Press Release
TARA-002 Demonstrates 68% Complete Response Rate at Six Months in BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
Favorable safety and tolerability profile with no Grade 3 or greater treatment-related adverse events
Company expects to complete enrollment of the BCG-Unresponsive cohort of the ADVANCED-2 trial in 2H 2026
Company to host conference call and webcast tomorrow at
“These interim data are highly encouraging with respect to TARA-002’s efficacy and safety,” said Raj Satkunasivam, MD, MS, FRCSC, Urologic Oncologist, Associate Professor at
“TARA-002 continues to demonstrate impressive and durable response rates with excellent safety and tolerability,” said
Updated Interim Results
BCG-Unresponsive Cohort
The interim analysis of the BCG-Unresponsive cohort includes 35 evaluable participants, of whom, 22 were evaluable at six months and 15 were evaluable at 12 months as of a
- The CR rate at any time was 65.7% (23 of 35)
- The CR rate was 68.2% (15 of 22) at six months and 33.3% (5 of 15) at 12 months
- Among responders:
- The Kaplan-Meier (KM) estimated probability of maintaining a CR for six months was 71.1% (95% CI: 46.7, 95.5)
- 100% (5 of 5) maintained their CR from nine to 12 months
- 61.5% (8 of 13) of re-induced patients converted to a CR at six months
BCG-Naïve Cohort
The interim analysis of the BCG-Naïve cohort includes 29 evaluable participants, 27 of whom, were evaluable at six months and 19 were evaluable at 12 months as of a
- The CR rate at any time was 72.4% (21 of 29)
- The CR rate was 66.7% (18 of 27) at six months and 57.9% (11 of 19) at 12 months
- Among responders:
- The KM estimated probability of maintaining a CR for six months was 73.1% (95% CI: 52.9, 93.4)
- 100% (11 of 11) maintained their CR from nine to 12 months
- 66.7% (4 of 6) of re-induced patients converted to a CR at six months
Safety and Tolerability
The majority of treatment-related adverse events (TRAEs) were Grade 1 and transient with no Grade 3 or greater TRAEs and no related serious adverse events as assessed by study investigators. No patients discontinued treatment due to TRAEs. The most common TRAEs were dysuria, bladder spasm, fatigue and micturition urgency. Most bladder irritations resolved shortly after administration or within a few hours to a few days.
“The data generated to date in these high-risk NMIBC patient populations highlight TARA-002’s potential as a meaningful addition to the treatment landscape,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “In addition to demonstrating impressive efficacy and safety, TARA-002 overcomes the limitations of existing NMIBC treatments that burden patients as well as urologists and their practices. We look forward to continuing to advance TARA-002’s clinical development as we work to bring this treatment to patients.”
Protara expects to complete enrollment of the BCG-Unresponsive registrational cohort of the ADVANCED-2 trial in the second half of 2026. Enrollment is complete in the BCG-Naïve cohort of the ADVANCED-2 trial with 31 patients, and the Company remains on track to initiate the ADVANCED-3 registrational trial in BCG-Naïve patients in the second half of 2026.
Conference Call and Webcast
Protara will host a conference call and webcast tomorrow at 8:00 a.m. ET to review the data reported this evening.
About ADVANCED-2
ADVANCED-2 (NCT05951179) is a Phase 2 open-label trial assessing intravesical TARA-002 in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive (Cohort B N=75-100) or BCG-Naïve (Cohort A N=31). Trial subjects receive an induction course, with or without a reinduction, of six weekly intravesical instillations of TARA-002, followed by a maintenance course of three weekly instillations every three months.
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, Breakthrough and Fast Track Designations by the U.S. Food and Drug Administration. TARA-002 is a first-in-class TLR2/NOD2 agonist and novel immunopotentiator derived from inactivated Streptococcus pyogenes with a mechanism of action that includes the activation of innate and adaptive immune pathways. When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma, IL-6, IL-10 and IL-12. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.
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 by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
Bladder cancer is the sixth most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses, representing approximately 65,000 patients in the U.S. 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 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, 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
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Company Contact:
Justine.OMalley@protaratx.com
646-817-2836
Source: Protara Therapeutics