NervGen Pharma (NGEN) HCW Home with NervGen Conference summary

Technology platform and mechanism of action

• NVG-291 is a 35-amino acid peptide delivered via subcutaneous injection, designed to neutralize the inhibitory effects of chondroitin sulfate proteoglycans (CSPGs) and promote neuronal regeneration in spinal cord injury (SCI).

• The drug targets chronic motor incomplete cervical SCI, aiming to restore function by enabling remyelination and neuroplasticity without removing CSPGs.

• CSPG upregulation is implicated in other neurotraumas and neurodegenerative disorders, suggesting broader therapeutic potential.

Clinical trial results and patient impact

• The CONNECT trial (phase I-B/IIa) enrolled chronic SCI patients (1–10 years post-injury) and showed statistically significant improvements in motor evoked potentials and fine motor function (GRASSP quantitative prehension).

• Patients receiving NVG-291 had a 3.7-point improvement in GRASSP at 12 weeks versus 0.4 in placebo, with further gains at 16 weeks.

• Two-thirds of participants reported improved bladder control, and 56% saw reduced muscle spasticity; some regained the ability to complete the 10-meter walk test.

• No serious adverse events occurred; mild, sporadic injection site reactions were the only notable side effect.

Phase III RESTORE trial design and regulatory engagement

• RESTORE is a single registrational phase III trial enrolling 150 patients, powered to detect a 2.5-point difference in GRASSP at 12 weeks, with a 1:1 randomization.

• The primary endpoint is functional improvement in hand function (GRASSP), with secondary endpoints including Patient and Clinician Global Impression of Change, SCIM-III, and Modified Ashworth Scale.

• The FDA has been highly collaborative, emphasizing the importance of functional outcomes and patient-centric measures.

• Enrollment is limited to patients 1–10 years post-injury to exclude spontaneous recovery, but broader use is anticipated post-approval.