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 committee of the European Medicines Agency (EMA) is leaning toward not recommending a conditional approval of Amylyx Pharmaceuticals’ AMX0035 for treating amyotrophic lateral sclerosis (ALS) in the European Union. The drug is already marketed in the U.S. under the name Relyvrio (sodium phenylbutyrate and taurursodiol) and…
A new technology powered by artificial intelligence (AI) may one day help doctors diagnose people in the early stages of amyotrophic lateral sclerosis (ALS), according to data from a clinical trial. Developed by Pison, the technology was able to detect involuntary muscle twitches, or fasciculations, and changes in…
There are currently up to 18,000 people living with amyotrophic lateral sclerosis (ALS) in the U.S., with national costs likely to reach $1.02 billion each year, a study has found. Researchers also observed that men, white people, and veterans appear more likely to develop the disease. “However, large-scale, population-based…
Enrollment is still ongoing for a Phase 2 clinical trial testing the safety and efficacy of Corcept Therapeutics‘ cortisol modulator, called dazucorilant, in people with amyotrophic lateral sclerosis (ALS). The DAZALS trial (NCT05407324), launched in November 2022, is expected to enroll an estimated 198 adults with…
Pridopidine, an oral small molecule, failed to meet its primary goal of improving physical function across amyotrophic lateral sclerosis (ALS) patients who took part in a Phase 2/3 clinical trial. Changes in secondary endpoints measuring muscle strength and respiratory function also did not significantly differ between people taking pridopidine…
Kadimastem’s experimental cell-based therapy AstroRx was safe and significantly slowed disease progression in the first three months among people with amyotrophic lateral sclerosis (ALS), a Phase 1/2 clinical study shows. The findings are in line with initial data released by the company in 2020, with AstroRx more…
A new formulation of MediciNova’s investigational amyotrophic lateral sclerosis treatment ibudilast (MN-166) given via an intravenous (into the vein) infusion was found to be safe and well tolerated in healthy volunteers. That’s according to data from a recently completed Phase 1 clinical trial that tested the safety and…
ANX005, an experimental medication being developed by Annexon Biosciences for neurodegenerative and autoimmune diseases, resulted in a slowing of disease progression in adults with amyotrophic lateral sclerosis (ALS). That’s according to preliminary data from eight patients taking part in a Phase 2a clinical study (NCT04569435), whose main…
Masitinib, an oral medicine developed by AB Science, has won orphan drug status for treating amyotrophic lateral sclerosis (ALS) in Switzerland. The status is granted by the Swiss Agency for Therapeutic Products (Swissmedic) to therapies that seek to treat life-threatening or chronic diseases affecting no more than…
