RARE Daily

Kala Acquires Combangio to Expand its Pipeline Targeting Rare Ocular Surface Diseases

November 16, 2021

Kala Pharmaceuticals said it has acquired Combangio, a private, clinical-stage company developing regenerative biotherapies for severe ocular surface diseases.

Photo: Mark Iwicki, CEO of Kala

The acquisition gives Kala Combangio’s novel investigational secretome therapy, now known as KPI-012, which is in development to address the complex wound healing process in persistent corneal epithelial defect (PCED) and other severe ocular diseases driven by impaired corneal healing.

Under the terms of the agreement, Kala acquired all of the outstanding equity of Combangio, and the former Combangio equity holders are entitled to receive an upfront payment of an aggregate of $5.0 million in cash, subject to customary adjustments, and an aggregate of 7.8 million shares of Kala common stock with an aggregate value of approximately $16.1 million.

The former Combangio equity holders are also entitled to receive up to an aggregate of $105 million in cash and Kala stock upon the achievement by KPI-012 of specified development, regulatory and sales milestones and tiered royalties on worldwide net sales of KPI-012, if any, at a rate in the mid to high single digits. In addition, the former Combangio equity holders are entitled to receive a percentage rate in the high single digits of any income received by Kala from a commercial out-license of KPI-012.

“KPI-012 is currently in development for PCED, with potential application across a wide range of orphan diseases of the eye,” said Mark Iwicki, CEO of Kala. “This product candidate is a natural fit with our R&D and commercial expertise, and along with our internal pipeline provides an additional opportunity to leverage our deep ophthalmic expertise to address substantial, underserved markets.”

KPI-012 is a novel bone marrow-derived mesenchymal stem cell (MSC) secretome comprised of biomolecules secreted by MSCs, including protease inhibitors, growth factors and neurotrophic factors, processed into a topical ocular solution. KPI-012 is currently in clinical development for the treatment of PCED, which is defined as a persistent non-healing corneal defect or wound that is refractory to conventional treatments. It is a rare disease with an estimated incidence in the United States of 100,000 cases and 238,000 cases in the United States, European Union, and Japan combined. PCED can have various etiologies including neurotrophic keratitis, epithelial debridement, microbial/viral keratitis, corneal transplant, limbal stem cell deficiency and trauma, and can lead to corneal ulceration, perforation, scarring, infection, and significant vision loss.

KPI-012 has received Orphan designation for the treatment of PCED by the U.S. Food and Drug Administration.

In a phase 1b clinical trial, seven of eight PCED patients treated with KPI-012 twice-daily showed improvement in their PCED, with six of the eight achieving complete healing during the treatment period, which ranged from one to eight weeks. Four of eight patients had complete healing within one week of treatment with the other two that achieved complete healing doing so within two to four weeks of initiation of treatment with KPI-012. All six of the healed patients remained healed through the end of follow-up, which ranged between eight and 19 weeks. There was also significant pain relief in the six patients who reported pain at baseline, with all six achieving a zero-pain score within three weeks of initiation of dosing with KPI-012. KPI-012 was well-tolerated in the trial with no treatment-related safety issues observed.

“The phase 1b clinical data are very encouraging for PCED as well as for other rare ocular diseases that involve impaired corneal wound healing. We look forward to working together with our extensive network of corneal specialists to advance this program through clinical development and regulatory approval.” said Kim Brazzell, chief medical officer of Kala.

Kala plans to submit an investigational new drug (IND) application to the FDA and, subject to regulatory clearance, initiate a phase 2/3 trial of KPI-012 in the third quarter of 2022. Kala believes this trial could serve as the first of two required pivotal trials.

In addition to PCED, Kala is evaluating other orphan diseases driven by impaired corneal wound healing, such as thermal/chemical injury, corneal ulcers, ocular graft vs host disease, Stevens-Johnson syndrome and limbal cell deficiency, and is looking to initiate clinical evaluation for an additional indication after the IND submission and initiation of the PCED trial.

In conjunction with the acquisition, Mark Blumenkranz, chairman of Combangio, has been appointed to the Kala board of directors, and Darius Kharabi, president and CEO of Combangio, has been appointed as chief business officer of Kala, both effective immediately.

The board of both companies have approved the transaction and the transaction closed simultaneously with execution of definitive agreements on November 15, 2021.

Author: Rare Daily Staff

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