NervGen Pharma (NGEN) investor relations material

Pipeline overview and program updates

• NVG-291 is advancing as a once-daily, at-home subcutaneous injection for chronic cervical motor incomplete spinal cord injury, with the pivotal phase III RESTORE study now enrolling patients following FDA alignment and initial site activation.

• NVG-291 is positioned as a potential first-in-class therapy for chronic spinal cord injury, with no current FDA-approved drugs for this indication.

• The program has advanced through key milestones, including proof-of-concept efficacy in the CONNECT study, positive Phase 1b/2a CONNECT SCI data, FDA Type C and End-of-Phase 2 meetings, and a NASDAQ listing under ticker NGEN in January 2026.

• NVG-291's mechanism targets CSPG-driven inhibition, with preclinical data supporting potential expansion into other CNS disorders such as stroke, traumatic brain injury, Alzheimer's disease, and multiple sclerosis.

• The company is capitalized with $61.1M as of June 30, 2026, expected to fund operations through the Phase 3 RESTORE topline readout in 1H 2028.

Clinical trial data and development milestones

• Phase I and I-B/II-A CONNECT studies demonstrated NVG-291’s safety, tolerability, and efficacy in improving neural connectivity and function in chronic SCI patients, with no dose-limiting toxicities or serious adverse events.

• CONNECT study met its co-primary endpoint, showing statistically significant improvement in motor evoked potential amplitude in the hand, and functional gains in GRASP quantitative prehension exceeding the minimally important difference.

• NVG-291 demonstrated significant improvements in hand function (GRASSP quantitative prehension), gait recovery, and patient-reported outcomes versus placebo.

• Improvements in hand function, walking, and GRASSP scores were maintained after treatment discontinuation, with patient-reported outcomes and blinded exit interviews confirming meaningful quality-of-life benefits.

• All NVG-291 participants met the composite gait responder criterion, with significant differences in coordination, mechanical effort, and postural stability.

R&D strategy and innovation priorities

• The R&D focus is on targeting chondroitin sulfate proteoglycans (CSPGs) to enable neural regeneration, a mechanism validated across multiple preclinical models and supported by decades of research.

• The Phase 3 RESTORE study is FDA-aligned, focusing on clinically meaningful hand function improvement as the primary endpoint.

• RESTORE retains core design elements from CONNECT SCI, with robust statistical power and rigorous oversight of outcome measures.

• The program integrates patient and caregiver perspectives into clinical development and endpoint selection.

• The company is building a senior leadership team with clinical, regulatory, and financial expertise to execute late-stage development and commercialization.