Protara Therapeutics Provides Regulatory Update for TARA-002 for the Treatment of Lymphatic Malformations
April 23 2021 - 7:00AM
Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical stage company
developing transformative therapies for the treatment of cancer and
rare diseases with significant unmet needs, today announced a path
forward related to TARA-002 for the treatment of Lymphatic
Malformations (LMs), which are rare malformations of the lymphatic
vasculature for which there is no U.S. Food and Drug Administration
(FDA)-approved treatment. Based on feedback from the FDA, the
Company intends to complete confirmatory, large-scale, GMP
manufacturing comparability in the second half of 2021 and
subsequently initiate a clinical study in pediatric LM patients
pending alignment with FDA on study design.
“With the benefit of the recent FDA feedback, we will work with
the agency to align on a clinical study in pediatric LM patients,
which, we believe, combined with the existing dataset for OK-432
(the originator compound for TARA-002), which demonstrated
treatment effect and support for strong safety profile in over 500
LM patients, should provide a robust data package for this rare
disease,” said Jesse Shefferman, Chief Executive Officer of Protara
Therapeutics. “We have already begun preparation to initiate a
clinical study in LMs and we look forward to continued
collaboration with FDA to achieve our goal of delivering the first
approved medication for LMs to these patients and their
physicians.”
TARA-002 is derived from the same cell bank as OK-432, a broad
immunopotentiator approved in Japan and Taiwan for the treatment of
LMs, where it is currently the standard of care. In 2020, Protara
successfully demonstrated initial manufacturing comparability
between TARA-002 and the originator compound OK-432, which has been
studied in more than 500 patients in one of the largest Phase 2
trials ever conducted in LMs. TARA-002 has been granted Rare
Pediatric Disease designation by the FDA for the treatment of
LMs.
About Lymphatic Malformations
Lymphatic malformations (LMs) are rare, congenital malformations
of lymphatic vessels resulting in the failure of these structures
to connect or drain into the venous system. Most LMs are present in
the head and neck region and are diagnosed in early childhood
during the period of active lymphatic growth, with more than 50%
detected at birth and 90% diagnosed before the age of 3 years. The
most common morbidities and serious manifestations of the disease
include compression of the upper aerodigestive tract, including
airway obstruction requiring intubation and possible tracheostomy
dependence; intralesional bleeding; impingement on critical
structures, including nerves, vessels, lymphatics; recurrent
infection, and cosmetic and other functional disabilities.
About Protara Therapeutics
Protara is committed to identifying and advancing transformative
therapies for people with cancer and rare diseases with limited
treatment options. Protara’s portfolio includes its lead program,
TARA-002, an investigational cell-based therapy being developed for
the treatment of non-muscle invasive bladder cancer and lymphatic
malformations, and IV Choline Chloride, an investigational
phospholipid substrate replacement therapy for the treatment of
intestinal failure-associated liver disease. For more information,
visit www.protaratx.com
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that are not historical facts are “forward looking statements”
within the meaning of the Private Securities Litigation Reform Act
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concerning, among other things: statements regarding Protara’s
business strategy, including its plans with respect to clinical
studies and anticipated timing, Protara’s development objectives
for its product candidates and related interactions with the FDA.
Because such statements are subject to risks and uncertainties,
actual results may differ materially from those expressed or
implied by such forward-looking statements. Factors that contribute
to the uncertain nature of the forward-looking statements include
risks and uncertainties associated with: Protara’s development
programs, including the initiation and completion of non-clinical
studies and clinical trials and the timing of required filings with
the FDA and other regulatory agencies; the impact of the COVID-19
pandemic on Protara’s business and the global economy; general
market conditions; changes in the competitive landscape; changes in
Protara’s strategic and commercial plans; Protara’s ability to
obtain sufficient financing to fund its strategic plans and
commercialization efforts; the loss of key members of management;
and the risks and uncertainties associated with Protara’s business
and financial condition in general, including the risks and
uncertainties described more fully under the caption “Risk Factors”
and elsewhere in Protara’s filings and reports with the United
States Securities and Exchange Commission. All forward-looking
statements contained in this press release speak only as of the
date on which they were made and are based on management's
assumptions and estimates as of such date. Protara undertakes no
obligation to update any forward-looking statements, whether as a
result of the receipt of new information, the occurrence of future
events or otherwise, except as required by law.
Company Contact:
Justine O’MalleyProtara
TherapeuticsJustine.omalley@protaratx.com646-817-2836
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