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Clinical Trial News

Tune Therapeutics' TUNE-401, a First-in-Class Epigenetic Silencer, Enters Clinical Trials for Chronic Hepatitis B

  • Tune Therapeutics has received approval to begin a Phase 1b clinical trial in New Zealand for TUNE-401, an epigenetic silencer targeting chronic hepatitis B (HBV).
  • TUNE-401 aims to achieve a functional cure by permanently repressing both integrated HBV DNA (intDNA) and covalently closed circular DNA (cccDNA) in hepatocytes.
  • Pre-clinical data presented at AASLD 2024 demonstrated near-complete repression of HBV DNA and reduction of hepatitis B surface antigen levels with TUNE-401.
  • The Phase 1b trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of TUNE-401 in adults with chronic HBV, with initial studies in Auckland.

Zerlasiran Shows Durable Lp(a) Reduction in ALPACAR-360 Trial

  • Zerlasiran, a gene-silencing therapy, demonstrated sustained reductions in lipoprotein(a) [Lp(a)] levels through 60 weeks in the ALPACAR-360 trial.
  • The Phase 2 trial showed zerlasiran reduced Lp(a) levels by 81.3% to 85.6% at 36 weeks, with consistent reductions maintained through 60 weeks.
  • Zerlasiran was well-tolerated, with mostly mild injection site reactions, suggesting its potential as a long-term, infrequent-dose therapy for high Lp(a).
  • The trial's findings support the progression of zerlasiran into Phase 3 trials for patients with elevated Lp(a) and high cardiovascular risk.

Ziftomenib Shows Promise in AML: Kura Oncology and Kyowa Kirin Advance to Phase 3 Trials

  • Kura Oncology and Kyowa Kirin's ziftomenib demonstrates statistically significant efficacy in Phase 2 trial for relapsed/refractory NPM1-mutant acute myeloid leukemia (AML).
  • The companies plan to submit a New Drug Application (NDA) to the FDA in the second quarter of 2025, seeking approval for ziftomenib in R/R NPM1-m AML.
  • Phase 3 trials are set to begin in the second half of 2025, evaluating ziftomenib in combination with intensive and non-intensive chemotherapy regimens for newly diagnosed AML.
  • FDA feedback supports potential accelerated approval pathways in both intensive and non-intensive treatment settings, based on MRD negative CR and CR endpoints.

First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

NCT04067336RecruitingPhase 1
Kura Oncology, Inc.
Posted 9/12/2019

A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, or 7+3 in Patients With AML

NCT05735184RecruitingPhase 1
Kura Oncology, Inc.
Posted 7/18/2023

Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML

NCT07007312Not Yet RecruitingPhase 3
Kura Oncology, Inc.
Posted 9/1/2025

Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia

2023-510288-37-00RecruitingPhase 1
Kura Oncology Inc.
Posted 2/22/2024

FDA Approves Zanidatamab for HER2-Positive Biliary Tract Cancer

• The FDA granted accelerated approval to zanidatamab-hrii (Ziihera) for previously treated, unresectable or metastatic HER2-positive biliary tract cancer. • Approval was based on the phase 2b HERIZON-BTC-01 trial, which showed a 52% overall response rate in patients treated with the bispecific antibody. • Zanidatamab offers a new chemotherapy-free treatment option for patients with HER2-positive biliary tract cancer, addressing a high unmet need. • Updated data from ASCO 2024 demonstrated a median duration of response of 14.9 months and a median overall survival of 18.1 months in IHC 3+ patients.

A Study of ZW25 (Zanidatamab) in Subjects With Advanced or Metastatic HER2-Amplified Biliary Tract Cancers

NCT04466891CompletedPhase 2
Jazz Pharmaceuticals
Posted 10/1/2020

uniQure Doses First Patient in Phase I/IIa Trial of AMT-260 for Refractory Mesial Temporal Lobe Epilepsy

  • uniQure initiated the GenTLE Phase I/IIa clinical trial of AMT-260, a gene therapy for refractory mesial temporal lobe epilepsy (MTLE).
  • AMT-260 uses an AAV9 vector to deliver engineered miRNAs, reducing GluK2 protein expression in the hippocampus to decrease seizure activity.
  • The multi-center, open-label trial will assess the safety, tolerability, and efficacy of two AMT-260 doses in patients with refractory MTLE.
  • Preclinical studies showed AMT-260 reduced seizure frequency and GluK2 expression in epileptic mice and human hippocampal slices.

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

NCT06063850RecruitingPhase 1
UniQure Biopharma B.V.
Posted 6/12/2024

Science 37 Significantly Boosts Enrollment in GSK's Phase 3 PBC Trial Through Decentralized Approach

• Science 37 facilitated nearly half of the U.S. enrollment for GSK's Phase 3 trial on primary biliary cholangitis (PBC) using a direct-to-participant approach. • The decentralized trial design addressed challenges of rare disease research by expanding geographic access and reducing patient burden. • GSK leveraged Science 37's innovative services to reach patients in rural areas, overcoming barriers related to travel and frequent site visits. • The trial achieved an 82.3% completion rate for Part A, demonstrating the effectiveness of at-home participation in evaluating the investigational drug.

FDA Approves Ziihera (zanidatamab-hrii) for HER2-Positive Biliary Tract Cancer

• The FDA granted accelerated approval to Ziihera (zanidatamab-hrii) for previously treated, unresectable or metastatic HER2-positive biliary tract cancer (BTC). • Approval was based on a 52% objective response rate and 14.9 months median duration of response in the HERIZON-BTC-01 trial. • Ziihera is the first and only dual HER2-targeted bispecific antibody approved for HER2-positive BTC in the U.S., offering a new treatment option. • Zymeworks will receive a $25M milestone payment from Jazz Pharmaceuticals and is eligible for up to $500M in regulatory milestones.

REGENXBIO's RGX-202 DMD Gene Therapy Enters Pivotal Phase 3 Trial

  • REGENXBIO has dosed the first patient in the Phase 3 AFFINITY DUCHENNE trial evaluating RGX-202 for Duchenne muscular dystrophy (DMD).
  • RGX-202 demonstrated improved or stable function in treated patients compared to natural history controls, with significant improvements in NSAA scores.
  • The pivotal trial aims for accelerated approval in 2026, focusing on microdystrophin expression as the primary endpoint.
  • RGX-202 was well-tolerated in earlier phases, with no serious adverse events reported, offering promise for a broader DMD patient population.

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

NCT05693142RecruitingPhase 2
REGENXBIO Inc.
Posted 1/4/2023

Diamyd Medical's DIAGNODE-3 Trial Reaches Recruitment Milestone, Eyes Accelerated Approval

  • Diamyd Medical's DIAGNODE-3 trial, a Phase 3 study for Type 1 Diabetes, has recruited 180 patients, surpassing the target for its early readout planned in March 2026.
  • The early readout is designed to support a potential accelerated Biologics License Application (BLA) submission to the FDA, leveraging the Fast Track designation.
  • DIAGNODE-3 evaluates Diamyd®, an antigen-specific immunotherapy, in individuals with newly diagnosed Stage 3 Type 1 Diabetes, focusing on preserving endogenous insulin production.
  • The trial will continue recruitment to ensure comprehensive data collection from approximately 330 patients, followed for 24 months, to further assess Diamyd's efficacy.

Diamyd Medical's DIAGNODE-3 Trial Exceeds Recruitment Target, Anticipates Early Readout

  • Diamyd Medical's DIAGNODE-3 Phase 3 trial has recruited 180 patients, surpassing the target for its early readout planned in March 2026.
  • The early readout is intended to support a potential accelerated Biologics License Application (BLA) for Diamyd®.
  • DIAGNODE-3 assesses Diamyd®, an antigen-specific immunotherapy, in individuals with newly diagnosed Stage 3 Type 1 Diabetes, focusing on preserving insulin production.
  • The FDA has granted Diamyd® Fast Track designation and acknowledged C-peptide as a surrogate endpoint, potentially accelerating its approval pathway.

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