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Clinical Trial News

European Commission Approves Aflibercept Biosimilar OPUVIZ for Retinal Disorders

• The European Commission has granted approval to OPUVIZ, an aflibercept biosimilar developed by Samsung Bioepis, for treating several retinal disorders. • OPUVIZ is indicated for conditions including neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema and myopic choroidal neovascularisation. • A Phase 3 study demonstrated that OPUVIZ has equivalent efficacy, comparable safety, immunogenicity, and pharmacokinetics to reference aflibercept (Eylea). • This approval marks the second ophthalmology biosimilar approved in Europe within the Samsung Bioepis portfolio, potentially expanding patient access to more affordable treatment options.

Nurix Therapeutics' NX-5948 Receives EMA PRIME Designation for Relapsed/Refractory CLL

  • Nurix Therapeutics' NX-5948, a BTK degrader, has been granted PRIME designation by the EMA for relapsed or refractory chronic lymphocytic leukemia (CLL).
  • The PRIME designation aims to expedite the development and evaluation of NX-5948, offering enhanced support for Nurix's development plans.
  • NX-5948 is currently in Phase 1 trials, with pivotal trials planned for 2025, showing promise in patients resistant to BTK and BCL-2 inhibitors.
  • The drug's mechanism targets BTK degradation, potentially overcoming resistance seen with existing BTK inhibitors in heavily pretreated CLL/SLL patients.

A Study of NX-5948 in Adults With Relapsed/Refractory B-cell Malignancies

NCT05131022RecruitingPhase 1
Nurix Therapeutics, Inc.
Posted 4/13/2022

Organovo's FXR314 Demonstrates Liver Fat Reduction in Phase 2 MASH Trial

• Organovo's FXR314 showed statistically significant reduction in liver fat content in MASH patients compared to placebo in a Phase 2 trial. • The study demonstrated a favorable safety profile for FXR314, with significantly lower pruritus rates than other FXR agonists. • Improvements in hepatocellular damage and liver function were observed based on serological measures, without worsening of liver fibrosis. • FXR314 represents a promising oral therapeutic option for MASH, with potential for further evaluation due to its intestinal activating specificity.

Nurix Therapeutics' NX-5948 Receives FDA Fast Track for Waldenstrom's Macroglobulinemia

  • Nurix Therapeutics' NX-5948, a BTK degrader, has received FDA Fast Track designation for relapsed or refractory Waldenstrom's macroglobulinemia (WM) after prior BTK inhibitor therapy.
  • The Fast Track designation aims to expedite the development and review of NX-5948, addressing an unmet need for WM patients who have progressed on BTK inhibitors.
  • NX-5948 previously received Fast Track designation for CLL/SLL and PRIME designation from the EMA, highlighting its potential in B-cell malignancies.
  • Nurix anticipates sharing additional clinical data from the ongoing Phase 1b expansion cohort of NX-5948 in WM patients in 2025.

A Study of NX-5948 in Adults With Relapsed/Refractory B-cell Malignancies

NCT05131022RecruitingPhase 1
Nurix Therapeutics, Inc.
Posted 4/13/2022

Gene Therapy Advances: From FDA Clearances to Clinical Trial Progress in Early 2025

• The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant. • uniQure received the green light to proceed with the second dose cohort in their Phase 1/2 trial of AMT-162 for SOD1-ALS gene therapy. • Sarepta Therapeutics' Elevidys showed sustained benefit in ambulatory patients with Duchenne muscular dystrophy in Phase 3 trial results.

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults with PKP2 Mutation-associated ARVC

NCT06228924RecruitingPhase 1
Tenaya Therapeutics
Posted 3/26/2024

A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma

NCT04181827Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 6/12/2020

Autologous T-Cells Expressing a Second Generation CAR for Treatment of T-Cell Malignancies Expressing CD5 Antigen

NCT03081910RecruitingPhase 1
Baylor College of Medicine
Posted 11/1/2017

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

NCT05898620RecruitingPhase 1
Neurogene Inc.
Posted 6/13/2023

Infusion of Autologous T Cells Engineered to Target FSH Receptor in Recurrent Ovarian Cancer

NCT05316129RecruitingPhase 1
H. Lee Moffitt Cancer Center and Research Institute
Posted 4/28/2022

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT05096221CompletedPhase 3
Sarepta Therapeutics, Inc.
Posted 10/27/2021

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)

NCT06285643RecruitingPhase 2
AskBio Inc
Posted 6/11/2024

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943Active, Not RecruitingPhase 1
Spur Therapeutics
Posted 4/15/2022

A Trial of Vigil for Participants With Ovarian Cancer

NCT02346747Active, Not RecruitingPhase 2
Gradalis, Inc.
Posted 2/1/2015

Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy

NCT06302608Active, Not RecruitingEarly Phase 1
Xiamen Ophthalmology Center Affiliated to Xiamen University
Posted 2/8/2023

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

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

NCT06100276RecruitingPhase 1
UniQure Biopharma B.V.
Posted 8/1/2024

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

NCT05693142RecruitingPhase 2
REGENXBIO Inc.
Posted 1/4/2023

A Phase 1 Study of ADI-001 in Autoimmune Disease

NCT06375993RecruitingPhase 1
Adicet Therapeutics
Posted 11/10/2024

Study Evaluating SC291 in Subjects With Severe r/r B-cell Mediated Autoimmune Diseases (GLEAM)

NCT06294236RecruitingPhase 1
Sana Biotechnology
Posted 4/29/2024

Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome

NCT06518005RecruitingPhase 2
Genethon
Posted 11/8/2024

Nurix Therapeutics' BTK Degrader NX-5948 Receives EMA PRIME Designation for CLL/SLL

  • Nurix Therapeutics' NX-5948, a BTK degrader, has been granted PRIME designation by the EMA for relapsed or refractory CLL/SLL.
  • The PRIME designation aims to expedite the development and evaluation of NX-5948 for patients who have failed BTK and BCL-2 inhibitor therapies.
  • NX-5948 is currently in Phase 1 clinical trials, demonstrating promising early clinical benefits and activity against BTK inhibitor-resistant tumor cell lines.
  • Pivotal trials for NX-5948 are planned to commence in 2025, potentially offering a new treatment option for CLL/SLL patients with limited alternatives.

A Study of NX-5948 in Adults With Relapsed/Refractory B-cell Malignancies

NCT05131022RecruitingPhase 1
Nurix Therapeutics, Inc.
Posted 4/13/2022

FDA Approves Bimzelx (bimekizumab-bkzx) for Moderate to Severe Hidradenitis Suppurativa

  • The FDA has approved Bimzelx (bimekizumab-bkzx) as the first and only interleukin-17F and -17A inhibitor for treating moderate to severe hidradenitis suppurativa in adults.
  • Approval was based on the BE HEARD trials, demonstrating Bimzelx's efficacy and safety in improving hidradenitis suppurativa signs and symptoms, with sustained outcomes through 48 weeks.
  • In clinical trials, a significantly higher proportion of patients treated with Bimzelx achieved a 50% or greater improvement in Hidradenitis Suppurativa Clinical Response compared to placebo at week 16.
  • Bimzelx presents a new treatment option, addressing a critical unmet need, as existing treatments may not be effective for all individuals with hidradenitis suppurativa.

FDA Grants Fast Track Designation to Alentis Therapeutics' ALE.P02 for CLDN1-Positive Solid Tumors

• The FDA has granted Fast Track designation to ALE.P02, an anti-Claudin-1 (CLDN1) antibody-drug conjugate (ADC), for CLDN1-positive squamous solid tumors. • ALE.P02, a first-in-class ADC, targets the CLDN1 epitope on cancer cells and has shown promise in preclinical studies, demonstrating complete tumor regression in xenograft models. • A first-in-human phase 1/2 trial is set to begin in Q1 2025, evaluating ALE.P02 in patients with CLDN1-positive squamous solid tumors, following the FDA's IND clearance in October 2024.

Puma Biotechnology Initiates Phase II Trial of Alisertib for HR+/HER2- Metastatic Breast Cancer

• Puma Biotechnology has commenced the ALISCA™-Breast1 Phase II trial to evaluate alisertib combined with endocrine therapy for HR+/HER2- metastatic breast cancer. • The trial will enroll up to 150 patients previously treated with CDK 4/6 inhibitors and at least two prior lines of endocrine therapy. • The primary objective is to determine the optimal dose of alisertib in combination with endocrine therapy, assessing objective response rate and survival. • Puma plans to engage with regulatory agencies to explore an approval pathway for alisertib based on trial outcomes and biomarker analysis.

Evaluating how well and how safely Alisertib works with hormone therapy in treating patients with breast cancer that has come back or spread (hormone receptor positive, HER2 negative

2024-511497-79-00RecruitingPhase 2
Puma Biotechnology Inc.
Posted 11/19/2024

WHO Approves First Mpox Vaccine for Children Amid Outbreak Surge

  • The World Health Organization (WHO) has granted emergency use listing for the Lc16m8 mpox vaccine, the first approved for children aged one year and older.
  • This decision aims to increase access to vaccines in communities experiencing mpox outbreaks, particularly in regions like the Democratic Republic of Congo.
  • The Lc16m8 vaccine, developed by KM Biologics, is recommended as a single dose for individuals one year of age and older, offering a new tool in containing the outbreak.
  • Mpox cases among children have risen significantly, with the DRC reporting a 130% increase, highlighting the urgent need for pediatric vaccination options.

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