FDA grants fast track designation for KPI-012 by Kala Pharmaceuticals

Kala Pharmaceuticals KPI-012 is a treatment for PCED with potential to treat retinitis pigmentosa and Stargardt disease. (Image Credit: Adobe Stock/JHVEPhoto)

FDA granted Fast Track designation for Kala Pharamecuticals human mesenchymal stem cell secretome (MSC-S) therapy (KPI-012). The primary focus of this therapy is the treatment of persistent corneal epithelial defect (PCED), a rare and debilitating ocular condition, but Kala has also 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.

Kim Brazzell, PhD, head of research and development and chief medical officer at Kala Pharmaceuticals, pointed out that there is a significant unmet need for patients diagnosed with PCED, which she noted in a news release can result in a number of potential complications and sequelae, including vision impairment, infection, corneal perforation and significant pain and discomfort.

KPI-012 could represent a significant advancement in the treatment of PCED and could become the first approved treatment for PCED across all its various etiologies.

The PCED treatment approval process

According to the company, Fast Track is a process designed by the FDA to facilitate the development and expedite the review of drug candidates intended to treat serious conditions and for which nonclinical and/or clinical data demonstrate the potential to address unmet medical need. The purpose of this designation is to help speed development of new drugs, making them available to the patient more quickly. A company with a therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the product development plan. Therapeutic candidates with Fast Track designation may also be eligible for priority review and accelerated approval if supported by clinical data.

In February 2023, Kala dosed the first patient in its Corneal Healing After Secretome therapy (CHASE) Phase 2b trial evaluating KPI-012 for the treatment of PCED. In March 2023, Kala announced positive safety data from the first cohort of two patients that were treated with a high dose of KPI-012 (3 U/mL) four times per day (QID).

According to the company, both patients successfully completed at least one week of dosing with no safety issues observed and the trial has now advanced to Cohort 2. The second cohort is a multicenter, randomized, double-masked, vehicle-controlled, parallel-group study to evaluate the safety and tolerability of two doses of KPI-012 in 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. Kala is targeting reporting topline safety and efficacy data in the first quarter of 2024. If the results are positive, and subject to discussion with regulatory authorities, Kala 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 FDA.

According to the company, it expects to report top-line safety and efficacy data from the trial during the first quarter of 2024.