Clinical trialRSS5 days ago
A company called Neurizon Therapeutics has been approved to test a new liquid version of a drug called NUZ-001 in Australia. This drug is being developed to treat ALS, a disease that affects nerve cells and causes muscle weakness. The company is testing this liquid form in healthy volunteers, while a tablet version of the same drug is already being tested in a larger trial in the United States.
WHY IT MATTERSIf the liquid formulation proves safe and effective, it could offer ALS patients an easier-to-take alternative to a tablet, which may improve medication adherence for people with swallowing difficulties—a common symptom of ALS.
Clinical trialRSSMay 4
A new experimental drug called dazucorilant helped people with ALS live longer when given at a higher dose, according to a Phase 2 clinical trial. While the trial didn't fully meet all its main goals, the survival benefit was significant enough to be noteworthy. This suggests the drug may have promise for treating ALS, a disease that affects nerve cells controlling muscles.
WHY IT MATTERSIf dazucorilant advances to Phase 3 testing, ALS patients may have access to a new treatment option that could extend survival, which is critical since current ALS therapies have limited effectiveness.
Clinical trialRSSApr 30
A company called Vectory Therapeutics has received permission to test its experimental ALS drug, called VTx-002, at hospitals in the United Kingdom and European Union. The drug is already being tested in the United States as part of a Phase 1/2 clinical trial. This expansion means more patients in Europe may have the opportunity to participate in this early-stage study of a potential new ALS treatment.
WHY IT MATTERSALS patients in the U.K. and EU now have access to a first-in-human trial for a novel therapy, potentially expanding treatment options beyond currently available medications for this rapidly progressive disease.
Clinical trialRSSApr 28
Scientists are testing a new drug called RAG-17 for people with a specific type of ALS (a disease that weakens muscles over time) caused by a SOD1 gene mutation. Early results show the drug was safe at different doses and made important disease markers improve. This is early-stage testing, but it's a promising first step.
WHY IT MATTERSIf you have SOD1-ALS, this is one of the first treatments specifically designed for your genetic form of the disease, and early signs suggest it may slow the damage happening in your body.
Clinical trialRSSApr 22
A new drug called masitinib helped ALS patients live longer than expected. In a clinical trial, over 40% of patients treated with masitinib survived for at least 5 years from when their disease started. This is much better than the typical outcome, where fewer than 25% of ALS patients usually live that long.
WHY IT MATTERSIf masitinib gains approval, ALS patients may have access to a treatment that could significantly extend their survival time compared to current standard care.
Clinical trialRSSApr 22
AB Science is preparing to test an experimental drug called masitinib for ALS (a disease that affects nerve cells that control muscles). To protect their investment in this large clinical trial, the company bought special insurance that would reimburse them if the trial doesn't go as planned or costs more than expected.
WHY IT MATTERSThis insurance arrangement signals that AB Science is confident enough in masitinib to move forward with Phase 3 testing in ALS patients, which is a critical step toward potentially bringing a new treatment option to people living with this progressive disease.