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Clinical Trial News

PolTREG Establishes U.S. Subsidiary Immuthera and Receives Positive EMA Opinion for Type 1 Diabetes Cell Therapy

CAR-T
  • PolTREG established Immuthera as a U.S. subsidiary in Delaware and submitted a Pre-IND application to the FDA to advance its T-regulatory cell therapies in the American market.
  • The European Medicines Agency's Paediatric Committee issued a positive opinion on PolTREG's Pediatric Investigation Plan for PTG-007, an autologous Treg therapy for preventing symptomatic type 1 diabetes in children.
  • The EMA committee recommended expanding the eligible patient population from ages 6-16 years to 3-18 years, suggesting the therapy demonstrates exceptional safety profile.
  • PolTREG operates one of Europe's largest GMP-certified manufacturing facilities with over 2,100 sqm of laboratory space and is the first company worldwide to administer Treg therapies to patients.

Olverembatinib Shows Promising Results in Multiple Philadelphia Chromosome-Positive ALL Studies

  • A prospective clinical trial demonstrated that olverembatinib combined with venetoclax and reduced-intensity chemotherapy achieved a 62.0% complete molecular response rate at 3 months in newly diagnosed Ph+ ALL patients.
  • The combination therapy showed excellent survival outcomes with 1-year overall survival and event-free survival rates of 93.1% and 89.1% respectively, without requiring intensive chemotherapy or immunotherapy.
  • Additional Phase II studies revealed olverembatinib's efficacy in various Ph+ ALL treatment settings, including first-line therapy with VP regimen achieving 100% overall response rate and relapsed/refractory disease treatment.
  • Transcriptomic analysis provided mechanistic evidence for the complementary action between tyrosine kinase inhibitors and venetoclax, supporting the rational combination approach.

Olverembatinib Combined With Reduced-Intensity Chemotherapy and Venetoclax for de Novo Ph+ ALL

NCT05594784CompletedPhase 2
Institute of Hematology & Blood Diseases Hospital, China
Posted 10/8/2022

UK Flair Trial Demonstrates Superior Outcomes with Chemotherapy-Free Combination Therapy for Chronic Lymphocytic Leukaemia

Targeted Therapy
  • The UK-wide Flair trial involving 786 patients showed that 94% of chronic lymphocytic leukaemia patients receiving ibrutinib plus venetoclax were alive with no disease progression after five years, compared to 79% on ibrutinib alone and 58% on standard chemotherapy.
  • The chemotherapy-free combination therapy achieved superior bone marrow clearance rates, with 66% of patients showing no detectable cancer after two years versus 48% on chemotherapy and none on ibrutinib monotherapy.
  • Researchers from Leeds Teaching Hospitals described the results as a "milestone" that could reshape treatment approaches for the most common adult leukaemia, offering more effective and tolerable personalized medicine options.

Adolore BioTherapeutics Reports Positive Preclinical Data for Gene Therapy Targeting Chronic Knee Pain

  • Adolore BioTherapeutics published preclinical data in Molecular Therapy demonstrating biosafety and efficacy of its rdHSV-CA8* gene therapy for chronic knee pain from osteoarthritis.
  • The gene therapy delivered via intra-articular injection showed profound analgesia lasting over 6 months without functional impairments or toxicity in mouse models.
  • The therapy works by activating Kv7 voltage-gated potassium channels to reduce nerve excitability, offering a potential non-opioid alternative for chronic pain treatment.
  • The company's lead program ADB-102 is fully funded by NIH/NINDS HEAL program with first-in-human studies expected to commence in 2026.

Samsung Biologics Expands Into Preclinical Research with Launch of Samsung Organoids Drug Screening Platform

  • Samsung Biologics launched Samsung Organoids, an advanced drug screening service utilizing three-dimensional cell culture systems that closely mimic human organs to provide clinically relevant insights for drug discovery and development.
  • The new platform supports precision screening to predict patient responses and accelerate timelines toward investigational new drug (IND) filings through data-driven analysis of candidate molecules.
  • This launch marks Samsung Biologics' expansion into preclinical research, with services now spanning target discovery, lead selection, preclinical development, and clinical trial planning to support the entire drug lifecycle.
  • The organoid technology is emerging as a new research model due to its high similarity to live tissues and potential applications in biomarker discovery and drug efficacy prediction.

NHS App to Feature Clinical Trial Matching System as UK Targets 150-Day Setup Times

  • The NHS App will introduce a new "Be Part of Research" feature that automatically matches patients with clinical trials based on their health data and interests, sending push notifications for relevant studies.
  • The UK government aims to reduce clinical trial setup times from the current 250 days to 150 days by March 2026, compared to Spain's 100-day benchmark.
  • The initiative includes making trial performance data public for NHS trusts and prioritizing funding for top-performing sites, while introducing standardized national contracts to reduce bureaucracy.
  • The program specifically encourages participation from under-represented communities including young, Black, and South Asian populations to improve diversity in clinical research.

Hengrui and Kailera Present Phase 2 Data for Dual GLP-1/GIP Agonist and Oral GLP-1 Therapies at ADA 2025

  • Jiangsu Hengrui Pharmaceuticals and Kailera Therapeutics will present six abstracts at the American Diabetes Association 85th Scientific Sessions showcasing clinical progress of their metabolic disease portfolio.
  • The presentations include Phase 2 data for injectable dual GLP-1/GIP receptor agonist HRS9531 (KAI-9531) in type 2 diabetes and obesity, with trials extending up to 52 weeks.
  • Clinical results will also cover oral small molecule GLP-1 receptor agonist HRS-7535 (KAI-7535) and an oral formulation of the dual agonist HRS9531.
  • The data demonstrates the companies' advancement in next-generation therapies for obesity and diabetes, including both injectable and oral delivery mechanisms.

Axcelead and A2 Healthcare Form Strategic Partnership to Accelerate Drug R&D in Japan

Drug Discovery
  • Axcelead and A2 Healthcare announced a strategic partnership to establish an integrated support framework covering drug discovery, non-clinical studies, clinical trials, regulatory submissions, and manufacturing in Japan.
  • The collaboration combines Axcelead's drug discovery services and mRNA CDMO capabilities with A2 Healthcare's clinical development expertise to address drug loss and strengthen Japan's pharmaceutical R&D ecosystem.
  • The partnership will provide comprehensive support to academia, biotech ventures, and pharmaceutical companies from Japan and abroad advancing drug development in the Japanese market.
  • A2 Healthcare's J-STEP initiative and Boston office expansion complement the alliance's goal of enhancing Japan's drug discovery capabilities and facilitating international pharmaceutical market entry.

Novel BTK Degrader BGB-16673 Achieves 84.8% Response Rate in Heavily Pretreated CLL/SLL Patients

Targeted Therapy
  • BGB-16673, a novel BTK protein degrader, demonstrated an 84.8% overall response rate in 66 heavily pretreated patients with relapsed/refractory chronic lymphocytic leukemia and small lymphocytic lymphoma in the phase 1/2 CaDAnCe-101 trial.
  • The treatment showed efficacy across high-risk patient subgroups, including those with BTK mutations and prior exposure to multiple BTK and BCL2 inhibitors, with responses observed regardless of baseline mutations and unfavorable biological features.
  • At a median follow-up of 15.6 months, the drug was well-tolerated with manageable safety profile, and achieved a one-year progression-free survival rate of 77.4% in this challenging patient population.
  • The 200-mg dose level, selected for expansion, showed particularly promising results with a 93.8% overall response rate and median duration of exposure of 16.2 months.

A Dose-Escalation and Expansion Study of BGB-16673 in Participants With B-Cell Malignancies

NCT05006716RecruitingPhase 1
BeiGene
Posted 9/13/2021

Intellia's CRISPR Gene Therapy Shows 98% Reduction in HAE Attacks After Three Years

Gene EditingOrphan DrugRare Disease
  • Intellia Therapeutics reported three-year follow-up data showing lonvoguran ziclumeran achieved a 98% mean reduction in monthly hereditary angioedema attack rates across all 10 Phase 1 patients.
  • All patients remained attack-free and treatment-free for a median of 23 months, demonstrating the potential for lonvo-z to become the first one-time therapy for HAE patients.
  • The company's Phase 3 HAELO trial completed screening ahead of schedule, with results expected in the first half of 2026 and a planned U.S. launch in 2027.
  • The CRISPR-based therapy targets the KLKB1 gene to prevent HAE attacks and has received multiple regulatory designations including FDA Orphan Drug and RMAT status.

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

NCT06634420Active, Not RecruitingPhase 3
Intellia Therapeutics
Posted 1/15/2025

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830Active, Not RecruitingPhase 1
Intellia Therapeutics
Posted 12/10/2021

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