On May 13, 2026 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage biotechnology company developing transformative therapies for the treatment of cancer and rare diseases, reported a business update and announced financial results for the first quarter ended March 31, 2026.

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"We’re pleased with the productive discussions we’ve had with the FDA around TARA-002 in LMs," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We intend to submit a BLA for TARA-002 in LMs based on the results of the pivotal STARBORN-1 trial in the second half of 2027. Later this month, we look forward to presenting updated safety and durability data from the STARBORN-1 trial at the ISSVA World Congress and hosting an investor webinar with KOL perspectives."

Mr. Shefferman added, "In NMIBC, we continue to establish TARA-002’s potential as a differentiated treatment through a robust clinical data set demonstrating excellent safety, promising efficacy and encouraging durability, as well as a convenient and tolerable method of administration, in both Bacillus Calmette-Guérin (BCG)-Unresponsive and BCG-Naive patients. Looking ahead, we remain focused on completing enrollment in the BCG-Unresponsive registrational cohort in the ADVANCED-2 trial and initiating the ADVANCED-3 registrational trial in BCG-Naïve patients, both in the second half of the year. Additionally, our THRIVE-3 program for IV Choline Chloride remains on track, and we expect to announce interim results in the second half of 2026."

Recent Progress and Highlights

TARA-002 in LMs

Under Breakthrough Therapy designation, Protara is engaged in an ongoing dialogue with the FDA and has received confirmation that the review of TARA-002 has been moved from the Office of Vaccines Research and Review to the Office of Therapeutic Products, which has significant experience in pediatric rare disease and is the review division for TARA-002 in NMIBC.
Based on engagement with the FDA, the Company intends to submit a BLA for TARA-002 in LMs based on the results of the pivotal STARBORN-1 trial in the second half of 2027 and will continue to submit safety and efficacy data from the trial on an ongoing basis to support the FDA’s evaluation of the risks and benefits of TARA-002 in LMs.
Protara plans to host a virtual investor webinar discussing TARA-002 in LMs at 4:30 pm ET on May 19, 2026. The event will provide an overview of TARA-002 in LMs, KOL perspectives on the unmet need and TARA-002’s potential role in the treatment paradigm. The live event and accompanying slides can be accessed visiting the Events and Presentations section of the Company’s website View Source A replay of the webcast will be archived for a limited time following the event.
The Company will present updated interim safety and durability data from the ongoing Phase 2 STARBORN-1 trial evaluating TARA-002 in macrocystic and mixed cystic LMs in a poster session at the ISSVA World Congress on May 20, 2026 in Philadelphia, Pennsylvania.
TARA-002 in NMIBC

The Company expects to complete enrollment of the BCG-Unresponsive cohort of the ADVANCED-2 trial in the second half of 2026.
The Company will present updated 12-month landmark results for TARA-002 in BCG-Naïve NMIBC patients in Cohort A of the ADVANCED-2 trial during a poster presentation at the AUA Annual Meeting on May 15, 2026 in Washington, D.C.
The Company is planning a proposed registrational trial in BCG-Naïve and potentially BCG-Exposed patients. Protara continues to engage with the FDA on aspects of the analysis plan and intends to initiate the ADVANCED-3 trial in the second 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.
First Quarter 2026 Financial Results

As of March 31, 2026, unrestricted cash and cash equivalents and marketable debt securities totaled $177.4 million. 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 first quarter of 2026 increased to $13.6 million from $9.1 million for the prior year period. This increase was primarily due to a $2.2 million increase in direct expenses for our product candidates and a $2.2 million increase in indirect expenses not directly attributable to one specific product candidate. The increase in direct expenses was primarily due to higher ongoing costs associated with the ADVANCED-2 trial for NMIBC as well as start-up costs related to the ADVANCED-3 trial for NMIBC.
General and administrative expenses for the first quarter of 2026 increased to $6.1 million from $5.0 million for the prior year period. The increase was primarily due to personnel-related expenses, including stock-based compensation.
For the first quarter of 2026, Protara incurred a net loss of $17.8 million, or $0.31 per share, compared with a net loss of $11.9 million, or $0.29 per share, for the same period in 2025. Net loss for the first quarter of 2026 included approximately $1.4 million in stock-based compensation expenses.
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, Orphan Drug, Breakthrough Therapy 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. 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.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the sixth most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses, or 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 Lymphatic Malformations

Lymphatic Malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Protara’s focus is on macrocystic and mixed cystic LMs, for which there are no currently approved therapies. They are most frequently 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 and lymphatics; recurrent infection; and cosmetic and other functional disabilities. TARA-002 has been granted Rare Pediatric Disease, Orphan Drug, Breakthrough Therapy and Fast Track designations by the FDA for the treatment of LMs.

About IV Choline Chloride for Patients on Parenteral Support

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 and 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 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 parenteral nutrition and has 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 Protara a U.S. patent claiming a choline composition and a U.S. patent claiming a method of treating choline deficiency with a choline composition, each with a term expiring in 2041.

(Press release, Protara Therapeutics, MAY 13, 2026, View Source [SID1234665639])