KALA BIO, Inc. reported less-than-stellar clinical news for its lead investigational candidate in development for treating persistent corneal epithelial defect (PCED): a phase 2b trial on ophthalmic solution failed to meet its primary endpoint.
Oh dear. Let’s back up a moment and start with the basics.
We’ll begin with a look at this investigational therapeutic: KPI-012.
What it is: A human mesenchymal stem cell secretome (MSC-S) therapy derived from human bone marrow composed of formulated biofactors—growth factors, protease inhibitors, and matrix proteins—intended to promote ocular surface tissue repair.
- Why this composition matters: These biofactors are intended to (potentially) correct impaired corneal healing—a notable root cause of severe ocular diseases.
Also: KPI-012 was formulated to utilize KALA’s proprietary MSC-S platform, designed as a cell-free approach for disease management.
And which ocular diseases is KPI-012 intended to target?
Aside from PCED, the formulation is listed as still in the preclinical stage for limbal stem cell deficiency and other rare corneal diseases.
Gotcha. Now to its clinical journey thus far.
Up until this point, the therapeutic’s trajectory toward potential regulatory approval was on track.
In fact, the FDA previously granted KPI-012 Fast Track designation and Orphan Drug designation for its PCED indication.
As for its clinical evaluation history: KPI-012 was reported to be well-tolerated with a favorable safety profile following positive results from a phase 1b study, according to KALA.
- This phase 2b trial was intended to be the next step in its promising future.
Tell me about the study.
Initiated in 2023, the Corneal Healing After SEcretome therapy (CHASE) phase 2b trial (NCT05727878) was designed as a multicenter, randomized, double-masked, vehicle-controlled, parallel-group study.
- Its purpose: To evaluate the safety and efficacy of KPI-012 when topically administered to PCED patients.
- The participants: 79 patients (aged 18+ and diagnosed with PCED) were enrolled across 37 clinical sites in the U.S. and Latin America
- The setup: Patients were divided into four groups to receive either KPI-012 (in one of two doses) or a placebo, topically administered four times a day (QID)
- The duration: 56 days
As for the primary outcome: This was a complete healing of PCED, as determined by corneal fluorescein staining photographs analyzed by a masked central reading center (measured at Day 56).
… and what went wrong?
The company’s topline data readout included mixed results.
- The good: KPI-102 was well-tolerated with no treatment-related serious adverse events observed.
- The bad: The study did not meet its primary endpoint of complete PCED healing at Week 8 (Day 56)—and failed to achieve statistical significance for key secondary efficacy endpoints (plus showed no meaningful difference between either KPI-012 or placebo treatment arms)
Based on the demonstrated lack of efficacy, there is no reason to advance the candidate in its evaluations, according to Kim Brazzell, PhD, Head of Research & Development and chief medical officer.
Yikes. So what’s next for the clinical program?
Nothing. Unfortunately, it appears to be the end of the (clinical) road for KPI-012, according to KALA.
- As such: The company has decided to “cease development” of its lead asset (along with its MSC-S platform).
And that’s it?
For KIO-102’s PCED indication, yes. It’s currently unclear whether preclinical evaluations will continue for those two other proposed indications we mentioned earlier.
In the interim: KALA shared plans to evaluate its strategic options as part of a broader evaluation with its “secured lender” (see here for a look at all the company’s investors)—and will be taking steps to “preserve cash.”
Preserve cash? What does that translate to for the company’s future?
The company hinted at potentially cost-saving measures, such as a workforce reduction.
- And according to some reports, this may translate to cutting 51% of its staff.
We’ll keep you posted on further developments over the coming months.