AbbVie Receives Orphan Drug and Fast Track Designations from the U.S. Food and Drug Administration for Elezanumab, an Investigational Monoclonal Antibody RGMa Inhibitor, for the Treatment of Spinal Cord Injury

- Elezanumab (ABT-555) is an investigational treatment being evaluated in neurological disorders, including treatment following spinal cord injury

- Orphan Drug Designation is given to a drug or biologic for the treatment, diagnosis or prevention of a rare disease or condition¹

- Fast Track Designation facilitates the development and expedites the review of drugs to treat serious conditions and fill unmet medical needs²

News provided by

AbbVie

Sep 28, 2020, 08:00 ET

NORTH CHICAGO, Ill., Sept. 28, 2020 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Fast Track designations for elezanumab (ABT-555), an investigational treatment for patients following spinal cord injury.

Elezanumab is a monoclonal antibody of the human immunoglobulin (Ig)G1 isotype that binds selectively to repulsive guidance molecule A (RGMa). RGMa is an inhibitor of axonal outgrowth and recognized as an important factor in inhibiting neuronal regeneration and functional recovery following central nervous system (CNS) damage. Elezanumab is being investigated to treat spinal cord injuries, multiple sclerosis and acute ischemic stroke. It is currently in a phase 2 study (NCT04295538) for the treatment of spinal cord injury.

"AbbVie is committed to delivering therapies that make a meaningful difference in patients' lives," said Michael Gold, MD, Vice President, Neuroscience Development. "Spinal cord injuries result in devastating lifelong physical, emotional and economic consequences. The FDA's Orphan Drug and Fast Track Designation for spinal cord injury patients signals an important step forward in AbbVie's ongoing commitment to investigating innovative scientific approaches with the hope of bringing new treatment options to patients."

Innovative Partnerships for Spinal Cord Injury Patients
Currently AbbVie is partnering with the Shirley Ryan AbilityLab, a global leader in physical medicine and rehabilitation, and MC10, a health digital solutions company, in a pilot study involving 20 spinal cord injury patients. The pilot study will inform the ongoing Phase 2 study of elezanumab by testing optimal biosensor placement to capture surface electromyography (sEMG), among other assessments. The pilot study will be completed in approximately two months.

AbbVie is also partnering with United Spinal Association and the North American Spinal Cord Injury Consortium to support spinal cord injury awareness and incorporate spinal cord injury community perspectives into our clinical research and outreach.

About Spinal Cord Injury
A spinal cord injury often causes permanent changes in motor function, sensation and other body functions below the site of the injury.³ Cervical spinal cord injuries are the most common and debilitating, with many occurring in younger people, typically male adults, with 43 as the average age at the time of injury.⁴ A spinal cord injury after age 65 is most often caused by a fall.⁵ Signs and symptoms of spinal cord injuries include loss of movement; loss of sensation, including the ability to feel heat, cold and touch; loss of bowel or bladder control; exaggerated reflex activities or spasms; changes in sexual function, sexual sensitivity and fertility; pain or an intense stinging sensation caused by damage to the nerve fibers in the spinal cord; and difficulty breathing, coughing or clearing secretions from lungs.⁶

About Orphan Drug and Fast Track Designations
Orphan Drug Designation is given to a drug or biologic for the treatment, diagnosis or prevention of a rare disease or condition.⁷ The FDA uses a fast track process to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.⁸

About the Shirley Ryan AbilityLab
Shirley Ryan AbilityLab, formerly the Rehabilitation Institute of Chicago (RIC), is the global leader in physical medicine and rehabilitation for adults and children with the most severe, complex conditions — from traumatic brain and spinal cord injury to stroke, amputation and cancer-related impairment. The organization expands and accelerates leadership in the field that began at RIC in 1953. The quality of its care and research has led to the designation of "No. 1 Rehabilitation Hospital in America" by U.S. News & World Report every year since 1991. The organization offers three clinical service lines: inpatient rehabilitation for those requiring complex medical and nursing care, intensive Day Rehabilitation in an outpatient setting, and Outpatient therapy and physician consultation services. Upon opening in March 2017, the $550 million, 1.2-million-square-foot Shirley Ryan AbilityLab became the first-ever "translational" research hospital in which clinicians, scientists, innovators and technologists work together in the same space, surrounding patients, discovering new approaches and applying (or "translating") research real time. This unique model enables patients to have 24/7 access to the brightest minds, the latest research and the best opportunity for recovery. Shirley Ryan AbilityLab is a 501 (c)(3) nonprofit organization. For more information, go to www.sralab.org.

About MC10
MC10 is a privately held company focused on improving human health through digital solutions. The company combines conformal BioStamp sensors with clinical analytics to unlock novel insights from physiological data collected from the home or in clinical settings. Our flagship product, BioStamp nPoint, is intended for the clinical research community. MC10 is headquartered in Lexington, MA. Visit MC10 online at mc10inc.com or follow us on LinkedIn.

About United Spinal Association
United Spinal Association is the largest non-profit organization dedicated to enhancing the quality of life of all people living with spinal cord injuries and disorders (SCI/D), including veterans, and providing support and information to loved ones, care providers and professionals. United Spinal has over 70 years of advocacy experience educating and empowering wheelchair users impacted by paralyzing conditions and mobility disabilities to achieve and maintain the highest levels of independence, health and personal fulfillment. United Spinal has over 50,000 members, 54 chapters, close to 200 support groups and more than 100 rehabilitation facilities and hospital partners nationwide. United Spinal Association is also a VA-recognized veterans service organization (VSO) serving veterans with disabilities of all kinds. Resources United Spinal provides include a Pathways to Employment (PTE) program, Accessibility Services Consulting aiding ADA compliance and accessibility, a virtual Resource Center and AskUs service offering personalized peer support, and New Mobility magazine, a premier disability lifestyle publication. The organization's headquarters is in Kew Gardens, NY with a government relations office in Washington, DC. https://unitedspinal.org/

About the North American Spinal Cord Injury Consortium (NASCIC)
The North American Spinal Cord Injury Consortium (NASCIC) has a focus to build collaboration and a unified voice among the spinal cord injury community, mainly those living with the condition and those organizations that represent them, within North America.

About Elezanumab (ABT-555)
Elezanumab is a monoclonal antibody RGMa inhibitor being investigated to treat spinal cord injuries, multiple sclerosis and acute ischemic stroke.

About AbbVie in Neuroscience
At AbbVie, our commitment to preserve the personhood of those living with neurologic and psychiatric disorders is unwavering. Every challenge in this uncharted territory makes us more determined and drives us harder to discover and deliver solutions for patients, care partners and clinicians. AbbVie's Neuroscience portfolio consists of approved therapies and a robust pipeline in neurologic and psychiatric disorders, including Alzheimer's disease, bipolar disorder and depression, major depressive disorder, migraine, multiple sclerosis, Parkinson's disease, post-stroke spasticity, schizophrenia, and stroke.

We have a strong investment in neuroscience research, with our Foundational Neuroscience Center in Cambridge, Massachusetts, and our Neuroscience Discovery site in Ludwigshafen, Germany, where our research and perseverance in these challenging therapeutic areas is yielding a deeper understanding of the pathophysiology of neurologic diseases, and identifying targets for potential disease-modifying therapeutics aimed at making a difference in people's lives. For more information, please visit www.abbvie.com.

About AbbVie
AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking Statements
Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions, among others, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, failure to realize the expected benefits from AbbVie's acquisition of Allergan plc ("Allergan"), failure to promptly and effectively integrate Allergan's businesses, competition from other products, challenges to intellectual property, difficulties inherent in the research and development process, adverse litigation or government action, changes to laws and regulations applicable to our industry and the impact of public health outbreaks, epidemics or pandemics, such as COVID-19. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2019 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.

¹ FDA. https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/frequently-asked-questions-faq-about-designating-orphan-product. Accessed September 1, 2020.

² FDA. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track. Accessed September 1, 2020.

³ Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/spinal-cord-injury/symptoms-causes/syc-20377890. Accessed September 1, 2020.

⁴ Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/spinal-cord-injury/symptoms-causes/syc-20377890. Accessed September 1, 2020.

⁵ Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/spinal-cord-injury/symptoms-causes/syc-20377890. Accessed September 1, 2020.

⁶ Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/spinal-cord-injury/symptoms-causes/syc-20377890. Accessed September 1, 2020.

⁷ FDA. https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/frequently-asked-questions-faq-about-designating-orphan-product. Accessed September 1, 2020.

⁸ FDA. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track. Accessed September 1, 2020.

SOURCE AbbVie