Seneca Biopharma, Germantown, Md., issued a press release announcing its recent meeting with the FDA to discuss elements of a final design and protocol for a Phase 3 trial investigating NSI-566, its stem cell therapy for amyotrophic lateral sclerosis (ALS).
NSI-566 is Seneca’s lead neural stem cell candidate for the treatment of ALS. Relying on its feedback from the FDA, the company believes that its Phase 1 and 2 trials (NCT01348451) and (NCT01730716) fully support conducting a phase 3 study of NSI-566.
Dr. David Recker, Seneca’s Chief Medical Officer, said the company received excellent guidance from the FDA towards trial design. Seneca is now finalizing protocol for the FDA’s review prior to the onset of its Phase 3 trial.
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a rare and fatal disorder causing paralysis that leads to eventual respiratory failure. It is the most common type of motor neuron disease.
Approximately six thousand cases are diagnosed each year in the United states. A report by the CDC in 2018 estimated that sixteen to seventeen thousand people were diagnosed with ALS in the United States.
About NSI-566
The investigational therapy NSI-566 is a neural stem cell therapy using human spinal cord stem cells. These stem cells have the ability to become specialized cells during their development.
Upon injection into the spinal cord, the NSI-566 cells are transformed into neurons that support the motor neurons which are damaged by ALS. They integrate into the patient’s neurons and form synapses (connections).
In addition, the integrated neurons secrete molecules found to activate motor neuron survival and growth.
The FDA had previously granted orphan drug status to NSI-566. The grant gives Seneca exclusivity for a specified period plus financial incentives for its clinical trial investigations.
The Phase 1 and Phase 2 trials
NSI-566 demonstrated encouraging results in both Phase 1 and 2 trials when tested for efficacy, safety and maximum tolerated dose. Patients will continue to participate in the follow-up portion of the trials where they will be monitored for up to twenty-four months.
A Comparison of Trial Data
As a comparison, researchers referred to ALS patients’ outcome and survival from three years of previous ALS trial data. A rating scale using questionnaires and a statistical analysis were other measurements used for comparison.
At the two-year mark, people who received NSI-566 transplants had substantially higher scores than those in the two rating groups. However, there was minimal difference in survival between the groups.
Seneca Biopharma’s Company Profile
As a biopharmaceutical company, Seneca focuses on developing treatments for disorders that have a high unmet medical need. Seneca is developing NSI-566 to treat paralysis in ALS, chronic spinal cord injury, and stroke.
