On August 3, 2023 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, 2023 and provided a business update (Press release, Protara Therapeutics, AUG 3, 2023, View Source [SID1234633770]).

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"Following the positive preliminary data from the dose escalation portion of the ADVANCED-1 trial of TARA-002 in patients with high-grade non-muscle invasive bladder cancer (NMIBC), we have seen rapid enrollment in the expansion portion of the trial and anticipate sharing preliminary results in the first half of 2024," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are building on this momentum, and plan to initiate two additional trials for TARA-002 later this year while continuing to maintain a disciplined approach to investments. We are in a solid financial position with cash runway into 2025."

Recent Highlights

TARA-002 in NMIBC

In April 2023 at the American Urological Association Annual Meeting, the Company announced positive preliminary results from the Phase 1a dose-escalation component of the ADVANCED-1 clinical trial of TARA-002, its investigational cell-based therapy, for the treatment of patients with high-grade NMIBC. The clinical data indicate that TARA-002 was generally well tolerated and anti-tumor activity was observed, including tumor regression in all three evaluable patients with carcinoma in situ (CIS), including one heavily pre-treated Bacillus Calmette-Guérin (BCG)-unresponsive patient who achieved a complete response (CR).

In May 2023, the Company dosed the first patients in its Phase 1b ADVANCED-1EXP study, an open-label expansion trial evaluating intravesical TARA-002 at the 40KE1 dose in 12 CIS patients, including BCG-naïve, BCG-unresponsive, and BCG-inadequately treated patients. Dosing continues to progress in the trial, with preliminary results expected in the first half of 2024. The primary endpoint of the trial is the CR rate at three months.

In the fourth quarter of 2023, the Company plans to initiate ADVANCED-2, a Phase 1b/2 open-label trial evaluating intravesical TARA-002 in up to 102 patients with high-grade CIS, including 27 patients with CIS (± Ta/T1), BCG-Naïve, or BCG-experienced, who have not received intravesical BCG for at least 24 months prior to CIS diagnosis, and 75 patients with BCG-unresponsive CIS (± Ta/T1).
TARA-002 in Lymphatic Malformations (LMs)

In April 2023, the Company received regulatory clearance from the U.S. Food and Drug Administration (FDA) to commence STARBORN-1, a Phase 2 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed-cystic LMs. Trial start-up activities are well underway at the ten pediatric centers of excellence participating in the trial, and initiation is expected in the fourth quarter of 2023.
IV Choline Chloride Program

Protara is concluding its prospective prevalence study to enhance understanding of the incidence of choline deficiency in patients dependent on parenteral nutrition. The Company continues to engage with the FDA and plans to use both regulatory feedback and results from the prospective study to inform next steps for the IV Choline Chloride development program.
Second Quarter 2023 Financial Results

As of June 30, 2023, cash, cash equivalents and restricted cash were $80.4 million. The Company expects its current cash and cash equivalents will be sufficient to fund its planned operations into 2025.
Research and development expenses for the second quarter of 2023 increased to $7.2 million from $3.1 million during the second quarter of 2022, primarily reflecting an increase in expenses related to clinical and non-clinical trial activities for TARA-002.
General and administrative expenses for the second quarter of 2023 decreased to $4.9 million from $5.6 million for the prior year period, primarily due to lower employee related expenses.
For the second quarter of 2023, Protara reported a net loss of $11.3 million, or $1.00 per share, compared with a net loss of $8.5 million, or $0.76 per share, for the same period in 2022. Net loss for the second quarter of 2023 included approximately $1.6 million of 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 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 abnormal cells.

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 3 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 (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN). Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. IV Choline Chloride has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PN patients.