Protara Therapeutics, Inc.
(Nasdaq: TARA), a
clinical-stage company developing transformative therapies for the
treatment of cancer and rare diseases, today announced positive
preliminary results from the Phase 1a dose-escalation component of
its ongoing ADVANCED-1 clinical trial of TARA-002, the Company’s
investigational cell-based therapy, for the treatment of patients
with high-grade non-muscle invasive bladder cancer (NMIBC). The
clinical data indicate that TARA-002, a novel intravesical
monotherapy, was generally well tolerated and showed anti-tumor
activity in high-grade NMIBC patients. The data will be featured
during a moderated poster session at the American Urological
Association (AUA) 2023 Annual Meeting being held in Chicago from
April 28, 2023 to May 1, 2023.
“These promising results suggest TARA-002 may
provide meaningful benefit to patients with NMIBC, who currently
have limited treatment options,” said Neal Shore, M.D., Medical
Director, Carolina Urologic Research Center, Chief Medical Officer,
GenesisCare US, and study investigator. “These data show favorable
tolerability and initial evidence of anti-tumor activity, thus
serving as an impetus to advance TARA-002 into larger, later-stage
trials.”
“We are highly encouraged by preliminary results
from the dose-escalation component of ADVANCED-1 and look forward
to deepening our understanding of TARA-002’s potential in the
ongoing expansion trial, which is currently enrolling NMIBC
patients with carcinoma in situ (CIS),” said Jathin Bandari, M.D.,
Chief Medical Officer of Protara Therapeutics. “We plan to
initiate larger clinical trials in NMIBC patients with CIS who are
Bacillus Calmette-Guérin (BCG)-naïve and BCG-unresponsive in the
second half of this year.”
Preliminary Results
- TARA-002 was generally well
tolerated at all three dose levels evaluated in the trial, and no
dose limiting toxicities were observed. A maximum tolerated dose
was not determined, and dose escalation remains ongoing in
exploratory cohorts. The Company has selected the 40KE1 dose for
use in subsequent clinical trials.
- The majority of reported adverse
events were Grades 1 and 2 across all dose levels, and
treatment-related adverse events, as assessed by study
investigators, were in line with typical responses to bacterial
immunopotentiation, and included fatigue, headache, fever, and
chills. The most common urinary symptoms were urinary urgency,
urinary frequency, urinary tract pain/burning, incomplete emptying,
and bladder spasm. Most bladder irritations resolved soon after
administration or in a few hours to a few days.
- A total of nine patients were
enrolled in the study, including three patients with CIS who
reached the three-month efficacy assessment. Of those three
patients with CIS, one heavily pre-treated BCG-unresponsive patient
achieved a complete response (CR) at the 20KE dose, and tumor
regression was observed in the other two patients.
A copy of the AUA poster will be available in
the Events and Presentations section of the Company’s website:
https://ir.protaratx.com. The Company plans to present complete
results from the ADVANCED-1 study at a subsequent medical
conference.
Patient enrollment is ongoing in the open-label
expansion trial (ADVANCED-1EXP), which is evaluating intravesical
TARA-002 at the 40KE dose in 12 CIS patients, including BCG-naïve,
BCG-unresponsive, and BCG-inadequately treated patients.
Clinical Development Plan
Update
Based on these results, Protara is advancing the
clinical development of TARA-002 for the treatment of NMIBC. The
Company plans to initiate ADVANCED-2, a Phase 1b/2 open-label trial
evaluating intravesical TARA-002 in up to 102 patients with CIS.
The Phase 1b trial is expected to enroll 27 patients with CIS (±
Ta/T1), BCG-Naïve or BCG-experienced, who have not received
intravesical BCG for at least 24 months prior to CIS diagnosis. The
Phase 2 trial is expected to enroll 75 patients with
BCG-unresponsive CIS (± Ta/T1). ADVANCED-2 is expected to initiate
in the second half of 2023.
About ADVANCED-1
ADVANCED-1 is a Phase 1 dose-finding, open-label
trial (NCT05085977) evaluating TARA-002 in treatment-naïve and
treatment-experienced NMIBC patients with carcinoma in situ (CIS)
and high-grade papillary tumors (Ta). In the initial dose
escalation phase of the trial, patients received six weekly
intravesical doses of TARA-002 evaluating the 10KE, 20KE and 40KE
doses. The primary objective of the trial was to evaluate the
safety, tolerability and preliminary signs of anti-tumor activity
of TARA-002, with the goal of establishing a recommended dose for a
future Phase 2 clinical trial.
About TARA-002
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 Non-Muscle Invasive Bladder
Cancer
Bladder cancer is the 6th most common cancer
in the United States, with NMIBC representing
approximately 80% of bladder cancer diagnoses. Approximately 65,000
patients are diagnosed with NMIBC in the United
States each year. NMIBC is cancer found in the tissue that
lines the inner surface of the bladder that has not spread into the
bladder muscle.
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.
Reference:
1. Klinische Einheit, or KE, is a German term indicating a
specified number of dried cells in a vial.
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 non-clinical studies and clinical trials, and statements
regarding the anticipated safety or efficacy of Protara’s product
candidates. 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 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; 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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