The pivotal Phase 3 clinical trial testing BrainStorm Cell Therapeutic’s cell-based therapy NurOwn in amyotrophic lateral sclerosis (ALS) patients continues with only occasional scheduling changes to treatment due to the COVID-19 pandemic. Patient dosing is expected to be complete by July, the company announced. “BrainStorm made…
Tikomed has secured $5.5 million in financing from investors to support the ongoing clinical development of ILB, its lead therapy candidate for degenerative neurological diseases, including amyotrophic lateral sclerosis (ALS). The funding also will help develop Ibsolvmir, a therapy to increase the effectiveness of transplanting cells to treat patients…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
The European Medicines Agency (EMA) granted orphan drug status to Cytokinetics‘ reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS), the company announced. The move follows fast track designation granted in December by the U.S. Food and Drug Administration for the same indication, and provides the…
In the coming months, Alexion Pharmaceuticals will launch a pivotal Phase 3 clinical trial to test its complement system inhibitor Ultomiris (ravulizumab) in people with amyotrophic lateral sclerosis (ALS) whose motor symptoms began in the prior three years. The CHAMPION-ALS trial — which follows an investigational new…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Cytokinetics and Astellas Pharma‘s reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS), the companies announced. Reldesemtiv, a fast skeletal muscle troponin activator designed to improve muscle function with minimal nerve…
The U.S. Food and Drug Administration has agreed to expand Tiglutik, a liquid oral form of riluzole, to include giving it via a feeding tube to treat amyotrophic lateral sclerosis (ALS), ITF Pharma, the company marketing Tiglutik, announced. The approval of administering the therapy via percutaneous endoscopic…
Anelixis Therapeutics has completed its Phase 1 clinical trial testing AT-1501, an investigational antibody, in healthy volunteers and eight amyotrophic lateral sclerosis (ALS) patients. The company also announced it has secured the funds to proceed to a Phase 2 clinical trial of AT-1501. The results of the Phase…
Kadimastem plans to submit an amendment to the protocol of its ongoing Phase 1/2a trial in Israel assessing the use of AstroRx, an “off-the-shelf” stem cell therapy, for the treatment of amyotrophic lateral sclerosis (ALS). The company wants to change the planned dosage in one of the…
ProMIS Neurosciences is advancing its program for amyotrophic lateral sclerosis (ALS) by generating antibodies that specifically target the toxic TDP-43 clumps — a hallmark of the condition. The antibody candidates may work as therapies for both ALS and frontotemporal dementia (FTD), the company says. As a result…
Get regular updates to your inbox.