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Clinical Trial News

Prilenia's Pridopidine Under EMA Review for Huntington's Disease Treatment

  • The EMA is reviewing Prilenia Therapeutics' application for pridopidine, an oral therapy for Huntington's disease, based on clinical data suggesting it may slow disease progression.
  • Pridopidine targets the sigma-1 receptor, aiming to modulate cellular pathways crucial for nerve cell function, potentially easing symptoms and slowing the progression of Huntington's disease.
  • While a Phase 3 trial showed mixed results, subgroup analyses indicated benefits in patients not taking certain medications, supporting the application for approval.
  • If approved, pridopidine could be available in Europe by the second half of 2025, offering a new therapeutic approach to slow disease progression for Huntington's patients.

A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease

NCT02006472CompletedPhase 2
Prilenia
Posted 2/28/2014

PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD

NCT04556656CompletedPhase 3
Prilenia
Posted 10/16/2020

Why BET Inhibitors Fail in Estrogen-Driven Breast Cancer and How to Improve Them

  • A new study explains why BET inhibitors, promising for other cancers, are ineffective against estrogen receptor-positive breast cancer, revealing they only partially block oncogene activation.
  • Researchers found that cancer-related genes depend on the BET protein itself, not just its bromodomain, and BRD4 can activate MYC without relying on its bromodomain.
  • The study identified that BRD4 interacts with the Mediator complex, crucial for gene transcription and estrogen receptor function, independent of the bromodomain.
  • Scientists are now focusing on a more thorough understanding of the BRD4-Mediator complex structure and exploring combination therapies to target both the bromodomain and transcriptional backdoors.

Switching to Ranibizumab Biosimilar Maintains Vision in nAMD Patients

  • A study in India showed that patients with neovascular age-related macular degeneration (nAMD) maintained visual acuity after switching from aflibercept to ranibizumab biosimilar.
  • The research indicated a significant decrease in average central macular thickness (CMT) from baseline to the switch, which persisted throughout the 12-month follow-up period.
  • While subretinal fluid (SRF) increased moderately after switching, no serious adverse events were reported, suggesting the biosimilar is a viable, more affordable alternative.
  • The findings support the potential of biosimilar anti-VEGF therapy to broaden access to effective AMD treatment, especially for patients facing financial constraints.

Erythropoietin Fails to Show Long-Term Neuroprotective Benefits in Preterm Infants

  • A follow-up study of preterm infants treated with erythropoietin (rhEpo) showed no significant differences in neurodevelopmental outcomes at 10 years compared to placebo.
  • The study adds to a growing body of evidence from phase III trials that erythropoietin does not provide the hoped-for neuroprotective benefits in high-risk newborns.
  • Researchers emphasize the importance of well-powered phase III trials and preclinical studies in multiple animal models before advancing therapies to human infants.
  • The findings highlight the challenges of translating promising preclinical data into effective clinical treatments for preterm infants.

Does Erythropoietin Improve Outcome in Very Preterm Infants?

NCT00413946CompletedPhase 2
Swiss Neonatal Network
Posted 1/1/2006

Immunis' IMMUNA Receives FDA Clearance for Phase II Sarcopenic Obesity Trial

  • Immunis has received FDA clearance for a Phase II clinical trial of IMMUNA, a secretome therapy targeting sarcopenic obesity.
  • The trial aims to assess IMMUNA's efficacy in improving mobility and independence in individuals affected by sarcopenic obesity.
  • IMMUNA is designed to reduce fat while preserving muscle mass, contrasting with GLP-1 drugs that may lead to muscle loss.
  • Preclinical data supports IMMUNA's potential to increase lean mass, reduce fat mass, and enhance muscle function.

Leapfrog Bio's Platform Screens Phase II Drugs for Targeted Cancer Therapies

  • Leapfrog Bio utilizes a platform screening drugs that have passed Phase II trials to identify effective treatments for cancers with specific genetic mutations.
  • The Precision PGx platform aims to expedite the development of targeted therapies by repurposing clinically-vetted molecules for genetically-defined patient populations.
  • Leapfrog Bio's approach focuses on cancers driven by loss-of-function mutations, addressing a significant gap in current cancer treatment options.
  • The company plans to initiate clinical trials within 18 months, targeting lung, colon, and bladder cancers with a lead drug identified through its platform.

Bayer's BAY 2927088 Shows Promise in HER2-Mutant NSCLC

  • Bayer's BAY 2927088 demonstrated a 72.1% objective response rate in HER2-mutant NSCLC patients in an early to mid-stage study.
  • Patients with HER2 YVMA insertions showed a 90% ORR, with a median duration of response of 9.7 months and PFS of 9.9 months.
  • The Phase I/II SOHO-01 study indicates a manageable safety profile for BAY 2927088, supporting further investigation in advanced NSCLC.
  • Bayer has initiated a Phase III study (SOHO-02) to evaluate BAY 2927088 as a first-line therapy for advanced HER2-mutant NSCLC.

Gene Therapy Shows Promise for Inherited Retinal Dystrophy

  • A Phase I/II clinical trial of a gene therapy for Bothnia dystrophy, a rare inherited retinal disease, demonstrated improved vision in treated patients.
  • The gene therapy uses an AAV8 vector to deliver a corrected RLBP1 gene, addressing the underlying genetic defect in Bothnia dystrophy.
  • The treatment was generally well-tolerated, with mostly mild to moderate adverse events, offering hope for restoring vision in those affected by hereditary blindness.
  • Researchers are planning a larger, placebo-controlled study to further evaluate the therapy's efficacy and safety for potential market approval.

Decentralized Clinical Trials Show Promise and Challenges in Expanding Patient Diversity

  • A recent study reveals that Decentralized Clinical Trials (DCTs) can improve clinical trial access, but some solutions may present new challenges, especially for underserved communities.
  • The use of local labs in DCTs is associated with a higher enrollment of non-White participants, suggesting increased convenience and reduced travel burden.
  • Virtual visits and televisits in DCTs correlate with a lower percentage of Black participants, potentially due to disparities in technology access and comfort.
  • Research misconduct risks in DCTs include data integrity concerns and challenges with PI oversight, necessitating robust safeguards and monitoring systems.

Telo Genomics Evaluates MRD Assay in Multiple Myeloma with TELO-DMRD Study

  • Telo Genomics is assessing its minimal residual disease (MRD) biomarker assay technologies in multiple myeloma (MM) patients as part of the TELO-DMRD clinical validation study.
  • The study utilizes Adaptive Biotechnologies’ clonoSEQ assay technology to validate the sensitivity of Telo's MRD assay for MRD enumeration.
  • Telo's MRD clinical trials with McGill University aim to develop two prognostic tests for monitoring myeloma MRD using liquid biopsy-based methods.
  • The MRD global testing market is expected to reach USD 4.1 billion by 2032, highlighting the growing importance of MRD assessment in oncology.

Telo Genomics Technology to Detect & Profile Multiple Myeloma MRD

NCT05530096Recruiting
Sir Mortimer B. Davis - Jewish General Hospital
Posted 5/1/2024

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