Vienna, Virginia-based CEL-SCI Corporation, a biopharmaceutical company focused on research and development directed at disease treatments that utilize the immune system’s natural defense processes, announced that its investigational LEAPS rheumatoid arthritis vaccine candidate has been accepted for a new technology commercialization and niche analysis program managed by Foresight Science & Technology for the U.S. National Institutes of Health (NIH).
CEL-SCI’s focus on developing unique therapies that address unmet medical needs led to development of the Ligand Epitope Antigen Presentation System, or LEAPS, an immunotherapy platform technology. The LEAPS product candidate CEL-2000 has shown promise for development as a therapeutic vaccine for rheumatoid arthritis (RA) in animal models of the disease.
LEAPS is a CEL-SCI patented platform technology designed to stimulate antigen-specific immune responses in T-cells using synthetic peptides. LEAPS constructs physically link the antigenic peptide with a T-cell binding ligand and are delivered directly to the recipient’s immune system by injection or mucosal absorption, potentially enhancing T-cell responses to a particular antigen.
CEL-SCI’s selection into the NIH commercialization program, including market entry strategy and launch tactics, is an extension of the Phase I Small Business Innovation Research (SBIR) grant in the amount of $225,000 awarded to CEL-SCI in July 2014 from the NIH’s National Institute of Arthritis Muscoskeletal and Skin Diseases (NIAMS) to fund further development of LEAPS technology as a potential treatment for rheumatoid arthritis.
The development work was conducted at Rush University Medical Center in Chicago, Illinois, in the laboratories of Tibor Glant, MD, PhD, the Jorge O. Galante Professor of Orthopedic Surgery; Katalin Mikecz, MD, PhD, a Professor of Orthopedic Surgery & Biochemistry; and Allison Finnegan, PhD, a Professor of Medicine.
The NIH grant was awarded based on preliminary data by Dr. Glant’s team in collaboration with CEL-SCI showing that administration of a proprietary peptide using CEL-SCI’s LEAPS technology prevented development, and lessened the severity, including inflammation, of experimental RA when it was administered after the disease was induced in the animals. This data was presented in May 2013 by Daniel Zimmerman, PhD , CEL-SCI’s Senior Vice President of Research, Cellular Immunology, at the symposium on “Therapeutic Approaches to Autoimmunity” during the American Association of Immunologists (AAI) 100th annual meeting in Honolulu, Hawaii.
“These findings, in conjunction with the results from previously conducted studies with LEAPS vaccines in other RA models, suggest that LEAPS vaccines may be used as a therapeutic treatment for different types of RA. LEAPS vaccines may be advantageous to other therapies because the LEAPS vaccines act early on the immune system and inhibit the production of disease-promoting inflammatory cytokines, unlike anti-Tumor necrosis factor alpha (TNFa) therapy which generally acts late and neutralizes only one individual inflammatory cytokine out of many involved in the disease process. The successful conclusion of this round of studies in this autoimmune disease could take LEAPS closer to human studies and open its development to various other autoimmune diseases, such as multiple sclerosis, uveitis, colitis (Inflammatory Bowel disease) and certain types of diabetes,” Dr. Zimmerman said in a release.
The NIH grant funded studies in a well-established mouse model for Th1 Proteoglycan induced arthritis (PGIA) as developed by Drs. Glant and Mikecz and recently expanded to a Th17 PGIA by Drs. Finnegan and Glant. These two PGIA models are significant in that they more closely approximate human disease with the concurrent presence of rheumatoid factor and anti citrulline peptide antibodies and spondylitis that are not seen in most arthritis models.
Rheumatoid arthritis is a chronic inflammatory disease that mainly targets the synovial membrane, cartilage and bone, affecting about 1% of the global population and associated with significant morbidity and increased mortality. CEL-SCI notes that anti-TNF related therapies are the current standard treatment of patients with advanced RA, but over half of the RA patients do not respond to current anti-TNF drugs such as etanercept (Enbrel) and infliximab (Remicade).
The company observes that only a select subset of SBIR grant recipients are additionally awarded the commercialization and launch niche analysis program, which will examine the market in depth for the LEAPS rheumatoid arthritis vaccine. This analysis will include interviews with experts and end-users, recommendations for a market entry, launch tactics and revenue projections. According to Visiongain, the world rheumatoid arthritis drug market will generate revenues of $38.5 billion in 2017.
“As we advance the clinical development program for our LEAPS rheumatoid arthritis vaccine, we are very pleased that the NIH has recognized the potential market value of this product,” says Dr. Zimmerman. “We believe the commercialization and program niche analysis will be of value to CEL-SCI as we apply for a Phase II SBIR grant to continue to support our efforts of bringing LEAPS into human clinical trials.”
CEL-SCI is also developing its pre-clinical LEAPS technology for the potential treatment of pandemic influenza in hospitalized patients.
Rush University Medical Center