Kala Pharmaceuticals announced that the FDA has accepted its investigational drug application (IND) for KPI-012, its lead product candidate initially in development for treating (PCED).
What is persistent corneal epithelial defect?
PCED can be defined as a persistent non-healing corneal defect or wound that is refractory to conventional treatments. It is a rare disease with an estimated incidence in the U.S. of 100,000 cases per year linking to a myriad of etiologies including neurotrophic keratitis, viral/microbial keratitis, limbal stem cell deficiency, severe dry eye disease, and chemical/mechanical trauma.
I need info on KP1-012.
KPI-012 is a mesenchymal stem cell secretome (MSC-S) derived from human bone marrow that contains formulated biofactors such as growth factors, protease inhibitors, and matrix proteins. These properties give it the potential to correct impaired corneal healing that’s at the root of severe ocular diseases.
Any clinical trials to know about?
Kala plans to begin enrollment of approximately 90 PCED patients for a Phase 2b trial in the first quarter of 2023. The multicenter, randomized,double-masked, vehicle-controlled, parallel-group study will assess the safety and efficacy of two doses of KPI-012 ophthalmic solution in comparison to vehicle when topically dosed four times per day for 56 days. The primary endpoint will be a complete PCED healing measured by corneal fluorescein staining. (via)
When can we expect results?
Kala plans on reporting topline data within the first quarter of 2026. If positive, the company anticipates using the trial as one of two pivotal trials needed to submit a Biologics License Application (BLA) to the FDA.
From a supply chain perspective, Kala’s MSC-S good manufacturing practice (GMP) would be manufactured from a proprietary cell bank and well-defined chemistry, manufacturing, and controls (CMS) process, which would enable consistent lot-to-lot safety, stability and biopotency.
What’s the significance?
KPI–012 has the potential to become the first FDA-approved treatment for PCED across numerous etiologies. Futuristically, Kala might pursue anterior segment follow-on indications for KPI-012—which might include partial limbal stem cell deficiency and moderate to severe Sjögren’s Syndrome.