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Clinical Trial News

First Patient Treated with Novel B-Cell Targeting Therapy for Myasthenia Gravis in Phase III Trial

Rare Disease
  • The first patient worldwide has been treated with Remibrutinib, a novel B-cell targeting therapy for Myasthenia Gravis, marking the start of an international Phase III clinical trial at HonorHealth Research Institute.
  • This represents the first time a drug has targeted B cells in Myasthenia Gravis treatment, offering a new mechanism of action that blocks a different part of the neuro-muscular-junction pathway than existing therapies.
  • The rare autoimmune disease affects fewer than 200 people per million, causing muscle weakness symptoms that severely impact daily activities like brushing teeth and getting dressed.
  • If successful, the trial could lead to FDA approval for Remibrutinib in Myasthenia Gravis, with the drug already approved for certain cancer types.

Alzamend Neuro Completes $5 Million Financing to Advance Five Phase II Trials for AL001 Lithium Therapy

  • Alzamend Neuro completed a $5 million private placement months ahead of schedule to fund five Phase II clinical trials of AL001 "Lithium in Brain" studies at Massachusetts General Hospital.
  • The company's AL001 drug candidate uses patented ionic cocrystal technology to deliver lithium via a therapeutic combination of lithium, salicylate and L-proline for treating neurological and psychiatric conditions.
  • Five Phase II trials are planned across multiple indications, with the first healthy subject trial initiated in May 2025 and additional trials for bipolar disorder, Alzheimer's disease, major depressive disorder and PTSD expected to commence through Q4 2025.
  • The financing targets treatment solutions for over 43 million Americans afflicted with Alzheimer's disease, bipolar disorder, major depressive disorder and post-traumatic stress disorder.

ZIRCON Phase 3 Trial Demonstrates High Accuracy of Novel PET Tracer for Kidney Cancer Detection

  • The ZIRCON phase 3 trial evaluated 89Zr-TLX250 PET tracer for characterizing small renal masses, achieving greater than 85% accuracy in identifying clear cell renal cell carcinoma.
  • The novel molecular imaging approach demonstrated exceptional positive predictive value of 93-95% for ccRCC, with all positive cases indicating malignancy even when not specifically ccRCC.
  • This represents a significant advance in noninvasive kidney cancer diagnosis, potentially reducing unnecessary interventions while providing actionable clinical information for treatment decisions.

89Zr-TLX250 for PET/CT Imaging of ccRCC- ZIRCON Study

NCT03849118CompletedPhase 3
Telix Pharmaceuticals (Innovations) Pty Limited
Posted 8/15/2019

CDC Sponsors First Clinical Trial of Needle-Free Rotavirus Vaccine Using Dissolvable Microarray Patch Technology

  • Emory University and Micron Biomedical have launched the first CDC-sponsored clinical trial of a rotavirus vaccine delivered via dissolvable microarray patch technology, marking a significant milestone in needle-free vaccine administration.
  • The Phase I trial will evaluate CC24, a novel inactivated rotavirus vaccine developed by the CDC, using Micron's painless patch technology that eliminates cold chain requirements and medical waste.
  • The study addresses a critical global health need, as current oral rotavirus vaccines show reduced effectiveness in low- and middle-income countries where childhood diarrheal deaths from rotavirus remain highest.
  • The trial will recruit 50 healthy adults aged 18-45 to assess safety, reactogenicity, and immunogenicity, with results informing future pediatric studies for this life-saving vaccine innovation.

Teva and Fosun Pharma Form Strategic Partnership for Novel Anti-PD1-IL2 Cancer Immunotherapy TEV-56278

Oncology
  • Teva Pharmaceutical and Fosun Pharma announced a strategic partnership to develop TEV-56278, an anti-PD1-IL2 ATTENUKINE therapy currently in Phase 1 trials for various cancers including melanoma.
  • Under the agreement, Fosun Pharma receives exclusive rights to develop, manufacture and commercialize TEV-56278 in China, Hong Kong, Macau, Taiwan and select Southeast Asian countries while Teva retains global rights elsewhere.
  • TEV-56278 represents a novel cancer immunotherapy approach designed to selectively deliver IL-2 to PD-1+ T cells within tumors, potentially amplifying anti-tumor activity while minimizing systemic toxicities.
  • Preclinical data has demonstrated tumor regression, enhanced T-cell infiltration, and durable immune memory, with the therapy being evaluated both as monotherapy and in combination with pembrolizumab.

Debiopharm Initiates First-in-Human Trial of CD37-Targeted ADC Debio 1562M for Relapsed/Refractory AML

Monoclonal AntibodyOncologyTargeted Therapy
  • Debiopharm has dosed the first patient in a Phase 1/2 trial of Debio 1562M, a first-in-class CD37-targeted antibody-drug conjugate for relapsed/refractory acute myeloid leukemia patients.
  • The compound utilizes Debiopharm's proprietary Trifecta approach, combining naratuximab antibody, MultiLink™ linker technology, and a microtubule inhibitor payload to target CD37 antigens on leukemic cells.
  • AML represents a significant unmet medical need with only 32% five-year overall survival and median survival as low as 7 months in certain populations.
  • Preclinical studies demonstrated Debio 1562M showed anti-leukemic activity across all AML subtypes and superior activity compared to current standard-of-care therapies.

A Study to Assess the Safety, Tolerability, and Antileukemic Activity of Debio 1562M in Participants With Acute Myeloid Leukemia (AML)

NCT06969430RecruitingPhase 1
Debiopharm International SA
Posted 5/30/2025

AbbVie's VERONA Trial for High-Risk Myelodysplastic Syndrome Fails to Meet Primary Survival Endpoint

  • AbbVie's Phase 3 VERONA trial evaluating venetoclax plus azacitidine in newly diagnosed higher-risk myelodysplastic syndrome failed to meet its primary endpoint of overall survival with a hazard ratio of 0.908 and p-value of 0.3772.
  • The global randomized controlled trial compared venetoclax in combination with azacitidine versus azacitidine plus placebo, with no new safety signals observed during the study.
  • The negative results do not impact venetoclax's current approved indications for chronic lymphocytic leukemia, small lymphocytic lymphoma, and acute myeloid leukemia in elderly patients or those unsuitable for standard chemotherapy.
  • Full trial results will be presented at a future medical congress or publication, with participating patients being informed by their treating physicians about the study outcomes.

A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

NCT02942290Active, Not RecruitingPhase 1
AbbVie
Posted 1/12/2017

A Study to Evaluate the Safety and Effectiveness of Upadacitinib Tablets in Adult and Adolescent Participants With Severe Alopecia Areata

NCT06012240RecruitingPhase 3
AbbVie
Posted 10/11/2023

Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome

NCT04401748Active, Not RecruitingPhase 3
AbbVie
Posted 9/10/2020

Penumbra Completes Enrollment in Landmark STORM-PE Trial Testing Thrombectomy for Pulmonary Embolism

  • Penumbra has completed enrollment of 100 patients in the pivotal STORM-PE randomized controlled trial, comparing computer assisted vacuum thrombectomy plus anticoagulation versus anticoagulation alone for acute intermediate-high risk pulmonary embolism.
  • The first-of-its-kind trial aims to provide level 1 clinical evidence on whether endovascular therapy with CAVT is superior to medical therapy alone for this critically ill patient population.
  • Pulmonary embolism affects an estimated 900,000 Americans annually and can be life-threatening, with 10-30 percent of individuals dying within one month of diagnosis.
  • The trial results will inform treatment guidelines for intermediate-high risk PE patients, where current treatment strategies are not well defined despite the condition being a leading cause of cardiovascular morbidity and mortality.

Comparison of Two Pulmonary Embolism Treatments

NCT05684796Active, Not RecruitingNot Applicable
Penumbra Inc.
Posted 11/27/2023

Innovative Molecules Completes Phase 1b Enrollment for Novel HSV Inhibitor IM-250

  • Innovative Molecules GmbH has completed enrollment in the Phase 1b portion of its clinical trial evaluating IM-250, a next-generation helicase-primase inhibitor for recurrent genital herpes.
  • The placebo-controlled study assesses safety, efficacy, and pharmacokinetics of once-weekly oral dosing in patients with recurrent genital herpes.
  • IM-250 represents potential innovation in a market that has seen little therapeutic advancement in over four decades.
  • Topline results from the Phase 1b portion are expected in the second half of 2025.

Xlife Sciences Expands Asian Footprint Through Strategic Partnerships in Digital Health and Precision Oncology

AI
  • Xlife Sciences AG has signed strategic partnerships with Korean companies Huraypositive Corp. and OncoMASTER Inc. to accelerate digital health and precision oncology innovation across Asia.
  • The collaboration leverages OncoMASTER's AI-powered platform built on clinical data from over 10,000 advanced cancer patients, representing the largest such dataset in Asia.
  • The partnership aims to bridge academic innovation with clinical translation while enabling Korean companies to access European commercialization opportunities through Xlife Sciences' network.
  • This marks a significant expansion of Xlife Sciences' international presence, focusing on personalized healthcare solutions and AI-driven cancer treatment prediction technologies.

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