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Clinical Trial News

Tectonic Therapeutic's TX45 Shows Promise in Phase Ia Trial for Pulmonary Hypertension

  • Tectonic Therapeutic reported positive topline data from its Phase Ia trial of TX45 for Group 2 pulmonary hypertension with preserved ejection fraction (PH-HFpEF).
  • The trial demonstrated that TX45 was safe and well-tolerated in healthy subjects, with minimal adverse events and no immune-mediated clearance observed.
  • A strong pharmacokinetic/pharmacodynamic (PK/PD) relationship was established, enabling the selection of appropriate doses for the upcoming Phase II trial.
  • The Phase II trial, named APEX, has commenced subject screening and will evaluate the effect of TX45 on PH-HFpEF patients with topline results expected in 2026.

Stanford University Initiates Phase 1 Trial of B7-H3-Targeted CAR-T Therapy in Pediatric Solid Tumors

  • Stanford University has commenced a Phase 1 clinical trial to assess the safety and efficacy of CAR-T cell therapy targeting the B7-H3 antigen in pediatric patients with solid tumors.
  • The CAR-T cells are engineered from the patient's own T cells and enhanced with dasatinib to improve their ability to target and destroy cancer cells expressing B7-H3.
  • The trial is enrolling patients aged 2-26 years with relapsed or refractory neuroblastoma, Wilm’s tumor, osteosarcoma, Ewing sarcoma, and soft tissue sarcomas.
  • Researchers aim to identify markers that predict CAR-T cell efficacy, potentially paving the way for improved treatments for solid tumors in the future.

Autologous B7-H3 Chimeric Antigen Receptor T Cells in Relapsed/Refractory Solid Tumors

NCT06500819RecruitingPhase 1
Crystal Mackall, MD
Posted 7/11/2024

NKT2152 Shows Promise in Advanced Kidney Cancer: Phase 1/2 Trial Results

  • NKT2152, a novel oral HIF2α inhibitor, demonstrated a 20% objective response rate in heavily pretreated patients with advanced clear cell renal cell carcinoma (RCC).
  • The dose-escalation phase of the study showed a 26.3% objective response rate, with responses observed across various risk categories and prior treatment histories.
  • Median progression-free survival was 7.392 months in the overall population, with a 12-month PFS rate of 41.8%, indicating durable responses in a subset of patients.
  • The safety profile of NKT2152 was consistent with its class, supporting further evaluation of loading and maintenance dosing regimens in the dose expansion phase.

A Study of NKT2152, a HIF2α Inhibitor, in Patients with Advanced Clear Cell Renal Cell Carcinoma

NCT05119335Active, Not RecruitingPhase 1
NiKang Therapeutics, Inc.
Posted 10/26/2021

NervGen Pharma Highlights Clinical Trial Challenges in Spinal Cord Injury at U2FP Symposium

  • NervGen Pharma's CMO, Dr. Daniel Mikol, will present at the U2FP Symposium, addressing challenges in translating SCI animal model results to human clinical trials.
  • NVG-291, NervGen's therapeutic peptide, targets nervous system repair mechanisms and has shown promise in preclinical models of spinal cord injury.
  • A Phase 1b/2a trial (NCT05965700) is underway to evaluate NVG-291's efficacy in chronic and subacute cervical spinal cord injury, focusing on motor function improvements.
  • The trial assesses corticospinal connectivity changes using electrophysiological and MRI imaging, with funding support from Wings for Life.

NVG-291 in Spinal Cord Injury Subjects

NCT05965700RecruitingPhase 1
NervGen Pharma
Posted 8/8/2023

FibroBiologics Partners with Southern Star Research for Diabetic Foot Ulcer Trial in Australia

  • FibroBiologics has engaged Southern Star Research as its CRO in Australia to advance clinical trials.
  • The collaboration will initially focus on preparatory work for a Phase 1/2 trial of CYWC628 for diabetic foot ulcers.
  • Australia's clinical trial infrastructure, regulatory environment, and R&D incentives make it an attractive location.
  • CYWC628 is an allogeneic fibroblast cell-based therapy designed to accelerate wound healing in diabetic foot ulcers.

Ipsen's Iqirvo Receives European Approval for Primary Biliary Cholangitis

  • The European Commission has granted conditional approval to Ipsen's Iqirvo (elafibranor) for treating primary biliary cholangitis (PBC) in adults.
  • Iqirvo is approved for use with ursodeoxycholic acid (UDCA) in patients with inadequate response or as a monotherapy for those who cannot tolerate UDCA.
  • The approval is based on the Phase III ELATIVE trial, which showed significant treatment benefits compared to placebo in reducing liver damage markers.
  • Iqirvo is the first new therapy approved for PBC in the EU in nearly a decade, offering a new treatment choice for patients.

Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)

NCT04526665Active, Not RecruitingPhase 3
Ipsen
Posted 9/24/2020

Cetylpyridinium Chloride Shows Promise in Inhibiting Hepatocellular Carcinoma Growth and Metastasis

  • Cetylpyridinium chloride (CPC) demonstrates significant anti-tumor activity against hepatocellular carcinoma (HCC) cells, inhibiting proliferation, invasion, and metastasis both in vitro and in vivo.
  • CPC promotes apoptosis in HCC cells by modulating the expression of apoptosis-related genes and suppresses epithelial-mesenchymal transition (EMT) by regulating EMT marker expression.
  • In vivo studies show that CPC effectively inhibits HCC tumor growth and lung metastasis in mice, suggesting its potential as a therapeutic agent for liver cancer.
  • Cancer cells are more sensitive to CPC than normal cells, indicating a potential for lower side effects compared to traditional chemotherapeutic agents.

BRAIN Biotech AG Secures €128.88 Million Royalty Monetization Deal for Deucrictibant

  • BRAIN Biotech AG заключила сделку по монетизации роялти на сумму до 128,88 млн евро с Royalty Pharma в отношении исследуемого фармацевтического соединения деукриктибант.
  • BRAIN Biotech получит авансовый платеж в размере 18,41 млн евро, потенциальные промежуточные платежи за нормативное соответствие в размере до 18,42 млн евро и долгосрочные промежуточные платежи, связанные с продажами, в размере до 92,05 млн евро.
  • Деукриктибант, часть конвейера BioIncubator компании, в настоящее время находится на стадии клинической разработки компанией Pharvaris NV для лечения наследственного ангионевротического отека.
  • Чистая выручка от этой транзакции будет использована BRAIN Biotech для финансирования будущего роста и погашения кредитов.

FDA Approves First Self-Administered Flu Vaccine, FluMist, for At-Home Use

• The FDA has approved FluMist, a nasal spray flu vaccine, for self-administration, marking the first flu vaccine that can be administered at home. • FluMist is approved for individuals aged 2 to 49 and will be available through an online pharmacy with a prescription, starting next flu season. • The approval is based on studies showing that adults can safely and effectively administer the vaccine themselves or to children with proper instructions. • This new option aims to improve convenience, flexibility, and accessibility to seasonal influenza vaccination, potentially increasing vaccination rates.

FDA Approves First Treatments for Niemann-Pick Type C, Offering New Hope to Patients

  • The FDA has approved two new treatments for Niemann-Pick Type C, a rare and fatal genetic disorder, marking a significant milestone for patients and families.
  • Miplyffa (arimoclomol) by Zevra Therapeutics, in combination with miglustat, is approved for neurological symptoms in patients aged 2 and older.
  • Aqneursa (levacetylleucine) by IntraBio Inc. has also been approved as a stand-alone therapy for neurological manifestations of Niemann-Pick disease type C.
  • These approvals underscore the FDA's commitment to supporting the development of new treatments for rare diseases, providing hope for improved management of symptoms.

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288RecruitingPhase 3
IntraBio Inc
Posted 6/30/2022

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

NCT02612129CompletedPhase 2
ZevraDenmark
Posted 6/14/2016

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