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Clinical Trial News

RetinalGenix Partners with LabCorp to Launch DNA/GPS Platform for Genetic and Retinal Disease Screening

Precision Medicine
  • RetinalGenix Technologies has entered into an agreement with LabCorp to support the rollout of its DNA/RNA/GPS Pharmaco-Genetic Mapping platform for early detection of ocular and systemic diseases.
  • The platform enables anonymous genetic testing and retinal imaging at LabCorp locations nationwide, analyzing blood, tears, nasal secretions, and saliva using proprietary algorithms.
  • The cost-effective methodology aims to reduce reliance on expensive diagnostic procedures like MRIs and CT scans while improving access to early disease detection.
  • High-resolution retinal imaging will be introduced as an additional service to boost diagnostic accuracy and enable mass screening by general practitioners and standard eye clinics.

PRD Therapeutics Initiates First-in-Human Trial of SOAT2-Selective Inhibitor PRD001 for Rare Cholesterol Disorders

Rare Disease
  • PRD Therapeutics has initiated dosing in the first-ever clinical trial of PRD001, a first-in-class SOAT2-selective inhibitor targeting homozygous familial hypercholesterolemia and metabolic dysfunction-associated fatty liver disease.
  • The Phase 1 study represents a breakthrough as the first clinical trial of an SOAT2-selective inhibitor, distinguishing it from previous SOAT1/2 dual or SOAT1-selective inhibitor trials.
  • Preclinical studies in animal models demonstrated that PRD001 effectively lowers blood and liver lipids while suppressing fatty liver and atherosclerosis progression without adverse events.
  • The oral therapy has potential to become the first effective treatment for HoFH patients who suffer from absent or extremely low LDL receptor activity.

A First-in-Human Study of PRD001

NCT07034183RecruitingPhase 1
PRD Therapeutics, Inc.
Posted 6/19/2025

BioPorto Launches ProNephro AKI Diagnostic Test in US Market Through Roche Partnership

  • BioPorto A/S announced the commercial launch of ProNephro AKI (NGAL) in the United States after receiving its first purchase order through distribution partner Roche Diagnostics.
  • The diagnostic test is FDA 510(k) cleared for risk stratification of moderate-to-severe acute kidney injury in patients aged 3 months to 21 years using Roche's cobas c501 analyzers.
  • This milestone represents BioPorto's entry into the US kidney diagnostics market, leveraging NGAL biomarker technology to enable earlier AKI detection than current standard of care measurements.
  • The launch establishes BioPorto's commercial platform for kidney diagnostics in the US healthcare system through widespread hospital and laboratory access via Roche's analyzer network.

FDA-Approved Tovorafenib Offers New Treatment Option for Pediatric Low-Grade Glioma with BRAF V600 Mutations

Targeted TherapyDrug Approval
  • Tovorafenib (Ojemda) is approved for treating low-grade glioma that has returned after previous treatments failed, specifically targeting tumors with BRAF V600 mutations.
  • The oral medication is available for adults and children as young as 6 months old, administered once weekly as either tablets or oral suspension.
  • Treatment requires genetic testing to confirm BRAF V600 mutations before initiation and regular monitoring for potential side effects including bleeding, liver problems, and growth impacts in children.
  • Common side effects include fatigue, gastrointestinal symptoms, cold-like symptoms, and skin reactions, with serious adverse events requiring immediate medical attention.

Study for Patients With Hairy Cell Leukemia to Test the Targeted Agent Tovorafenib in Combination With Rituximab and Compare the Combination With Current Standard Treatment

NCT06965114Not Yet RecruitingPhase 1
National Cancer Institute (NCI)
Posted 9/30/2025

A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

NCT05828069RecruitingPhase 2
National Cancer Institute (NCI)
Posted 3/28/2024

Patient-Derived Esophageal Cancer Organ Chips Accurately Predict Chemotherapy Response in Clinical Study

  • Researchers developed patient-specific esophageal adenocarcinoma organ chips that accurately predicted chemotherapy responses in all eight patients tested, overcoming limitations of traditional organoid models.
  • The microfluidic platform integrates tumor cells with patient-matched stromal fibroblasts and mimics physiological drug delivery through the tumor microenvironment.
  • Results showed perfect correlation between chip responses and patient outcomes, with the platform capable of stratifying patients into responders and non-responders within 12 days of biopsy.

Gene Therapy Normalizes Brain Blood Flow in Sickle Cell Disease Patients, Reducing Stroke Risk

  • Gene therapy treatment significantly improved brain blood flow in three sickle cell disease patients, reducing elevated flow speeds by 22% to 43% and reaching normal levels.
  • The therapy demonstrated more substantial and long-lasting protective effects on brain blood flow compared to standard treatments like hydroxyurea or blood transfusions.
  • Results provide preliminary evidence that gene therapy could protect against stroke risk in sickle cell patients and support inclusion of high-risk patients in future clinical trials.
  • The study represents the closest physiological evidence that gene therapy could be effective for patients with neurovascular disease who are at risk of stroke.

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

NCT04443907TerminatedPhase 1
Novartis Pharmaceuticals
Posted 8/25/2020

Novel VMS Therapy Shows Early Promise in Improving Quality of Life for Breast Cancer Patients

  • A novel therapy for vasomotor symptoms (VMS) demonstrated acceptable tolerability with fatigue, somnolence, and diarrhea as the most common side effects affecting 10% and 5% of patients respectively.
  • Patients experienced notable improvements in sleep quality and overall quality of life as early as week 4, with continued benefits through week 12 of treatment.
  • The therapy shows potential to address medication adherence challenges, as 25-30% of breast cancer patients discontinue endocrine therapy due to adverse effects like hot flashes and sleep disruption.
  • Long-term safety data remains limited due to the 2-year study duration, with researchers emphasizing the need for extended follow-up to assess cancer recurrence and mortality outcomes.

BioCryst Pharmaceuticals Divests European ORLADEYO Rights for $264 Million to Achieve Debt-Free Status

Rare Disease
  • BioCryst Pharmaceuticals sold its European commercial rights to ORLADEYO, a C1 inhibitor therapy for hereditary angioedema, to Neopharmed Gentili for up to $264 million on June 27, 2025.
  • The $250 million upfront payment will eliminate BioCryst's remaining $249 million term debt and save approximately $70 million in annual interest payments.
  • The transaction is expected to reduce operating expenses by $50 million annually while positioning BioCryst to end 2027 with $700 million in cash and no term debt.
  • BioCryst retains global royalty benefits from European sales and plans to reinvest savings into its rare disease pipeline, including pediatric ORLADEYO and BCX17725 for Netherton syndrome.

STENTiT Initiates First-in-Human Trial of Regenerative Stent for Chronic Limb-Threatening Ischemia

  • STENTiT has successfully implanted its first Resorbable Fibrillated Scaffold (RFS) in a patient with chronic limb-threatening ischemia below-the-knee as part of the VITAL-IT 1 clinical study.
  • The bioresorbable microfiber stent provides immediate structural support while facilitating natural vascular tissue regeneration through cell infiltration and gradually dissolves over time.
  • The VITAL-IT 1 study will evaluate up to 10 patients at the Medical University of Graz over 24 months to assess safety and feasibility of this regenerative approach.
  • Chronic limb-threatening ischemia affects approximately 3.5 million people in Europe and the United States, resulting in over 250,000 amputations annually.

Initial Feasibility Study of the STENTiT Resorbable Fibrillated Scaffold (RFS). The RFS is Intended to Restore Lumen Patency and Blood Flow to Infrapopliteal Arteries. The RFS is a Fully Electrospun Tubular Device With a Fibrillated Microarchitecture and Designed for Transcatheter Delivery

NCT07006467RecruitingNot Applicable
Stentit
Posted 6/3/2025

Regeneron Navigates EYLEA Decline with Pipeline Diversification and Regulatory Wins

Drug ApprovalOncology
  • Regeneron reported challenging Q1 2025 results with total revenues declining 4% to $3.029 billion, primarily driven by EYLEA franchise headwinds from biosimilar competition and pricing pressures.
  • The company's diversification strategy shows promise with Dupixent global sales rising 19% to $3.67 billion and EU approval of Lynozyfic for multiple myeloma, demonstrating pipeline strength beyond ophthalmology.
  • Strategic moves including a $7 billion infrastructure investment, inaugural dividend of $0.88 per share, and $1.05 billion share buyback program signal management confidence in long-term growth prospects.
  • Key regulatory catalysts ahead include FDA decisions on EYLEA HD expanded indications with August 2025 target date and potential approvals for odronextamab in follicular lymphoma.

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