The FDA has granted Kala Pharmaceuticals Fast Track designation status for KPI-012, its lead product candidate, for the treatment of persistent corneal epithelial defect (PCED)
Remind me what Fast Track designation means.
This process is a request made by a company to the FDA for facilitating the development and expediting the review of drugs to treat serious medical conditions and fill an unmet need; essentially, it’s a way to make new drugs available to patients faster.
Unlike a Breakthrough Therapy designation, this can be requested with non-clinical data and/or preliminary clinical evidence.
Talk about KPI-012.
KP1-012 is a human mesenchymal stem cell secretome (MSC-S) therapy 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.
Didn’t we hear about it earlier this year?
Yup! In January 2023, the FDA accepted Kala’s investigational new drug (IND) application for KPI-012.
Any clinical trials?
Yes! In February 2023, Kala dosed the first patient (of an estimated 90 participants) in its Corneal Healing After SEcretome therapy (CHASE) phase 2b, multicenter, randomized, double-masked, vehicle-controlled, parallel-group study (NCT05727878).
The trial is assessing the safety and efficacy of two doses (3 U/mL and 1 U/mL) of KPI-012 ophthalmic solution in comparison to a vehicle when topically dosed four times per day (QID) for 56 days.
Primary endpoint includes a complete PCED healing measured by corneal fluorescein staining.
Any data to report yet?
Kala released positive safety data in March 2023 from two patients (cohort 1) dosed with a high level of KPI-012 (3 U/mL) QID. Each patient completed week 1 of dosing successfully with no safety issues.
What comes next?
The CHASE study will progress to cohort 2, which will assess the two doses of KPI-012 versus vehicle. The dosing regimen and primary endpoint will follow that of cohort 1.
When can we expect results?
Kala expects to report topline safety and efficacy data by Q1 2024.
Pending positive data, the company hopes to use the results as the first of two pivotal trials to support a potential Biologics License Application (BLA) submission to the FDA.
Significance?
KPI–012 has the potential to become the first FDA-approved treatment for PCED across numerous etiologies.