SPG302 / Spinogenix - LARVOL DELTA

Clinical results of a Phase 2a trial evaluating the synaptogenic small molecule SPG302 in ALS participants (ALS-MND 2025) - P1 | "SPG302 was safe and well tolerated with drug exposure profiles similar to healthy volunteers. Baseline NfL measures showed prediction of outcome, and trends toward improved ECAS scores were observed. During the DBPC period, ALS signature deficits in resting state EEG power were significantly improved with SPG302 compared to placebo and showed a regional correspondence to affected brain regions."

Clinical • P2a data • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Orthopedics • Plasma NfL • TARDBP

Preliminary Phase 2 Data Support Therapeutic Development of Synaptic Regenerative Drug SPG302 in ALS (NeurologyLive) - "In terms of efficacy, preliminary data showed that 82% of SPG302-treated patients demonstrated stable or improved rate of disease progression at the end of treatment, as assessed through the ALS Functional Rating Scale-Revised (ALSFRS-R). When compared with historical controls from the PRO-ACT database (n = 299), patients on the investigational drug exhibited a 76% slower rate of decline through 6 months."

P2a data • Amyotrophic Lateral Sclerosis

Spinogenix Announces Publication of Preclinical Study Demonstrating Neuroprotective Effects of SPG302 in a Model of Glaucoma (PRNewswire) - "In the study, three-month-old mice were treated daily with SPG302 for eight weeks following microbead injection to induce elevated IOP. SPG302 administration significantly improved RGC survival and axonal integrity while preserving visual function as assessed by electroretinography, a clinically translatable measure of retinal function."

Preclinical • Glaucoma

SPG302 protects retinal ganglion cells and preserves visual function by preserving synaptic activity in a mouse model of glaucoma. (PubMed, Exp Eye Res) - "SPG302 treatment effectively preserved synaptic integrity by reversing these changes. These findings highlight the therapeutic potential of SPG302 for protecting RGCs and preserving vision by modulating synaptic activity in glaucomatous neurodegeneration."

Journal • Preclinical • CNS Disorders • Glaucoma • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • DLG4 • SYP

Findings From First Cohort of SPG302 in Phase 2 Alzheimer Disease Trial: Sharron Gargosky, PhD (NeurologyLive) - P2 | N=24 | NCT06427668 | Sponsor: Spinogenix | "SPG302...currently being investigated in a phase 2 trial for its safety and potential efficacy among adult patients with mild-to-moderate Alzheimer disease (AD) in Australia. In the first completed cohort of the study, findings showed that once-daily treatment with SPG302 was safe and well tolerated in patients with AD over the course of 28 weeks, revealing early signs of cognitive improvements. Coauthor Sharron Gargosky, PhD, and colleagues, presented these results at the recently concluded 2025 Alzheimer’s Association International Conference, held July 27-30, in Toronto, Canada....She also discussed the clinically meaningful cognitive improvements reported, with early effects apparent as soon as 1 week and sustained over a 24-week open-label extension. Overall, she highlighted that these results support further development of SPG302 both as a monotherapy and as an adjunctive therapy to standard of care in AD."

P2 data • Alzheimer's Disease

SPG302-SCZ-201: Study of SPG302 in Adults With Schizophrenia (clinicaltrials.gov) - P2 | N=32 | Recruiting | Sponsor: Spinogenix | Trial completion date: Oct 2025 ➔ Feb 2026 | Trial primary completion date: Jun 2025 ➔ Dec 2025

Trial completion date • Trial primary completion date • CNS Disorders • Psychiatry • Schizophrenia

Intermediate-size Patient Population Expanded Access Protocol (clinicaltrials.gov) - P=N/A | N=0 | Available | Sponsor: Spinogenix

New trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Study of SPG302 in Healthy Volunteers and ALS Participants (clinicaltrials.gov) - P1 | N=88 | Completed | Sponsor: Spinogenix | Active, not recruiting ➔ Completed | Trial completion date: Dec 2025 ➔ Jun 2025

Trial completion • Trial completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

Extension Study of Participants From SPG302-ALS-001 (clinicaltrials.gov) - P2 | N=16 | Enrolling by invitation | Sponsor: Spinogenix | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • Amyotrophic Lateral Sclerosis • CNS Disorders

Spinogenix Announces Full Enrollment in its Phase 2 Trial Evaluating SPG302 as a First-in-Class Synaptic Regenerative Therapy for Alzheimer's Disease (PRNewswire) - "Spinogenix...announced full enrollment of its Phase 2 clinical trial in Australia evaluating SPG302 for the treatment of people with Alzheimer's disease (AD)....Spinogenix is completing a randomized, double-blind, placebo-controlled Phase 2 study of SPG302 (NCT06427668) to assess the safety, tolerability, and clinical efficacy of SPG302 in adult participants with mild-to-moderate AD. Two dose cohorts are being evaluated in a total of 24 participants. The first cohort has completed 24 weeks of treatment (inclusive of a double-blind phase and an open label extension period). Results from this cohort will be presented at the Alzheimer's Association International Conference (AAIC) in Toronto on July 27-31, 2025. In parallel, the U.S. Food and Drug Administration (FDA) cleared Spinogenix's Investigational New Drug (IND) application for SPG302 for the treatment of people with AD."

Enrollment closed • IND • P2 data • Alzheimer's Disease

Spinogenix's SPG302, the First Synaptic Regenerative Therapy to Treat ALS, Granted Orphan Drug Designation by the European Medicines Agency (PRNewswire) - "Spinogenix...announced that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) to SPG302 for the treatment of people living with Amyotrophic Lateral Sclerosis (ALS). SPG302 is being developed as the first synaptic regenerative approach for the treatment of ALS, with the potential to restore function in cognition, movement, and respiration."

Orphan drug • Amyotrophic Lateral Sclerosis

Spinogenix Announces FDA-Authorized Expanded Access Program for SPG302, the First Synaptic Regenerative Therapy to Treat ALS (PRNewswire) - "Spinogenix, Inc...today announced that it has received notification from the Food and Drug Administration (FDA) to begin its Expanded Access Program (EAP) for SPG302 in the United States (U.S.) for people living with Amyotrophic Lateral Sclerosis (ALS) that meet program eligibility criteria....Spinogenix recently completed a Phase 2 ALS trial in Australia, and patients have been offered a continuation of treatment in an open label extension."

FDA event • Trial status • Amyotrophic Lateral Sclerosis

Spinogenix Announces Open Enrollment for Phase 2 US Trial Following FDA Clearance of IND Application for SPG302, the First Synaptic Regenerative Therapy for Schizophrenia (PRNewswire) - "Spinogenix, Inc...announced open enrollment for its Phase 2 trial of SPG302 for the treatment of people with schizophrenia, following clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). The U.S.-based trial evaluating the efficacy, safety, and tolerability of once-daily dosing of SPG302 expands on trials underway in Australia."

Enrollment open • IND • Schizophrenia

Extension Study of Participants From SPG302-ALS-101 (clinicaltrials.gov) - P2 | N=16 | Not yet recruiting | Sponsor: Spinogenix

New P2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Extension Study of Participants From SPG302-ALZ-101 (clinicaltrials.gov) - P2 | N=24 | Enrolling by invitation | Sponsor: Spinogenix | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • Alzheimer's Disease • CNS Disorders

Extension Study of Participants From SPG302-ALZ-101 (clinicaltrials.gov) - P2 | N=24 | Not yet recruiting | Sponsor: Spinogenix

New P2 trial • Alzheimer's Disease • CNS Disorders

Study of SPG302 in Healthy Volunteers and ALS Participants (clinicaltrials.gov) - P1 | N=112 | Active, not recruiting | Sponsor: Spinogenix | Recruiting ➔ Active, not recruiting

Enrollment closed • Amyotrophic Lateral Sclerosis • CNS Disorders

Spinogenix Announces Launch of a Phase 2 Clinical Trial Evaluating SPG302 for the Treatment of Schizophrenia (GlobeNewswire) - "Spinogenix, Inc...announced that with the approval of the Australia Human Research Ethics Committee (HREC), multiple sites are currently enrolling participants in a Phase 2 clinical trial to evaluate SPG302 as a novel treatment for schizophrenia."

Trial status • Schizophrenia

SPG302-SCZ-201: Study of SPG302 in Adults With Schizophrenia (clinicaltrials.gov) - P2 | N=32 | Recruiting | Sponsor: Spinogenix | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Psychiatry • Schizophrenia

SPG302-ALZ-101: Study of SPG302 in Adult Participants With Mild-to-Moderate Alzheimer's Disease (AD) (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Spinogenix | Not yet recruiting ➔ Recruiting

Enrollment open • Alzheimer's Disease • CNS Disorders

Spinogenix Announces Open Enrollment for Phase 2 Study Evaluating SPG302 for the Treatment of Alzheimer’s Disease (GlobeNewswire) - "Spinogenix...announced that enrollment is now open in Australia for their Phase 2 clinical trial evaluating SPG302 for the treatment of people with Alzheimer’s disease (AD), the most prevalent form of dementia worldwide....The Phase 2 trial will evaluate the safety, tolerability, clinical efficacy, pharmacokinetics, and pharmacodynamics of a once-a day pill of SPG302 in adult participants with mild-to-moderate Alzheimer’s disease. Enrollment is currently open at St. Vincents Hospital, Sydney, NSW, Australia with plans for a second site opening soon at Flinders Medical Center, Adelaide, SA, Australia."

Enrollment open • Alzheimer's Disease

Spinogenix Announces Approval from the Australia Human Research Ethics Committee to Initiate a Phase 2 Human Clinical Trial of SPG302 for the Treatment of Alzheimer’s Disease (GlobeNewswire) - "Spinogenix...announced that the initiation of Spinogenix’s Phase 2 trial to evaluate SPG302 for the treatment of adult participants with mild-to-moderate Alzheimer’s disease (AD) was approved in Australia. The Phase 2, multicenter study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical efficacy of SPG302 in adult AD participants. The clinical trial will consist of two parts: a pilot, placebo-controlled, randomized safety and preliminary efficacy cohort (Part A) and an expansion cohort (Part B)....SPG302 has completed the Phase 1 safety study in healthy subjects in Australia and is currently being evaluated in ALS patients in Australia, with plans to enroll patients in the U.S. following recent FDA IND clearance."

Trial status • Alzheimer's Disease • Amyotrophic Lateral Sclerosis

Study of SPG302 in Healthy Volunteers and ALS Participants (clinicaltrials.gov) - P1 | N=112 | Recruiting | Sponsor: Spinogenix | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: May 2024 ➔ May 2025

Trial completion date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

Spinogenix Announces FDA Clearance of IND Application for SPG302, a Novel Therapy for the Treatment of ALS (GlobeNewswire) - "Spinogenix, Inc...announced that the U.S. Food and Drug Administration (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."

IND • Trial status • Amyotrophic Lateral Sclerosis • CNS Disorders

Study of SPG302 in Adults With Schizophrenia (clinicaltrials.gov) - P2 | N=32 | Not yet recruiting | Sponsor: Spinogenix

New P2 trial • CNS Disorders • Psychiatry • Schizophrenia