Author Archives: Marta Figueiredo PhD

Phase 1b Trial of Oral DNL343 in ALS Patients Begins Dosing

A Phase 1b clinical trial evaluating the safety and pharmacological profile of DNL343, Denali Therapeutics’ investigational oral therapy, in adults with amyotrophic lateral sclerosis (ALS) has started dosing participants. The study (NCT05006352) is currently recruiting patients at the Centre for Human Drug Research (CHDR) in Leiden, Netherlands.

Amylyx Plans to File for Approval of AMX0035 in US

Amylyx Pharmaceuticals plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) in the coming months for approval of its investigational oral therapy AMX0035 for amyotrophic lateral sclerosis (ALS). The decision follows recent discussions with the FDA, including a pre-NDA meeting held…

Subtle Changes in MicroRNA-218 May Fuel ALS

Dropping the levels of microRNA-218 (miR-218) — a small molecule that regulates the activity of other genes — below a certain threshold leads to severe motor neuron damage, paralysis, and death in a mouse model of amyotrophic lateral sclerosis (ALS), a study shows. The findings, combined with the fact that…

Masitinib Helps Keep Inflammatory Mast Cells From Spinal Cord

Mast cells, a type of immune cell, infiltrate the spinal cord of amyotrophic lateral sclerosis (ALS) patients and rodent models of the disease, exhibiting pro-inflammatory features that are likely involved in motor neuron and microvasculature damage, a study suggests. Notably, treating these mice models with masitinib — an experimental oral…

Phase 3 Trial of Ultomiris in ALS Patients Stopping for Lack of Efficacy

Alexion Pharmaceuticals is stopping further work on Ultomiris (ravulizumab) — its approved therapy for complement-associated rare blood disorders — as a potential treatment of amyotrophic lateral sclerosis (ALS) based on interim data from the CHAMPION-ALS Phase 3 clinical trial. While top-line results were expected by mid-2022, the study’s independent data…

COMBAT-ALS Trial Still Enrolling in North America

MediciNova is still recruiting adults with early-stage amyotrophic lateral sclerosis (ALS) for the COMBAT-ALS Phase 2b/3 clinical trial evaluating its investigational oral therapy MN-166 (ibudilast). Patients are being enrolled at 19 sites across North America; more information about contacts and locations is available here. “We continue to…

Radicava Prolongs Survival, Small ALS Study Shows

Long-term treatment with Radicava (edaravone) is generally safe and nearly doubles survival time in adults with amyotrophic lateral sclerosis (ALS), according to a small real-life study in Japan. These data add to increasing, but still limited, evidence showing the therapy prolongs the life of ALS patients and…

TP73 Gene Mutations May Be Risk Factor for Sporadic ALS

Rare mutations in the TP73 gene, which is involved in the regulation of a cell’s life cycle, may put a person at greater risk of amyotrophic lateral sclerosis (ALS), according to a genetic analysis of nearly 2,900 sporadic ALS patients. Such mutations were found to affect the maturation and survival of…

New Israeli Production Site for NurOwn Certified for Quality

BrainStorm Cell Therapeutics announced that a new cleanroom facility for manufacturing NurOwn, its experimental cell-based therapy for amyotrophic lateral sclerosis (ALS), has received good manufacturing practice (GMP) certification from the Israel Ministry of Health. GMPs are standards set to ensure that batches of a medicine are produced with consistent high…