Catalyst Pharmaceuticals is launching a proof-of-concept Phase 2 trial of its investigational drug Firdapse (amifampridine phosphate), intended to relieve symptoms in ambulatory patients with spinal muscular atrophy (SMA) type 3.
If the treatment proves effective and safe in this group, which will include about 12 patients, Catalyst plans to continue the development of the drug while seeking orphan drug status with regulatory authorities — a designation that makes the development cheaper and faster.
The study will be led by Drs. Lorenzo Maggi and Giovanni Baranello at the Fondazione Istituto Neurologico Carlo Besta, a well-known referral center for SMA patients in Milan, Italy.
“We are very pleased that Drs. 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,” Patrick J. McEnany, CEO of Catalyst, said in a press release.
The trial will randomly assign patients to Firdapse or a control treatment. To get more robust results, patients will then switch treatments. Researchers will evaluate not only the safety of the treatment, but also its ability to improve symptoms.
Firdapse is a drug acting at the so-called neuromuscular junction — the spot at which a motor neuron connects to the muscle — by improving the signaling between a nerve and muscle.
Studies in both animal models and SMA patients show there are abnormalities in the neuromuscular junctions, seen before nerves start to degenerate and patients start developing symptoms. In fact, researchers believe that the changes in the nerve-muscle connection may contribute to the neurodegeneration.
Researchers have observed neuromuscular junction abnormalities in patients with SMA type 3 — a factor that contributed to Catalyst’s choice to focus on this patient group.
In addition to improving symptoms, researchers hope that the improved signaling at the neuromuscular junction may slow down the progression of neurodegeneration.
The trial will run through the first half of 2019.
“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 program required to obtain approval of Firdapse for this indication,” McEnany said.