Protara Therapeutics Announces Third Quarter 2020 Financial Results and Business Overview
- On Track to Complete GMP Batch Runs in Mid-2021 to Confirm Comparability Between TARA-002 and OK-432 -
- Expect to Initiate Phase 1 Trial for TARA-002 in Patients with Non-Muscle Invasive Bladder Cancer in 2021 -
- Requested Meeting with FDA to Discuss Path Forward for TARA-002 in Lymphatic Malformations -
- Strong Cash Position of $166M as of
“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
- 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 Applicationsto 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).
- 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
$16.87per share, and 4,148 shares of non-voting Series 1 Convertible Preferred Stock, at a public offering price of $16,873.54per 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 millionof 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 millionin personnel and related costs, (ii) $0.8 millionin chemistry manufacturing and controls (CMC) expenses and (iii) $0.3 millionin 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 millionin stock-based compensation expense, (ii) $0.6 millionin insurance expense and (iii) $0.7 millionin personnel and related costs.
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
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
About Non-Muscle Invasive Bladder Cancer
Bladder cancer is the 6th most common cancer in
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.
Protara is committed to identifying and advancing transformative therapies for people with cancer and rare diseases with limited treatment options. Protara’s portfolio includes its lead program, TARA-002, an investigational cell-based therapy being developed for the treatment of non-muscle invasive bladder cancer and lymphatic malformations, and IV Choline Chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure-associated liver disease. For more information, visit www.protaratx.com.
Statements contained in this press release regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, Protara’s development plans for its product candidates , including its plans regarding the timing or outcome of existing or future non-clinical studies and clinical trials, Protara’s expectations regarding interactions or upcoming filings with the FDA, statements regarding the anticipated safety or efficacy of Protara’s product candidates and, Protara’s financial footing. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s sales, revenue, expense and other financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; the impact of the COVID-19 pandemic on Protara’s business and the global economy; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; the loss of key members of management; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption "Risk Factors" and elsewhere in Protara's filings and reports with the
|PROTARA THERAPEUTICS, INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash and cash equivalents||$||165,904,797||$||564,124|
|Deferred offering costs||-||121,712|
|Prepaid expenses and other current assets||1,160,257||78,057|
|Total current assets||167,115,054||763,893|
|Property and equipment, net||760,548||458,591|
|Liabilities and Stockholders' Equity (Deficit)|
|Right-of-use liability, current||34,079||-|
|Total current liabilities||3,886,773||3,350,443|
|Right-of-use liability, long-term||394,721||-|
|Commitments and Contingencies (Note 6)|
|Stockholders' Equity (Deficit)|
|Series 1 Convertible Preferred Stock, 8,028 and 0 shares authorized at
|Common Stock, 10,521,840 and 2,627,533 common shares issued and outstanding as of|
|Additional Paid in Capital||232,567,265||10,651,073|
|Total Stockholders' Equity (Deficit)||194,625,763||(2,127,959||)|
|Total Liabilities and Stockholders' Equity (Deficit)||$||198,907,257||$||1,222,484|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|For the Three Months Ended
||For the Nine Months Ended
|Research & development||$||2,796,214||$||1,098,617||$||8,330,727||$||3,163,179|
|General & administrative||5,265,965||1,255,466||17,156,952||2,147,635|
|Total operating expenses||8,062,179||2,354,083||25,487,679||5,310,814|
|Other income, net|
|Interest income, net||(92,094||)||-||(317,307||)||-|
|Total other income, net||(92,094||)||-||(317,307||)||-|
|Weighted Average Shares Outstanding, basic and diluted||6,324,295||2,564,429||5,910,849||2,560,444|
|Net loss per share, basic and diluted||$||(1.26||)||$||(0.92||)||$||(4.26||)||$||(2.07||)|
Source: Protara Therapeutics