Catalyst Pharmaceuticals has started a Phase II proof-of-concept clinical trial of Firdapse (amifampridine phosphate) being evaluated in patients suffering from spinal muscular atrophy (SMA) Type 3.

Amifampridine is designed to increase transmission at the neuromuscular junction (NMJ) and in this patient population, the drug is expected to preserve the NMJ and motor neurons.

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The adequate, well-controlled, double-blind, two-period, cross-over, outpatient Phase II trial will investigate the safety, tolerability, and efficacy of Firdapse as a symptomatic treatment in ambulatory patients.

Being led by Italy’s Fondazione Istituto Neurologico Carlo Besta researchers Lorenzo Maggi and Giovanni Baranello, the trail will include 12 subjects who will be assessed for primary outcome variable of symptomatic relief.

“We intend to submit an application for orphan drug designation and to pursue a clinical programme required to obtain approval of Firdapse for this indication.”

Catalyst Pharmaceuticals CEO Patrick McEnany said: “We are very pleased that doctors Maggi and Baranello and their team at the Fondazione Istituto Neurologico Carlo Besta have agreed to conduct this study for Catalyst of Firdapse in patients diagnosed with spinal muscular atrophy Type 3.

“If the results from this trial support the safety and efficacy of Firdapse as a treatment for ambulatory patients with SMA Type 3, we intend to submit an application for orphan drug designation and to pursue a clinical programme required to obtain approval of Firdapse for this indication.”

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During the Phase II trial, the firm intends to assess if the increased NMJ function by amifampridine would slow SMA’s nerve degeneration pathology progression.

The firm expects to report top-line data from this trial in the first half of 2019.