On March 11, 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 fourth quarter and year ended December 31, 2020 and provided a business update (Press release, Protara Therapeutics, MAR 11, 2021, View Source [SID1234576475]).
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"We believe 2021 will be a transformative year for Protara, and we are entering it with strong momentum," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We remain on track to commence a Phase 1 study of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC), a pressing area of unmet need, by the end of the year. We believe that TARA-002 has the opportunity to play a meaningful role in the current NMIBC treatment landscape."
Mr. Shefferman continued, "We are in discussions with the U.S. Food and Drug Administration’s (FDA) Division of Vaccines and Related Products to establish a path forward to file our Biological License Application (BLA) for TARA-002 in lymphatic malformations (LMs). We are encouraged by the progress to date and, at the FDA’s request, have submitted the full Clinical Study Report (CSR) of a randomized Phase 2 study of OK-432 (the originator compound of TARA-002) in LMs led by the University of Iowa. We look forward to continuing our dialogue with the FDA."
Recent Highlights and Upcoming Milestones
TARA-002 in NMIBC
Protara remains on track to complete select non-clinical studies to characterize local toxicity of intravesical administration of TARA-002 in the first half of 2021, with an Investigational New Drug (IND) application submission anticipated in the second half of 2021. Subject to FDA acceptance of the IND application, the Company plans to commence a Phase 1 study 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
Protara plans to utilize the robust dataset for OK-432 (the originator compound of TARA-002) in LMs to support a potential filing. In connection with Protara’s request to discuss a potential BLA submission for TARA-002 in LMs, the FDA Division of Vaccines and Related Products has requested a CSR summarizing the totality of a randomized Phase 2 study of OK-432 in LMs led by the University of Iowa. The Company has submitted the CSR to the FDA and continues to prepare for a potential BLA filing in the second half of 2021, or to initiate additional clinical work as required.
IV Choline Chloride in intestinal failure associated liver disease (IFALD)
Following a successful meeting with the FDA in 2020 regarding the registration package for IV Choline Chloride, the Company is currently undertaking 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 February 2021, the Company announced the appointment of Cynthia Smith to its Board of Directors. Ms. Smith brings to Protara over 20 years of diverse leadership experience within the healthcare industry, most recently serving as Chief Commercial Officer at ZS Pharma.
Fourth Quarter and Full Year 2020 Financial Results
As of December 31, 2020, cash, cash equivalents and restricted cash were $169.4 million.
Research and development expenses for the fourth quarter of 2020 increased to $3.7 million from $0.7 million for the prior year period, and for the full year increased to $12.0 million compared to $3.9 million for 2019. The fourth quarter and full year increases were primarily due to increases in personnel and related costs, manufacturing and regulatory expenses as the company advanced its clinical programs supporting TARA-002.
General and administrative expenses for the fourth quarter of 2020 increased to $5.3 million from $1.8 million for the prior year period, and for the full year increased to $22.5 million compared to $4.0 million for 2019. The fourth quarter and full year increases were primarily due to increases in stock-based compensation expense, insurance expense and personnel and related costs supporting the company’s growth.
For the fourth quarter of 2020, Protara reported a net loss of $8.8 million, or $0.79 per share, compared with a net loss of $2.5 million, or $0.96 per share, for the same period in 2019. Net loss for the year ended December 31, 2020 was $34.0 million, or $4.70 per share, compared with a net loss of $7.8 million, or $3.04 per share, for the year ended December 31, 2019. Net loss for the fourth quarter included approximately $2.3 million of stock-based compensation expenses. Net loss for the year ended December 31, 2020 included $9.7 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 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. The current standard of care for high-grade NMIBC includes intravesical Bacillus Calmette-Guerin (BCG), which has been the subject of multiple global supply shortages in the past decade.
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.