Author Archives: Patricia Inacio PhD

Amylyx to Seek AMX0035’s Approval in Europe

Amylyx Pharmaceuticals is planning to file an application by the end of this year requesting that the European Medicines Agency (EMA) approve AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS). This follows a recent announcement that a similar application will be submitted to Health…

First Patient Enrolled in Phase 2a Trial of Engensis

The first patient has been enrolled in a Phase 2a clinical trial investigating Helixmith’s gene therapy Engensis (VM202) for the treatment of amyotrophic lateral sclerosis (ALS), the company announced. The REViVALS-1A study (NCT04632225) intends to recruit 18 patients across four clinical sites in the U.S. Enrollment is open…

Dosing Begins in Phase 2 Study of Pegcetacoplan in ALS Patients

A first patient has been dosed in the Phase 2 trial investigating pegcetacoplan (APL-2), Apellis Pharmaceuticals‘ candidate therapy for amyotrophic lateral sclerosis (ALS), the company and the Swedish Orphan Biovitrum (Sobi) announced. The potentially pivotal trial, called MERIDIAN (NCT04579666), is currently enrolling patients…

Scribe, Biogen Partner to Develop Gene Therapy for ALS, Other Neurological Diseases

Scribe Therapeutics and Biogen, a biotechnology company focused on neurological diseases, have established a research partnership to develop new gene therapies for amyotrophic lateral sclerosis (ALS) using the gene-editing technology CRISPR-Cas9. The collaboration will focus initially on the development and commercialization of CRISPR-based therapies that address some…

Phase 3 Study of Oral Edaravone for ALS Resumes Patient Enrollment

The Phase 3 clinical trial testing an oral suspension formulation of edaravone for amyotrophic lateral sclerosis (ALS) has resumed dosing and patient recruitment, its developer, Mitsubishi Tanabe Pharma America (MTPA), announced. The trial (NCT04165824) was launched in November, but was paused in March due to the COVID-19 pandemic. The study,…