On March 8, 2023 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 full year and fourth quarter ended December 31, 2022, and provided a business update (Press release, Protara Therapeutics, MAR 8, 2023, View Source [SID1234628317]).
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"With key data and milestones expected from our clinical programs for TARA-002, including data from the Phase 1a portion of the ADVANCED-1 trial in non-muscle invasive bladder cancer (NMIBC) in the second quarter of 2023, we believe this year will be a particularly exciting time for Protara," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We look forward to leveraging data from the ADVANCED-1 trial to inform the design of further clinical studies in our NIMBC program. For our program in lymphatic malformations (LMs), a highly underserved pediatric population for which we believe TARA-002 could serve as a meaningful intervention, we have begun Phase 2 clinical trial start up activities and anticipate initiating the trial in the second half of this year. We believe we are well positioned to successfully execute on our pipeline programs and look forward to providing updates in due course."
Recent Highlights
TARA-002 in NMIBC
In December 2022, the Company’s Phase 1 ADVANCED-1 clinical trial of TARA-002, Protara’s investigational cell-based immunopotentiator for the treatment of NMIBC, was featured in a Trials in Progress poster at the Annual Meeting of the Society of Urologic Oncology.
The Company expects to report data from the Phase 1a portion of the trial in the second quarter of 2023 and move rapidly into the Phase 1b expansion portion of the trial, which will evaluate safety and efficacy in patients with carcinoma in situ (CIS).
TARA-002 in LMs
The Company has initiated study start up activities for a Phase 2 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed-cystic LMs.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)
Protara’s prospective study to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition is ongoing with results expected in the third quarter of 2023.
The Company plans to leverage results from the prospective study, as well as its completed retrospective study, to inform next steps for the IV Choline Chloride development program.
Corporate Updates
In January 2023, Protara announced the appointment of Patrick Fabbio as Chief Financial Officer. Mr. Fabbio brings to Protara more than 30 years of experience at various life science and pharmaceutical companies and most recently served as President and Chief Financial Officer at NYSE-listed Rafael Holdings, Inc.
Fourth Quarter and Full Year 2022 Financial Results
As of December 31, 2022, cash, cash equivalents and marketable debt securities totaled $102.3 million. The Company expects its cash, cash equivalents, and marketable debt securities will be sufficient to fund its planned operations and data milestones into 2025.
Research and development expenses for the fourth quarter of 2022 increased to $5.0 million from $4.1 million for the prior year period, and for the full year decreased to $16.8 million compared to $21.1 million for 2021. The fourth quarter increase was primarily due to an increase in non-clinical studies performed in the quarter versus the comparable period. The full year decrease was primarily due to a reduction in clinical manufacturing expenses.
General and administrative expenses for the fourth quarter of 2022 decreased to $5.0 million from $6.2 million for the prior year period, and for the full year decreased to $20.7 million compared to $26.4 million for 2021. The fourth quarter and full year decreases were primarily due to a reduction in stock based compensation expense and market development activities.
For the fourth quarter of 2022, Protara reported a net loss of $39.0 million, or $3.46 per share, compared with a net loss of $10.2 million, or $0.91 per share, for the same period in 2021. Net loss in the fourth quarter of 2022 included a non-cash goodwill impairment charge of $29.5 million associated with the accounting for the reverse merger transaction in January of 2020. Net loss for the year ended December 31, 2022 was $66.0 million, or $5.86 per share, compared with a net loss of $47.3 million, or $4.21 per share, for the year ended December 31, 2021. Net loss for the fourth quarter included approximately $1.4 million of stock-based compensation expenses. Net loss for the year ended December 31, 2022 included approximately $6.7 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-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, granulocyte colony-stimulating factor, and granulocyte-macrophage colony-stimulating factor 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.