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Clinical Trial News

BioCardia Seeks Japan PMDA Approval for CardiAMP Cell Therapy in Heart Failure Treatment

  • BioCardia submitted its CardiAMP autologous cell therapy to Japan's PMDA for clinical consultation, marking a significant step toward potential regulatory approval in the key Japanese market.
  • The submission includes comprehensive clinical data from the CardiAMP HF Trial, which demonstrated statistically significant reduction in all-cause death and major adverse cardiac events in heart failure patients.
  • The FDA Breakthrough-designated therapy uses patients' own bone marrow cells delivered via minimally invasive catheter to increase capillary density and reduce tissue fibrosis.
  • Successful PMDA alignment could enable BioCardia to submit for market approval in Japan and potentially engage in post-marketing studies next year.

CardiAMP Heart Failure II Trial for Patients With Ischemic Heart Failure With Reduced Ejection Fraction

NCT06258447RecruitingNot Applicable
BioCardia, Inc.
Posted 8/1/2024

CardiAMP™ Cell Therapy for Heart Failure Trial

NCT02438306Active, Not RecruitingPhase 3
BioCardia, Inc.
Posted 12/1/2016

Gate Bioscience Partners with Lilly in $856 Million Deal to Develop Novel Molecular Gate Therapeutics

Drug Discovery
  • Gate Bioscience announced a collaboration and license agreement with Eli Lilly to discover and develop molecular gate therapeutics, a new class of small molecule drugs that eliminate disease-causing proteins at their source.
  • The partnership combines Gate's molecular gate drug discovery engine with Lilly's expertise in small molecule therapeutics to target difficult-to-drug proteins for diseases with high unmet medical need.
  • Under the agreement terms, Gate will receive an upfront payment, equity investment, milestone payments, and tiered royalties with a total deal value up to $856 million.
  • Gate's molecular gate technology works by recognizing and blocking specific proteins from crossing cellular channels, causing them to be degraded instead of secreted into the body.

GNT Pharma Advances First-in-Class Dual-Action Stroke Therapy Nelonemdaz to Global Phase 3 Trials

  • GNT Pharma received regulatory approval from South Korea's MFDS to initiate global Phase 3 clinical trials for Nelonemdaz, the world's first dual-action neuroprotectant for ischemic stroke treatment.
  • The multinational trial will enroll 378 patients with severe ischemic stroke across more than 20 leading stroke centers in Korea, USA, and Australia, evaluating functional independence at 12 weeks.
  • Previous Phase 2 and 3 trials involving 704 Korean stroke patients demonstrated that Nelonemdaz improved disability outcomes by 4.3-fold when administered within 60 minutes of emergency room arrival.
  • Nelonemdaz represents a paradigm shift by simultaneously inhibiting NR2B-selective NMDA receptors and scavenging free radicals, addressing two critical drivers of brain cell death following ischemic stroke.

CDx Diagnostics Receives FDA Breakthrough Device Designation for AI-Powered WATS3D Esophageal Cancer Diagnostic System

AI
  • CDx Diagnostics received FDA Breakthrough Device Designation for its WATS3D diagnostic system, a first-of-a-kind AI-powered digital pathology platform for diagnosing Barrett's esophagus and early neoplastic changes.
  • The system demonstrated superior effectiveness compared to the Seattle biopsy protocol in large multi-center studies involving over 23,000 patients for detecting dysplasia and esophageal cancer.
  • The WATS3D Test leverages artificial intelligence and 3D digital imaging to identify precancerous and cancerous cells, potentially detecting early-stage disease missed by conventional biopsies.
  • The diagnostic platform has analyzed over 400,000 patient samples and aims to improve early detection of patients at risk for esophageal cancer progression.

TCG Labs Soleil Raises $400M to Build Single-Asset Biotech Startups for Strategic Partnerships

  • TCG Labs Soleil, a venture firm collaborating with The Column Group, has raised $400 million to continue launching single-drug biotechnology startups focused on achieving proof-of-concept data for strategic partnerships.
  • The firm has already founded 10 portfolio companies targeting cancer, immune diseases, cardiometabolic disorders, and other conditions since its formation last year.
  • Led by Jin-Long Chen, founder of NGM Biopharmaceuticals and former Amgen research head, the company operates through both a venture fund and an evergreen R&D hub with expanded capabilities in Shanghai.
  • The single-asset approach aims to enable faster decision-making with better capital efficiency compared to broader platform strategies, positioning programs for partnerships at optimal value points.

Krystal Biotech's KB304 Gene Therapy Achieves 100% Response Rate in Phase 1 Décolleté Wrinkle Trial

  • Jeune Aesthetics' KB304 gene therapy demonstrated 100% investigator-reported improvement in décolleté wrinkles at all time points in the PEARL-2 Phase 1 trial, compared to 28.6% with placebo at three months.
  • The treatment uses HSV-1-based vectors to deliver collagen and elastin genes directly to skin cells, representing the first randomized controlled trial investigating combined COL3 and elastin supplementation for aesthetic applications.
  • KB304 showed broad improvements across multiple skin attributes including elasticity, hydration, and radiance, with 81.8% of patients reporting satisfaction with wrinkle appearance versus 14.3% on placebo.
  • The company plans to advance KB304 into Phase 2 development for décolleté wrinkles, targeting an area with no FDA-approved injectable treatments currently available.

A Study Assessing KB304 for the Treatment of Wrinkles in Women

NCT06724900Active, Not RecruitingPhase 1
Krystal Biotech, Inc.
Posted 11/20/2024

IDEAYA Biosciences to Present Phase 2 Darovasertib Data for Uveal Melanoma at ESMO 2025

Precision MedicineOncology
  • IDEAYA Biosciences will present Phase 2 clinical data on neoadjuvant darovasertib in primary uveal melanoma at the 2025 ESMO meeting in Berlin.
  • The presentation will include data from over 90 patients across both plaque brachytherapy and enucleation-eligible cohorts, focusing on enucleation prevention and vision preservation.
  • Darovasertib has received FDA Breakthrough Therapy Designation for neoadjuvant uveal melanoma in subjects requiring enucleation.
  • The company has initiated a multi-site, global randomized Phase 3 neoadjuvant registrational trial (OptimUM-10) in primary uveal melanoma in Q3 2025.

Neoadjuvant Darovasertib in Primary Uveal Melanoma

NCT07015190Not Yet RecruitingPhase 3
IDEAYA Biosciences
Posted 9/30/2025

(Neo)Adjuvant IDE196 (Darovasertib) in Patients With Localized Ocular Melanoma

NCT05907954RecruitingPhase 2
IDEAYA Biosciences
Posted 7/3/2023

Roche Explores Direct-to-Consumer Drug Sales Model to Reduce US Patient Costs

  • Roche is considering selling prescription medicines directly to US consumers to lower patient costs, following government pressure to reduce drug prices.
  • The direct-to-consumer model would bypass pharmacy benefit managers, which CEO Thomas Schinecker says would help reduce costs quickly.
  • This follows similar moves by Bristol Myers and Pfizer with their blood thinner Eliquis, and existing programs by Eli Lilly and Novo Nordisk for weight-loss drugs.
  • Industry sources suggest small-molecule drugs for diabetes, cardiovascular conditions, and respiratory diseases are most viable for direct-to-consumer distribution.

Sanner Opens First U.S. Manufacturing Facility in North Carolina, Expanding Global CDMO Operations

  • Sanner Group has launched its first North American manufacturing facility in Greensboro, North Carolina, featuring a 60,500 square foot GMP-certified site with cleanrooms and advanced injection molding capabilities.
  • The facility is designed to produce critical injection-molded components for medical devices and pharmaceutical packaging, along with desiccant solutions for the North American pharmaceutical and nutraceutical industries.
  • The new facility strengthens Sanner's position as a Contract Development and Manufacturing Organization (CDMO) for the healthcare industry, combining global quality standards with local responsiveness.
  • The investment includes 20,000 square feet of expansion space and represents part of Sanner's broader global expansion strategy following recent facility openings in Germany and China.

Mass General Brigham Study Links Glipizide to 13% Higher Cardiovascular Risk in Type 2 Diabetes Patients

Real World Evidence
  • Mass General Brigham researchers analyzed data from nearly 50,000 type 2 diabetes patients and found glipizide associated with a 13% increase in cardiovascular risk compared to DPP-4 inhibitors.
  • The five-year study examined patients with moderate cardiovascular risk treated with different sulfonylureas or DPP-4 inhibitors alongside metformin across 10 study sites nationwide.
  • Glipizide showed higher incidence of heart failure, related hospitalization and death, while glimepiride and glyburide demonstrated relatively smaller and less clear cardiovascular effects.
  • The findings highlight the importance of evaluating individual drugs within pharmaceutical classes separately rather than treating them as equivalent therapeutic options.

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