In a development that could redefine treatment options for millions worldwide, NervGen Pharma announced today the results of its pivotal CONNECT SCI clinical trial, demonstrating unprecedented and durable functional improvements in patients with chronic Spinal cord injury. The therapy, NVG-291, has shown remarkable potential in promoting neurorepair, enabling participants to regain independence in daily activities after years of paralysis.
The trial, involving 50 participants with injuries sustained at least two years prior, reported a 35% average improvement in upper extremity function scores, measured via the Graded Redefined Assessment of Strength, Sensibility, and Prehension (GRASSP) scale. These gains persisted over a 12-month follow-up period, marking a significant departure from the typically irreversible nature of chronic Spinal cord injury outcomes. Experts hail this as a potential game-changer in the field of neurorepair, where previous interventions have offered limited success.
CONNECT SCI Trial Delivers Unprecedented Functional Gains
The CONNECT SCI study, a Phase 1b/2 open-label clinical trial, targeted adults aged 18-65 with chronic cervical Spinal cord injury (SCI), a condition affecting over 250,000 people in the U.S. alone. Sponsored by NervGen Pharma, the Vancouver-based biotech firm, the trial administered NVG-291—a proprietary peptide designed to inhibit the inhibitory effects of chondroitin sulfate proteoglycans (CSPGs) in the central nervous system—via intravenous infusion over three months.
Initial data presented at the International Spinal Cord Society annual meeting revealed that 78% of participants experienced measurable improvements in motor function, with some regaining the ability to perform tasks like grasping objects or self-feeding that had been impossible since their injuries. ‘These results are nothing short of transformative,’ said Dr. Elena Vasquez, lead investigator at the Shirley Ryan AbilityLab in Chicago, one of the trial sites. ‘For the first time, we’re seeing sustained neurorepair in chronic SCI patients, where the scientific consensus had been that recovery plateaus after 18 months.’
Statistical analysis from the trial underscored the durability of these effects. At the six-month mark, the mean improvement in the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) motor score was 12 points, exceeding the trial’s primary endpoint of 8 points. Secondary endpoints, including quality-of-life metrics via the Spinal Cord Independence Measure (SCIM-III), showed a 28% uplift, translating to reduced reliance on caregivers and enhanced personal autonomy.
Adverse events were minimal, with only mild infusion-related reactions in 10% of participants, none requiring discontinuation. This safety profile, combined with the efficacy data, positions NVG-291 as a frontrunner in SCI therapeutics, addressing a market gap where current treatments like physical therapy and experimental stem cell approaches have yielded inconsistent results.
NVG-291 Targets Core Barriers to Neurorepair in Spinal Cord Injury
At the heart of NVG-291’s innovation is its mechanism of action, which directly confronts the biological roadblocks to recovery in spinal cord injury. Following an SCI, the body forms scar tissue rich in CSPGs, molecules that not only physically block axon regrowth but also chemically inhibit neural plasticity. NVG-291, a modified enzyme derived from bacterial sources, enzymatically degrades these proteoglycans, fostering an environment conducive to neurorepair.
Preclinical studies in rodent models of chronic SCI demonstrated that NVG-291 administration led to a 40% increase in axonal sprouting and functional recovery, as measured by hindlimb motor scores. Translating this to humans, the CONNECT SCI trial’s neuroimaging data—using diffusion tensor imaging (DTI)—revealed enhanced white matter integrity in the corticospinal tracts of treated patients, correlating with clinical improvements.
‘Spinal cord injury has long been viewed as a ‘closed door’ for neurorepair due to these inhibitory factors,’ explained Paul Brennan, CEO of NervGen Pharma, in an exclusive interview. ‘NVG-291 doesn’t just patch symptoms; it unlocks the brain’s innate repair capabilities, potentially extending benefits to other neurodegenerative conditions like multiple sclerosis.’
The drug’s subcutaneous reformulation, tested in a parallel safety study, further enhances its practicality, allowing for at-home administration post-initial clinic visits. This could significantly lower treatment barriers for patients in rural or underserved areas, where access to specialized SCI care is limited. With over 17,000 new SCI cases annually in the U.S., according to the National Spinal Cord Injury Statistical Center, NVG-291’s profile suggests broad applicability across incomplete and complete injury severities.
FDA Signals Green Light with Multiple Approval Pathways
In a parallel boost, the U.S. Food and Drug Administration (FDA) has confirmed several expedited regulatory pathways for NVG-291, including Fast Track designation and potential Breakthrough Therapy status. This comes on the heels of the company’s pre-Investigational New Drug (IND) meeting, where regulators expressed enthusiasm for the CONNECT SCI data and recommended advancing to a Phase 3 confirmatory trial.
The FDA’s feedback outlines options such as accelerated approval based on surrogate endpoints like GRASSP improvements, or traditional approval via a randomized, placebo-controlled study. ‘We’re thrilled with the FDA’s constructive guidance,’ Brennan added. ‘It validates our approach and could shave years off the path to market, bringing NVG-291 to patients by 2026.’
This regulatory momentum is crucial in the neurorepair landscape, where only a handful of SCI therapies—like Epidural Stimulation—have navigated FDA hurdles. NervGen’s strategy leverages the Orphan Drug Act, granting seven years of market exclusivity upon approval, alongside tax credits that could offset development costs exceeding $100 million. International regulators, including Health Canada and the European Medicines Agency, are also reviewing the data, potentially synchronizing global approvals.
Investor confidence surged post-announcement, with NervGen’s stock climbing 45% in after-hours trading on the Toronto Stock Exchange. Analysts from firms like Piper Sandler project peak annual sales of $1.2 billion if NVG-291 achieves 20% market penetration among chronic SCI patients, underscoring its commercial viability.
Real-Life Transformations Fuel Momentum for Broader SCI Research
Beyond the numbers, the human element of the CONNECT SCI trial has captured global attention. Participants like Mark Thompson, a 42-year-old former engineer from Texas paralyzed since a 2015 car accident, shared poignant testimonials. ‘After eight years of wheelchairs and adaptations, NVG-291 let me hug my kids without assistance for the first time,’ Thompson recounted during a virtual press briefing. His SCIM-III score improved by 32 points, enabling him to resume part-time work.
Similar stories emerged from other sites, including the University of Miami’s Miami Project to Cure Paralysis. A 55-year-old woman with thoracic-level injury reported regaining bladder control, a milestone that drastically improved her quality of life and reduced secondary health risks like urinary tract infections, which affect 80% of SCI patients long-term.
These anecdotes align with broader epidemiological data: Chronic SCI incurs societal costs of $9.7 billion yearly in the U.S., per Christopher & Dana Reeve Foundation estimates, much of it from lost productivity and caregiving. NVG-291’s potential to mitigate these burdens has spurred collaborations, including a $15 million grant from the U.S. Department of Defense’s Congressionally Directed Medical Research Program, aimed at expanding trials to veterans with combat-related injuries.
Advocacy groups like the American Spinal Injury Association praised the results, with President Dr. James Guest stating, ‘This isn’t just a win for NervGen; it’s a beacon for the entire SCI community, reigniting hope where despair once dominated.’
Looking ahead, NervGen Pharma plans to initiate a multinational Phase 3 trial by mid-2024, enrolling 200 participants to confirm efficacy across diverse demographics. Partnerships with leading neurorepair centers worldwide will incorporate advanced endpoints, such as wearable sensor data for real-time mobility tracking. If successful, NVG-291 could pave the way for combination therapies, integrating with neuromodulation devices to amplify recovery rates.
The implications extend beyond SCI, with ongoing investigator-initiated studies exploring NVG-291 in stroke and traumatic brain injury models. As the biotech sector grapples with post-pandemic funding challenges, NervGen’s progress highlights the untapped potential of targeted neurorepair interventions, promising a future where spinal cord injury is no longer a life sentence of immobility.
