NervGen Receives FDA Breakthrough Therapy Designation for NVG-291 in Chronic Spinal Cord Injury

Preliminary clinical evidence showed functional hand-use improvements, with no serious adverse events or treatment discontinuations.

Rhea-AI Summary

NervGen (NASDAQ: NGEN) received FDA Breakthrough Therapy designation for NVG-291 to treat chronic traumatic cervical spinal cord injury with incomplete motor function. The designation followed review of safety and efficacy data from the chronic cohort of the Phase 1b/2a CONNECT SCI study. Preliminary evidence showed improvements on a clinically significant endpoint measuring functional hand use. Participant assessments of change and blinded interviews supported gains in everyday function and independence compared with placebo. NVG-291 was generally well tolerated, with no serious adverse events or treatment discontinuations.

The Phase 3 RESTORE registrational study is enrolling participants, with site activation continuing in the United States and Canada. The designation supports expedited development and review, including eligibility for Priority Review and rolling submission of sections of a New Drug Application.

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• Moderate pointNVG-291 received FDA Breakthrough Therapy designation for chronic traumatic cervical motor-incomplete spinal cord injury.
• Moderate pointPreliminary CONNECT SCI evidence showed functional hand-use improvements on a clinically significant endpoint.
• Moderate pointPhase 3 RESTORE is enrolling, with site activation continuing in the United States and Canada.
• Minor pointParticipant assessments and blinded interviews supported everyday function and independence gains compared with placebo.
• Minor pointNVG-291 was generally well tolerated, with no serious adverse events or treatment discontinuations.
• Minor pointDesignation benefits include Priority Review eligibility, rolling application submission and FDA support for an efficient approval route.

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• None.

Historical Context

1 past event · Latest: May 26

1 event

1. May 26

   Clinical trial results

   24h Move

   +2.4%

   CONNECT SCI gait analyses reported statistically significant improvements versus placebo.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

breakthrough therapy designation, patient global impression of change, placebo, priority review, +1 more

5 terms

breakthrough therapy designation regulatory

"Breakthrough Therapy designation granted based on FDA’s review"

A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.

patient global impression of change medical

"Patient Global Impression of Change (PGIC)"

A patient global impression of change is a simple, patient-reported rating that asks whether a person’s overall health or symptoms have gotten better, worse, or stayed the same after treatment. For investors, it matters because this kind of direct feedback can influence regulators’ and doctors’ views of a therapy’s real-world benefit and therefore affect approval prospects, prescribing behavior and market acceptance — think of it as customer satisfaction for a medical treatment.

placebo medical

"participant-reported gains in everyday function and independence compared to placebo"

A placebo is an inactive pill, injection or procedure that looks and feels like the real treatment but contains no therapeutic ingredient, often called a sugar pill. Investors care because comparing a drug to a placebo reveals whether observed benefits come from the medicine itself or from expectation; clear superiority over placebo reduces regulatory and commercial risk, much like a blind taste test proves a new recipe really tastes better.

priority review regulatory

"eligibility for Priority Review"

Priority review is a regulatory fast-track that shortens the time an agency spends evaluating a drug, vaccine or medical device application so a decision comes sooner than normal. For investors, it matters because a faster review is like an express lane to market: it can speed revenue potential and reduce regulatory uncertainty, but it does not guarantee approval and still requires the product to meet safety and effectiveness standards.

new drug application regulatory

"sections of the New Drug Application"

A new drug application is a formal request submitted to government regulators seeking approval to market a new medicine. It is like a detailed proposal that shows the drug has been tested for safety and effectiveness. For investors, receiving approval signals that the drug may soon become available for sale, potentially leading to revenue growth and impacting the company's value.

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• Breakthrough Therapy designation granted based on FDA’s review of safety and efficacy data from the chronic cohort of the Phase 1b/2a CONNECT SCI study
• Phase 3 RESTORE registrational study enrolling participants, with site activation continuing in the United States and Canada

MIAMI, Nov. 12, 2026 (GLOBE NEWSWIRE) -- NervGen (NASDAQ: NGEN), a late-stage clinical biopharmaceutical company developing first-in-class neuroreparative therapeutics for spinal cord injury (SCI) and other neurotraumatic and neurologic conditions, today announced that the U.S. Food and Drug Administration (FDA) has granted NVG-291 Breakthrough Therapy designation for the treatment of chronic traumatic cervical motor-incomplete SCI. NVG-291 is being developed as potentially the first therapy to promote nerve regeneration in individuals living with chronic SCI.

Preliminary clinical evidence from the Phase 1b/2a CONNECT SCI study demonstrated substantial improvements on a clinically significant endpoint assessing functional hand use in individuals with chronic traumatic cervical motor-incomplete SCI. These findings were supported by Patient Global Impression of Change (PGIC) and blinded qualitative interviews documenting participant-reported gains in everyday function and independence compared to placebo. NVG-291 was generally well tolerated, with no serious adverse events or treatment discontinuations.

“Receiving Breakthrough Therapy designation for NVG-291 underscores the FDA’s recognition of the promising clinical data that NVG-291 has the potential to demonstrate substantial improvement over current treatments for individuals living with chronic SCI,” said Shamim Ruff, Chief Regulatory Affairs Officer of NervGen. “NVG-291 is designed to facilitate the body’s natural ability to regenerate and repair its nervous system, with the goal of achieving functional recovery even years after injury. We appreciate the FDA’s continued engagement and look forward to building on this collaboration as we advance our ongoing Phase 3 RESTORE study.”

“This designation brings renewed hope to the more than 300,000 individuals living with traumatic SCI in the United States and many more around the world,” said Andrew Wagner, CEO of Wings for Life USA. “For someone living with a long-standing injury, even small gains in function can make a profound difference to quality of life and everyday independence. Wings for Life is committed to funding clinical research that advances the possibility for recovery. As a supporter of the CONNECT SCI study, we are encouraged by the progress of NVG-291 and share the SCI community’s urgency to translate promising clinical findings into treatments that improve people’s lives.”

Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on one or more clinically significant endpoints. The benefits of Breakthrough Therapy designation include eligibility for Priority Review, rolling submission of sections of the New Drug Application and FDA’s organizational commitment to help determine an efficient route to approval.

About the Phase 3 RESTORE Study
RESTORE is a multicenter, randomized, double-blind, placebo-controlled Phase 3 registrational study evaluating the efficacy and safety of NVG-291 in adults living with chronic traumatic cervical motor-incomplete SCI. The study is expected to enroll approximately 150 participants across the United States and Canada. The study’s primary endpoint assesses improvement in hand function, a leading recovery priority for individuals with cervical SCI, as measured by GRASSP Quantitative Prehension. Secondary endpoints and blinded participant interviews will provide additional insight into functional independence and quality of life. Eligible participants are one to ten years following a traumatic SCI, have a motor level at C7 or higher on both sides and have an American Spinal Injury Association (ASIA) Impairment Scale classification of C or D. Participants will be randomized 1:1 to receive NVG-291 or placebo by once-daily subcutaneous injection for 12 weeks, followed by a four-week observation period. An optional 12-week open-label extension is planned following study completion. For additional information, including eligibility criteria and study locations, visit ClinicalTrials.gov (identifier: NCT07799532).

About Spinal Cord Injury

Spinal cord injury (SCI) disrupts communication between the brain and body and can result in lasting impairments in voluntary movement, sensation and autonomic function. Approximately 312,000 people in the United States are living with traumatic SCI, and about 18,500 new injuries occur each year. For individuals with cervical SCI, regaining arm and hand function is a leading recovery priority because of its importance to everyday independence and quality of life.

Following SCI, increased levels of chondroitin sulfate proteoglycans (CSPGs), molecules found in the extracellular matrix, contribute to an environment that inhibits nervous system repair. CSPGs act through receptors, including protein tyrosine phosphatase sigma (PTPσ), to inhibit nerve fiber regrowth and sprouting, limiting the potential for recovery. There are currently no FDA-approved pharmacologic therapies to restore lost neurologic function in individuals living with chronic SCI.

About NervGen
NervGen (NASDAQ: NGEN) is a late-stage clinical biopharmaceutical company developing first-in-class neuroreparative therapeutics for spinal cord injury (SCI) and other neurotraumatic and neurologic conditions. The Company’s mission is to transform the lives of individuals living with SCI by enabling the nervous system to repair itself. NervGen’s lead therapeutic candidate, NVG-291, is a subcutaneously administered neuroreparative peptide designed to target the inhibitory CSPG-PTPσ pathway. NVG-291 is the first pharmacologic candidate to improve function, independence, and quality of life in chronic SCI, as observed in the Phase 1b/2a CONNECT SCI study. NVG-291 has received Breakthrough Therapy designation from the FDA for the treatment of chronic traumatic cervical motor-incomplete SCI. It has also received Fast Track designation from the FDA and Orphan Drug designation from the European Medicines Agency for the treatment of SCI. Through NVG-291 and its next-generation candidate, NVG-300, NervGen is pursuing a pharmacologic approach to transform the treatment paradigm for neurotraumatic and neurologic conditions with significant unmet medical need. For more information, visit www.nervgen.com and follow NervGen on X and LinkedIn.

Cautionary Note Regarding Forward-Looking Statements
This news release contains “forward-looking information” and “forward-looking statements” within the meaning of applicable securities laws (collectively, “forward-looking statements”). Statements regarding the Company’s current and future plans, expectations and intentions, results, levels of activity, performance, goals or achievements, and other future events or developments constitute forward-looking statements. The words “may”, “will”, “would”, “should”, “could”, “expect”, “plan”, “intend”, “anticipate”, “believe”, “estimate”, “predict”, “likely” or “potential”, or the negative or other variations of these words or other comparable words or phrases, are intended to identify forward-looking statements. Forward-looking statements include, without limitation, statements relating to: the potential benefits of Breakthrough Therapy designation, including its potential to expedite the development and regulatory review of NVG-291, and the Company’s planned interactions with the FDA; the design, conduct, expected enrollment and anticipated timelines of the RESTORE study, including the planned open-label extension; the development and potential therapeutic benefits of the Company’s product candidates; and other factors described in the “Risk Factors” section of the Company’s most recently filed Annual Information Form and Annual Report on Form 40-F/A, its amended and restated management’s discussion and analysis for the year ended December 31, 2025, and subsequent filings with Canadian securities regulators and the U.S. Securities and Exchange Commission. These documents are available under the Company’s profile on SEDAR+ at www.sedarplus.ca and on the SEC’s website at www.sec.gov. Breakthrough Therapy designation does not guarantee regulatory approval of NVG-291, and results from earlier clinical studies may not be predictive of results from subsequent studies. Readers should not place undue reliance on forward-looking statements made in this news release. Unless otherwise stated, the forward-looking statements contained in this news release are made as of the date of this news release, and the Company has no intention and undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law. The forward-looking statements contained in this news release are expressly qualified by this cautionary statement.

Contacts
On365 Partners, Investor Relations
ir@nervgen.com
203.293.8091

FAQ

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What FDA designation did NervGen receive for NVG-291?

NVG-291 received Breakthrough Therapy designation for chronic traumatic cervical motor-incomplete spinal cord injury. The designation followed FDA review of safety and efficacy data from the chronic cohort of the Phase 1b/2a CONNECT SCI study and provides benefits intended to expedite development and review.