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Clinical Trial News

Denali Therapeutics Plans Accelerated Approval Filing for Tividenofusp Alfa in MPS II

• Denali Therapeutics plans to submit a Biologics License Application (BLA) to the FDA in early 2025 for tividenofusp alfa (DNL310) to treat Mucopolysaccharidosis type II (MPS II). • The FDA has indicated that cerebrospinal fluid heparan sulfate (CSF HS) can serve as a surrogate biomarker to support accelerated approval of tividenofusp alfa in MPS II. • Phase 1/2 data presented at SSIEM 2024 showed a 90% mean reduction in CSF HS and improvements in clinical outcomes with tividenofusp alfa treatment in MPS II patients. • Tividenofusp alfa is currently being evaluated in the Phase 2/3 COMPASS study, with plans for full approval contingent upon the study's completion.

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

NCT05371613RecruitingPhase 2
Denali Therapeutics Inc.
Posted 7/21/2022

IRLAB Strengthens Patent Portfolio for Parkinson's Drug Candidates Mesdopetam and Pirepemat

  • IRLAB has secured additional patent protection for mesdopetam, potentially extending market exclusivity into the early 2040s for this drug candidate targeting levodopa-induced dyskinesias in Parkinson's disease.
  • The newly approved patent covers a specific salt formulation of mesdopetam used in clinical development, complementing existing substance patents and strengthening the commercial value of the program.
  • IRLAB has also been granted a similar patent for pirepemat in the US, a drug candidate designed to reduce falls in Parkinson's patients, further expanding the company's robust patent portfolio for both promising treatments.

Clinical Trial Aims to Transform PSP Care with Innovative Platform Approach

  • A new clinical trial will concurrently test three drugs for progressive supranuclear palsy (PSP), a fatal neurodegenerative disorder with no current cure.
  • The adaptive platform trial design allows for efficient testing of new therapies, increasing the likelihood of patients receiving active treatment.
  • The trial prioritizes the enrollment of diverse populations and offers financial support to reduce barriers to participation.
  • Researchers aim to slow disease progression, with even a 20-30% reduction considered a meaningful impact on this relentlessly progressive condition.

Food Support Intervention Trial Launched to Reduce Food Insecurity and Healthcare Utilization in Low-Income Children

  • A clinical trial (NCT06560229) has been initiated to assess the impact of monthly food support on low-income families within a Medicaid Accountable Care Organization.
  • The primary goal is to determine if providing monthly grocery gift cards reduces food insecurity among pediatric patients and their families.
  • The study will also evaluate the intervention's effect on healthcare utilization, including unplanned readmissions and emergency department visits, over an 18-month period.
  • Participants will complete surveys at multiple time points to assess food insecurity, healthcare utilization, recovery, and medication access.

Giving Resources to lOw-income Children to Enhance RecoverY

NCT06560229Enrolling by InvitationNot Applicable
Children's Hospital Medical Center, Cincinnati
Posted 9/27/2024

FDA Authorizes Phase 2 Trial of Bezisterim for Neurological Symptoms of Long COVID

  • BioVie Inc. received FDA authorization for an Investigational New Drug application to study bezisterim (NE3107) in treating neurological symptoms associated with long COVID.
  • The Phase 2 trial will involve approximately 200 patients randomized to assess the safety and tolerability of bezisterim over three months, focusing on reducing neurocognitive symptoms.
  • Bezisterim, an orally bioavailable insulin sensitizer with anti-inflammatory properties, modulates NFkB activation, potentially addressing inflammation, a key driver of long COVID.
  • Current estimates suggest that 5.5% of US adults have long COVID, highlighting the urgent need for effective treatments, with bezisterim also under investigation for Alzheimer's and Parkinson's diseases.

Insulin-Sensitizing Anti-Inflammatory Small Molecule for Investigative Treatment of Dementia

NCT05227820CompletedPhase 2
Neurological Associates of West Los Angeles
Posted 1/19/2022

NE3107 Activity and Safety in Patients With Parkinson's Disease Using Levodopa

NCT05083260CompletedPhase 1
BioVie Inc.
Posted 1/4/2022

Theolytics Receives Grant to Advance THEO-260 Ovarian Cancer Trial

  • Theolytics has been awarded a £2m grant from Innovate UK to support a Phase I clinical trial of THEO-260 for advanced-stage platinum-resistant ovarian cancer.
  • The trial will assess the safety, tolerability, and optimal Phase II dosage of THEO-260 in patients with ovarian cancer.
  • The funding will also facilitate a biomarker study to validate THEO-260’s mechanism, which targets cancer and immune-suppressive stromal cells while promoting T cell activation.
  • THEO-260, an oncolytic adenovirus therapy, has demonstrated promising preclinical results, warranting further clinical investigation.

Grit Biotechnology's GT201 TIL Therapy Receives FDA Clearance for Advanced Solid Tumor Trial

• Grit Biotechnology's GT201, a genetically engineered tumor-infiltrating lymphocyte (TIL) therapy, has been cleared by the FDA for clinical trials in advanced solid tumors. • GT201 leverages Grit's StemTexp platform and StaViral transduction system to enhance T-cell survival and function without heavy reliance on IL-2. • Early clinical data presented at ASGCT and ASCO demonstrated GT201's stable in-vivo expansion and preliminary efficacy with a good safety profile in advanced solid tumor patients. • Grit Biotechnology is also advancing GT101, a non-genetically engineered TIL product, with plans to submit a biologics license application in China in 2025.

KalVista Seeks Global Approval for Oral HAE Treatment Sebetralstat

  • KalVista Pharmaceuticals has submitted Marketing Authorization Applications (MAAs) for sebetralstat in the UK, Switzerland, Australia, and Singapore.
  • Sebetralstat, an oral treatment for hereditary angioedema (HAE), could be the first on-demand oral medication for HAE attacks in patients 12 and older.
  • The MAAs are supported by Phase 3 KONFIDENT trial data, demonstrating sebetralstat significantly accelerated symptom relief compared to placebo.
  • A pediatric trial (KONFIDENT-KID) is underway, potentially positioning sebetralstat as the first oral on-demand therapy for children aged 2-11.

Athira Pharma's Fosgonimeton Fails to Meet Primary Endpoint in Phase II/III Alzheimer's Trial

• Athira Pharma's fosgonimeton did not meet the primary endpoint in the Phase II/III LIFT-AD trial for mild-to-moderate Alzheimer's disease. • The trial failed to show statistically significant improvement in cognition and function compared to placebo after 26 weeks of treatment. • Subgroup analyses showed some improvements in cognition and function in patients with moderate Alzheimer's and APOE4 carriers. • Despite the setback, Athira Pharma believes the data suggest potential benefits of HGF pathway modulation on neuronal health.

ATH-1017 for Treatment of Mild to Moderate Alzheimer's Disease

NCT04488419CompletedPhase 2
Athira Pharma
Posted 9/28/2020

ATH-1017 Treatment in Subjects With Parkinson's Disease Dementia or Dementia With Lewy Bodies (SHAPE Trial)

NCT04831281TerminatedPhase 2
Athira Pharma
Posted 1/20/2022

A Study to Evaluate the Safety and Efficacy of CT1812 in Subjects With Mild to Moderate Alzheimer's Disease.

NCT03507790CompletedPhase 2
Cognition Therapeutics
Posted 10/10/2018

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

NCT05524883Active, Not RecruitingPhase 1
Dyne Therapeutics
Posted 8/12/2022

Safety, Tolerability, and Immunogenicity Study of a 31-Valent Pneumococcal Conjugate Vaccine (VAX-31) in Adults

NCT06151288CompletedPhase 1
Vaxcyte, Inc.
Posted 11/8/2023

A Study of ATH-1017 in Mild to Moderate Alzheimer's Disease

NCT04491006CompletedPhase 2
Athira Pharma
Posted 11/23/2020

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