A potential disease-modifying treatment for osteoarthritis from Cynata Therapeutics

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A potential disease-modifying treatment for osteoarthritis from Cynata Therapeutics

Osteoarthritis is a chronic inflammatory joint disease that causes pain and disability, affecting over two million people in Australia and over 500 million people worldwide. Currently, there are no approved disease-modifying therapies for osteoarthritis, with treatment options limited to symptom management.

Cynata is developing CYP-004, our Cymerus™ off-the-shelf iPSC-derived MSC product for intra-articular injection (injection directly into a joint), as a potential treatment to reduce pain, inflammation and cartilage degeneration in people with osteoarthritis of the knee.

Phase 3 clinical trial – SCUlpTOR study

Our Phase 3 clinical trial in osteoarthritis, known as the SCUlpTOR trial (Stem Cells as a symptom- and strUcture-modifying Treatment for medial tibiofemoral OsteoaRthritis), represents one of the most advanced stem cell studies in this condition globally.

This randomised and placebo-controlled trial is being conducted the University of Sydney, under the leadership of Professor David Hunter, with funding provided under an Australian Government National Health and Medical Research Council (NHMRC) project grant.

Patient recruitment was completed in November 2023, with a total of 321 participants enrolled. All patients have now completed their study treatment – the follow-up period (two years after the first dose of study treatment) is expected to conclude in November 2025. We expect results in the first half of 2026.

Trial design:

The co-primary endpoints of the trial are:

  • The proportion of participants achieving patient-acceptable symptom state (PASS) for knee pain at 24 months
  • Central medial femorotibial (cMFT) cartilage thickness change from baseline to 24 months, as assessed magnetic resonance imaging (MRI)

If successful, CYP-004 could become the first approved disease-modifying therapy for osteoarthritis, which would be a significant advancement in the treatment of this debilitating condition. Delaying or avoiding the need for knee replacement surgery would offer substantial quality-of-life and economic benefits for patients worldwide.

Registry details: Osteoarthritis (CYP-004) – phase 3: https://anzctr.org.au/Trial/Registration/TrialReview.aspx?ACTRN=12620000870954