Kala Bio's CHASE trial misses primary endpoint, KPI-012 development discontinued

Kala Bio announced that CHASE (NCT05727878), its phase 2b clinical trial of KPI-012 for persistent corneal epithelial defect (PCED), did not meet its primary endpoint. Additionally, the company noted that the trial failed to show statistical significance in the key secondary efficacy endpoints.¹

Kala defines KPI-012 as an “ophthalmic solution of human bone marrow-derived secretome that contains a wide array of extracellular matrix components and biofactors, including growth factors, neurotrophic factors, and cytokines.”

PCED is defined as a “persistent non-healing corneal defect or wound that is refractory to conventional treatments.” According to Kala, only 100,000 cases are reported per year in the United States.

The company had previously announced enrollment in the trial was completed in July 2025. In the trial, 79 patients were randomized across 37 sites across the United States and Latin America.²

The trial was a multicenter, double-masked, vehicle-controlled, parallel-group study evaluating the safety and efficacy of KPI-012 ophthalmic solution at 2 different doses, 3 U/mL and 1 U/mL.²

According to the company, due to the failed results, it plans to cease development on KPI-012. KPI-012 had previously received both orphan drug and fast track designations from the FDA. In addition to ceasing production on KPI-012, the company also plans to end its mesenchymal stem cell secretome (MSC-S) platform.

The company plans to reduce its workforce, but no details on the number of workers or a timeline were released by the company. The company also noted it plans to perform “other cost-saving measures,” which it did not detail.¹

Kim Brazzell, PhD, head of R&D and chief medical officer of Kala Bio, commented on the failed trial in a press release from the company.

“We are disappointed to see the results of the CHASE study given the positive results KPI-012 yielded in the Phase 1b study. KPI-012 continued to be well-tolerated and demonstrated a favorable safety profile but did not demonstrate the efficacy results that would warrant advancing the program for treatment of front-of-the-eye diseases. We would like to thank all of the patients and investigators who participated in the CHASE trial,” said Brazzell.

Data was presented from the phase 1b trial at ARVO 2022, and in the trial, rapid and complete wound healing was seen in 6 of 8 patients with various PCED etiologies with KPI-012 treatment. Additionally, it was safe and well-tolerated.³

As of writing, Kala’s stock has dropped by approximately 90%, going from about $19 a share to $1.90 a share.⁴

References:

1. KALA BIO Announces Topline Results from CHASE Phase 2b Clinical Trial Evaluating KPI-012 for the Treatment of Persistent Corneal Epithelial Defect (PCED) Did Not Meet Primary Endpoint. Published September 29, 2025. Accessed September 29, 2025. https://www.globenewswire.com/news-release/2025/09/29/3157682/0/en/KALA-BIO-Announces-Topline-Results-from-CHASE-Phase-2b-Clinical-Trial-Evaluating-KPI-012-for-the-Treatment-of-Persistent-Corneal-Epithelial-Defect-PCED-Did-Not-Meet-Primary-Endpoin.html

2. Filkins K. KALA Bio completes enrollment of CHASE clinical trial. Published July 12, 2025. Accessed September 29, 2025. https://www.ophthalmologytimes.com/view/kala-bio-completes-enrollment-of-chase-clinical-trial

3. Sanchez-Huerta V, Quiroz-Mercado H, Graue-Hernandez E, et al; Results of a Phase 1b clinical trial of KPI-012, a novel secretome therapy, in participants with Persistent Corneal Epithelial Defect (PCED). Presented at ARVO 2022. https://www.kalarx.com/assets/pdf/KPI-012_Ph1b_Study_Results_ARVO_2022.pdf

4. KALA Bio (KALA) Stock Plummets 92% After Failed Trial. Published September 29, 2025. Accessed September 29, 2025. https://www.gurufocus.com/news/3123738/kala-bio-kala-stock-plummets-92-after-failed-trial