Combangio Awarded $15 Million to Support Ongoing KPI-012 Program for the Treatment of PCED

Kala Pharmaceuticals, Inc. recently announced the California Institute for Regenerative Medicine (CIRM) has awarded Combangio, Inc., a wholly owned subsidiary of Kala, a $15-million grant to support its ongoing KPI-012 program for the treatment of persistent corneal epithelial defect (PCED). The grant includes funding for the CHASE (Corneal Healing After SEcretome therapy) Phase 2b clinical trial as well as product and process characterization and analytical development for the program.

“Consistent with CIRM’s mission to accelerate the development of potentially transformative medicines to improve the care of patients globally, we are excited to support the KPI-012 program for the treatment of PCED,” said Maria T. Millan, MD, President and CEO of CIRM. “KPI-012’s differentiated product profile has the potential to deliver rapid and sustained wound healing with broad-based application across all underlying PCED etiologies. We look forward to partnering with the company to bring this regenerative therapy to the thousands of patients in need.”

KPI-012 is a human mesenchymal stem cell secretome (MSC-S), being developed for the treatment of PCED. Based on its multifactorial mechanism of action and preclinical and clinical data generated to-date, Kala believes KPI-012 may represent a significant advancement in the treatment of PCED and could become the first approved treatment for PCED across all its various etiologies.

The CHASE Phase 2b clinical trial includes two patient cohorts. In March 2023, the company reported positive safety data from the first patient cohort, evaluating the safety of the high dose of KPI-012 (3 U/mL) dosed topically four times per day (QID). The second patient cohort is currently enrolling in a multicenter, randomized, double-masked, vehicle-controlled, parallel-group study to evaluate the safety and efficacy of two doses of KPI-012 ophthalmic solution (3 U/mL and 1 U/mL) versus vehicle dosed topically QID for 56 days in approximately 90 patients. The primary endpoint of the trial is the complete healing of the PCED as measured by corneal fluorescein staining. Topline safety and efficacy data is targeted in the first quarter of 2024. If the results are positive, and subject to discussion with regulatory authorities, the company believes this trial could serve as the first of two pivotal trials required to support the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA).

“We are honored that CIRM has recognized KPI-012’s potential to correct impaired corneal healing,” said Mark Iwicki, Chief Executive Officer and Chairman of Kala Pharmaceuticals. “We believe this grant not only serves as further validation for KPI-012 and our MSC-S platform, but will provide us with important funding to advance our ongoing Phase 2b trial, which could serve as the first of two pivotal trials needed to support the submission of a BLA to the FDA.”

Persistent corneal epithelial defect, which is defined as a persistent non-healing corneal defect or wound that is refractory to conventional treatments, is a rare disease with an estimated incidence in the United States of 100,000 cases per year and 238,000 cases per year in the United States, European Union and Japan combined. PCED can have various etiologies, including neurotrophic keratitis, surgical epithelial debridement, microbial/viral keratitis, corneal transplant, limbal stem cell deficiency and mechanical and chemical trauma and, if left untreated, can lead to infection, corneal ulceration or perforation, scarring, opacification and significant vision loss.

Based on a multifactorial mechanism of action and preclinical and clinical data generated to-date, Kala believes KPI-012 may represent a significant advancement in the treatment of PCED and could become the first approved treatment for PCED across all its various etiologies.

Kala is a clinical-stage biopharmaceutical company dedicated to the research, development and commercialization of innovative therapies for rare and severe diseases of the eye. Kala’s biologics-based investigational therapies utilize Kala’s proprietary mesenchymal stem cell secretome (MSC-S) platform. Kala’s lead product candidate, KPI-012, is a human MSC-S, which contains numerous human-derived biofactors, such as growth factors, protease inhibitors, matrix proteins and neurotrophic factors that can potentially correct the impaired corneal healing that is an underlying etiology of multiple severe ocular diseases. KPI-012 is currently in clinical development for the treatment of persistent corneal epithelial defect (PCED), a rare disease of impaired corneal healing, for which it has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration. Kala is also targeting the potential development of KPI-012 for the treatment of Limbal Stem Cell Deficiency and ocular manifestations of moderate-to-severe Sjögren’s and has initiated preclinical studies to evaluate the potential utility of its MSC-S platform for retinal degenerative diseases, such as Retinitis Pigmentosa and Stargardt Disease. For more information, visit www.kalarx.com.