Clinical Trial News
UNITY Biotechnology Announces 48-Week Results from Phase 2 ENVISION Study of UBX1325 in Patients with Wet Age-Related Macular Degeneration
UNITY Biotechnology revealed the 48-week outcomes of its Phase 2 ENVISION study, focusing on UBX1325's efficacy and safety in treating wet age-related macular degeneration (AMD). The study highlighted that patients maintained vision gains with a combination of UBX1325 and aflibercept, and a significant portion of UBX1325-treated patients achieved a treatment-free interval of over 24 weeks.
Highlighted Clinical Trials:
Unity Biotechnology, Inc.
Posted 3/2/2022
Actinogen Optimizes XanaMIA Phase 2b Alzheimer's Disease Trial Design
Actinogen Medical Limited has announced optimizations to its XanaMIA Phase 2b trial for mild-to-moderate Alzheimer's disease, reducing costs by AUD 30 million and speeding up initial results. The trial now focuses on a 10 mg dose group, enrolling 220 patients, and includes Australian sites for the first 100 patients. Additionally, the XanaCIDD trial for cognitive impairment associated with depression has expanded to include UK sites, with results expected by mid-2024.
Resminostat Receives Orphan Drug Designations in US and EU for CTCL Treatment
- Resminostat (Kinselby), a histone deacetylase (HDAC) inhibitor developed by 4SC AG, has been granted Orphan Drug Designation (ODD) by the FDA for cutaneous T-cell lymphoma (CTCL).
- The European Medicines Agency (EMA) has also granted Orphan Drug Designation to resminostat for the treatment of CTCL, providing 10 years of market exclusivity in the EU.
- 4SC anticipates submitting a Marketing Authorisation Application (MAA) to the EU for Kinselby (resminostat) in Q1 2024, marking a significant step toward potential approval.
Xeltis Initiates Pivotal Trial for aXess Graft in Hemodialysis Patients
- Xeltis has commenced a pivotal European clinical trial to assess the safety and efficacy of its aXess graft in patients with end-stage renal disease requiring hemodialysis.
- The prospective, single-arm study aims to enroll 110 patients across up to 25 European centers, monitoring them over five years post-implantation.
- aXess, designed for immediate use and to transform into a living blood vessel, has shown promising preliminary results in a first-in-human trial.
- The trial seeks to validate aXess as a potentially safer and longer-lasting solution, reducing complications associated with repeated dialysis punctures.
Novel Targets and Strategic Sequencing: The Evolving Landscape of T-Cell Redirection Therapies in Multiple Myeloma
- CAR T-cell therapies and bispecific antibodies targeting BCMA and GPRC5D are transforming multiple myeloma treatment, with ciltacabtagene autoleucel showing unprecedented efficacy with a median PFS of 34.9 months in heavily pretreated patients.
- Disease progression rate is a critical factor in therapy selection, with rapidly progressing patients better suited for "off-the-shelf" bispecific antibodies, while patients with indolent disease may benefit more from CAR T-cell therapies despite longer manufacturing times.
- Strategic sequencing of therapies is crucial, as prior BCMA-directed bispecific antibody treatment significantly reduces the efficacy of subsequent BCMA-targeted CAR T-cell therapy, highlighting the importance of preserving T-cell fitness and considering antigen loss.
Highlighted Clinical Trials:
Janssen Research & Development, LLC
Posted 2/1/2021
Pfizer
Posted 2/2/2021
Janssen Research & Development, LLC
Posted 11/7/2019
Celgene
Posted 12/13/2017
Celgene
Posted 4/16/2019
Janssen Research & Development, LLC
Posted 6/12/2020
Janssen Research & Development, LLC
Posted 9/17/2020
Janssen Research & Development, LLC
Posted 6/29/2018
AI-Powered HASTEN Tool Accelerates Virtual Drug Screening 10-Fold
- Finnish researchers have developed an ML-powered tool called HASTEN that reduces virtual drug screening time from 6 months to under 10 days for 1.56 billion molecules.
- The system successfully identified 90% of top-scoring drug candidates when screening just 1% of the compound library against bacterial and viral targets.
- Orion Pharma and University of Eastern Finland researchers have made their datasets public to advance computational drug discovery research.
Leucid Bio's Lateral NKG2D CAR-T Therapy LEU011 Receives MHRA Approval for Solid Tumor Clinical Trial
- Leucid Bio has received MHRA clinical trial authorization for LEU011, a novel lateral CAR-T therapy targeting NKG2D ligands expressed in over 80% of human tumors.
- The Phase 1/2 AERIAL trial will evaluate LEU011 in patients with relapsed or refractory solid tumors, with the first patient expected to be dosed in Q4 2023.
- LEU011 previously received the Innovation Passport designation from MHRA, highlighting its potential as a breakthrough therapy for solid tumors with limited treatment options.
Travere Therapeutics' Filspari Nears, But Misses, Primary Endpoint in IgA Nephropathy Trial
- Travere Therapeutics' Filspari narrowly missed statistical significance in a Phase 3 trial for IgA nephropathy, a rare kidney disorder, with a p-value of 0.058.
- The trial involved 404 patients randomized to receive Filspari or irbesartan, an existing blood pressure medication used to manage the disease.
- While early data showed a 50% reduction in urine protein levels, the 24-month data on kidney function decline narrowly missed the primary endpoint.
- Filspari received accelerated approval in February based on earlier biomarker data, with full approval contingent on these confirmatory trial results.
FDA Grants Orphan Drug Designation to Immix Biopharma's NXC-201 for AL Amyloidosis
- Immix Biopharma's NXC-201, a CAR-T cell therapy, receives Orphan Drug Designation from the FDA for treating Amyloid Light Chain (AL) Amyloidosis.
- The designation provides Immix Biopharma with benefits including potential market exclusivity, tax credits, and a waiver for prescription drug user fees.
- NXC-201 is currently in Phase 1b/2a clinical trials and has demonstrated a 100% hematologic response rate in AL amyloidosis patients in early trials.
- AL Amyloidosis, a rare disorder affecting 30,000-40,000 individuals in the US and Europe, has a market projected to reach $6 billion by 2025.
Highlighted Clinical Trials:
Immix Biopharma, Inc.
Posted 2/3/2023
Hadassah Medical Organization
Posted 1/1/2021
First RoActemra Biosimilar Tyenne Receives EU Approval, Marking Milestone for Fresenius Kabi
- The European Commission has granted approval for Tyenne, the first biosimilar version of Roche's blockbuster drug RoActemra (tocilizumab), developed by Fresenius Kabi for multiple inflammatory conditions and severe COVID-19.
- Tyenne is approved for all RoActemra indications, including rheumatoid arthritis, systemic juvenile idiopathic arthritis, and cytokine release syndrome, representing a significant advancement in accessible treatment options.
- This approval marks Fresenius Kabi's third biosimilar success and aligns with their Vision 2026 strategy to generate €1 billion in additional revenue every three years.