Cytonics Completes Enrollment for Phase 1 Study of CYT-108 in Knee Osteoarthritis Patients

Cytonics Corporation, a private biotechnology firm specializing in biologic treatments for inflammatory musculoskeletal conditions, has completed enrollment for its Phase 1 clinical trial of CYT-108. CYT-108 is a recombinant variant of the alpha-2-macroglobulin (A2M) blood serum protease inhibitor, designed as a potential disease-modifying therapy for osteoarthritis (OA).

The clinical trial, the first of its kind in humans, is a multi-center, double-blind, randomized controlled trial involving 22 patients. These patients, who have unilateral, mild-to-moderate primary OA of the knee, were evenly divided into treatment and placebo groups. Over six months, the trial will evaluate both the safety and efficacy of CYT-108 when administered via intra-articular injection directly into the affected joints. The primary safety endpoints focus on the drug's tolerability, while secondary efficacy endpoints will assess improvements in self-reported pain and mobility. Additionally, an exploratory endpoint aims to quantify peptide fragments in blood serum as a biomarker for cartilage degradation.

So far, CYT-108 has been well-tolerated by all patients, with no drug-related adverse events reported. Joey Bose, President & CEO of Cytonics Corporation, expressed excitement over meeting the enrollment target ahead of schedule. This achievement is anticipated to expedite the clinical study report and subsequent Investigational New Drug (IND) filing with the U.S. Food and Drug Administration (FDA) in 2025. Bose highlighted the efficient recruitment processes at their clinical sites in Australia and expressed eagerness to continue collaborating with these researchers and clinicians as they move into Phase 2 of the study.

CYT-108 was engineered by manipulating the "bait" region of the A2M protein to enhance its affinity for specific proteases implicated in the development of OA. This unique characteristic allows CYT-108 to inhibit a broad spectrum of proteases, distinguishing it from other treatments that target only a single protease. This makes CYT-108 a promising candidate for a disease-modifying therapy for OA.

The development of CYT-108 is built on the success of Cytonics' Autologous Protease Inhibitor Concentrate (APIC) system. The APIC system, a 510(k) medical device, selectively enriches autologous A2M for intra-articular injection into joints affected by articular cartilage damage. It has been successfully used since its FDA clearance in 2015. Its clinical and commercial success has paved the way for the development of CYT-108, as it demonstrates that high concentrations of A2M can be safely introduced into arthritic joints without significant side effects.

Founded in 2006, Cytonics Corporation is dedicated to developing molecular diagnostics and therapies for chronic musculoskeletal diseases such as OA. The company's flagship product was the Fibronectin-Aggrecan Complex Test (FACT), which helps identify the source of joint pain and guides physicians in selecting appropriate treatments. Following FACT, Cytonics developed the APIC system to concentrate A2M from patients' blood and inject the concentrate into damaged joints. This system has treated over 8,000 patients nationwide, aiding many to avoid invasive joint replacement surgeries.

Cytonics has garnered over $25 million in private funding and received $1.8 million in grants from the National Institutes of Health (NIH) to support innovative research. The company's current focus is on the Phase 1 clinical trials for CYT-108. If approved, CYT-108 could become the first and only disease-modifying therapy for OA.