Phase 1 trial

PharmAust announced that it soon will engage with U.S. Food and Drug Administration (FDA) regulators regarding development plans for monepantel, its investigational therapy for amyotrophic lateral sclerosis (ALS) and motor neuron disease (MND). If the pre-investigational new drug meeting goes well, the company intends to file a…

The biotechnology company ProJenX recently received $15 million in funding to advance its experimental oral treatment prosetin for amyotrophic lateral sclerosis (ALS). This Series A financing round is essentially the next round of funding after seed money was used to rapidly develop the investigational treatment. Both rounds…

A first healthy volunteer has been dosed in a Phase 1 clinical study evaluating how safe and well tolerated is CVN293, a treatment candidate for amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease, the therapy’s developer, Cerevance, announced. The treatment, given orally, is designed to block a protein…

VRG50635, an experimental oral therapy being developed by Verge Genomics for amyotrophic lateral sclerosis (ALS), was found to be safe and well-tolerated in healthy volunteers, new Phase 1 trial data show. Top-line data from the trial (ISRCTN14792372) also showed a promising tolerability and pharmacological profile supporting once-daily…

A recently completed Phase 1 trial studying NPT520-34 — an investigational small molecule being developed by Neuropore Therapies for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS) — has shown the candidate to be safe and well-tolerated in a group of healthy volunteers. The…

AL-S Pharma AG, a biotech company jointly founded and financed by Neurimmune and TVM Capital Life Science, has enrolled the first patient in its Phase 1 clinical trial assessing the safety and tolerability of AP-101, an experimental therapy for amyotrophic lateral sclerosis (ALS). AP-101 is a human…

Injection of human neural stem cells into the spinal cord of people with amyotrophic lateral sclerosis (ALS) was found safe and did not cause adverse effects even two years after the transplant, results from a Phase 1 clinical trial show. Trial findings were published in the study, “Results…

NPT520-34, an investigational small molecule for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS), is being tested on healthy individuals in a new Phase 1 clinical trial, its developer Neuropore Therapies has announced. Brain inflammation is a common feature among many neurodegenerative disorders, including Parkinson’s,…

CuATSM, one of the lead investigational therapies of Collaborative Medicinal Development (CMD), may slow disease progression and improve the respiratory and cognitive function of patients with amyotrophic lateral sclerosis (ALS), a Phase 1 trial shows. The findings of the multicenter, open-label, dose-finding study (NCT02870634) were announced by…

Results from a Phase 1 clinical trial reveal that giving patients infusions of a specialized immune cell may be a viable option to safely slow the progression of amyotrophic lateral sclerosis (ALS). That finding was reported in the study “Expanded autologous regulatory T-lymphocyte infusions in ALS,” published…