Cynata Therapeutics (CYP) Study update summary

Overview of clinical and pivotal trial results

• Phase II acute graft-versus-host disease (GVHD) and phase III osteoarthritis (OA) trials both failed to meet primary endpoints, with no statistically significant differences between active and control groups in efficacy measures.

• Both products demonstrated safety and were well tolerated, with adverse event profiles comparable to control and no significant safety concerns identified.

• High placebo response rates in both trials, especially OA, undermined the ability to demonstrate efficacy.

• The GVHD trial's outcome was unexpected given positive phase I results, while the OA trial had no prior company data.

• Additional outcome measures from the OA trial, such as KOOS and quality of life, are still being analyzed and will be reported later.

Detailed trial findings

• In the OA trial, 51.7% of active group and 48.1% of placebo group reached the pain threshold (PASS) at 24 months (p=0.5907), negating statistical significance.

• Mean cartilage thickness loss was 0.27mm (active) vs 0.21mm (control), p=0.1453, with no evidence of reduced cartilage loss in the active group.

• Both groups in the OA trial showed substantial and durable pain reduction over two years, but no significant difference between arms.

• In the GVHD trial, overall response rate was 57.7% (active) vs 54.8% (control), p > 0.9999, with no significant difference.

• Secondary endpoints and overall survival rates in GVHD showed similar patterns, with no meaningful group differences (Day 100 OS: 88.1% active vs 82.3% control, p=0.5137).

Study design and methodology

• Phase 3 SCULPTOR trial evaluated CYP-004 in knee osteoarthritis patients using a randomized, placebo-controlled design, led by Professor David Hunter at the University of Sydney with NHMRC funding.

• Patients received intra-articular injections of either CYP-004 or saline placebo.

• CYP-004 is an off-the-shelf iPSC-derived MSC product candidate.