Clinical Trial News
FDA Issues New Draft Guidance on Allogeneic Cell-Based Medical Products
The FDA has released a new draft guidance titled 'Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products', aimed at assisting sponsors in determining appropriate cell safety testing for investigational new drug applications or biologics license applications. The guidance applies to allogeneic cell-based products regulated by CBER's Office of Therapeutic Products, including genetically modified cells, and excludes cell substrates used in manufacturing non-cell-based products. Comments on the draft are accepted until July 29, 2024.
MindBio Therapeutics Advances MB22001 Microdosing for Depression with Phase 2 Trials
- MindBio Therapeutics has completed a Phase 2a trial of MB22001 for major depressive disorder, showing a 60% reduction in depressive symptoms and a 53% remission rate.
- The company has initiated a Phase 2b trial of MB22001, a proprietary form of LSD, involving take-home microdosing for patients with major depressive disorder.
- MindBio is also exploring a dual listing on a senior exchange in the United States or Australia, following positive clinical trial results.
- Prior trials of MB22001 showed statistically significant improvements in sleep quality, mood, and overall well-being, suggesting potential as a disruptive treatment for depression.
Jacobio Pharma's Glecirasib Shows Promise in KRAS G12C-Mutated NSCLC
- Jacobio Pharma's glecirasib demonstrated a confirmed objective response rate (cORR) of 47.9% in second-line non-small cell lung cancer (NSCLC) patients.
- The Phase II registrational study of glecirasib showed a median progression-free survival (mPFS) of 8.2 months and a median overall survival (mOS) of 13.6 months.
- Glecirasib has a manageable safety profile, with only 5.0% of patients discontinuing treatment due to treatment-related adverse events (TRAEs).
- Glecirasib received orphan drug designation for pancreatic cancer from the FDA and breakthrough therapy designation from China's CDE.
GSK Reports Strong Q1 2024 Driven by Pipeline Progress and Key Therapeutic Areas
- GSK's Q1 2024 demonstrates robust performance, fueled by advancements in its pipeline and positive Phase III data across multiple therapeutic areas.
- The company highlights strengthened growth prospects in infectious diseases, HIV, respiratory/immunology, and oncology, indicating a diversified approach.
- GSK anticipates continued momentum throughout 2024, projecting meaningful growth in both sales and earnings based on recent R&D achievements.
- The report emphasizes forward-looking statements are subject to uncertainties, including market conditions and regulatory changes, advising investor caution.
Chime Biologics and Mabgeek Achieve PPQ Milestone for MG-K10, Advancing Atopic Dermatitis and Asthma Therapies
- Chime Biologics and Mabgeek completed Process Performance Qualification (PPQ) for MG-K10, a humanized anti-IL-4Rα monoclonal antibody, crucial for regulatory compliance and manufacturing validation.
- MG-K10 targets Th2-mediated inflammatory diseases like atopic dermatitis and asthma, with Phase III clinical trials underway, and is designed for once-monthly dosing.
- The collaboration focuses on commercial manufacturing and CMC development to accelerate MG-K10's commercialization in American and European markets, emphasizing strong efficacy and patient adherence.
- MG-K10 has shown potential as a best-in-class treatment, improving clinical signs and lung function in Phase II trials for atopic dermatitis and asthma, respectively.
SeaStar Medical Secures $3.6 Million NIH Grant for Heart Failure Device Study
SeaStar Medical has been awarded a $3.6 million grant by the NIH to conduct a clinical trial on its Selective Cytopheretic Device (SCD-ADULT) for treating severe chronic heart failure patients, aiming to bridge them to LVAD implantation.
Eton Pharmaceuticals Submits NDA to FDA for ET-400 (Hydrocortisone Oral Solution)
- Eton Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for ET-400, a patented hydrocortisone oral solution, aimed at treating adrenocortical insufficiency.
- The FDA review is expected to take 10 months, potentially leading to approval and market launch in the first quarter of 2025, addressing a critical need for children.
- ET-400 is protected by a U.S. patent expiring in 2043, with additional patent applications pending, highlighting its unique formulation and market exclusivity.
- Eton anticipates that ET-400, along with Alkindi Sprinkle, could achieve combined peak sales exceeding $50 million annually, reflecting strong demand from patients and physicians.
FDA Updates LUPKYNIS® Label with Three-Year Data and Modified Kidney Function Monitoring Guidelines for Lupus Nephritis
- The FDA has updated the label for LUPKYNIS® (voclosporin) to include three-year data from the AURORA 2 extension study, demonstrating sustained complete renal response in patients with lupus nephritis (LN).
- The updated label provides new guidance for monitoring kidney function, recommending quarterly eGFR assessments after the first year of treatment, aligning with standard clinical care.
- The safety profile of LUPKYNIS® remains consistent with previous findings from the AURORA clinical program, with no new safety concerns identified in the long-term data.
- A post hoc analysis showed that 20.1% of patients on LUPKYNIS achieved sustained complete renal response compared to 11.8% on placebo, highlighting the drug's long-term efficacy.
FDA Implements Historic Regulation of Laboratory-Developed Tests with New Final Rule
- The FDA has issued a final rule to regulate laboratory-developed tests (LDTs), marking a significant shift from its previous enforcement discretion approach to comprehensive oversight.
- The new regulation introduces a 4-year phase-out period and will require LDTs to meet the same standards as other in vitro diagnostic devices, with exceptions for certain existing tests and unmet needs.
- The rule aims to ensure test accuracy and patient safety, particularly for critical applications like cancer risk assessment and disease diagnosis, though it faces mixed reactions from industry stakeholders.
Iterum Therapeutics Resubmits NDA for Oral Sulopenem to Treat Uncomplicated UTIs
- Iterum Therapeutics has resubmitted its New Drug Application (NDA) to the FDA for oral sulopenem, targeting uncomplicated urinary tract infections (uUTIs) in adult women.
- The resubmission includes data from Phase 3 trials (REASSURE, SURE 1, SURE 2, SURE 3) demonstrating oral sulopenem's efficacy and safety compared to Augmentin and ciprofloxacin.
- If approved, oral sulopenem could be the first oral penem antibiotic in the U.S., offering a new treatment option for the estimated 40 million uUTIs annually.
- The FDA is expected to complete its review in early Q4 2024, provided the resubmitted NDA addresses prior deficiencies.