Cytonics Begins First Human Phase 1 Trial for CYT-108 in Knee Osteoarthritis Patients

JUPITER, Fla., July 15, 2024 – Cytonics Corporation, a private biotech firm focused on developing biological treatments for musculoskeletal conditions, has begun enrolling participants for its Phase 1 clinical trial of CYT-108. This drug is a recombinant form of the alpha-2-macroglobulin (A2M) blood serum protein, which inhibits proteases that contribute to cartilage degradation in osteoarthritis (OA). CYT-108 has been specifically designed to enhance its protease-inhibition capabilities against the main proteases responsible for breaking down cartilage in arthritic joints, while still maintaining its broad-spectrum anti-protease activity.

President and CEO Joey Bose commented, "Our first-in-human clinical trial marks the culmination of six years of preclinical research and development. During this period, we successfully tackled numerous technical challenges to develop CYT-108. The massive size and complex folding structure of the protein (720kD) made Good Manufacturing Practice (GMP) production a scientific achievement, thanks to the expertise of Lewis Hanna, PhD, our Chief Scientific Officer. Preclinical data shows promising signs that CYT-108 can protect not just cartilage tissue but also other joint structures involved in osteoarthritis. The development of CYT-108 benefits from the clinical and commercial success of our first-generation OA therapy, the Autologous Protease Inhibitor Concentrate (APIC) medical device, which has treated over 8,000 patients using autologous A2M concentrated from their own blood."

The Phase 1 trial aims to evaluate the safety and effectiveness of CYT-108 in patients with primary osteoarthritis of the knee. A single dose of CYT-108 will be administered into the joint at two different timepoints, 90 days apart. Patients will undergo monitoring every few weeks for an additional 90 days to assess safety, pharmacokinetics, and efficacy in alleviating joint pain, stiffness, and mobility issues.

CYT-108 is a recombinant variant of the natural A2M blood serum protein. The "bait" region of the protein, which interacts with proteases, has been engineered to heighten its affinity for specific proteases implicated in the molecular mechanisms of osteoarthritis. CYT-108’s ability to inhibit a broad range of proteases, as well as its specificity, sets it apart from other small molecule inhibitors that target single proteases. This makes CYT-108 a promising candidate for disease-modifying osteoarthritis therapy. The development of CYT-108 builds on the success of Cytonics' APIC system, a 510(k) medical device that enriches autologous A2M for injection into joints with damaged articular cartilage. APIC's clinical and commercial success lowers the risk of developing CYT-108 since it has been demonstrated that high concentrations of A2M can be safely introduced into arthritic joints.

Founded in 2006, Cytonics is a private research and development company dedicated to creating molecular diagnostics and treatments for chronic musculoskeletal diseases like osteoarthritis. The company's flagship product, the Fibronectin-Aggrecan Complex Test (FACT), helps identify the source of joint pain to guide treatment decisions. Cytonics then developed the APIC system, which concentrates therapeutic A2M from patients' blood for injection into damaged joints. APIC has treated over 8,000 patients nationwide, often preventing the need for invasive joint replacement surgeries. The company has raised over $25 million in private funding and received $1.8 million in grants from the National Institutes of Health (NIH) to support its innovative research. Currently, Cytonics is conducting Phase 1 clinical trials for CYT-108. If approved, CYT-108 could become the first disease-modifying therapy for osteoarthritis.