Protara Therapeutics Announces Third Quarter 2021 Financial Results and Business Overview
– TARA-002 Confirmatory Large-Scale GMP Comparability Complete –
– Strong Cash, Cash Equivalents, and
“We have made significant progress advancing our TARA-002 clinical programs in the third quarter, most notably the completion of our confirmatory large-scale GMP comparability work and the clearance of our Investigational New Drug (IND) application by the
Recent Highlights and Upcoming Milestones
TARA-002 in NMIBC
October 2021, Protara announced that the FDA cleared the Company’s IND application for TARA-002, an investigational cell-based therapy in development for the treatment of NMIBC. The confirmatory, GMP-scale comparability data for TARA-002 in relation to OK-432, the originator therapy for TARA-002, have been completed and were reviewed by the FDA as part of the clearance of the IND.
- The Company plans to commence a Phase 1 clinical trial by the end of 2021 to assess the safety, tolerability, and preliminary signs of anti-tumor activity of TARA-002 in adults with high-grade NMIBC.
TARA-002 in LMs
October 2021, the Company updated its IND submission for TARA-002 for the treatment of LMs with completed confirmatory, GMP-scale comparability data, and plans to engage the FDA on the design, and subsequently initiate a clinical trial in pediatric LM patients.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)
September 2021, the Company announced the completion of a retrospective prevalence study designed to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition (PN). The study found that approximately 30% of patients who are dependent on PN have cholestasis, despite the use of medical management in these patients.
- The Company plans to use the results from the completed retrospective study and ongoing prospective study to inform next steps for the IV Choline Chloride development program.
October 2021, the Company hired Mary Grendellas its new General Counsel. Ms. Grendellhas an extensive track record as a legal executive, most recently serving as Vice President, Deputy General Counsel and Corporate Secretary, at Intercept Pharmaceuticals. Previously, she held positions at Mylan (now part of Viatris) and Amgen following her early legal career at Sullivan & Cromwell and Covington & Burling. Ms. Grendellreceived her J.D. from the University of Pennsylvania Law Schooland her B.A. from Yale University.
Third Quarter 2021 Financial Results
- As of
September 30, 2021, cash, cash equivalents and marketable debt securities totaled $138.4 million.
- Research and development expenses for the third quarter of 2021 increased to
$4.1 millionfrom $2.8 millionduring the third quarter of 2020. The increased R&D expenses were primarily due to increases in non-clinical, clinical and regulatory expenses associated with TARA-002, headcount and stock-based compensation, and other employee-related expenses.
- General and administrative expenses for the third quarter of 2021 increased to
$6.7 millionfrom $5.3 millionduring the third quarter of 2020. The increase was primarily due to increases in headcount and employee-related expenses, development of commercial capabilities, and costs associated with the new corporate office in New York, NY.
- For the third quarter of 2021, Protara reported a net loss of
$10.8 million, or $0.96per share, compared with a net loss of $8.0 million, or $1.26per share, for the third quarter of 2020. Net loss for the third quarter of 2021 included approximately $2.7 millionof stock-based compensation expenses.
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.
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.
About Non-Muscle Invasive Bladder Cancer
Bladder cancer is the 6th most common cancer in the
About 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 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 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.
About Protara Therapeutics, Inc.
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, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials; statements related to expectations regarding interactions with the FDA, including anticipated alignment with the FDA on clinical trial design for TARA-002 in pediatric LM patients; Protara’s financial footing; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year. 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 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
|CONDENSED CONSOLIDATED BALANCE SHEETS
| (in thousands, except share and per share data)
|Cash and cash equivalents||$||40,742||$||168,598|
|Marketable debt securities, current||53,016||-|
|Prepaid expenses and other current assets||1,937||787|
|Total current assets||95,695||169,435|
|Restricted cash, non-current||745||745|
|Marketable debt securities, non-current||44,652||-|
|Property and equipment, net||1,729||1,240|
|Operating lease right-of-use asset||7,413||1,060|
|Other assets, non-current||946||1,160|
|Liabilities and Stockholders' Equity|
|Operating lease liability, current||840||88|
|Total current liabilities||3,688||2,915|
|Operating lease liability, non-current||6,603||999|
|Commitments and Contingencies (Note 6)|
|Series 1 Convertible Preferred Stock, 8,028 shares authorized at
|Common Stock, 11,235,731 and 11,211,840 shares issued and outstanding as of
|Additional Paid in Capital||254,218||245,992|
|Accumulated Other Comprehensive Income (Loss)||(39||)||-|
|Total Stockholders' Equity||170,406||199,243|
|Total Liabilities and Stockholders' Equity||$||180,697||$||203,157|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS|
|(in thousands, except share and per share data)|
|For the three months ended
||For the nine months ended
|Research & development||$||4,093||$||2,796||$||17,020||$||8,330|
|General & administrative||6,737||5,266||20,182||17,157|
|Total operating expenses||10,830||8,062||37,202||25,487|
|Other income, net|
|Interest income, net||(53||)||(92||)||(178||)||(317||)|
|Total other income, net||(53||)||(92||)||(178||)||(317||)|
|Other comprehensive gain (loss):|
|Unrealized gains (losses) on available-for-sale marketable debt securities||62||-||(39||)||-|
|Total other comprehensive gain (loss)||62||-||(39||)||-|
|Weighted Average Shares Outstanding, basic and diluted||11,235,507||6,324,295||11,231,513||5,910,849|
|Net loss per share, basic and diluted||$||(0.96||)||$||(1.26||)||$||(3.30||)||$||(4.26||)|
Source: Protara Therapeutics