Phase 1/2 clinical trial

WVE-004, Wave Life Sciences‘ investigational treatment for amyotrophic lateral sclerosis (ALS), significantly reduces toxic proteins associated with C9orf72 genetic mutations, but that doesn’t seem to translate into functional status gains in patients. Based on findings from the Phase 1b/2a FOCUS-C9 trial (NCT04931862), the company has decided to discontinue developing…

A Phase 1/2 clinical trial testing the investigational imaging agent 18F-OP-801 in healthy volunteers and people with amyotrophic lateral sclerosis (ALS) has enrolled its first patient. Ashvattha Therapeutics‘ agent is designed to detect inflammation in imaging exams like positron emission tomography (PET) or MRI scans. This is possible…

The U.S. Food and Drug Administration (FDA) has granted fast track status to Apic Bio’s experimental therapy APB-102, designed for amyotrophic lateral sclerosis (ALS) patients who carry mutations in the SOD1 gene that lead to misfolded proteins in cells. This designation accelerates the development of investigational therapies that…

A single infusion of AstroRx, an off-the-shelf, investigational cell therapy for amyotrophic lateral sclerosis (ALS), safely and significantly slows disease progression in the first three months following treatment, according to updated data from an ongoing Phase 1/2 trial. These benefits waned after six months, indicating…

Kadimastem has finished treating its second group of participants in a Phase 1/2a clinical trial testing the safety and preliminary efficacy of AstroRx, an investigational stem cell therapy for amyotrophic lateral sclerosis (ALS). AstroRx is an off-the-shelf cell therapy consisting…

Kadimastem’s experimental cell therapy — AstroRx — may slow disease progression in patients with amyotrophic lateral sclerosis (ALS), according to interim results in a first group of patients treated in a Phase 1/2a study. AstroRx consists of functional healthy astrocytes — cells that support and protect neurons…

MediciNova has completed enrollment for its Phase 1/2 clinical trial testing the investigational therapy MN-166 (ibudilast) in amyotrophic lateral sclerosis (ALS) patients. The trial (NCT02714036) has enrolled 35 ALS patients who will be treated with 100 mg daily doses (50 mg twice a day) of MN-166. The…

IONIS-SOD1Rx (BIIB067), an investigational therapy for amyotrophic lateral sclerosis (ALS) developed by Biogen and Ionis Pharmaceuticals, was shown to prolong survival and reverse neuromuscular damage in mouse and rat models…