On November 3, 2022 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 third quarter ended September 30, 2022 and provided a business update (Press release, Protara Therapeutics, NOV 3, 2022, View Source [SID1234623009]).

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"We continue to make meaningful progress with dose escalation in the ongoing Phase 1a portion of our ADVANCED-1 study of TARA-002 in non-muscle invasive bladder cancer (NMIBC), and look forward to ultimately utilizing these data, as well as data from our ongoing preclinical work, to design effective later-stage clinical trials in this indication," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are pleased to share that we have received initial feedback from the U.S. Food and Drug Administration (FDA) on our proposed Phase 2 study protocol evaluating TARA-002 in Lymphatic Malformations (LMs), and now expect to initiate the study in 2023. LMs represents a highly underserved rare pediatric indication for which we believe TARA-002 could serve as a much-needed treatment option."

Recent Highlights

TARA-002 in NMIBC

Dose escalation remains ongoing in the Phase 1a portion of the Company’s ADVANCED-1 clinical trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of NMIBC. The Phase 1a dose escalation portion of the trial is designed to identify a safe and tolerable dose of TARA-002 to be further evaluated in the Phase 1b expansion portion of the trial.
TARA-002 in LMs

Protara recently received initial feedback from the Vaccines and Related Products Division of the FDA on the protocol for the proposed Phase 2 study evaluating TARA-002 in LMs. The Company is in the process of finalizing the trial design and expects to commence study start-up activities in the first half of 2023.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

The Company’s prospective study to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition remains ongoing. Protara expects to use results from the prospective study, as well as its previously completed retrospective study, to inform next steps for the IV Choline Chloride development program.
Third Quarter 2022 Financial Results

As of September 30, 2022, cash, cash equivalents and marketable debt securities were $107.1 million. The Company now expects its current cash and cash equivalents will be sufficient to fund its planned operations into the second half of 2024.
Research and development (R&D) expenses for the third quarter of 2022 decreased to $3.5 million from $4.1 million during the third quarter of 2021. The decreased R&D expenses were primarily due to lower regulatory costs relative to regulatory expense in the comparable three months ended September 30, 2021, when the Company filed its Investigational New Drug application for TARA-002.
General and administrative expenses for the third quarter of 2022 decreased to $4.5 million from $6.7 million for the prior year period. The decrease was primarily due to a decrease of $1.2 million in stock-based compensation expense, a decrease of $0.2 million in compensation, benefits and other employee-related expenses, and a decrease of $0.6 million in market development expenses.
For the third quarter of 2022, Protara reported a net loss of $7.7 million, or $0.68 per share, compared with a net loss of $10.8 million, or $0.96 per share, for the same period in 2021. Net loss for the third quarter of 2022 included approximately $1.4 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and 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 Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully demonstrated 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, tumor necrosis factor (TNF)-alpha, granulocyte colony-stimulating factor, and granulocyte-macrophage colony-stimulating factor are secreted by immune cells to induce a strong local 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 and Intestinal Failure-associated Liver Disease (IFALD)

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have IFALD. 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. If approved, IV Choline Chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.