Caliway Biopharmaceuticals (6919) investor relations material

Disease overview and unmet needs

• Dercum's Disease is a chronic, recurrent, and severely debilitating rare disorder marked by painful lipomas, with symptoms unrelieved by weight loss and significant impact on daily life and mental health.

• No approved therapies exist; current treatments focus on symptom management with limited efficacy and substantial side effects, including surgery, analgesics, and immunomodulators.

• Liposuction can reduce pain but carries high risks, recurrence, and adverse effects, with pain relief diminishing over time.

CBL-514 phase 2 clinical trial design and analysis

• The phase 2b study enrolled 19 subjects with Dercum's Disease, randomized 1:1 to CBL-514 or placebo, with up to five treatments over 16 weeks and 8 weeks of follow-up.

• Analysis sets included full analysis, per protocol, safety, and as-treated populations, with robust statistical methods and corrective actions for dosing deviations.

Efficacy results

• CBL-514 demonstrated statistically significant pain reduction in treated lipomas at weeks 16, 20, and 24 compared to placebo across all analysis sets (all p < 0.0001).

• Over 90% of participants achieved ≥3-point pain reduction in at least 50% of treated lipomas, with 70% requiring only a single treatment.

• The magnitude and consistency of clinical benefit support a high probability of success in future pivotal phase 3 trials.