According to the company, it is on track to initiate a Phase 2b trial in the first quarter of 2023, with topline data expected in the first quarter of 2024.
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.
Kala Pharmaceuticals Inc. this week announced that the FDA has accepted an investigational new drug (IND) application for the company’s lead product candidate, KPI-012, a human mesenchymal stem cell secretome (MSC-S), initially in development for the treatment of persistent corneal epithelial defect (PCED).
“The acceptance of the KPI-012 IND is an important milestone for Kala, as we work to translate the promise of our MSC-S platform into better outcomes for people living with rare ocular surface diseases,” Kim Brazzell, PhD, head of R&D and chief medical officer of Kala Pharmaceuticals, said in a news release. “We are now turning our focus to clinical execution. We are working closely with investigators to initiate our Phase 2b clinical trial of KPI-012 for PCED in the first quarter of 2023.”
According to the news release, the Phase 2b clinical trial will be a multicenter, randomized, double-masked, vehicle-controlled, parallel-group study to evaluate the safety and efficacy of two doses of KPI-012 ophthalmic solution compared to vehicle when dosed topically four times per day (QID) for 56 days.
Moreover, the company noted in the news release that the trial is expected to enroll approximately 90 adult patients with PCED, and the primary endpoint of the trial will be complete healing of the PCED as measured by corneal fluorescein staining.
The company said it expects to initiate enrollment in the trial in the first quarter of 2023 and expects to report topline data in the first quarter of 2024. If the results are positive, 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.
In addition, following the FDA’s acceptance of the IND application for KPI-012 and under the terms of the private placement announced last month, Kala this week sold an aggregate of 43,478 shares of Series E Convertible Non-Redeemable Preferred Stock at a price of $575.00 per share to a life sciences focused-investor for gross proceeds of $25.0 million. In total, Kala raised aggregate gross proceeds of $31.0 million in the private placement.
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. 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 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.