Kala Pharmaceuticals Receives FDA Fast Track Designation for KPI-012, a Human MSC-S Therapy for Persistent Corneal Epithelial Defect
12 Abril 2023 - 7:00AM
Kala Pharmaceuticals, Inc. (NASDAQ:KALA), a clinical-stage
biopharmaceutical company dedicated to the research, development
and commercialization of innovative therapies for rare and severe
diseases of the eye, today announced that the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation for Kala’s
human mesenchymal stem cell secretome (MSC-S) therapy (KPI-012) for
the treatment of persistent corneal epithelial defect (PCED), a
rare and debilitating ocular condition.
“There is a significant unmet need for patients suffering from
PCED, which can lead to many complications and sequelae, including
vision impairment, infection, corneal perforation and significant
pain and discomfort. There are currently no approved pharmaceutical
treatments for the majority of PCED patients, and KPI-012’s
multifactorial mechanism of action is a potential solution to
address all underlying etiologies of PCED,” said Kim Brazzell,
Ph.D., Head of R&D and Chief Medical Officer at Kala
Pharmaceuticals. “We look forward to working with the Agency to
efficiently advance KPI-012 through the development and review
process to fulfill our greater mission of delivering a treatment to
the roughly 100,000 patients in the U.S., and thousands more
globally, suffering from PCED.”
Fast Track is a process designed by the FDA to facilitate the
development and expedite the review of drug candidates intended to
treat serious conditions and for which nonclinical and/or clinical
data demonstrate the potential to address unmet medical need. The
purpose of this designation is to help speed development of new
drugs, making them available to the patient more quickly. A company
with a therapeutic candidate that receives Fast Track designation
may be eligible for more frequent interactions with the FDA to
discuss the product development plan. Therapeutic candidates with
Fast Track designation may also be eligible for priority review and
accelerated approval if supported by clinical data.
In February 2023, Kala dosed the first patient in its CHASE
(Corneal Healing
After SEcretome therapy) Phase 2b
trial evaluating KPI-012 for the treatment of PCED. In March 2023,
Kala announced positive safety data from the first cohort of two
patients that were treated with a high dose of KPI-012 (3 U/mL)
four times per day (QID). Both patients successfully completed at
least one week of dosing with no safety issues observed and the
trial has now advanced to Cohort 2. The second cohort is a
multicenter, randomized, double-masked, vehicle-controlled,
parallel-group study to evaluate the safety and tolerability of two
doses of KPI-012 in ophthalmic solution (3 U/mL and 1 U/mL) versus
vehicle dosed topically QID for 56 days in approximately 90
patients. The primary endpoint of the trial is the complete healing
of the PCED as measured by corneal fluorescein staining. Kala is
targeting reporting topline safety and efficacy data in the first
quarter of 2024. If the results are positive, and subject to
discussion with regulatory authorities, Kala believes this trial
could serve as the first of two pivotal trials required to support
the submission of a Biologics License Application (BLA) to the
FDA.
About KPI-012 for Persistent Corneal Epithelial Defect
(PCED)
Persistent corneal epithelial defect, 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 and
238,000 cases per year in the United States, European Union and
Japan combined. 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, if left
untreated, can lead to infection, corneal ulceration or
perforation, scarring, opacification and significant vision
loss.
Based on a multifactorial mechanism of action and preclinical
and clinical data generated to-date, Kala believes KPI-012 may
represent a significant advancement in the treatment of PCED and
could become the first approved treatment for PCED across all its
various etiologies.
About Kala Pharmaceuticals
Kala is a clinical-stage biopharmaceutical company dedicated to
the research, development and commercialization of innovative
therapies for rare and severe diseases of the eye. Kala’s
biologics-based investigational therapies utilize Kala’s
proprietary mesenchymal stem cell secretome (MSC-S) platform.
Kala’s lead product candidate, KPI-012, is a human MSC-S, which
contains numerous human-derived biofactors, such as growth factors,
protease inhibitors, matrix proteins and neurotrophic factors that
can potentially correct the impaired corneal healing that is an
underlying etiology of multiple severe ocular diseases. KPI-012 is
currently in clinical development for the treatment of persistent
corneal epithelial defect (PCED), a rare disease of impaired
corneal healing, for which it has received Orphan Drug and Fast
Track designations from the U.S. Food and Drug Administration. Kala
is also targeting the potential development of KPI-012 for the
treatment of Limbal Stem Cell Deficiency and ocular manifestations
of moderate-to-severe Sjögren's and has initiated preclinical
studies to evaluate the potential utility of its MSC-S platform for
retinal degenerative diseases, such as Retinitis Pigmentosa and
Stargardt Disease. 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 potential advantages of KPI-012 and
its MSC-S platform; the clinical utility of KPI-012 for PCED;
Kala’s plans to pursue research and development of KPI-012 and its
MSC-S platform for other indications; the benefits of a Fast Track
designation by the FDA; and other statements containing the words
“anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,”
“plan,” “predict,” “project,” “target,” “potential,” “likely,”
“will,” “would,” “could,” “should,” “continue,” and similar
expressions 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:
uncertainties inherent in the initiation and conduct of preclinical
studies and clinical trials; uncertainties regarding availability
and timing of data from clinical trials; whether results of early
clinical trials or trials in different disease indications will be
indicative of the results of ongoing or future trials; whether
results of the Phase 1b clinical trial of KPI-012 will be
indicative of results for any future clinical trials and studies of
KPI-012, including the CHASE Phase 2b clinical trial; whether
interim data from a clinical trial will be predictive of the
results of the trial; uncertainties associated with regulatory
review of clinical trials and applications for marketing approvals;
Kala’s ability to retain and hire key personnel; the impact of
extraordinary external events, such as the current pandemic health
event resulting from the coronavirus (COVID-19), and their
collateral consequences; the sufficiency of cash resources and need
for additional financing and other important factors, any of which
could cause Kala’s actual results to differ from those contained in
the forward-looking statements, discussed in the “Risk Factors”
section of Kala’s Annual Report on Form 10-K and other filings Kala
makes with the Securities and Exchange Commission. These
forward-looking statements represent Kala’s views as of the date of
this press release and should not be relied upon as representing
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 Contact:
Hannah Deresiewiczhannah.deresiewicz@sternir.com
212-362-1200
KALA BIO (NASDAQ:KALA)
Gráfica de Acción Histórica
De May 2024 a Jun 2024
KALA BIO (NASDAQ:KALA)
Gráfica de Acción Histórica
De Jun 2023 a Jun 2024