On March 9, 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 fourth quarter and year ended December 31, 2021 and provided a business update (Press release, Protara Therapeutics, MAR 9, 2022, View Source [SID1234609790]).
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"Following a productive 2021, we are well poised to advance our pipeline in 2022, in particular, we are excited to have commenced our Phase 1 study of TARA-002 in non-muscle invasive bladder cancer (NMIBC), a significant step forward in our mission to bring a new immunotherapy to this patient population," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Discussions with the U.S. Food and Drug Administration (FDA) remain ongoing on the design of a clinical trial of TARA-002 in patients with lymphatic malformations (LMs), a rare pediatric indication for which there are currently no U.S. FDA-approved therapies. In addition, we continue to assess potential future indications and combinations for TARA-002."
Mr. Shefferman added, "Supported by a strong balance sheet, which includes ample runway to support our planned operations into mid-2024, we remain steadfast in our commitment to bringing meaningful new therapeutic options to pressing areas of high unmet need."
Recent Highlights
TARA-002 in NMIBC
In October 2021, the Company announced that the FDA cleared its Investigational New Drug (IND) application for TARA-002, an investigational cell-based therapy in development for the treatment of NMIBC. A Phase 1 clinical trial has commenced to assess the safety, tolerability, and preliminary signs of anti-tumor activity of TARA-002 in adults with high-grade NMIBC.
TARA-002 in LMs
In October 2021, the Company updated its IND submission for TARA-002 for the treatment of LMs with completed confirmatory, current Good Manufacturing Practices (cGMP) comparability data. The Company is engaged with the FDA to align on a development plan for TARA-002 in LMs.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)
The Company’s prospective prevalence study to enhance understanding of the incidence of IFALD and choline deficiency in patients dependent on parenteral nutrition remains ongoing. The Company plans 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.
Corporate Updates
In January 2022, Protara announced the appointment of Jathin Bandari, M.D., as Chief Medical Officer. Dr. Bandari is a practicing urologic oncologist, recently serving at the University of Rochester where he specializes in both minimally invasive urologic oncology and advanced open pelvic retroperitoneal cancer surgery, and where he maintains a faculty appointment. Dr. Bandari joined Protara in April 2020 and most recently was Vice President, Head of Clinical Development, and Interim Chief Medical Officer.
Fourth Quarter and Full Year 2021 Financial Results
As of December 31, 2021, cash, cash equivalents and marketable debt securities totaled $130.7 million. The Company expects its cash, cash equivalents, and marketable debt securities will be sufficient to fund its planned operations into mid-2024.
Research and development expenses for the fourth quarter of 2021 increased to $4.1 million from $3.7 million for the prior year period, and for the full year increased to $21.1 million compared to $12.0 million for 2020. The fourth quarter and full year increases were primarily due to increases in non-clinical, clinical and regulatory expenses associated with TARA-002, headcount and stock-based compensation, and other employee-related expenses.
General and administrative expenses for the fourth quarter of 2021 increased to $6.2 million from $5.3 million for the prior year period, and for the full year increased to $26.4 million compared to $22.5 million for 2020. The fourth quarter and full year increases were due to increases in headcount and employee-related expenses, market development capabilities, and costs associated with the new office in New York.
For the fourth quarter of 2021, Protara reported a net loss of $10.2 million, or $0.91 per share, compared with a net loss of $8.8 million, or $0.79 per share, for the same period in 2020. Net loss for the year ended December 31, 2021 was $47.3 million, or $4.21 per share, compared with a net loss of $34.0 million, or $4.70 per share, for the year ended December 31, 2020. Net loss for the fourth quarter included approximately $2.0 million of stock-based compensation expenses. Net loss for the year ended December 31, 2021 included $10.4 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-6, IL-8, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, and vascular endothelial growth factor (VEGF) 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.