US-based biopharmaceutical company Catalyst has begun enrolment for its second Phase III LMS-003 clinical trial of Firdapse to treat Lambert-Eaton myasthenic syndrome (LEMS).
Firdapse is amifampridine phosphate or 3,4-diaminopyridine phosphate (3,4-DAP) that has obtained an orphan drug designation in the US and orphan medicinal product designation in the European Union to treat LEMS.
LEMS is an autoimmune neuromuscular disease resulting in a decrease of the release of acetylcholine at the neuromuscular junction, triggering muscle weakness.
Firdapse acts by increasing acetylcholine release, thereby addressing muscle weakness caused by the disease.
The second Phase III, double-blind, placebo-controlled withdrawal LMS-003 trial will be conducted across clinical trial sites in the US.
The trial will test the safety and efficacy of Firdapse while treating approximately 28 subjects with LEMS.
It will share the same co-primary endpoints with the first Phase III trial, which tested Firdapse for the treatment of LEMS.
Catalyst CEO Patrick McEnany said: "The initiation of our second Phase III study of Firdapse in LEMS patients is a significant step forward on our path towards gaining approval for Firdapse.
"We believe we have a clearly defined development and regulatory pathway for Firdapse in the treatment of LEMS and remain focused on our goal of improving access to treatments for patients with this significant unmet need."
Additionally, the US Food and Drug Administration (FDA) has approved Catalyst to include patients from its expanded access programme as study subjects in this second trial.
The company is planning to re-submit a new drug application (NDA) for its product and is expecting the train results in the second half of next year.