Clinical Trial News
FDA Approves Alvotech/Teva's Ustekinumab Biosimilar, SELARSDI
- The FDA has approved SELARSDI (ustekinumab-aekn), a biosimilar to Johnson & Johnson’s STELARA® (ustekinumab), for treating moderate to severe plaque psoriasis and active psoriatic arthritis.
- SELARSDI, developed by Alvotech and to be commercialized by Teva in the U.S., is administered as a subcutaneous injection for adult and pediatric patients aged six years and older.
- This marks the second ustekinumab biosimilar approved by the FDA, following Amgen’s WEZLANA, and Alvotech and Teva settled with Johnson & Johnson to market it no later than February 21, 2025.
EU Approves Pfizer's Emblaveo: First-in-Class Antibiotic for Multi-Drug Resistant Infections
- The European Commission has approved Pfizer's Emblaveo (aztreonam-avibactam), marking the first approval of its class for treating serious multidrug-resistant Gram-negative bacterial infections in adults.
- Clinical trials demonstrated Emblaveo's effectiveness, with the ASSEMBLE study showing a 42% cure rate against metallo-beta-lactamase producing infections compared to 0% with standard therapy.
- The approval covers multiple serious conditions including complicated intra-abdominal infections, hospital-acquired pneumonia, and complicated urinary tract infections, offering new hope for patients with limited treatment options.
Health Canada Grants Priority Review for Trofinetide for Rett Syndrome Treatment
- Health Canada has accepted Acadia Pharmaceuticals' New Drug Submission for trofinetide for treating Rett syndrome and granted it Priority Review.
- Trofinetide, if approved, would be the first treatment option available in Canada for Rett syndrome, a rare neurodevelopmental disorder.
- The submission is supported by Phase 3 LAVENDER study results, demonstrating statistically significant improvements in key Rett syndrome symptoms.
- Priority Review is reserved for drugs treating serious conditions with substantial evidence of clinical effectiveness and improved benefit/risk profiles.
Abeona Therapeutics Receives Complete Response Letter from FDA for Pz-cel in RDEB
- The FDA issued a Complete Response Letter (CRL) for Abeona Therapeutics' Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to treat recessive dystrophic epidermolysis bullosa (RDEB).
- The CRL cites the need for additional Chemistry, Manufacturing, and Controls (CMC) information, specifically regarding validation requirements for manufacturing and release testing methods.
- The FDA did not identify any deficiencies related to the clinical efficacy or safety data and did not request new clinical trials to support pz-cel approval.
- Abeona plans to resubmit the BLA in the third quarter of 2024 after addressing the CMC information requests outlined in the CRL.
Highlighted Clinical Trials:
Abeona Therapeutics, Inc
Posted 10/5/2010
Abeona Therapeutics, Inc
Posted 1/10/2020
FDA Approves CAR-T Therapies Abecma and Carvykti for Earlier Use in Multiple Myeloma
- The FDA has approved Abecma (idecabtagene vicleucel) for relapsed or refractory multiple myeloma after two or more prior lines of therapy, expanding its availability to more patients.
- Carvykti (ciltacabtagene autoleucel) has also been approved by the FDA for multiple myeloma after at least one prior line of therapy, marking it as the first BCMA-targeted therapy for first relapse.
- Both approvals are based on Phase 3 trial data demonstrating significant reduction in disease progression or death compared to standard of care regimens in earlier lines of treatment.
- These approvals offer the potential for treatment-free periods and improved outcomes for patients with multiple myeloma, addressing a critical unmet need in the treatment landscape.
Highlighted Clinical Trials:
Janssen Research & Development, LLC
Posted 6/12/2020
Janssen Research & Development, LLC
Posted 11/7/2019
Janssen Research & Development, LLC
Posted 8/29/2016
Stichting European Myeloma Network
Posted 12/14/2018
Metformin's Potential in Slowing Aging to be Examined in TAME Trial
- The TAME Trial will investigate metformin's potential to prevent age-related diseases like heart disease, cancer, and cognitive decline in healthy older adults.
- Metformin, a widely used diabetes drug, has shown anti-inflammatory effects and reduced risk of cancer, dementia, and cardiovascular issues in observational studies.
- The trial aims to understand metformin's mechanisms, such as improving cellular energy through autophagy and reducing inflammation and oxidative stress.
- Funding for the TAME Trial, estimated at $45-70 million, is primarily sourced from philanthropists and the National Institute on Aging due to metformin's generic status.
Fate Therapeutics Treats First Lupus Patient with Off-the-Shelf CAR-T Cell Therapy FT819
- Fate Therapeutics has treated the first patient with systemic lupus erythematosus using FT819, an off-the-shelf CD19-targeted CAR-T cell therapy derived from induced pluripotent stem cells.
- The 27-year-old patient with refractory SLE received 360 million FT819 cells after conditioning chemotherapy and was discharged after three days without notable adverse events.
- Phase 1 data from B-cell malignancy studies demonstrated that FT819 achieves rapid and sustained CD19+ B cell depletion, tissue infiltration, and immune reconstitution mechanisms crucial for autoimmune disease treatment.
- The iPSC-derived platform offers advantages over traditional CAR-T therapies by eliminating the need for patient apheresis and providing off-the-shelf availability for broader patient access.
Highlighted Clinical Trials:
Fate Therapeutics
Posted 3/28/2024
FDA Clears IND for Arbor Biotechnologies' ABO-101 Gene-Editing Therapy for Primary Hyperoxaluria Type 1
- Arbor Biotechnologies received FDA clearance for its Investigational New Drug (IND) application for ABO-101, a gene-editing therapeutic targeting primary hyperoxaluria type 1 (PH1).
- The Phase 1/2 redePHine study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of ABO-101 in adult and pediatric PH1 patients.
- ABO-101 uses a CRISPR-based approach to knock down the HAO1 gene in the liver, aiming for durable reduction in oxalate levels, addressing a high unmet need in PH1 treatment.
- ABO-101, a one-time therapy, comprises lipid nanoparticles encapsulating mRNA encoding a Type V CRISPR Cas12i2 nuclease and a guide RNA targeting the human HAO1 gene.
AI Tool Predicts Responses to Cancer Therapy Using Single-Cell Omics
A groundbreaking AI tool named PERCEPTION has been developed to predict patient responses to cancer drugs at single-cell resolution, offering a new approach to personalized cancer treatment by utilizing rich information from single-cell omics.
Pembrolizumab Improves Overall Survival in Clear Cell Renal Cell Carcinoma
- Adjuvant pembrolizumab demonstrates a significant 38% reduction in the risk of death compared to placebo in patients with clear cell renal cell carcinoma (ccRCC) post-nephrectomy.
- The KEYNOTE-564 study reveals a 91.2% overall survival rate at 48 months in the pembrolizumab group versus 86.0% in the placebo group, indicating a clinically meaningful survival benefit.
- Pembrolizumab was associated with a higher incidence of serious adverse events, but no deaths were attributed to the therapy, suggesting a manageable safety profile.
- These findings support adjuvant pembrolizumab as a standard intervention after surgery for ccRCC patients at increased risk of recurrence, potentially expanding survival options.
Highlighted Clinical Trials:
Merck Sharp & Dohme LLC
Posted 6/9/2017