On August 9, 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 second quarter ended June 30, 2022 and provided a business update (Press release, Protara Therapeutics, AUG 9, 2022, View Source [SID1234617940]).
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"We are pleased with the continued progress of the ADVANCED-1 study of TARA-002 in non-muscle invasive bladder cancer (NMIBC)," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We remain committed to unlocking the full potential of TARA-002 in this underserved patient population, and look forward to sharing our progress in the coming quarters. In parallel, we are keenly focused on finalizing the design of a clinical trial of TARA-002 in patients with lymphatic malformations (LMs), a rare pediatric indication for which we believe TARA-002 has the potential to serve as a much-needed intervention."
Recent Highlights
TARA-002 in NMIBC
Enrollment is progressing in the Company’s Phase 1 ADVANCED-1 clinical trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of NMIBC.
In June 2022, a Trials in Progress poster related to the ADVANCED-1 Phase 1 trial was featured at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
TARA-002 in LMs
Protara continues to engage with the Vaccines and Related Products Division of the U.S. Food and Drug Administration (FDA) regarding a development path for TARA-002 in LMs. Following receipt of preliminary guidance from the FDA and in consultation with the Company’s external scientific advisory board, Protara plans to initiate a Phase 2 clinical trial of TARA-002 in LMs, subject to alignment with the agency on a trial protocol.
Protara announced that the European Medicines Agency (EMA) recently granted orphan drug designation (ODD) to TARA-002 for the treatment of LMs.
In May 2022, the Company announced results of a retrospective analysis of OK-432, the originator compound for TARA-002, for the treatment of LMs. Results from the analysis, which were presented during a poster presentation at the International Society for the Study of Vascular Anomalies World Congress 2022, showed that OK-432 met the primary efficacy endpoint of clinical success (defined as complete [90%-100%] or substantial [60%-89%] reduction in LM volume measured radiographically) in treating LMs and demonstrated a favorable safety profile.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)
The Company’s prospective study to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition remains ongoing. Protara 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.
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.
Second Quarter 2022 Financial Results
As of June 30, 2022, cash, cash equivalents and marketable debt securities were $112.8 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 second quarter of 2022 decreased to $3.1 million from $5.9 million during the second quarter of 2021. The decreased R&D expenses were primarily due to decreases in manufacturing expenses associated with TARA-002.
General and administrative expenses for the second quarter of 2022 decreased to $5.6 million from $6.9 million for the prior year period. The decrease was primarily due to decreases in stock-based compensation.
For the second quarter of 2022, Protara reported a net loss of $8.5 million, or $0.80 per share, compared with a net loss of $12.8 million, or $1.14 per share, for the same period in 2021. Net loss for the second quarter of 2022 included approximately $3.8 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.