KALA BIO (KALA) Piper Sandler 36th Annual Healthcare Conference summary

Strategic focus and clinical development

• Emphasis on mesenchymal stem cell secretomes for rare ophthalmic diseases, with KPI-012 as the lead product in phase IIb/pivotal trial targeting persistent corneal epithelial defects (PCED).

• Top-line data for KPI-012 expected in Q2 2025, aiming for a broad PCED indication and billion-dollar market potential.

• IND filed in November 2022, with FDA alignment on CMC and potency assay programs for phase III and BLA.

• Leadership team has prior success with FDA-approved ophthalmic products and experience in rare diseases and cell therapies.

• Previous divestiture of non-core assets to focus on biologics for rare ophthalmic conditions.

Product platform and mechanism of action

• KPI-012 is a topical ocular formulation derived from human bone marrow mesenchymal stem cell secretome, containing growth factors, protease inhibitors, matrix proteins, and neurotrophic agents.

• Designed to address multiple pathways in corneal wound healing, supporting a multifactorial approach for PCED and other rare ocular diseases.

• Orphan drug and fast-track designations obtained, with potential applications in limbal stem cell deficiency and inherited retinal diseases.

• Secretome platform offers benefits of cell therapy without associated safety and logistical challenges.

Market opportunity and competitive landscape

• PCED affects approximately 100,000 patients in the U.S. and 240,000 across the U.S., Europe, and Japan.

• Only one approved product, Oxervate, addresses about one-third of PCED cases; KPI-012 aims for a broader indication.

• Oxervate sales reached $700 million in 2023 and are projected to approach $1 billion, highlighting significant market potential.

• KPI-012's multifactorial mechanism could address all PCED etiologies, filling a major unmet need.