Grant Award Details

Phase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect
Grant Type: 
Grant Number: 
CLIN2-14516
Project Objective: 
  • Persistent corneal epithelial defect, or PCED, is a non-healing corneal wound refractory to standard treatments. KPI-012 is a topical eye drop containing the components of allogeneic human bone-marrow mesenchymal stem cell (MSC) secretome. The active components include growth factors, protease inhibitors, matrix proteins, and neurotrophic factors, all of which have been shown to facilitate corneal healing in preclinical and clinical studies independent of injury (mechanical, chemical) or etiology (diabetic neurotrophy, Steven-Johnson Syndrome, infectious keratitis). KPI-012 demonstrated promising safety and efficacy in two Phase 1/2 clinical studies for PCED.
  • Combangio is employing CIRM funding to complete a Phase 2b trial of KPI-012 for treatment of persistent corneal epithelial defect (PCED) by topical ocular instillation to assess safety, tolerability, efficacy, as well as to support the manufacturing and product development required to prepare for a Phase 3 trial in late 2024.
  • The trial includes up to 42 sites. Approximately 250 participants are planned to be screened and 90 randomized to High Dose, Low Dose, or Vehicle in an approximate 1:1:1 ratio (approximately 60 treated with active drug) in Cohort 2 for this trial.
  • Assay development, therapeutic product manufacture and stability studies are also to be conducted in support of the current P2 trial and future P3 trial.
  • The outcome of the project is a CSR and EOP2 meeting with FDA in preparation for a pivotal Ph3 trial.
Investigator: 
Name: 
Institution: 
Type: 
PI
Disease Focus: 
Corneal Damage
Vision Loss
Human Stem Cell Use: 
Adult Stem Cell
Award Value: 
$15,000,000
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • Phase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect
Public Abstract: 

Therapeutic Candidate or Device

KPI-012 is a human bone marrow mesenchymal stem cell secretome formulated as a topical ophthalmic therapeutic

Indication

Treatment of persistent corneal epithelial defect, a cornea defect refractory to conventional treatments that can lead to blindness

Therapeutic Mechanism

KPI-012 is intended to restore normal corneal wound healing, providing a curative solution to chronic corneal epithelial lesions and avoiding pathologic sequalae associated with loss of vision. The anticipated benefits are restoration of vision, reduction or elimination of pain, reduced treatment burden, and increased quality of life.

Unmet Medical Need

No effective PCED therapies exist that address all etiologies, as they do not target the multiple dysregulated pathways in persistent wounds. With its ability to target multiple healing pathways, KPI-012 has the potential to heal PCEDs, thereby helping to protect vision and reduce pain.

Project Objective

Phase 2b trial completed and results published

Major Proposed Activities

  • • Complete a Phase 2b clinical study to demonstrate safety and efficacy of the therapeutic candidate
  • • Conduct product and process characterization studies to support product development
  • • Evaluate and develop additional analytical methods for product comparability studies
Statement of Benefit to California: 

One of the major contributing factors to development of PCED is diabetes. Over 3 million Californians have diabetes, approximately 43% of whom are at risk for diabetic keratopathy and associated PCED. The availability of an approved curative treatment for PCED will improve quality of life by avoiding loss of vision and its associated loss in productivity for this population.

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