On May 5, 2022 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 first quarter ended March 31, 2022 and provided a business update (Press release, Protara Therapeutics, MAY 5, 2022, View Source [SID1234613680]).
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"The recent commencement of patient dosing in the Phase 1 ADVANCED-1 trial of TARA-002 for non-muscle invasive bladder cancer (NMIBC) marked an important step toward our goal of bringing a much needed, novel therapeutic option to this underserved population," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Beyond NMIBC, we are encouraged to have recently held a meeting with the U.S. Food and Drug Administration (FDA), where we received preliminary guidance regarding a development path for TARA-002 in lymphatic malformations (LMs). We are currently planning to initiate a Phase 2 clinical trial in this indication subject to alignment with FDA on a clinical trial protocol. We also remain very engaged with the LMs treating community as we work to design a clinical study that will further progress our program for this rare pediatric indication that currently lacks any FDA-approved therapies."
Recent Highlights
TARA-002 in NMIBC
In March 2022, the Company announced that the first patient was dosed in its Phase 1 ADVANCED-1 clinical trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of NMIBC.
TARA-002 in LMs
The Company continues to engage with the Vaccines and Related Products Division of the FDA and recently received preliminary guidance regarding a potential development path for TARA-002 in LMs. The Company is planning to initiate a Phase 2 clinical trial of TARA-002 in this indication subject to alignment with the agency on a clinical trial protocol.
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 remains ongoing. The Company expects to use results from the prospective study, as well as its previously completed retrospective study, to inform next steps for the IV Choline Chloride development program.
With respect to the IV Choline Chloride program, in April 2022, the U.S. Patent and Trademark Office issued to the Company a patent claiming a sterile aqueous choline salt composition with a term expiring in 2041. The Company expects such patent to be listed in the FDA’s "Orange Book" in the event of the FDA’s approval of the Company’s IV Choline Chloride product candidate.
First Quarter 2022 Financial Results
As of March 31, 2022, cash, cash equivalents and restricted cash were $118.5 million. The Company expects its current cash and cash equivalents will be sufficient to fund its planned operations into mid-2024.
Research and development (R&D) expenses for the first quarter of 2022 decreased to $5.3 million from $7.0 million during the first quarter of 2021. The decreased R&D expenses were primarily due to decreases in manufacturing and non-clinical expenses associated with TARA-002.
General and administrative expenses for the first quarter of 2022 decreased to $5.6 million from $6.5 million for the prior year period. The decrease was primarily due to decreases in stock-based compensation.
For the first quarter of 2022, Protara reported a net loss of $10.8 million, or $0.96 per share, compared with a net loss of $13.5 million, or $1.20 per share, for the same period in 2021. Net loss for the first quarter of 2022 included approximately $1.9 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.