Protara Therapeutics reported interim clinical data, regulatory updates, and its financial results for the fourth quarter and full year ended December 31, 2025.

Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage biotechnology company developing transformative therapies for the treatment of cancer and rare diseases, today provided a business update and announced financial results for the fourth quarter and full year ended December 31, 2025.

"Over the last few months, we reported meaningful clinical and regulatory progress across our portfolio of late-stage programs and are building on this momentum as we advance toward several important milestones in 2026," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "For TARA-002 in non-muscle invasive bladder cancer (NMIBC), the clinical dataset continues to support its potential as a differentiated intravesical therapy with compelling efficacy, a favorable safety profile and a streamlined approach to administration in the clinical setting for both Bacillus Calmette-Guérin (BCG)-Unresponsive and BCG-Naïve patients. We remain on track to complete enrollment in our BCG-Unresponsive registrational cohort in the ADVANCED-2 trial and to initiate the ADVANCED-3 registrational trial in BCG-Naïve patients in the second half of 2026."

Mr. Shefferman added, "For our rare disease programs, we reported positive interim results for TARA-002 in lymphatic malformations (LMs), followed by the receipt of Breakthrough Therapy and Fast Track designations for TARA-002 in LMs from the U.S. Food and Drug Administration (FDA). We are working with the FDA to bring this promising treatment to patients expeditiously and expect to provide a regulatory update in the first half of 2026. In addition, our THRIVE-3 registrational trial of intravenous (IV) Choline Chloride for patients on parenteral support remains on track, with interim results expected in the second half of 2026. With cash runway into 2028, we are well positioned to continue executing across our pipeline and advance our mission of delivering transformational therapies for patients with cancer and rare diseases."

Recent Progress and Highlights

TARA-002 in NMIBC

At the ASCO Genitourinary Cancers Symposium in February 2026, the Company announced updated interim results from the ADVANCED-2 trial in evaluable NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are BCG-Unresponsive and BCG-Naïve. As of the January 28, 2026 data cutoff:

In the BCG-Unresponsive cohort of patients, TARA-002 demonstrated a complete response (CR) rate at any time of 65.7% (23/35) with CR rates of 68.2% (15/22) at six months and 33.3% (5/12) 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). Additionally, among responders, 100% (5 of 5) maintained their CR from nine to 12 months. Re-induction therapy was successful with 61.5% (8 of 13) of re-induced patients converting to a CR at six months.

In the BCG-Naïve cohort of patients, TARA-002 demonstrated a CR rate at any time of 72.4% (21/29) with CR rates of 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). In addition, among responders, 100% (11 of 11) maintained their CR from nine to 12 months. Re-induction therapy was successful with 66.7% (4 of 6) of re-induced patients converting to a CR at six months.

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 Company has completed enrollment of the BCG-Naïve cohort and expects to complete enrollment of the BCG-Unresponsive cohort in the second half of 2026.

The Company remains on track to commence ADVANCED-3, a registrational trial of TARA-002 compared to intravesical chemotherapy in BCG-Naïve patients (who have never been exposed and those who have not received BCG within the last 24 months and are ineligible to receive BCG, have a contraindication to BCG, cannot tolerate BCG, do not have access to BCG or refuse BCG) in the second half of 2026.

TARA-002 in LMs

In January 2026, the Company announced that the FDA granted TARA-002 Fast Track and Breakthrough Therapy designations for the treatment of pediatric patients with macrocystic and mixed cystic LMs. TARA-002 was previously granted Rare Pediatric Disease designation for the treatment of LMs.

The Company remains on track to share a regulatory update on the registrational path for TARA-002 in LMs in the first half of 2026.

IV Choline Chloride for Patients on Parenteral Support (PS)

THRIVE-3, the Company's registrational Phase 3 clinical trial, is ongoing, and the Company expects to report interim results in the second half of 2026.

Corporate Update

Fourth Quarter and Full Year 2025 Financial Results

As of December 31, 2025, unrestricted cash and cash equivalents and marketable debt securities totaled $197.9 million, including proceeds from the Company's $86.3 million public offering in December 2025. The Company expects its cash and cash equivalents and marketable debt securities will be sufficient to fund its planned operations and milestones into 2028.

Research and development expenses for the fourth quarter of 2025 increased to $13.1 million from $9.5 million for the prior year period, and for the full year increased to $42.6 million compared to $31.7 million for 2024. The fourth quarter and full year increases were primarily due to the start-up and advancement of clinical trials across our portfolio.

General and administrative expenses for the fourth quarter of 2025 increased to $6.0 million from $4.8 million for the prior year period, and for the full year increased to $21.9 million compared to $17.5 million for 2024. The fourth quarter and full year increases were primarily due to increases in personnel-related expenses and other general and administrative expenses primarily related to professional and consulting services.

For the fourth quarter of 2025, Protara incurred a net loss of $17.3 million, or $0.37 per share, compared with a net loss of $12.8 million, or $0.48 per share, for the same period in 2024. Net loss for the year ended December 31, 2025 was $57.4 million, or $1.34 per share, compared with a net loss of $44.6 million, or $2.17 per share, for the year ended December 31, 2024. Net loss for the fourth quarter of 2025 included approximately $1.0 million in stock-based compensation expenses. Net loss for the year ended December 31, 2025 included approximately $3.8 million in stock-based compensation expenses.

About ADVANCED-2

ADVANCED-2 (NCT05951179) is a Phase 2 open-label trial assessing intravesical TARA-002 in non-muscle invasive bladder cancer (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 received 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. The BCG-Unresponsive cohort is designed to be registrational based on the FDA's August 2024 Draft Guidance for Industry on BCG-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment.

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 FDA. 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 within the bladder wall. 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 the 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.

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