Kala Pharmaceuticals to Present Clinical Data for KPI-012, its Mesenchymal Stem Cell Secretome… | News

— Data demonstrates rapid and complete wound healing, with six out of eight patients (75%) achieving complete healing within four weeks; All remained cured through end of follow-up — — All patients with pain at baseline reported no pain at week 3 — — KPI-012 was well tolerated and no safety issues no significant was observed — — In the process of submitting Investigational New Drug (IND) application and launching the phase 2/3 trial in Q4 2022 —

ARLINGTON, Mass., May 01, 2022 (GLOBE NEWSWIRE) — Kala Pharmaceuticals, Inc. (NASDAQ: KALA), a commercial-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies for eye diseases , today reported clinical data from a Phase 1b trial of KPI-012, its new acellular secretome therapy for the treatment of serious eye diseases caused by impaired wound healing. As previously described, treatment with KPI-012 was well tolerated and resulted in significant improvements in patients with various etiologies of persistent corneal epithelial defect (PCED), with complete recovery from PCED in six of eight evaluable patients. The data will be presented on Tuesday, May 3 during a poster session at the 2022 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO).

“The clinical activity seen to date with KPI-012 is encouraging,” said Valeria Sánchez-Huerta, MD FACS, Medical Director of Asociación para Evitar la Ceguera en México (Association for the Prevention of Blindness in Mexico ) and investigator in the phase 1b trial. “PCED is an impaired corneal wound healing disease that, if left untreated, can lead to infection, corneal perforation, and irreversible vision loss. Achievement of rapid and complete wound healing, as well as reduction of PCED-related pain and improvement in visual acuity and corneal opacity, in patients with a range of etiologies under underlying is remarkable, especially after such a short duration of treatment. Based on these early data, I believe KPI-012 could become the first treatment to treat PCED across all etiologies and I look forward to further evaluating its potential in future studies.

PCED, which is defined as a persistent corneal abnormality or sore that does not heal and 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 can lead to corneal ulceration, perforation , stromal scarring, secondary infections and significant vision loss.

Healing after corneal injury follows a highly coordinated process involving growth factors, cell signaling, proliferation, migration, and extracellular matrix remodeling. In patients with PCED, there is an imbalance of key biomolecules, including growth factors and cytokines, resulting in significant inflammation, impaired innervation, and disruption of the protective layers of the corneal epithelium and of the stroma. KPI-012 was designed specifically to address this imbalance: it is a novel mesenchymal stem cell (MSC) secretome derived from human bone marrow containing numerous human biomolecules including protease inhibitors, matrix proteins , growth factors and neurotrophic factors, which provide a multifactorial mechanism of action to treat corneal wound healing disorders in many etiologies.

“We are thrilled to present these exciting data from the first KPI-012 clinical trial,” said Kim Brazzell, Ph.D., head of research and development and medical director at Kala Pharmaceuticals. “These data, which served as the basis for our acquisition of Combangio last year, highlight the potential of KPI-012 to offer a new approach to the treatment of PCED, as well as other rare diseases forward. and the back of the eye. Our goal remains to advance KPI-012 into a Phase 2/3 trial for PCED later this year, as we aim to provide new and better options for people living with serious ocular surface diseases.

The poster presentation is now available on the Kala Pharmaceuticals website at https://investors.kalarx.com/presentations.

ARVO Presentation Highlights

The phase 1b, prospective, open-label, single-arm clinical trial enrolled 12 patients, including three in a preliminary safety cohort and nine in an efficacy cohort. Within the efficacy cohort, patients presented with PCED of various etiologies and durations ranging from 15 to 871 days. Patients were treated with KPI-012 twice daily for up to four weeks, with follow-up at two, four and 12 weeks after their last treatment dose. The key efficacy endpoint was complete healing of corneal defects assessed by corneal staining. Other efficacy parameters included reduction in defect size, visual acuity and corneal opacity. Safety measures included tolerance/pain, intraocular pressure and adverse events.

Eight patients were evaluable for efficacy assessment; one participant was ineligible due to an unrelated adverse event. Improvement was seen in seven of eight evaluable patients, with six of eight achieving complete healing by the end of week 4, including four patients who were healed by the end of week 1 and one patient who was healed at the end of week 2 The six cured patients remained cured until the end of the follow-up period. Additionally, improvement in PCED lesion size was observed in the two patients who did not experience complete healing. In the eight patients, the mean improvement in lesion size between the start and end of treatment was -16.23 mm. KPI-012 was well tolerated in the trial.

Clinical development plans

Kala plans to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) and, subject to regulatory approval, initiate a Phase 2/3 clinical trial of the KPI- 012 in PCED patients in Q4 2022 Kala believes this trial could be the first of two pivotal trials required. The FDA has granted orphan designation KPI-012 for the treatment of PCED and the company believes it may also qualify for fast-track and breakthrough designations.

In addition, Kala believes that KPI-012’s multifactorial mechanism of action also makes it a technology platform and is evaluating KPI-012 for potential extension to rare disease indications of the front of the eye, such as deficiency. in limbal stem cells and Sjögren’s syndrome, as well as certain rare diseases of the back of the eye, such as retinitis pigmentosa and optic neuritis.

About Kala Pharmaceuticals, Inc.

Kala is a commercial-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies for eye diseases. Kala has applied its AMPPLIFY® mucus-penetrating particle (MPP) drug delivery technology to two eye therapies, EYSUVIS® (loteprednol etabonate ophthalmic suspension) 0.25% and INVELTYS® (loteprednol etabonate ophthalmic suspension). loteprednol) 1%. The Company also has a pipeline of development programs, including a clinical-stage secretome product candidate, KPI-012, initially targeting persistent corneal epithelial defects (PCEDs) and several preclinical New Chemical Entity (NCE) development programs. ) owners aimed at addressing unmet medical needs, including diseases of the front and back of the eye. For more information about 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 contained in this press release regarding Kala’s future expectations, plans and prospects, including but not limited to statements about Kala’s expectations regarding KPI-012, future development or commercialization of the KPI -012, the conduct and timing of clinical trials, Kala’s plans to advance its pipeline of preclinical development programs aimed at treating diseases in the front and back of the eye, are forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements due to a variety of important factors, including those discussed in the “Risk Factors” section of Kala’s Annual Report on Form 10-K, the most recently filed quarterly report on Form 10-Q and other documents filed by Kala with the Securities and Exchange Commission. These forward-looking statements represent the views of the company as of the date of this release and should not be relied upon as representing the views of Kala as of any date subsequent to the date hereof. Kala undertakes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Investor contacts:

Jill Steier [email protected] 781-996-5252

Hannah Deresiewicz [email protected] 212-362-1200

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