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Clinical Trial News

Real-World Evidence Guides Extended Dosing Strategies for Next-Generation Anti-VEGF Therapies in Retinal Disease

Real World Evidence
  • Retinal specialists are successfully extending treatment intervals beyond traditional 12-week limits with newer anti-VEGF agents like faricimab and aflibercept 8 mg, with some physicians comfortable reaching 16-20 weeks in appropriate patients.
  • Real-world data reveals that switching from aflibercept 2 mg to 8 mg typically allows 2-4 week extensions in treatment intervals, with benefits improving over time rather than immediately after switching.
  • Patient compliance drops significantly beyond 4-month intervals, leading physicians to identify a "sweet spot" of 3-4 month treatment cycles that balance disease control with patient convenience.
  • Loading dose strategies differ between treatment-naive and previously treated patients, with complete reloading often unnecessary when switching to newer agents in experienced patients.

Biohaven Initiates Pivotal Phase II/III Trial of Brain-Penetrant TYK2/JAK1 Inhibitor BHV-8000 for Early Parkinson's Disease

  • Biohaven has launched a global Phase II/III trial of BHV-8000, a first-in-clinic brain-penetrant TYK2/JAK1 inhibitor targeting neuroinflammation in early Parkinson's disease.
  • The randomized, placebo-controlled trial will enroll 550 patients across 185 sites in 13 countries to evaluate 10mg and 20mg doses against placebo.
  • BHV-8000 demonstrated good safety profile in Phase I studies with no serious adverse events and showed target engagement with significant reductions in inflammatory biomarkers.
  • The trial addresses a critical unmet need as no disease-modifying therapies currently exist for Parkinson's disease, which affects over 10 million people globally.

A Study to Determine if BHV-8000 is Effective, Safe and Tolerable as a Treatment for Adults Living With Early Parkinson's Disease

NCT06976268RecruitingPhase 2
Biohaven Therapeutics Ltd.
Posted 5/28/2025

Ochre Bio Strengthens Leadership with Former Eli Lilly and AstraZeneca Executive as RNA Therapeutics Company Expands

AIDrug Discovery
  • Oxford-based Ochre Bio has appointed Dr. Elaine Sullivan, former VP at Eli Lilly and AstraZeneca, as Senior Independent Non-Executive Director to support the company's RNA therapeutics development for chronic liver disease.
  • The company has expanded its scientific team with key hires including Dr. Julian Maller as Head of In Silico Biology and Dr. Sarah Batey as Head of Portfolio Development & Scientific Operations.
  • Ochre Bio combines AI with human data to discover RNA therapeutics and operates across Oxford, New York, and Taipei with multi-million-pound partnerships with GSK and Boehringer Ingelheim.

Rona Therapeutics Reports 99% Lipoprotein(a) Reduction with Annual-Dose RNAi Therapy RN026

  • Rona Therapeutics announced preclinical data for RN026, an RNAi therapy targeting lipoprotein(a), achieving 99% reduction in non-human primate studies with potential annual dosing.
  • A single 2mg/kg subcutaneous injection maintained 95% Lp(a) reduction at 98 days and sustained ~80% reduction through 154 days in non-human primates.
  • The therapy demonstrated dual cardiovascular benefits with 25% LDL-C reduction and showed no adverse findings in repeated dose toxicity studies.
  • RN026 targets an estimated 10-20% of the global population with elevated Lp(a) levels, addressing multiple cardiovascular diseases including ASCVD and aortic stenosis.

Inspira Technologies Accelerates Global Rollout of FDA-Cleared ART100 Respiratory Support System

  • Inspira Technologies announced a strategic shift to accelerate commercialization of its FDA-cleared ART100 system following successful clinical implementations in U.S. medical centers.
  • The company is engaged in advanced sales discussions with multiple locations, with deliveries expected to begin in the second half of 2025.
  • The ART100 system is approved for cardiopulmonary bypass in the U.S. and ECMO procedures outside the U.S., serving as an alternative to mechanical ventilation.
  • Inspira continues developing its next-generation ART500 system and HYLA blood sensor while prioritizing near-term revenue from ART100 commercialization.

Vima Therapeutics Emerges with $60M to Develop First Oral Therapy for Dystonia

  • Vima Therapeutics launched with $60 million Series A funding led by Atlas Venture to advance VIM0423, a potential first-in-class oral therapy for dystonia targeting muscarinic cholinergic receptors.
  • The company's lead candidate addresses an unmet medical need for over 100,000 Americans with isolated dystonia, as current treatments fail to target the underlying cause of the condition.
  • VIM0423 is currently in Phase 1 clinical trials with Phase 2 studies planned for the fourth quarter of 2025, representing a novel approach to movement disorder treatment.
  • The therapy aims to overcome limitations of existing muscarinic receptor drugs by providing improved pharmacology, efficacy, and safety profiles for patients with this disabling neurological condition.

FDA Approves 7-Year Post-Market Study for BrainSee Alzheimer's Progression Prediction Tool

AIFDA Approval
  • The FDA has approved Darmiyan's 7-year post-market surveillance study for BrainSee, the first FDA-approved AI-powered tool that predicts Alzheimer's disease progression in patients with mild cognitive impairment.
  • BrainSee combines standard brain MRI, cognitive assessments, age, and biological sex to determine whether patients over 55 with amnestic mild cognitive impairment will progress to clinical Alzheimer's disease within five years.
  • The study will validate BrainSee's effectiveness across diverse patient populations and support pharmaceutical companies in identifying optimal candidates for next-generation Alzheimer's clinical trials.
  • Unlike traditional tests that detect amyloid beta plaques, BrainSee addresses the critical gap where roughly 40% of amyloid-positive patients remain stable and may not benefit from anti-amyloid drugs.

Beyfortus Demonstrates Superior Real-World Efficacy Against RSV Compared to Maternal Vaccination

Monoclonal AntibodyReal World Evidence
  • Sanofi's Beyfortus reduced infant RSV hospitalizations by 69% in Spain's immunization program, significantly outperforming the UK's maternal RSVpreF vaccination program which achieved only 26.7% reduction.
  • New durability data from the HARMONIE Phase 3b study show Beyfortus maintained 82.7% efficacy against RSV hospitalizations through six months, exceeding the typical five-month RSV season length.
  • The REACH study represents the first multi-country public health impact analysis comparing different RSV prevention strategies, providing crucial real-world evidence for infant immunization programs.
  • With over six million infants immunized across 40 studies spanning four continents, Beyfortus has established the largest body of real-world evidence for infant RSV protection.

Lantern Pharma's LP-184 Demonstrates Significant Survival Benefits in Pediatric Brain Cancer Models

Rare DiseaseOrphan Drug
  • LP-184 showed remarkable survival improvements in mouse models of atypical teratoid rhabdoid tumors (ATRT), with median survival increasing by 345% in one model (p<0.0001).
  • Independent research from Johns Hopkins University validates Lantern Pharma's FDA Rare Pediatric Disease Designation for LP-184 in treating this aggressive pediatric brain cancer.
  • The drug demonstrated potent anti-tumor activity across multiple ATRT cell lines with strong blood-brain barrier penetrance and no apparent toxicity in preclinical studies.
  • Lantern Pharma plans to initiate a pediatric Phase I clinical trial for LP-184 in brain tumors in late 2025 or early 2026.

Study of LP-184 in Patients with Advanced Solid Tumors

NCT05933265RecruitingPhase 1
Lantern Pharma Inc.
Posted 6/9/2023

uniQure's Gene Therapy AMT-260 Achieves 92% Seizure Reduction in First Drug-Resistant Epilepsy Patient

Rare Disease
  • uniQure's investigational gene therapy AMT-260 demonstrated a 92% reduction in seizure frequency in the first patient treated for drug-resistant mesial temporal lobe epilepsy during five months of follow-up.
  • The patient experienced no serious adverse events and went seizure-free for the final 60 days of the monitoring period, showing promising early safety and efficacy signals.
  • AMT-260 works by delivering engineered microRNAs to suppress the GRIK2 gene and aberrant GluK2 expression, targeting a key mechanism believed to trigger seizures in refractory MTLE.
  • The GenTLE Phase I/IIa trial is actively enrolling patients across 12 U.S. sites, addressing a significant unmet need for over 600,000 Americans with temporal lobe epilepsy.

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

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

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