On August 11, 2025 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the second quarter ended June 30, 2025, and provided a business update (Press release, Protara Therapeutics, AUG 11, 2025, View Source [SID1234655074]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"As we look toward the second half of the year, we believe we are well positioned to continue to advance our pipeline of potentially transformative therapies for the treatment of patients with cancer and rare diseases," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We continue to execute on our ADVANCED-2 trial of TARA-002 in non-muscle invasive bladder cancer (NMIBC) and look forward to presenting interim results in the first quarter of 2026. In addition, we remain on track to dose the first patient in our THRIVE-3 registrational trial of IV Choline Chloride in patients dependent on parenteral support (PS) by the end of this quarter and expect to provide an interim update from the STARBORN-1 trial in pediatric patients with lymphatic malformations (LMs) in the fourth quarter of this year."

Recent Progress and Highlights

TARA-002 in NMIBC

The Company expects to report interim results from approximately 25 six-month evaluable NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are BCG-Unresponsive at a medical conference in the first quarter of 2026.
Following discussions with the U.S. Food and Drug Administration (FDA), the Company expects to provide an update on next steps in its BCG-Naïve program in the second half of 2025.
Protara continues to investigate subcutaneous dosing through priming and maintenance combined with intravesical dosing, as well as exploring combination treatments with TARA-002 in NMIBC patients with CIS.
IV Choline Chloride for Patients on Parenteral Support (PS)

The Company remains on track to dose the first patient in THRIVE-3, a registrational Phase 3 clinical trial, in the third quarter of 2025. In addition, following the recent clinical trial approval by the European Union Clinical Trials Regulation, the Company expects to begin enrollment across the EU in the coming months. THRIVE-3 is a seamless Phase 2b/3 trial with a dose confirmation portion (n=24) followed by a double-blinded, randomized, placebo-controlled portion to assess the efficacy and safety of intravenous (IV) Choline Chloride over 24 weeks in adolescents and adults on long-term PS when oral or enteral nutrition is not possible, insufficient, or contraindicated (n=105). IV Choline Chloride was previously granted Fast Track designation by the FDA.
TARA-002 in LMs

Dosing remains ongoing in the Phase 2 STARBORN-1 trial of TARA-002 in pediatric patients with macrocystic and mixed cystic LMs and the Company intends to provide an interim update from the trial in the fourth quarter of 2025. The Company previously announced the completion of the study’s first safety cohort, in which TARA-002 showed promising results and was generally well-tolerated.
Corporate Update

In June 2025, Protara announced the appointment of William "Bill" Conkling as Chief Commercial Officer. He brings to Protara more than two decades of experience developing and commercializing novel cancer and rare disease therapeutics.
In June 2025, the Company announced it has been added as a member of the broad-market Russell 3000 Index as part of the annual reconstitution, effective at the open of U.S. equity markets on June 30, 2025.
Second Quarter 2025 Financial Results

As of June 30, 2025, unrestricted cash and cash equivalents and investments in marketable debt securities totaled $145.6 million. The Company expects its cash, cash equivalents, and investments in marketable debt securities will be sufficient to fund operations into mid-2027.
Research and development expenses for the second quarter of 2025 increased to $10.8 million from $6.4 million for the prior year period. The increase was primarily due to a $3.9 million increase in clinical trial activities for TARA-002 and IV Choline Chloride.
General and administrative expenses for the second quarter of 2025 increased to $5.8 million from $4.3 million for the prior year period. This increase was primarily due to an increase of $0.6 million in personnel-related expenses and $0.5 million in market development-related expenses.
For the second quarter of 2025, Protara incurred a net loss of $15.0 million, or $0.35 per share, compared with a net loss of $9.5 million, or $0.45 per share, for the same period in 2024.
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 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 pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma IL-6, IL-10, 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.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

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 Lymphatic Malformations (LMs)

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride

IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function that also play an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 78% of patients dependent on PS are choline-deficient and of those approximately 63% have some degree of liver dysfunction, which can lead to hepatic failure. Every year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term PS. IV Choline Chloride has the potential to become the first U.S. Food and Drug Administration (FDA) approved IV choline formulation for PS patients. It has been granted Orphan Drug Designation by the FDA for the prevention and/or treatment of choline deficiency in patients on long-term PN and been granted Fast Track Designation as a source of choline when oral or enteral nutrition is not possible, insufficient, or contraindicated. The U.S. Patent and Trademark Office has issued us a U.S. patent claiming a choline composition and a U.S. patent claiming a method for treating choline deficiency with a choline composition, each with a term expiring in 2041.