KALA BIO (KALA) Jefferies 2024 Global Healthcare Conference summary

Strategic focus and pipeline progress

• Lead product KPI-012, based on mesenchymal stem cell secretomes, is in a phase IIb pivotal trial for persistent corneal epithelial defects (PCED), with top-line data expected by end of 2024.

• Additional pipeline includes a planned trial for limbal stem cell deficiency and research into inherited retinal diseases.

• Received a $15 million grant from CIRM in 2023, supporting ongoing development.

• Cash reserves are projected to last through Q3 2025.

• Management team has a track record of developing and divesting approved ophthalmic products.

Product mechanism and clinical rationale

• KPI-012 utilizes biomolecules secreted by human bone marrow-derived mesenchymal stem cells, formulated as a topical ocular solution.

• The product contains growth factors, protease inhibitors, and matrix proteins to promote corneal healing and address multiple etiologies.

• Offers a multifactorial mechanism of action, potentially avoiding safety and logistical issues of cell therapy.

• Orphan Drug and Fast Track designations have been granted.

Market opportunity and unmet needs

• PCED affects over 100,000 patients in the U.S. and 200,000+ in the U.S. and Japan combined.

• Only one approved product (Oxervate) currently addresses a subset of PCED, with nearly $1 billion in 2023 sales.

• KPI-012 aims for a broader PCED indication, with a more patient-friendly administration compared to Oxervate.

• Significant unmet need exists for both PCED and limbal stem cell deficiency, each with large patient populations and limited treatment options.