Kala Pharmaceuticals, Inc. (NASDAQ:KALA), a commercial-stage
biopharmaceutical company focused on the discovery, development and
commercialization of innovative therapies for diseases of the eye,
today reported clinical data from a Phase 1b trial of KPI-012, its
novel, cell-free secretome therapy for the treatment of severe
ocular diseases driven by impaired healing. As previously
disclosed, treatment with KPI-012 was well tolerated and resulted
in significant improvements in patients with various persistent
corneal epithelial defect (PCED) etiologies, with complete healing
of the PCED in six of eight evaluable patients. The data will be
presented on Tuesday, May 3 in a poster session at the 2022
Association for Research in Vision and Ophthalmology (ARVO) Annual
Meeting.
“The clinical activity observed to date with KPI-012 is
encouraging,” said Valeria Sánchez-Huerta, M.D. FACS, Medical
Director at Asociación para Evitar la Ceguera en México
(Association to Prevent Blindness in Mexico) and an investigator in
the Phase 1b trial. “PCED is a disease of impaired corneal healing
which, if left untreated, can lead to infection, corneal
perforation and irreversible vision loss. Achieving rapid and
complete wound healing, as well as a reduction in PCED-related
pain, and an improvement in visual acuity and corneal opacity, in
patients with a range of underlying etiologies is remarkable,
particularly after such a short treatment duration. Based on these
early data, I believe KPI-012 could become the first treatment to
address PCED across all etiologies and I look forward to further
evaluating its potential in later-stage studies.”
PCED, 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. 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, which results in significant inflammation,
impaired innervation and disruption of the protective corneal
epithelial and stromal layers. KPI-012 was designed specifically to
address this imbalance: it is a novel, human bone marrow-derived
mesenchymal stem cell (MSC) secretome containing numerous human
biomolecules, including protease inhibitors, matrix proteins,
growth factors and neurotrophic factors, that provide a
multifactorial mechanism of action to address impaired corneal
healing across numerous etiologies.
“We are pleased to present these exciting data from the first
KPI-012 clinical trial,” said Kim Brazzell, Ph.D., Head of Research
and Development and Chief Medical Officer at Kala Pharmaceuticals.
“These data, which served as the foundation for our acquisition of
Combangio last year, highlight the potential of KPI-012 to deliver
a novel approach to treating PCED, as well as other rare front and
back of the eye diseases. Our goal remains to advance KPI-012 into
a Phase 2/3 trial for PCED later this year, as we aim to deliver
new and better options to people living with severe ocular surface
diseases.”
The poster presentation is now available on the Kala
Pharmaceuticals website at
https://investors.kalarx.com/presentations.
Highlights from the ARVO Presentation
The single-arm, prospective, open-label Phase 1b clinical trial
enrolled 12 patients, including three who were enrolled in a safety
lead-in cohort and nine enrolled 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 twice daily KPI-012 for up to four weeks, with follow-up
occurring at two, four and 12 weeks after their last dose of
therapy. The key efficacy endpoint was complete healing of the
corneal defects evaluated by corneal staining. Other efficacy
endpoints included reduction in defect size, visual acuity, and
corneal opacity. Safety measures included tolerability/pain,
intraocular pressure and adverse events.
Eight patients were evaluable for efficacy assessment; one
participant was ineligible due to a non-treatment related adverse
event. Improvement was seen in seven of the eight evaluable
patients, with six of the 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 by the end of Week 2. All
six healed patients remained healed through the end of the
follow-up period. In addition, improvement in PCED lesion size was
observed in both patients who did not experience full wound
healing. Across all eight patients, the mean improvement in lesion
size from baseline to 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 clearance, initiate a Phase 2/3 clinical trial of
KPI-012 in PCED patients in the fourth quarter of 2022. Kala
believes this trial could serve as the first of two required
pivotal trials. The FDA has granted KPI-012 Orphan Designation for
the treatment of PCED and the Company believes it could also meet
the criteria for fast-track and breakthrough designations.
In addition, Kala believes the multifactorial mechanism of
action of KPI-012 also makes it a platform technology and is
evaluating KPI-012 for potential expansion
to indications for rare front of the eye diseases,
such as limbal stem cell deficiency and Sjogren’s Syndrome, as well
as select rare back of the eye diseases, 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 diseases of the eye. Kala has applied its
AMPPLIFY® mucus-penetrating particle (MPP) Drug Delivery
Technology to two ocular therapies, EYSUVIS® (loteprednol
etabonate ophthalmic suspension) 0.25% and
INVELTYS® (loteprednol etabonate ophthalmic suspension) 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 (PCED) and multiple
proprietary new chemical entity (NCE) preclinical development
programs targeted to address unmet medical needs, including both
front and back of the eye diseases. 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 KPI-012, the future development or
commercialization of KPI-012, conduct and timelines of clinical
trials, Kala’s plans to progress its pipeline of preclinical
development programs targeted to address front and back of the eye
diseases, 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
those discussed in the “Risk Factors” section of Kala’s Annual
Report on Form 10-K, most recently filed Quarterly Report on Form
10-Q and other filings Kala makes with the Securities and Exchange
Commission. These forward-looking statements represent the
Company’s views as of the date of this release and should not be
relied upon as representing the 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 Contacts:
Jill Steierjill.steier@kalarx.com 781-996-5252
Hannah Deresiewiczhannah.deresiewicz@sternir.com
212-362-1200
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