Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company
developing transformative therapies for the treatment of cancer and
rare diseases, today announced that it has received regulatory
clearance from the U.S. Food and Drug Administration (FDA) on its
planned Phase 2 STARBORN-1 trial evaluating TARA-002, an
investigational cell-based immunopotentiator, for the treatment of
pediatric patients with lymphatic malformations (LMs). Trial
initiation is expected in the fourth quarter of 2023.
“There are currently no FDA-approved treatments for LMs, a rare,
serious condition mostly affecting children,” said Jesse
Shefferman, Chief Executive Officer of Protara Therapeutics. “We
are pleased to take the next step on our journey to deliver
TARA-002 to these young patients and look forward to initiating
this Phase 2 trial in pediatric patients with LMs.”
“LMs is a devastating disease for young children and there is a
significant need for treatment options. Current management of these
malformations often includes surgical excision, which can be a
difficult procedure and have a high rate of complication and
recurrence, especially in very young children,” said Richard Smith,
M.D., Professor of Otolaryngology in the University of Iowa Carver
College of Medicine and Director of Molecular Otolaryngology and
Renal Research Laboratories and the Iowa Institute of Human
Genetics. “As the lead investigator of the largest trial ever
completed in LMs using OK-432, the predecessor therapy to TARA-002,
I am very encouraged by the progress of TARA-002 as a potential
treatment for this serious disease.”
STARBORN-1 is a Phase 2 single-arm, open-label, prospective
clinical trial to evaluate the safety and efficacy of intracystic
injection of TARA-002 for the treatment of macrocystic and mixed
cystic LMs (≥ 50% macrocystic disease) in participants six months
to less than 18 years of age. Following completion of an age
de-escalation safety lead-in, the trial will enroll approximately
30 patients who will receive up to four injections of TARA-002
spaced approximately six weeks apart.
The primary endpoint of the trial is the proportion of
participants with macrocystic LMs and mixed cystic LMs who
demonstrated clinical success, defined as having either a complete
response (90% to 100% reduction from baseline in total LM volume)
or substantial response (60% to less than 90% reduction in total LM
volume) eight weeks after the last injection, as measured by
axial imaging.
TARA-002 is an investigational cell therapy based on the broad
immunopotentiator, OK-432, which was originally granted marketing
approval by the Japanese Ministry of Health and Welfare as an
immunopotentiating cancer therapeutic agent. This cell therapy is
currently approved in Japan and Taiwan for LMs and has been used to
successfully treat thousands of pediatric patients. In addition,
OK-432 was studied in the largest ever conducted Phase 2 trials in
LMs, in which the therapy was administered via a now-closed
compassionate use program led by the University of Iowa to over 500
pediatric and adult patients in the United States.
TARA-002 has been granted Rare Pediatric Disease designation by
the FDA for the treatment of LMs.
About TARA-002 in LMs
TARA-002 is an investigational cell therapy in development for
the treatment of NMIBC and of LMs for which it has been granted
Rare Pediatric Disease Designation by the U.S. Food and Drug
Administration. TARA-002 was developed from the same master cell
bank of genetically distinct group A Streptococcus pyogenes as
OK-432, a broad immunopotentiator marketed as Picibanil® in Japan
and approved in Taiwan by Chugai Pharmaceutical Co., Ltd. Protara
has successfully shown manufacturing comparability between TARA-002
and OK-432.
When TARA-002 is administered, it is hypothesized that innate
and adaptive immune cells within the cyst or tumor are activated
and produce a strong immune cascade. Neutrophils, monocytes and
lymphocytes infiltrate the abnormal cells and various cytokines,
including interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon
(IFN)-gamma, and tumor necrosis factor (TNF)-alpha are secreted by
immune cells to induce a strong inflammatory reaction and destroy
the abnormal cells.
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 three 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,
Inc.Protara is committed to advancing transformative
therapies for people with cancer and rare diseases. 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 patients dependent on parenteral nutrition. For more
information, visit www.protaratx.com.
Forward-Looking StatementsStatements contained
in this press release regarding matters that are not historical
facts are "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995. Protara may, in
some cases, use terms such as “predicts,” “believes,” “potential,”
“proposed,” “continue,” “designed,” “estimates,” “anticipates,”
“expects,” “plans,” “intends,” “may,” “could,” “might,” “will,”
“should” or other words or expressions referencing future events,
conditions or circumstances that convey uncertainty of future
events or outcomes to identify these forward-looking statements.
Such forward-looking statements include but are not limited to,
statements regarding Protara’s intentions, beliefs, projections,
outlook, analyses or current expectations concerning, among other
things: Protara’s business strategy, including its development
plans for its product candidates and plans regarding the timing or
outcome of existing or future clinical trials; statements related
to expectations regarding interactions with the FDA, including
potential alignment with the FDA on a development path for TARA-002
in pediatric LM patients; Protara’s financial position; statements
regarding the anticipated safety or efficacy of Protara’s product
candidates; and Protara’s outlook for the remainder of the year.
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 that Protara’s financial guidance may not be as expected, as
well as 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; 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; having to use cash in ways or on timing
other than expected; the impact of market volatility on cash
reserves; the loss of key members of management; the impact of
general U.S. and foreign, economic, industry, market,
regulatory, political or public health conditions; 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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