On May 6, 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 first quarter ended March 31, 2021 and provided a business update (Press release, Protara Therapeutics, MAY 6, 2021, View Source [SID1234579268]).
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"We remain on track to file an Investigational New Drug (IND) application and initiate our Phase 1 clinical study for TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC) by the end of the year. Patients suffering from NMIBC have limited approved treatment options, and with the current standard-of-care facing a long-term supply shortage, there is an urgent need for effective therapies for these patients," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We look forward to completing GMP scale up and comparability and then aligning with the Food and Drug Administration (FDA) on the design of a clinical study for TARA-002 in patients with Lymphatic Malformations (LMs). This new study, together with the extensive data for OK-432, the originator compound for TARA-002, should provide a robust data package for TARA-002 in this rare pediatric indication for which there is no U.S. FDA-approved therapy."
Recent Highlights and Upcoming Milestones
TARA-002 in NMIBC
Protara remains on track to submit an IND application in the second half of 2021. Subject to the successful completion of select non-clinical studies, which are expected to be completed in the first half of 2021, and 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
Based on feedback from the FDA, the Company intends to complete confirmatory, large-scale, GMP manufacturing comparability in the second half of 2021 and, upon alignment with FDA on study design, subsequently initiate a clinical study in pediatric LM patients.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)
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 April 2021, the Company announced the appointment of Martín Sebastian Olivo, M.D. as Chief Medical Officer. Dr. Olivo brings to Protara more than 15 years of experience in oncology translational and clinical research and global drug development, most recently serving as Vice President, Breast Cancer Clinical Development Lead at Gilead Sciences, Inc. (formerly Immunomedics, Inc.).
First Quarter 2021 Financial Results
As of March 31, 2021, cash, cash equivalents and investments totaled $155 million.
Research and development expenses for the first quarter of 2021 increased to $7.0 million from $3.1 million during the first 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 first quarter of 2021 decreased to $6.5 million from $7.1 million for the prior year period. The decrease was primarily due to one-time expenses related to the reverse merger, which occurred in the first quarter of 2020, off-set by an increase in personnel and related costs supporting the Company’s growth.
For the first quarter of 2021, Protara reported a net loss of $13.5 million, or $1.20 per share, compared with a net loss of $10.1 million, or $1.81 per share, for the same period in 2020. Net loss for the first 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 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).
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.