Protara Therapeutics, Inc.
(Nasdaq: TARA), a
clinical-stage company developing transformative therapies for the
treatment of cancer and rare diseases, today announced that dosing
is now underway in the Phase 2 STARBORN-1 trial evaluating
TARA-002, an investigational cell-based immunopotentiator, for the
treatment of pediatric patients with lymphatic malformations (LMs).
“There is a pressing need for an effective
FDA-approved therapy to treat LMs, a rare condition mainly
affecting children. There are currently no such agents that are FDA
approved specifically for this indication,” said Nancy Bauman, MD,
Children's National Medical Center: Children's Research Institute,
Washington DC, and investigator for the STARBORN-1 trial. “Having
witnessed first-hand in prior studies the significant benefit
observed with OK-432, the predecessor of TARA-002, I firmly believe
that TARA-002 has the potential to play a meaningful role in this
vulnerable patient population.”
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. Including 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.
“Initiation of the STARBORN-1 trial marks an
important step forward for our LMs program and, more importantly,
the young patients impacted by this rare condition,” said Jesse
Shefferman, Chief Executive Officer of Protara Therapeutics. “We
are committed to bringing TARA-002 to patients suffering from this
devastating disease and look forward to advancing this study.”
The primary endpoint of the trial is the
proportion of participants with macrocystic and mixed cystic LMs
who demonstrate 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) 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 with this
rare condition. 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.
TARA-002 has been granted Rare Pediatric Disease designation by
the U.S. Food and Drug Administration (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 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 TARA-002 in LMs
TARA-002 is an investigational cell therapy in
development for the treatment of non-muscle invasive bladder cancer
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 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 for
patients dependent on parenteral nutrition. For more information,
visit www.protaratx.com.
Forward-Looking Statements
Statements 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; 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
Protara Therapeutics (NASDAQ:TARA)
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