Protara Therapeutics Announces Results from a Retrospective Study Evaluating the Prevalence of Cholestasis in Patients Dependent on Parenteral Nutrition
September 13 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 the completion of a retrospective prevalence study
designed to enhance understanding of the incidence of intestinal
failure associated liver disease (IFALD) in patients dependent on
parenteral nutrition (PN). The study found that approximately 30%
of patients who are dependent on PN have cholestasis, a hallmark
pathology of IFALD, despite the use of current medical management
in these patients. The Company is currently developing intravenous
(IV) Choline Chloride, an investigational phospholipid substrate
replacement therapy, for the treatment of patients receiving PN who
have IFALD.
“In addition to steatosis, we know that cholestasis is a core
feature of IFALD and data from this prevalence study further
underscore the significant need for an effective intervention for
patients with IFALD, which carries a particularly poor prognosis in
the absence of an intestine-liver transplant,” said Alan Buchman,
M.D., Professor of Clinical Surgery and Medical Director,
Intestinal Rehabilitation and Transplant Center, University of
Illinois at Chicago. “Patients who are dependent on PN are unable
to absorb sufficient levels of choline, an essential component of
several metabolic processes, ultimately resulting in the
development of IFALD. Results from the previously completed Phase 2
study of IV Choline Chloride support the clinical potential of
choline substrate replacement therapy to treat IFALD.”
“Findings from this retrospective study
reinforce that there are a significant number of patients dependent
on PN who are suffering from IFALD who may potentially benefit from
treatment with IV Choline Chloride,” said Jesse Shefferman, Chief
Executive Officer of Protara Therapeutics. “We remain committed to
strengthening the understanding of the unmet need in this
devastating disease, for which there are no approved
therapies.”
In order to further characterize the prevalence and needs of
IFALD patients dependent on PN, the Company recently initiated a
prospective, multi-center, cross-sectional observational study that
will assess the prevalence of choline deficiency, as well as
cholestasis and steatosis, in approximately 300 patients dependent
on PN.
The Company expects to use the results from the completed
retrospective study and ongoing prospective study to inform next
steps for its IV Choline Chloride development program. As
previously announced, the Company held a positive end of Phase 2
meeting with the U.S. Food and Drug Administration (FDA) and
received feedback on the design of studies necessary to complete
the registration package for IV Choline Chloride for the treatment
of IFALD, including a Phase 1 pharmacokinetic study and a Phase 3
placebo-controlled study.
Retrospective Study Results
The retrospective, observational study was conducted in
partnership with a large home health organization and examined data
from 468 patients dependent on PN for six months or more. The
primary endpoint of the study was to identify the proportion of
patients dependent on PN with suspected liver disease defined as
serum alkaline phosphatase (ALP) levels greater than 1.5 times the
upper limit of normal (ULN). The study evaluated ALP levels from
baseline up to 36 months to determine if there is a progressive
component to cholestasis in IFALD and the degree to which medical
management affected ALP levels. ALP is an established biomarker for
cholestasis and a clinically meaningful indicator of IFALD severity
and progression. Prolonged elevation of ALP is indicative of
ongoing hepatocellular injury.
Key findings are summarized below:
- Approximately 31% of all patients, irrespective of baseline
levels, presented with ALP levels greater than 1.5 times the ULN at
any given time over a 30-month period.
- Approximately 28% of all patients had persistent ALP elevations
greater than 1.5 times the ULN at 36 months.
- At baseline, approximately 23% of patients presented with ALP
levels greater than 1.5 times the ULN.
- In these patients, approximately 76% presented with greater
than 1.5 times the ULN at any given time over a 30-month period and
approximately 59% had persistent ALP elevations greater than 1.5
times the ULN at 36 months.
- While medical management demonstrated some improvement in ALP
levels, it was not sufficient for managing ALP levels over the long
term in patients on PN.
- Results support further exploration in this patient population
to determine rates of choline deficiency and steatosis.
About IV Choline Chloride and Intestinal
Failure-associated Liver Disease (IFALD)
IV Choline Chloride is an investigational,
intravenous (IV) phospholipid substrate replacement therapy
initially in development for patients receiving parenteral
nutrition (PN) who have IFALD. Choline is a known important
substrate for phospholipids that are critical for healthy liver
function. Because PN patients cannot sufficiently absorb adequate
levels of choline and no available PN formulations contain
sufficient amounts of choline to correct this deficiency, PN
patients often experience a prolonged progression to hepatic
failure and death, with the only known intervention being a dual
small bowel/liver transplant. If approved, IV Choline Chloride
would be the first approved therapy for IFALD. It has been granted
Orphan Drug Designations (ODDs) by the FDA for the treatment of
IFALD and the prevention of choline deficiency in PN patients.
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 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 plans with respect to the IV Choline Chloride program
and the impact of the study results discussed in this press release
on such plans, Protara’s beliefs regarding the benefit of IV
Choline Chloride for patients dependent on PN who are suffering
from IFALD, Protara’s development plans for its product candidates
and related expectations regarding interactions or upcoming filings
with the FDA and plans regarding the timing or outcome of existing
or future non-clinical studies and clinical trials. 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 sales, revenue, expense and other 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; 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; 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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