Spinogenix Announces FDA Clearance of IND Application Novel Therapy for the Treatment of ALS

Spinogenix, Inc. recently announced the US FDA has cleared the Investigational New Drug (IND) application for its Phase 1/2 clinical trial of SPG302 for the treatment of people with Amyotrophic Lateral Sclerosis (ALS). The trial will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of SPG302 dosed as a once-a-day pill in ALS patients.

“Having completed the Phase 1 safety study in healthy subjects in Australia, we are thrilled to have gained FDA acceptance of our US IND for SPG302 in ALS,” said Stella Sarraf, PhD, Spinogenix Chief Executive Officer and Founder. “SPG302’s unique approach to regenerate synapses offers a fundamentally different treatment modality, focusing on synapse loss, which is central to ALS. Current treatments have not sufficiently met the needs of ALS patients, as slowing disease progression alone is not enough. We are committed to advancing SPG302 with the hope of providing a new, transformative therapeutic that can significantly improve the lives of those battling this devastating disease.”

SPG302 has been granted US FDA Orphan Drug Designation (ODD) for the treatment of ALS and has received preclinical support from the US National Institutes of Health (NIH) and the Department of Defense (DoD). SPG302 is currently undergoing a Phase 1/2 study in Australia, which has completed dosing of healthy volunteer cohorts, showing dose proportionality, excellent tolerability and plasma levels aligned with efficacy in animal models. Dosing of ALS patients in Australia began in April 2024. Additional information on the trial may be found on ClinicalTrials.gov (NCT05882695).

Dr. Merit Cudkowicz, Chair of the Massachusetts General Hospital Department of Neurology, Director, Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, Julieanne Dorn Professor of Neurology at Harvard Medical School, and Spinogenix Advisory Board member, added “ALS is a complex and varied disease, affecting cognitive and motor functions as well as speech and respiration. Spinogenix’s new approach works at the synaptic level to regenerate synapses. This first study in people with ALS is an important step towards determining whether SPG302 helps recover lost functions in motor and cognitive symptom domains.”

SPG302 is a once-a-day pill being developed as a regenerative treatment for ALS and other neurodegenerative diseases that has the unique ability restore synapses, the key connections between neurons that allow people to think, plan, remember, and control motor functions. The synaptic regenerative activity of SPG302 represents a first-in-class approach to treating ALS and has the potential to reverse declines in cognitive, respiratory, and motor function.

ALS, also called Lou Gehrig’s disease, is a neurodegenerative disease that results in the progressive loss of motor neurons that control voluntary muscles. ALS affects as many as 30,000 people in the United States, with approximately 5,000 new cases diagnosed each year. ALS is the most common form of motor neuron diseases, characterized by progressive paralysis and, almost invariably, death by respiratory failure within 2-5 years of diagnosis. Around half of people with ALS develop at least mild difficulties with thinking and behavior, and about 15% develop frontotemporal dementia. There is no known cure for ALS.

Spinogenix is dedicated to developing transformative therapeutics for conditions involving the loss or dysfunction of synapses. Our lead clinical-stage synaptic regenerative candidate is a first-in-class therapeutic designed to reverse synapse loss and improve cognitive and motor functions in neurodegenerative and neuropsychiatric diseases such as ALS, Alzheimer’s disease, and schizophrenia. In parallel, we are also developing a synaptic function therapeutic designed to improve behavior in Fragile X Syndrome. For more information, visit www.spinogenix.com.