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
reached alignment with the U.S. Food and Drug Administration (FDA)
on a registrational path forward for intravenous (IV) Choline
Chloride, an investigational phospholipid substrate replacement.
The Company had previously been pursuing an indication in
intestinal failure-associated liver disease (IFALD) and following
feedback from FDA, will now pursue a broader indication in patients
on parenteral nutrition (PN) who are or may become unable to
synthesize choline from oral or enteral nutrition sources. The
Company plans to advance the development of IV Choline Chloride as
a source of choline for adult and adolescent patients on long-term
PN. The FDA has granted IV Choline Chloride Orphan Drug Designation
for the prevention of choline deficiency in PN patients.
“There are currently no IV formulations of
choline available or in development for PN patients. The FDA
recognizes this as a serious unmet need and has been instrumental
in helping us define an efficient regulatory path to provide a
much-needed source of IV choline for these patients,” said Jesse
Shefferman, Chief Executive Officer of Protara Therapeutics. “We
look forward to advancing the clinical development of IV Choline
Chloride, which we believe has the potential to become the first
FDA approved IV choline therapy for patients dependent on PN. In
parallel, we remain keenly focused on advancing our lead product
candidate, TARA-002, for patients with non-muscle invasive bladder
cancer and lymphatic malformations.”
“Approximately 80 percent of PN-dependent
patients are choline-deficient and have some degree of liver
damage, which may be reversible with effective supplementation,”
said Palle Bekker Jeppesen M.D., Ph.D., Clinical Professor and Head
of the Department of Intestinal Failure and Liver Diseases,
Rigshospitalet, Copenhagen University Hospital, Copenhagen,
Denmark. “Top professional medical societies in both the U.S. and
Europe recognize the impact of long-term choline deficiency on
liver health, particularly, the development of liver disease with
progressive steatosis, fibrosis, and eventually end-stage liver
cirrhosis, and recommend treatment with choline. Access to an IV
formulation of choline has the potential to make a meaningful
impact for intestinal failure patients for whom oral or enteral
choline supplementation is not an option.”
Choline is recommended for patients on PN by the
American Society for Parenteral and Enteral Nutrition (ASPEN)
Recommendations for Changes in Commercially Available Parenteral
Multivitamin and Multi–Trace Element Products, as well as by the
European Society for Clinical Nutrition and Metabolism (ESPEN) in
their Guideline on Home Parenteral Nutrition.
The FDA indicated that a single study with an
endpoint of restoring choline levels in PN patients could serve as
the basis for a regulatory filing for IV Choline Chloride. Based on
this feedback, the Company intends to assess the safety and
efficacy of IV Choline Chloride in its planned seamless Phase 2b/3
double-blinded, randomized, placebo-controlled THRIVE-3 study in
adolescents and adults on long-term PN when oral or enteral
nutrition is not possible, insufficient, or contraindicated. The
Phase 2b portion of the study, which will seek to establish the
pharmacokinetics (PK) profile of IV Choline Chloride, will enroll
approximately 24 patients who will receive one of three daily doses
of IV Choline Chloride for 24 weeks. During the randomized,
double-blind Phase 3 portion of the study, approximately 100
patients will receive either high dose IV Choline Chloride, low
dose Choline Chloride, or placebo for 24 weeks. The primary
endpoint of this portion of the study will seek to demonstrate IV
Choline Chloride as a durable source of choline. Secondary
endpoints will assess the impact of choline replacement on liver
function. All patients will be eligible to enter an open-label
extension. The Company intends to initiate this study in the first
half of 2025.
In previous studies, treatment with IV Choline
Chloride successfully increased plasma choline concentrations in
patients on PN and was also shown to improve steatosis, a key
marker of liver injury.
About IV Choline Chloride
IV Choline Chloride is an investigational,
intravenous (IV) phospholipid substrate replacement therapy
initially in development for patients receiving parenteral
nutrition (PN). Choline is a known important substrate for
phospholipids that are critical for healthy liver function and also
plays an important role in modulating gene expression, cell
membrane signaling, brain development and neurotransmission, muscle
function, and bone health. PN patients are unable to synthesize
choline from enteral nutrition sources, and there are currently no
available PN formulations containing choline. Approximately 80
percent of PN-dependent patients are choline-deficient and have
some degree of liver damage, which can lead to hepatic failure.
There are currently no available PN formulations containing
choline. In the U.S. alone, there are approximately 40,000 patients
on long-term parenteral nutrition who would benefit from an IV
formulation of choline. IV Choline Chloride has the potential to
become the first FDA approved IV choline formulation for PN
patients. IV Choline Chloride has been granted Orphan Drug
Designation by the FDA for the prevention of choline deficiency in
PN patients. The Company was issued a U.S. patent claiming a
choline composition with a term expiring in 2041.
About Protara Therapeutics, Inc.
Protara is a clinical-stage biotechnology
company committed to advancing transformative therapies for people
with cancer and rare diseases. Protara’s portfolio includes its
lead candidate, TARA-002, an investigational cell-based therapy in
development for the treatment of non-muscle invasive bladder cancer
(NMIBC) and lymphatic malformations (LMs). The Company is
evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients
with carcinoma in situ (CIS) who are unresponsive or naïve to
treatment with Bacillus Calmette-Guérin (BCG), as well as a Phase 2
trial in pediatric patients with LMs. Additionally, Protara is
developing IV Choline Chloride, an investigational phospholipid
substrate replacement for patients on parenteral nutrition who are
otherwise unable to meet their choline needs via oral or enteral
routes. 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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