucin GmbH
Posted 2/2/2017

Novel Inhalable Crizotinib Nanoparticles Show Promise for Targeted Lung Cancer Treatment

Targeted Therapy
  • Researchers have developed an innovative dry powder inhalation therapy containing Crizotinib-loaded nanoparticles designed to deliver targeted treatment directly to the lungs for metastatic non-small cell lung cancer.
  • The formulation achieved impressive technical specifications with 82.3% encapsulation efficiency, 56.2% fine particle fraction, and enhanced anticancer activity compared to free drug administration.
  • This approach aims to reduce systemic toxicity associated with conventional Crizotinib therapy while improving drug localization and patient compliance through non-invasive inhalation delivery.
  • The technology represents a significant advancement in precision oncology, potentially transforming treatment strategies for ALK and ROS1 mutation-positive lung cancers.

Cellectar Biosciences Raises $5.8 Million Through Warrant Exercise to Advance Cancer Drug Pipeline

Orphan DrugTargeted TherapyOncology
  • Cellectar Biosciences secured approximately $5.8 million in gross proceeds through institutional investor warrant exercises, with Ladenburg Thalmann serving as exclusive placement agent.
  • The funding will support the company's Phase 1b clinical study of CLR 121125 in triple-negative breast cancer and preparation for European regulatory filing.
  • The company's proprietary Phospholipid Drug Conjugate platform targets next-generation cancer treatments with improved efficacy and safety profiles.
  • Cellectar's lead asset iopofosine I 131 has received six Orphan Drug, four Rare Pediatric Drug, and two Fast Track designations from the FDA.

Pentixapharm's CXCR4-Targeted Radiopharmaceuticals Show Promise in Primary Aldosteronism and Bladder Cancer

Targeted TherapyPrecision Medicine
  • Pentixapharm presented extensive clinical data at EANM 2025 demonstrating [⁶⁸Ga]Ga-PentixaFor's potential to improve diagnosis in Primary Aldosteronism, a frequently underdiagnosed cause of secondary hypertension.
  • The diagnostic candidate showed high potential to significantly improve patient subtyping compared to adrenal venous sampling, the current invasive gold standard for Primary Aldosteronism.
  • First-in-human data for therapeutic candidate [¹⁷⁷Lu]Lu-PentixaTher in bladder cancer patients demonstrated initial signs of therapeutic activity, further validating CXCR4 as a clinically relevant target.
  • The findings showcase the versatility of Pentixapharm's CXCR4-targeted platform across cardiovascular, endocrine, and oncologic diseases.

UC Davis Scientists Develop Precision Gene Therapy for Kaposi's Sarcoma Using Viral "Trojan Horse" Strategy

Precision MedicineTargeted Therapy
  • UC Davis researchers have developed a highly targeted gene therapy that uses an adeno-associated virus to selectively destroy cancer cells infected with Kaposi's sarcoma-associated herpesvirus (KSHV) while sparing healthy tissue.
  • The therapy exploits the viral protein LANA, found only in KSHV-infected cells, to activate a modified thymidine kinase enzyme that converts ganciclovir into a potent cancer-killing agent.
  • Preclinical studies in mouse models demonstrated significant tumor growth reduction with no detectable side effects, offering hope for safer treatment of KSHV-related cancers affecting immunocompromised patients.
  • The research, published in Molecular Therapy Oncology, represents a paradigm shift toward precision oncology by turning the virus's own biology against itself.

Revolution Medicines Appoints Alan Sandler as Chief Development Officer to Advance RAS-Targeted Cancer Pipeline

Targeted TherapyOncology
  • Revolution Medicines has appointed Alan Sandler, M.D. as chief development officer to lead development of RAS(ON) inhibitors for RAS-addicted cancers.
  • The company also named regional general managers Alicia Gardner for the U.S. and Gerwin Winter for Europe to prepare for potential commercial launches.
  • Dr. Sandler brings extensive oncology drug development experience from ALX Oncology, Mirati Therapeutics, and Genentech to advance the company's clinical-stage pipeline.
  • The leadership additions support Revolution Medicines' preparation for potential approvals of daraxonrasib and other RAS-targeted therapies.

Aro Biotherapeutics Completes Enrollment in Phase 1b Trial of Novel siRNA Therapy for Late-Onset Pompe Disease

Rare DiseaseOrphan DrugTargeted Therapy
  • Aro Biotherapeutics has completed enrollment in its Phase 1b clinical trial of ABX1100, a first-in-class targeted siRNA therapy for late-onset Pompe disease patients currently receiving enzyme replacement therapy.
  • ABX1100 represents a novel therapeutic approach using Centyrin-siRNA conjugate technology to inhibit glycogen synthase 1 (GYS1), targeting the root cause of glycogen accumulation in muscle tissue.
  • The trial evaluates safety, tolerability, and preliminary therapeutic activity over 20 weeks, with results expected to inform later-phase clinical studies for this orphan drug-designated treatment.
  • Current enzyme replacement therapy requires intravenous infusions lasting up to 6 hours multiple times monthly, highlighting significant unmet medical need in this rare neuromuscular disorder.

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

NCT06109948RecruitingEarly Phase 1
Aro Biotherapeutics
Posted 10/19/2023

Kelun-Biotech's Next-Generation RET Inhibitor A400/EP0031 Receives NDA Acceptance in China for NSCLC Treatment

Targeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncology
  • Sichuan Kelun-Biotech announced that China's NMPA accepted its New Drug Application for A400/EP0031, a next-generation selective RET inhibitor for treating RET-fusion positive non-small cell lung cancer.
  • The acceptance is based on positive Phase 2 results from the KL400-I/II-01 study, which demonstrated favorable efficacy in both treatment-naïve and pretreated NSCLC patients, including those with brain metastases.
  • A400/EP0031 previously received FDA Fast Track designation in March 2024 and is being developed globally through a partnership with Ellipses Pharma for markets outside Greater China.
  • The drug addresses an unmet medical need for RET-fusion positive NSCLC patients, who represent 1.4% to 2.5% of Chinese NSCLC cases and derive limited benefit from conventional treatments.

A Study of KL590586 in Patients With Advanced Solid Tumors

NCT05265091Active, Not RecruitingPhase 1
Sichuan Kelun Pharmaceutical Research Institute Co., Ltd.
Posted 6/1/2021

A Study of EP0031 in Patients With Advanced RET-altered Malignancies

NCT05443126RecruitingPhase 1
Ellipses Pharma
Posted 9/30/2022

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