On August 5, 2021 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases with significant unmet needs, reported financial results for the second quarter ended June 30, 2021 and provided a business update (Press release, Protara Therapeutics, AUG 5, 2021, View Source [SID1234585916]).

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"We continue to make significant progress executing against our development plans for TARA-002 in non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs), and we look forward to achieving multiple important milestones in the second half of this year," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "With the successful completion of required non-clinical studies for TARA-002, we remain on track to submit our Investigational New Drug (IND) application and initiate our Phase 1 clinical study by year-end. In addition, our Good Manufacturing Practice (GMP) scale up and comparability work remain on track to be completed by year-end, and we look forward to working with the U.S. Food and Drug Administration (FDA) on a clinical trial of TARA-002 in patients with LMs, a rare pediatric indication for which there are currently no U.S. FDA-approved therapies."

Recent Highlights and Upcoming Milestones

TARA-002 Comparability

The Company remains on track to complete confirmatory, large-scale GMP manufacturing comparability by year-end.
TARA-002 in NMIBC

Protara successfully completed required non-clinical IND-enabling studies to characterize local toxicity of intravesical administration of TARA-002. The Company remains on track to submit an IND application in the second half of 2021 and, subject to the FDA acceptance of the IND application, the Company plans to commence a Phase 1 trial by the end of 2021 to assess the safety and tolerability of TARA-002 in patients with NMIBC, including patients with carcinoma in situ (CIS).
TARA-002 in LMs

Following the completion of confirmatory, large-scale GMP manufacturing comparability, the Company plans to align with 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)

The Company is currently executing a prevalence study in partnership with a large home health organization in the U.S. to enhance understanding of the appropriate patient population and will use this information to define the next steps for the development program.
Corporate Update

In June 2021, the Company announced the appointment of Jane Huang, M.D., to its Board of Directors. Dr. Huang is an experienced biotech executive and proven leader throughout the development lifecycle of multiple oncology therapeutics globally and currently serves as Chief Medical Officer, Hematology at BeiGene, Ltd.
Second Quarter 2021 Financial Results

As of June 30, 2021, cash, cash equivalents and investments totaled $145 million.

Research and development expenses for the second quarter of 2021 increased to $5.9 million from $2.5 million during the second quarter of 2020. The increased R&D expenses were primarily due to increases in manufacturing and regulatory expenses associated with TARA-002.

General and administrative expenses for the second quarter of 2021 increased to $6.9 million from $4.8 million during the second quarter of 2020. The increase was primarily due to increases in stock-based compensation, headcount, and costs associated with the new Company headquarters in New York, NY.

For the second quarter of 2021, Protara reported a net loss of $12.8 million, or $1.14 per share, compared with a net loss of $7.1 million, or $1.22 per share, for the second quarter of 2020. Net loss for the second quarter of 2021 included approximately $3.0 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (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 initial 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 non-muscle invasive bladder cancer (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

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 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.