On November 12, 2020 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 third quarter ended September 30, 2020 (Press release, Protara Therapeutics, NOV 12, 2020, View Source [SID1234573137]).
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"The third quarter marked a highly productive time for the Company, notably with the Food and Drug Administration’s (FDA) confirmation of initial comparability between TARA-002 and OK-432, and separately the Company’s identification of an acceptable development path for TARA-002 in non-muscle invasive bladder cancer (NMIBC), an oncology indication with high unmet need," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We believe TARA-002 has the potential to serve as a much needed intervention for NMIBC patients lacking alternative therapeutic options, and we look forward to the expected commencement of our clinical program in NMIBC in 2021. Finally, we have requested a meeting with the FDA Division of Vaccines and Related Products, the review division which the Investigational New Drug (IND) application was initially opened by the University of Iowa, to discuss a potential Biological License Application (BLA) filing for TARA-002 in lymphatic malformations (LMs)."
Recent Highlights
TARA-002
In September 2020, Protara announced the following updates for the TARA-002 program:
FDA Confirmation of Initial Comparability Between TARA-002 and OK-432. Following a pre-IND engagement with the Office of Tissues and Advanced Therapies (OTAT) division of the Center for Biologics Evaluation and Research (CBER), the FDA agreed that Protara has successfully demonstrated initial manufacturing comparability between TARA-002 and OK-432 and that the in-process and release protocols employed by Protara to demonstrate initial comparability are appropriate to utilize for GMP-Scale comparability testing. Good Manufacturing Practice (GMP) scale-up is currently in process and the Company expects to complete three such large-scale batch runs to confirm comparability in mid-2021.
Clinical Development Path in NMIBC. The Company reached alignment with the FDA on a proposed clinical development plan to evaluate TARA-002 in patients with NMIBC. Advancement into the clinic will be supported by existing and ongoing non-clinical studies as well as historical clinical safety and efficacy data for OK-432. Subject to the successful completion of select non-clinical studies to characterize local toxicity of intravesical administration of TARA-002, as well as acceptance of an IND filing, the Company plans to commence a Phase 1 study in 2021 to assess the safety and tolerability of TARA-002 in patients with NMIBC, including patients with carcinoma in situ (CIS), with results expected in 2022.
Regulatory Path in LMs. Protara has requested a meeting with the FDA Division of Vaccines and Related Products Applications to discuss the regulatory path for TARA-002 in LMs. The Company plans to utilize the robust dataset for OK-432 in LMs to support a BLA filing for TARA-002 in LMs.
IV Choline Chloride
Prevalence Study Underway to Assess Incidence of Liver Disease in Patients Dependent on Parenteral Nutrition (PN). The Company recently commenced a prevalence study to assess the incidence of liver disease in patients dependent on PN in the home care setting. The Company believes the study will enhance its understanding of the population of patients who may potentially benefit from IV Choline Chloride, its investigational phospholipid substrate replacement therapy currently in development for the treatment of patients receiving PN who have intestinal failure-associated liver disease (IFALD).
Corporate Update
Raised $151 Million in Concurrent Public Offerings. Protara recently announced the closing of concurrent but separate underwritten public offerings of 4,600,000 shares of its common stock, at a public offering price of $16.87 per share, and 4,148 shares of non-voting Series 1 Convertible Preferred Stock, at a public offering price of $16,873.54 per share. In addition, the underwriters exercised their overallotment option in full to purchase an additional 690,000 shares of common stock at a public offering price of $16.87. Aggregate net proceeds to Protara were approximately $151 million, after deducting underwriting discounts and offering expenses.
Third Quarter 2020 Results from Operations
As of September 30, 2020, cash and restricted cash were $166.0 million.
Protara reported a net loss of $8.0 million for the third quarter of 2020 as compared to a net loss of $2.4 million for the three months ended September 30, 2019. The third quarter of 2020 included approximately $2.8 million of stock-based compensation expense.
Research and Development expenses were $2.8 million for the third quarter of 2020, an increase of $1.7 million as compared to the three months ended September 30, 2019. The increase was primarily due to an increase of (i) $0.6 million in personnel and related costs, (ii) $0.8 million in chemistry manufacturing and controls (CMC) expenses and (iii) $0.3 million in regulatory expenses.
General and Administrative expenses were $5.3 million for the third quarter of 2020, which represented an increase of $4.0 million as compared to the three months ended September 30, 2019. The increase was primarily related to an increase of (i) $2.6 million in stock-based compensation expense, (ii) $0.6 million in insurance expense and (iii) $0.7 million in personnel and related costs.
A Form 10-Q containing the full financial statements was filed this morning and is available for viewing on Protara’s website at www.protaratx.com or www.sec.gov.
About TARA-002
TARA-002 is an investigational cell therapy in development for the treatment of lymphatic malformations (LMs) and non-muscle invasive bladder cancer (NMIBC). 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. TARA-002 has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration for the LMs indication.
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 2 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.