On November 4, 2021Protara 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, 2021 and provided a business update (Press release, Protara Therapeutics, NOV 4, 2021, View Source [SID1234594493]).

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"We have made significant progress advancing our TARA-002 clinical programs in the third quarter, most notably the completion of our confirmatory large-scale GMP comparability work and the clearance of our Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for our non-muscle invasive bladder cancer (NMIBC) clinical development program. NMIBC is one of the most recurrent and difficult to treat cancers with limited treatment options, and we look forward to initiating our Phase 1 clinical trial of TARA-002 in adults with high-grade NMIBC by the end of the year," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are also working to align with the FDA 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."

Recent Highlights and Upcoming Milestones

TARA-002 in NMIBC

In October 2021, Protara announced that the FDA cleared the Company’s IND application for TARA-002, an investigational cell-based therapy in development for the treatment of NMIBC. The confirmatory, GMP-scale comparability data for TARA-002 in relation to OK-432, the originator therapy for TARA-002, have been completed and were reviewed by the FDA as part of the clearance of the IND.
The Company plans to commence a Phase 1 clinical trial by the end of 2021 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, GMP-scale comparability data, and plans to engage the FDA on the design, and subsequently initiate a clinical trial in pediatric LM patients.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

In September 2021, the Company announced the completion of a retrospective prevalence study designed to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition (PN). The study found that approximately 30% of patients who are dependent on PN have cholestasis, despite the use of medical management in these patients.
The Company plans to use the results from the completed retrospective study and ongoing prospective study to inform next steps for the IV Choline Chloride development program.
Corporate Update

In October 2021, the Company hired Mary Grendell as its new General Counsel. Ms. Grendell has an extensive track record as a legal executive, most recently serving as Vice President, Deputy General Counsel and Corporate Secretary, at Intercept Pharmaceuticals. Previously, she held positions at Mylan (now part of Viatris) and Amgen following her early legal career at Sullivan & Cromwell and Covington & Burling. Ms. Grendell received her J.D. from the University of Pennsylvania Law School and her B.A. from Yale University.
Third Quarter 2021 Financial Results

As of September 30, 2021, cash, cash equivalents and marketable debt securities totaled $138.4 million.

Research and development expenses for the third quarter of 2021 increased to $4.1 million from $2.8 million during the third quarter of 2020. The increased R&D expenses 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 third quarter of 2021 increased to $6.7 million from $5.3 million during the third quarter of 2020. The increase was primarily due to increases in headcount and employee-related expenses, development of commercial capabilities, and costs associated with the new corporate office in New York, NY.

For the third quarter of 2021, Protara reported a net loss of $10.8 million, or $0.96 per share, compared with a net loss of $8.0 million, or $1.26 per share, for the third quarter of 2020. Net loss for the third quarter of 2021 included approximately $2.7 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

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 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 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.