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Clinical Trial News

AIML Innovations Secures Provisional Patents for AI-Powered ECG Signal Processing Technology

AI
  • AI/ML Innovations Inc. has filed comprehensive provisional patent applications with the USPTO through its subsidiary Neural Cloud Solutions, covering advanced neural network architectures and cloud-based ECG signal optimization workflows.
  • The patent filings protect key innovations within AIML's proprietary AI signal-processing platforms MaxYield™ and CardioYield™, including a dynamic ECG reporting engine that supports clinical workflows with customizable components.
  • These patents strengthen AIML's position in AI-assisted cardiology by protecting both algorithmic foundations and deployment infrastructure, supporting the company's global commercialization strategy.
  • The company maintains a growing regulatory portfolio including a 510(k) premarket notification and registered FDA Device Master File, demonstrating commitment to rigorous validation and broad interoperability.

AstraZeneca Exercises Option for Quell's CAR-Treg Therapy in Inflammatory Bowel Disease

  • AstraZeneca has selected and licensed Quell Therapeutics' lead CAR-Treg cell therapy candidate for inflammatory bowel disease, triggering a $10 million milestone payment.
  • This marks the second major milestone in the companies' 2023 collaboration, following a similar achievement in Type 1 diabetes in November 2024.
  • The engineered T-regulatory cell therapy utilizes Quell's proprietary Foxp3 Phenotype Lock technology to address underlying immune drivers of IBD.
  • AstraZeneca will advance the candidate through CTA/IND-enabling studies while Quell provides manufacturing support for first-in-human trials.

Gyre Therapeutics Initiates Phase 1 Trial of F230 for Pulmonary Arterial Hypertension in China

Rare Disease
  • Gyre Therapeutics has successfully dosed the first volunteer in a Phase 1 clinical trial evaluating F230, a novel endothelin A receptor antagonist, for treating pulmonary arterial hypertension in China.
  • F230 is a fully synthetic small molecule designed to selectively block the ETA receptor, targeting pulmonary vascular remodeling and pressure reduction in PAH patients.
  • This milestone marks Gyre's expansion into the PAH field, leveraging China's growing PAH market valued at $370 million in 2023 and projected to reach $480 million by 2031.
  • The Phase 1 trial will evaluate safety, tolerability, and pharmacokinetics in healthy volunteers as part of Gyre's fibrosis-first therapeutic strategy.

Day One Biopharmaceuticals Appoints Michael Vasconcelles as Head of R&D to Drive Pipeline Expansion

OncologyTargeted TherapyLeadership Change
  • Day One Biopharmaceuticals has appointed Michael Vasconcelles, M.D., as Head of Research and Development, bringing over 25 years of oncology research and development expertise to the company.
  • Dr. Vasconcelles will oversee the company's research, development, and medical affairs infrastructure while supporting OJEMDA's growth and expanding the pipeline of targeted therapies.
  • The appointment comes at a pivotal time for Day One as it transitions from focusing primarily on pediatric cancer to developing transformative medicines for patients of all ages with life-threatening diseases.
  • Dr. Vasconcelles previously served as Executive Vice President at ImmunoGen, where he led the transformation into a global biotech enterprise and optimized ELAHERE's commercialization strategy.

Curadev and Memorial Sloan Kettering Expand Collaboration to Advance Novel STING Agonist CRD3874-SI

Oncology
  • Curadev Pharma and Memorial Sloan Kettering Cancer Center are expanding their collaboration through MSK's Therapeutics Accelerator program to advance development of CRD3874-SI, a first-in-class allosteric STING agonist.
  • The collaboration builds on an ongoing Phase 1a/b clinical trial for sarcoma and Merkel cell carcinoma patients and aims to explore CRD3874-SI's potential in additional cancer types.
  • CRD3874-SI has demonstrated encouraging safety and efficacy profiles in first-in-human studies and is moving forward into multiple solid-tumor expansion cohorts.
  • The expanded partnership provides Curadev with MSK's expertise and institutional resources, including medical, clinical, and regulatory advice to accelerate drug development.

A Study of CRD3874-SI in People With Solid Tumors

NCT06021626RecruitingPhase 1
Memorial Sloan Kettering Cancer Center
Posted 8/25/2023

32 Biosciences Secures $6 Million Funding to Advance Gut Microbiome Diagnostic and Therapeutic Platform

AI
  • 32 Biosciences raised $6 million in funding to advance its AI-powered gut microbiome diagnostic platform and first-of-its-kind non-antibiotic antimicrobial therapy.
  • The company's diagnostic tool uses mass spectrometry and machine learning to quantitatively assess gut microbiome functional health and is targeted for FDA clearance by late 2026.
  • Their therapeutic candidate represents a novel approach to preventing gut-derived bacterial infections without contributing to antibiotic resistance, having shown effectiveness in animal models.
  • The funding will support clinical validation, regulatory submissions, and commercial readiness as the company prepares for Series A fundraising and Phase I clinical trials.

Vanda Pharmaceuticals Initiates First-in-Human Trial of Personalized ASO Therapy VCA-894A for Ultra-Rare CMT2S

Rare Disease
  • Vanda Pharmaceuticals has dosed the first patient in a first-in-human clinical trial evaluating VCA-894A, an antisense oligonucleotide therapy designed for a specific patient with Charcot-Marie-Tooth disease Type 2S.
  • VCA-894A targets a patient-specific IGHMBP2 gene variant and has demonstrated ability to restore gene expression levels in neuromuscular junction systems derived from the patient's own cells.
  • CMT2S is an extremely rare inherited neuromuscular disorder with an estimated prevalence of less than 1 in 1,000,000 worldwide, causing progressive muscle weakness and motor function loss.
  • This personalized medicine approach represents a potential breakthrough for addressing rare genetic disorders using nucleic acid-based platforms to restore function where crucial gene expression has been disrupted.

Insmed's TPIP Achieves Breakthrough Results in Phase 2b PAH Trial with 35% Reduction in Pulmonary Vascular Resistance

Rare Disease
  • Insmed's treprostinil palmitil inhalation powder (TPIP) met its primary endpoint with a statistically significant 35% placebo-adjusted reduction in pulmonary vascular resistance in patients with pulmonary arterial hypertension.
  • The once-daily inhaled therapy demonstrated sustained 24-hour efficacy with a 35.5-meter improvement in six-minute walk distance and 60% reduction in cardiac stress biomarker NT-proBNP.
  • TPIP was well-tolerated with 75% of patients achieving the maximum dose, prompting Insmed to engage with FDA for Phase 3 trial design beginning in 2025-2026.

Caliway Unveils CBL-514 Preclinical Data for GLP-1 Weight Rebound Prevention at BIO 2025

  • Caliway Biopharmaceuticals will present first-time preclinical data showing CBL-514 combined with GLP-1 therapies significantly reduces post-discontinuation weight regain and fat replenishment rates.
  • The company addresses a critical unmet need as only 10% of patients maintain weight loss after stopping GLP-1 treatment, representing a significant clinical challenge.
  • CBL-514 works through selective adipocyte apoptosis to target subcutaneous fat directly, potentially serving as a complementary therapy to enhance long-term GLP-1 treatment outcomes.
  • Caliway plans to submit a Phase 2 IND application to the FDA for this weight rebound management indication in Q4 2025.

Monte Rosa Therapeutics Receives FDA Clearance for MRT-8102, First NEK7-Targeted Molecular Glue Degrader for Inflammatory Diseases

  • Monte Rosa Therapeutics received FDA clearance for its Investigational New Drug application for MRT-8102, a NEK7-directed molecular glue degrader targeting inflammatory diseases driven by NLRP3 inflammasome and IL-1β dysregulation.
  • MRT-8102 demonstrated nanomolar-level NEK7 degradation with no off-target activity and achieved near-complete inhibition of downstream inflammatory markers in non-human primate studies.
  • The company plans to initiate a Phase 1 healthy volunteer study in the coming weeks, with clinical results including safety, pharmacokinetics, and pharmacodynamic data expected in the first half of 2026.
  • Preclinical toxicology studies showed a greater than 200-fold exposure margin over the projected human efficacious dose, supporting MRT-8102's potential safety profile for clinical development.

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