Clinical Trial News

FDA grants Fast Track designation to Abata Therapeutics' autologous Treg therapy for progressive MS, ABA-101. Orgenesis Inc. reports encouraging results from its CD19 CAR-T therapy trial for CD19+ acute lymphoblastic leukemia, with high response rates and low severe cytokine release syndrome incidence. Skyline Therapeutics' SKG1108, a gene therapy for retinitis pigmentosa, receives FDA Orphan Drug Designation.

CD47 inhibitor drugs, targeting cancer cell evasion, are in advanced clinical trials, with magrolimab and evorpacept leading. Combination therapies are key, aiming to enhance efficacy and overcome resistance. Market growth is driven by rising cancer incidence and immunotherapy use, attracting significant investments and partnerships. Challenges include managing toxicity and demonstrating therapeutic advantages. The field is rapidly expanding, with global competition intensifying.

KalVista Pharmaceuticals presented sebetralstat data at Bradykinin Symposium 2024, showing consistent efficacy and safety profiles across phase 2 and 3 trials, with rapid symptom relief and a safety profile comparable to placebo. Median time to treatment was 9 minutes for all attacks and 8 minutes for laryngeal attacks, with median time to symptom relief at 1.8 hours for all attacks and 1.3 hours for laryngeal attacks. Oral sebetralstat may address challenges of current injectable treatments for HAE.

Dr. Jim Omel, a multiple myeloma survivor, has been an advocate and expert for over 25 years, contributing invaluable insights to the DETERMINATION study on autologous stem cell transplants for newly diagnosed patients. His unique perspective as both a physician and patient has significantly impacted the trial's success and the development of tailored treatment approaches for diverse patient groups.

Study uses three cohorts (discovery, validation, testing) to discover, prioritize, validate, and test biomarkers for suicidality, integrating microarray and RNA sequencing data. Key steps include discovery of biomarkers tracking suicidality, prioritization through Convergent Functional Genomics, validation in postmortem suicide completers, and testing in independent psychiatric cohorts for predicting suicidality severity and future hospitalizations. Top candidate biomarkers are identified and tested for clinical utility, with a focus on gender-specific predictions and personalized medicine.

Study evaluates PMNs and NETs in PsA patients, finding distinct phenotypic and functional deficits compared to HCs, including lower expression of activation markers, reduced ROS production, diminished phagocytosis, and less granular enzyme and NET biomarker release. Elevated serum levels of neutrophil-related mediators and NET biomarkers in PsA patients suggest a distinct neutrophil signature associated with PsA.

Strides Pharma share price hit an all-time high after its Singapore subsidiary received USFDA approval for generic Theophylline extended-release tablets, to be manufactured in Bangalore.

Dasiglucagon for congenital hyperinsulinism has a PDUFA date of October 8, 2024, supported by phase 3 trials. Perioperative nivolumab-based regimen for resectable NSCLC also has a PDUFA date of October 8, 2024, based on the CheckMate -77T trial. Oclaiz (CAM2029) for acromegaly has a PDUFA date of October 21, 2024, supported by phase 3 studies. Sulopenem etzadroxil/probenecid for uncomplicated UTI has a PDUFA date of October 25, 2024, with data from the REASSURE trial.

IN8bio reduces headcount by 49% and suspends glioblastoma programme to focus on AML cell therapy INB-100, aiming for a registrational trial. The board and management team also cut cash compensation by 11%. IN8bio plans to add a control cohort to its ongoing Phase I trial and expand the Phase II dose cohort, with enrolment expected by H1 2025.

Sun Pharmaceutical Industries and Moebius Medical received FDA Fast Track designation for MM-II (Large Liposomes of DPPC and DMPC) to treat osteoarthritis knee pain, with Phase 3 clinical trials planned.