Clinical Trial News

HepaRegeniX's HRX-215, a first-in-class MKK4 inhibitor, shows promise in treating liver diseases by enhancing liver regeneration and preventing liver failure, as evidenced by clinical and preclinical data. Safe and well-tolerated in healthy volunteers, it aims to revolutionize liver surgery and transplantation, with Phase II studies planned.

A groundbreaking study led by WEHI and the Peter MacCallum Cancer Centre has identified two proteins critical for the development of B-cell acute lymphoblastic leukaemia, offering new treatment possibilities. By targeting these proteins, researchers were able to kill leukaemia cells and stop their growth in lab models, paving the way for future clinical trials.

I-Mab announced a strategic divestiture of its China assets, expected to close by March 2024, impacting its financials and pipeline. Key updates include uliledlimab's IND filing for NSCLC, givastomig's dose escalation study, and ragistomig's Phase 1 results. The company reported a net loss of RMB1,465.7 million for 2023, with cash reserves of RMB2.3 billion.

Gilgamesh Pharmaceuticals received a $14M NIDA grant for GM-3009, a cardiac-safe ibogaine analog for opioid use disorder treatment, aiming to eliminate ibogaine's cardiovascular risks and prove efficacy in Phase 1/1b trials, marking a potential breakthrough in addiction therapy.

Dimerix Limited announced successful interim Phase 3 trial results for DMX-200, a treatment for Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disease. The trial involved 72 patients, with plans to expand to more countries and include children over 12. DMX-200 was co-invented by Professor Kevin Pfleger, Dr James Williams, and Liddy McCall, aiming to address the high unmet need in FSGS treatment.

CAP-1002, an investigational cell therapy, has shown to slow declines in upper limb and heart function in Duchenne muscular dystrophy (DMD) patients over two years in the HOPE-2 study. A Phase 3 trial is ongoing, with potential FDA approval pathways being explored. CAP-1002 aims to benefit late-stage DMD patients by slowing skeletal and cardiac muscle function decline, demonstrating disease-modifying potential and a favorable safety profile.

Rezdiffra, the first FDA-approved medication for NASH, targets liver fibrosis and NASH resolution in noncirrhotic patients with moderate to advanced liver fibrosis. Approved based on Phase 3 MAESTRO-NASH trial results, it offers a new treatment paradigm, with ongoing studies to confirm clinical benefits and support full approval.

Zacks.com highlights biotech stocks, focusing on Q4 earnings and regulatory updates. Moderna beat earnings estimates, reaffirmed 2024 sales guidance, and expects RSV vaccine approval. RAPT Therapeutics faced a clinical hold due to a liver failure case. Iovance surged after FDA approval for melanoma treatment. Sarepta seeks label expansion for DMD drug. Regeneron's myeloma drug gets Priority Review.

The article provides an update on CRISPR-based therapies, highlighting the first-ever approval of a CRISPR-based medicine, Casgevy, for sickle cell disease and transfusion-dependent beta thalassemia. It discusses the progress, challenges, and future directions of CRISPR clinical trials across various diseases, including blood disorders, chronic bacterial infections, protein-folding diseases, inflammatory diseases, cancers, cardiovascular disease, HIV/AIDS, diabetes, and autoimmune diseases.

Clearside Biomedical discussed its Q4 2023 financial results and corporate updates, highlighting progress in its CLS-AX clinical development program for wet AMD, including the initiation and completion of the Phase IIb trial, Odyssey. The company emphasized its leadership in suprachoroidal drug delivery technology, collaborations, and anticipated data readouts in 2024. Financial updates included a cash balance sufficient to fund operations into Q3 2025.