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Clinical Trial News

EuMentis Therapeutics Receives FDA Clearance for Phase 2 Trial of Novel PDE10A Inhibitor EM-221 in Schizophrenia

  • EuMentis Therapeutics received FDA clearance for its IND application to initiate a Phase 2 clinical trial of EM-221, a novel PDE10A inhibitor for schizophrenia treatment.
  • EM-221 targets the PDE10A enzyme to modulate dopamine and glutamate signaling without direct receptor antagonism, potentially offering superior tolerability compared to traditional antipsychotics.
  • The company plans to begin dosing patients in the Phase 2 study during the second half of 2025, targeting both positive and negative symptoms of schizophrenia.
  • Schizophrenia affects nearly 4 million adults and adolescents in the U.S., representing a significant unmet medical need for safer and more effective treatments.

Actio Biosciences Receives FDA Clearance and Fast Track Designation for First-in-Class KCNT1 Epilepsy Treatment

Rare DiseasePrecision Medicine
  • The FDA has cleared Actio Biosciences' IND application and granted Fast Track designation for ABS-1230, a first-in-class oral KCNT1 inhibitor targeting rare pediatric epilepsy affecting approximately 2,500 individuals in the U.S.
  • ABS-1230 demonstrated the ability to inhibit all recurrently observed pathogenic KCNT1 mutations and rapidly suppress seizures in preclinical models, addressing the root cause of this devastating condition.
  • The company plans to initiate Phase 1 trials in healthy volunteers in fall 2025, followed by a proof-of-concept Phase 1b study in KCNT1-related epilepsy patients in early 2026.
  • KCNT1-related epilepsy is a severe and often fatal pediatric condition causing up to 20 seizures daily, with no current disease-modifying therapies available.

German Federal Patent Court Revokes Key Xarelto Patent, Opening Door for Generic Competition

  • The German Federal Patent Court revoked Bayer's crucial Xarelto patent EP 1 845 961 due to lack of inventive step, contradicting its earlier 2024 opinion that assumed the patent's validity.
  • The court's decision was influenced by Bayer's own clinical study conducted before the patent's priority date, which provided patients with dosage information that constituted prior art.
  • This ruling will likely lift preliminary injunctions against generic manufacturers like Stada and Aliud Pharma, allowing generic rivaroxaban products to enter the German market.
  • The patent, set to expire in January 2026, has faced challenges in 24 jurisdictions worldwide, with mixed results for Bayer's defense efforts across different countries.

Aldevron Establishes Innovation Center in Boston Biotech Hub to Accelerate Genomic Medicine Development

Gene Editing
  • Aldevron, a Danaher-owned leader in DNA, RNA, and protein manufacturing, has opened a new Innovation Center in Waltham, Massachusetts, positioning itself at the heart of the Boston biotech corridor.
  • The facility is designed to accelerate advancements in cell-free DNA, gene editing, and mRNA analytics, enabling faster collaboration with clients and shorter project timelines for advanced therapies.
  • The expansion builds on Aldevron's recent breakthrough in producing the world's first personalized CRISPR gene editing therapy for urea cycle disorder, demonstrating its capability to translate scientific discoveries into scalable solutions.
  • The strategic move leverages Boston's unmatched biotech ecosystem, including proximity to MIT, Harvard, and the Broad Institute, while benefiting from Danaher Corporation's $40 billion life sciences portfolio.

Protagenic Therapeutics Secures Japanese Patent for Modified Stilbenoid Epilepsy Treatment Through 2041

  • Protagenic Therapeutics received Japanese Patent JP 7714571B on July 18, 2025, providing exclusivity for modified stilbenoid compounds to treat epilepsy and seizures until March 31, 2041.
  • The patent builds on previous UK patent protection and covers specific compounds, formulations, and medical uses for epilepsy treatment within Japan's $85 billion pharmaceutical market.
  • The global epilepsy drug market is projected to grow from $11.29 billion in 2024 to $18.74 billion by 2034, representing a 5.20% compound annual growth rate.
  • The company plans to expand patent protection to additional territories, strengthening its intellectual property portfolio for this key pipeline asset.

ZUMA-7 Trial Demonstrates Survival Advantage for Second-Line CAR-T Therapy in Refractory DLBCL

CAR-T
  • The ZUMA-7 trial remains the only randomized CAR-T therapy study to demonstrate an overall survival benefit compared to standard of care in primary refractory diffuse large B-cell lymphoma.
  • Patients who received CAR-T therapy in second-line treatment showed superior outcomes compared to those who delayed treatment until third-line, highlighting the importance of early intervention.
  • CAR-T patients experienced faster recovery and return to baseline quality of life compared to those undergoing high-dose chemotherapy and autologous transplant.
  • Long-term follow-up data from pivotal trials show approximately 35% of patients achieve durable responses, suggesting potential cure in this subset with manageable long-term toxicity profiles.

Study of Effectiveness of Axicabtagene Ciloleucel Compared to Standard of Care Therapy in Patients With Relapsed/Refractory Diffuse Large B Cell Lymphoma

NCT03391466CompletedPhase 3
Kite, A Gilead Company
Posted 1/25/2018

GSK's TIM-3 Inhibitor Cobolimab Fails Phase III Trial in Advanced Lung Cancer

Monoclonal Antibody
  • GSK's anti-TIM-3 monoclonal antibody cobolimab failed to improve overall survival in the Phase III COSTAR trial for advanced non-small cell lung cancer patients previously treated with immunotherapy.
  • The study tested both triple therapy (cobolimab plus Jemperli and docetaxel) and double combination (Jemperli plus docetaxel) against docetaxel alone, with neither meeting the primary efficacy endpoint.
  • This setback adds to a growing list of failed TIM-3 checkpoint inhibitor programs from major pharmaceutical companies including Novartis, Roche, and Bristol Myers Squibb.
  • Despite the failure, cobolimab remains in Phase II studies for liver cancer, cervical cancer, and melanoma, though GSK has not confirmed its development plans.

Efficacy Comparison of Cobolimab + Dostarlimab + Docetaxel to Dostarlimab + Docetaxel to Docetaxel Alone in Participants With Advanced Non-small Cell Lung Cancer Who Have Progressed on Prior Anti-PD-(L)1 Therapy and Chemotherapy

NCT04655976Active, Not RecruitingPhase 3
GlaxoSmithKline
Posted 12/8/2020

VYNE Therapeutics' Repibresib Gel Fails to Meet Primary Endpoints in Phase 2b Vitiligo Trial

  • VYNE Therapeutics announced that its phase 2b trial of repibresib gel in nonsegmental vitiligo failed to meet primary and key secondary endpoints, with only 19.5% of patients achieving F-VASI50 improvement compared to 23.4% on vehicle.
  • The trial was impacted by an unexpectedly high vehicle effect and higher dropout rates in active treatment arms (up to 36.6%) compared to vehicle (10.6%).
  • Despite the setbacks, the highest dose (3%) showed nominally significant improvements in facial vitiligo scores (-43.6% vs -25.6% vehicle) and total vitiligo scores (-28.3% vs -16.2% vehicle).
  • The company will terminate the trial's extension phase and seek a development partner for repibresib while continuing to evaluate its BET inhibitor platform for immune-mediated diseases.

A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VYN201 Gel in Subjects With Non-segmental Vitiligo.

NCT06493578Active, Not RecruitingPhase 2
Vyne Therapeutics Inc.
Posted 6/4/2024

MAGENTIQ EYE Secures Series A Funding to Advance AI-Powered Colonoscopy Technology

AIFDA Approval
  • MAGENTIQ EYE completed its Series A funding round led by aMoon to accelerate commercialization of its FDA- and CE-approved AI colonoscopy software MAGENTIQ-COLO in the USA and Europe.
  • The company's AI-powered software significantly improves adenoma detection rates during colonoscopies and offers real-time polyp analysis with size and type estimation capabilities.
  • Funding will support ongoing clinical trials for diagnostic tools targeting ulcerative colitis, Barrett's esophagus dysplasia, and gastric intestinal metaplasia while expanding global market access.
  • The investment reflects growing commercial traction with dozens of daily procedures and a robust pipeline of trial and purchase requests across international markets.

Nautilus Biotechnology Partners with Allen Institute to Advance Alzheimer's Research Through Single-Molecule Proteomics

  • Nautilus Biotechnology has entered into an agreement with the Allen Institute to investigate the connection between tau protein and Alzheimer's disease using single-molecule proteomics technology.
  • The collaboration will employ Nautilus' Iterative Mapping method to identify novel tau proteoforms from human brain tissue and characterize phosphorylation patterns that may predict disease progression.
  • Nautilus' recent preprint demonstrates the platform's ability to analyze millions to billions of single-protein molecules with unprecedented resolution, providing actionable biological insights from brain samples of cognitively normal and impaired Alzheimer's patients.

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