Full Press Release Details
Protara Therapeutics Announces Positive Preliminary
Data from ADVANCED-1 Phase 1a
Dose Escalation Trial of TARA-002 in NMIBC Supporting Advancement into Phase 2
Clinical Development
Company's current resources are expected to fund operations into 2025 supporting the initiation of the ADVANCED-2 trial in BCG-na ve
CIS patients and BCG-unresponsive CIS patients in 2H'2023
New York, April 28, 2023 -- 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-Gu rin (BCG)-na ve and BCG-unresponsive in the second half of this year."
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-na ve,
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-Na ve 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
ADVANCED-1 is a Phase 1 dose-finding, open-label
trial (NCT05085977) evaluating TARA-002 in treatment-na ve 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.
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
About Non-Muscle Invasive Bladder Cancer
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.
1. Klinische Einheit, or KE, is a German term indicating a specified
number of dried cells in a vial.
Forward-Looking Statements
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safety or efficacy of Protara's product candidates. Because such statements are subject to risks and uncertainties, actual results
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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
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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.
Protara Therapeutics