Kala Pharmaceuticals Receives FDA Fast Track Designation for KPI-012, a Human MSC-S Therapy for Persistent Corneal Epithelial Defect

Rhea-AI Summary

Kala Pharmaceuticals (NASDAQ:KALA) announced that the FDA has granted Fast Track designation for its KPI-012 therapy, aimed at treating persistent corneal epithelial defect (PCED), a rare and serious eye condition affecting approximately 100,000 patients in the U.S. This designation aims to expedite the development and review process, facilitating more frequent interactions with the FDA. KPI-012, based on a human mesenchymal stem cell secretome (MSC-S) platform, seeks to address the unmet medical needs of PCED patients, for whom no approved treatments currently exist. Kala is advancing its Phase 2b clinical trial, with promising initial safety results reported, and aims to present topline data in Q1 2024, potentially using this trial as one of the two pivotal studies required for a Biologics License Application (BLA).

Positive

• KPI-012 receives Fast Track designation from the FDA, expediting its development.
• Initial safety data from Phase 2b trial indicates no issues with high-dose KPI-012.
• Potential to be the first approved treatment for PCED, addressing a significant unmet medical need.

Negative

• None.

ARLINGTON, Mass., April 12, 2023 (GLOBE NEWSWIRE) -- Kala Pharmaceuticals, Inc. (NASDAQ:KALA), a clinical-stage biopharmaceutical company dedicated to the research, development and commercialization of innovative therapies for rare and severe diseases of the eye, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Kala’s human mesenchymal stem cell secretome (MSC-S) therapy (KPI-012) for the treatment of persistent corneal epithelial defect (PCED), a rare and debilitating ocular condition.

“There is a significant unmet need for patients suffering from PCED, which can lead to many complications and sequelae, including vision impairment, infection, corneal perforation and significant pain and discomfort. There are currently no approved pharmaceutical treatments for the majority of PCED patients, and KPI-012’s multifactorial mechanism of action is a potential solution to address all underlying etiologies of PCED,” said Kim Brazzell, Ph.D., Head of R&D and Chief Medical Officer at Kala Pharmaceuticals. “We look forward to working with the Agency to efficiently advance KPI-012 through the development and review process to fulfill our greater mission of delivering a treatment to the roughly 100,000 patients in the U.S., and thousands more globally, suffering from PCED.”

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 CHASE (Corneal Healing After SEcretome therapy) 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). 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.

About KPI-012 for Persistent Corneal Epithelial Defect (PCED)

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 and 238,000 cases per year in the United States, European Union and Japan combined. 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 a 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.

About Kala Pharmaceuticals

Kala is a clinical-stage biopharmaceutical company dedicated to the research, development and commercialization of innovative therapies for rare and severe diseases of the eye. Kala’s biologics-based investigational therapies utilize Kala’s proprietary mesenchymal stem cell secretome (MSC-S) platform. Kala’s lead product candidate, KPI-012, is a human MSC-S, which contains numerous human-derived biofactors, such as growth factors, protease inhibitors, matrix proteins and neurotrophic factors that can potentially correct the impaired corneal healing that is an underlying etiology of multiple severe ocular diseases. KPI-012 is currently in clinical development for the treatment of persistent corneal epithelial defect (PCED), a rare disease of impaired corneal healing, for which it has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration. Kala is also targeting the potential development of KPI-012 for the treatment of Limbal Stem Cell Deficiency and ocular manifestations of moderate-to-severe Sjögren's and 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. For more information on Kala, please visit www.kalarx.com.

Forward Looking Statements:

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any statements in this press release about Kala’s future expectations, plans and prospects, including but not limited to statements about Kala’s expectations with respect to potential advantages of KPI-012 and its MSC-S platform; the clinical utility of KPI-012 for PCED; Kala’s plans to pursue research and development of KPI-012 and its MSC-S platform for other indications; the benefits of a Fast Track designation by the FDA; and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “likely,” “will,” “would,” “could,” “should,” “continue,” and similar expressions constitute forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and conduct of preclinical studies and clinical trials; uncertainties regarding availability and timing of data from clinical trials; whether results of early clinical trials or trials in different disease indications will be indicative of the results of ongoing or future trials; whether results of the Phase 1b clinical trial of KPI-012 will be indicative of results for any future clinical trials and studies of KPI-012, including the CHASE Phase 2b clinical trial; whether interim data from a clinical trial will be predictive of the results of the trial; uncertainties associated with regulatory review of clinical trials and applications for marketing approvals; Kala’s ability to retain and hire key personnel; the impact of extraordinary external events, such as the current pandemic health event resulting from the coronavirus (COVID-19), and their collateral consequences; the sufficiency of cash resources and need for additional financing and other important factors, any of which could cause Kala’s actual results to differ from those contained in the forward-looking statements, discussed in the “Risk Factors” section of Kala’s Annual Report on Form 10-K and other filings Kala makes with the Securities and Exchange Commission. These forward-looking statements represent Kala’s views as of the date of this press release and should not be relied upon as representing Kala’s views as of any date subsequent to the date hereof. Kala does not assume any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Investor Contact:

Hannah Deresiewicz
hannah.deresiewicz@sternir.com
212-362-1200

FAQ

What is KPI-012 and its significance for KALA?

KPI-012 is a therapy that has received Fast Track designation from the FDA to treat persistent corneal epithelial defect (PCED), a rare eye condition with no approved treatments.

How many patients in the U.S. are affected by persistent corneal epithelial defect?

Approximately 100,000 patients in the U.S. are affected by persistent corneal epithelial defect (PCED).

What were the initial results of the KPI-012 clinical trial?

Initial results from the Phase 2b clinical trial of KPI-012 showed no safety issues in the first cohort of patients.

When does KALA expect to report data from its KPI-012 trial?

KALA is targeting to report topline safety and efficacy data from the KPI-012 trial in the first quarter of 2024.

What is the next step if KPI-012 trial results are positive?

If the results are positive, KPI-012 could serve as one of the two pivotal trials required for submitting a Biologics License Application (BLA) to the FDA.