Protara Therapeutics, Inc.
(Nasdaq: TARA), a
clinical-stage company developing transformative therapies for the
treatment of cancer and rare diseases, today announced the results
of a retrospective analysis of OK-432, the originator compound for
TARA-002, Protara’s investigational therapy in development for the
treatment of lymphatic malformations (LMs). LMs are serious, rare,
congenital malformations of lymphatic vessels. The results from the
analysis, which were presented during a poster presentation at the
International Society for the Study of Vascular Anomalies (ISSVA)
World Congress 2022, showed that OK-432 was clinically successful
and generally well-tolerated in the treatment of both macrocystic
and mixed-cystic LMs.
“We are pleased to share these compelling
results, which are consistent with the robust body of approximately
30 years of patient experience with OK-432,” said Richard Smith,
M.D., Department of Otolaryngology, Carver College of Medicine,
University of Iowa, and author of the study. “There are currently
no FDA-approved treatments for LMs, which are usually diagnosed in
early childhood and can lead to serious complications. These data
provide continued support for the potential of TARA-002 to
ultimately serve as an effective intervention in this highly
underserved area.”
The retrospective analysis included 246 patients
from a Phase 2 randomized study, and 275 patients from an
open-label study. The majority of participants in both studies were
six months to 18 years of age. In the first study, patients were
randomized 2:1 to receive treatment immediately (immediate
treatment group [ITG]) or delayed by six months (delayed treatment
group [DTG]). In the open-label study, patients received four doses
of OK-432 approximately six weeks apart. The primary efficacy
endpoint was clinical success (defined as complete [90%-100%] or
substantial [60%-89%] reduction in LM volume measured
radiographically) in the ITG versus spontaneous resolution of the
LM in the DTG. Efficacy was assessed two weeks post-treatment in
the randomized study, and one to six months post-treatment in the
open-label study.
Key findings are summarized below:
- Approximately 69% of patients in
the randomized study ITG achieved clinical success after six
months, while only 7.5% of patients in the DTG showed spontaneous
resolution of LMs in the same time period (p<0.0001).
- 73.1% of patients in the open-label
study achieved clinical success.
- In the randomized and open-label
studies, 10 of 219 (4.6%) and 5 of 275 (1.8%) subjects,
respectively, were reported to have treatment emergent serious
adverse events that were assessed by the investigator as related to
study drug. The most severe adverse events (SAE) were airway
obstruction and facial paralysis due to swelling post-injection
that required tracheostomy and hospitalization. Both of these
events were reported as resolved. One SAE related to OK-432 led to
discontinuation (proptosis of the eye).
- Local/systemic reactions peaked in
the first few days and resolved within two weeks.
- Patients were followed for up to
three years post treatment with no significant safety
concerns.
“We are encouraged by the growing amount of
positive data supporting OK-432 in helping patients with LMs,” said
Jesse Shefferman, Chief Executive Officer of Protara Therapeutics.
“We look forward to utilizing the robust data set for OK-432 to
support further development of TARA-002 as we work toward our goal
of delivering the first approved medication for LMs in the
U.S.”
About TARA-002
TARA-002 is an investigational cell therapy in
development for the treatment of NMIBC and 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 Taiwan by Chugai
Pharmaceutical Co., Ltd. Protara has successfully demonstrated
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, tumor necrosis factor
(TNF)-alpha, granulocyte colony-stimulating factor, and
granulocyte-macrophage colony-stimulating factor, are secreted by
immune cells to induce a strong local 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 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.
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 footing; 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; the impact of
the COVID-19 pandemic on Protara’s business and the global economy
as well as the impact on Protara’s contract research organizations,
study sites or other clinical partners; 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 or political 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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