Spinogenix Gets FDA IND Clearance for ALS Therapy SPG302

Spinogenix, Inc., a clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics that restore synapses, announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for its Phase 1/2 clinical trial of SPG302. This trial aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of SPG302 in patients with Amyotrophic Lateral Sclerosis (ALS). SPG302 is being developed as a once-a-day pill to potentially regenerate synapses and reverse declines in cognitive and motor functions in ALS patients.

Spinogenix’s CEO and Founder, Dr. Stella Sarraf, expressed excitement over the FDA's acceptance of the IND application for SPG302 in the U.S. She emphasized that SPG302 offers a novel approach by focusing on synapse regeneration, which is crucial for treating ALS. Current therapies have not sufficiently addressed the needs of ALS patients, as slowing disease progression alone is inadequate. By advancing SPG302, Spinogenix aims to provide a transformative treatment that could significantly enhance the lives of those suffering from this debilitating disease.

SPG302 has already received Orphan Drug Designation (ODD) from the FDA for the treatment of ALS and has garnered preclinical support from the U.S. National Institutes of Health (NIH) and the Department of Defense (DoD). A Phase 1/2 study is currently underway in Australia, where dosing of healthy volunteer cohorts has been completed, demonstrating dose proportionality, excellent tolerability, and plasma levels consistent with efficacy observed in animal models. Dosing of ALS patients in Australia began in April 2024. More details about the trial can be found on ClinicalTrials.gov under the identifier NCT05882695.

Dr. Merit Cudkowicz, Chair of the Massachusetts General Hospital Department of Neurology and Director of the Sean M. Healey & AMG Center for ALS, highlighted the complexities of ALS, which affects cognitive and motor functions, as well as speech and respiration. Dr. Cudkowicz pointed out that Spinogenix's approach targets the synaptic level to regenerate synapses, making this first study in ALS patients a critical step in evaluating whether SPG302 can help recover lost functions in various symptom domains.

SPG302 stands out as a once-a-day pill developed for ALS and other neurodegenerative conditions with its unique capacity to restore synapses—key connections between neurons that facilitate thinking, planning, memory, and motor control. This synaptic regenerative activity represents a pioneering approach to treating ALS and holds promise for reversing cognitive, respiratory, and motor function declines.

Amyotrophic Lateral Sclerosis (ALS), commonly known as Lou Gehrig’s disease, is a neurodegenerative condition that leads to the progressive loss of motor neurons controlling voluntary muscles. In the United States, ALS affects approximately 30,000 individuals, with about 5,000 new cases diagnosed annually. ALS is the most prevalent motor neuron disease, marked by progressive paralysis and nearly always resulting in death from respiratory failure within two to five years of diagnosis. Around half of ALS patients experience cognitive and behavioral difficulties, with about 15% developing frontotemporal dementia. Currently, there is no known cure for ALS.

Spinogenix focuses on creating transformative treatments for conditions involving synapse loss or dysfunction. Their lead clinical-stage synaptic regenerative candidate, SPG302, is designed to reverse synapse loss and enhance cognitive and motor functions in neurodegenerative and neuropsychiatric diseases such as ALS, Alzheimer’s disease, and schizophrenia. Additionally, Spinogenix is developing a therapeutic to improve behavior in Fragile X Syndrome.

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