Full Press Release Details
Protara Therapeutics Announces Second Quarter
2021 Financial Results and Business Overview
- Successfully Completed IND-Enabling Studies
for TARA-002 in Patients with NMIBC; On Track to Initiate Phase 1 Trial by Year-End -
- TARA-002 GMP Scale up and Comparability Ongoing
and Expected to be Completed by Year-End-
- Company Plans to Engage with FDA to Determine
Clinical Trial Design for TARA-002 in Lymphatic Malformations -
- Strong Cash, Cash Equivalents, and Investments
Position of $145M as of June 30, 2021 -
NEW YORK, August 5, 2021 -- Protara
Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare
diseases with significant unmet needs, today announced financial results for the second quarter ended June 30, 2021 and provided a business
make significant progress executing against our development plans for TARA-002 in non-muscle invasive bladder cancer (NMIBC) and lymphatic
malformations (LMs), and we look forward to achieving multiple important milestones in the second half of this year," said Jesse
Shefferman, Chief Executive Officer of Protara Therapeutics.
"With the successful completion of required non-clinical studies for TARA-002, we remain on track to submit our Investigational
New Drug (IND) application and initiate our Phase 1 clinical study by year-end. In addition, our Good Manufacturing Practice (GMP) scale
up and comparability work remain on track to be completed by year-end, and we look forward to working with the U.S. Food and Drug Administration
(FDA) on a clinical trial of TARA-002 in patients with LMs, a rare pediatric
indication for which there are currently no U.S. FDA-approved therapies."
Highlights and Upcoming Milestones
Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)
Quarter 2021 Financial Results
is an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations
(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 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.
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.
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 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
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.
Forward-Looking Statements
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 plans with respect to certain clinical trials, GMP scale up and comparability work and anticipated timing, Protara's
development plans for its product candidates and related expectations regarding interactions or upcoming filings with the FDA, including
its planned IND filing for TARA-002 in NMIBC and plans regarding the timing or outcome of existing or future non-clinical studies and
clinical trials, 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 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 United States Securities and Exchange Commission. All forward-looking statements
contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates
as of such date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new
information, the occurrence of future events or otherwise, except as required by law.
PROTARA THERAPEUTICS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share data)
| As of | ||||||||
| June 30, 2021 | December 31, 2020 | |||||||
| (unaudited) | ||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 46,836 | $ | 168,598 | ||||
| Restricted cash | - | 50 | ||||||
| Marketable debt securities, current | 37,830 | - | ||||||
| Prepaid expenses and other current assets | 3,153 | 787 | ||||||
| Total current assets | 87,819 | 169,435 | ||||||
| Non-current assets: | ||||||||
| Restricted cash, non-current | 745 | 745 | ||||||
| Marketable debt securities, non-current | 60,378 | - | ||||||
| Property and equipment, net | 1,769 | 1,240 | ||||||
| Operating lease right-of-use asset | 7,648 | 1,060 | ||||||
| Goodwill | 29,517 | 29,517 | ||||||
| Other assets, non-current | 1,016 | 1,160 | ||||||
| Total assets | $ | 188,892 | $ | 203,157 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,403 | $ | 914 | ||||
| Accrued expenses | 1,439 | 1,913 | ||||||
| Operating lease liability, current | 826 | 88 | ||||||
| Total current liabilities | 3,668 | 2,915 | ||||||
| Non-current liabilities: | ||||||||
| Operating lease liability, non-current | 6,819 | 999 | ||||||
| Total liabilities | 10,487 | 3,914 | ||||||
| Commitments and Contingencies (Note 6) | ||||||||
| Stockholders' Equity | ||||||||
| Preferred Stock, $0.001 par value, authorized 10,000,000 shares: | ||||||||
| Series 1 Convertible Preferred Stock, 8,028 shares authorized at June 30, 2021 and December 31, 2020, 8,027 shares issued and outstanding as of June 30, 2021 and December 31, 2020. | - | - | ||||||
| Common Stock, $0.001 par value, authorized 100,000,000 shares: | ||||||||
| Common Stock, 11,233,856 and 11,211,840 shares issued and outstanding as of June 30, 2021 and December 31, 2020, respectively. | 11 | 11 | ||||||
| Additional Paid in Capital | 251,502 | 245,992 | ||||||
| Accumulated Deficit | (73,007 | ) | (46,760 | ) | ||||
| Accumulated Other Comprehensive Income (Loss) | (101 | ) | - | |||||
| Total Stockholders' Equity | 178,405 | 199,243 | ||||||
| Total Liabilities and Stockholders' Equity | $ | 188,892 | $ | 203,157 |
PROTARA THERAPEUTICS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
AND COMPREHENSIVE LOSS
(in thousands, except share and per share data)
| For the three months ended June 30, | For the six months ended June 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Operating expense: | ||||||||||||||||
| Research & development | $ | 5,887 | $ | 2,470 | $ | 12,927 | $ | 5,534 | ||||||||
| General & administrative | 6,905 | 4,796 | 13,445 | 11,891 | ||||||||||||
| Total operating expenses | 12,792 | 7,266 | 26,372 | 17,425 | ||||||||||||
| Operating loss | (12,792 | ) | (7,266 | ) | (26,372 | ) | (17,425 | ) | ||||||||
| Other income, net | ||||||||||||||||
| Interest income, net | (10 | ) | (126 | ) | (125 | ) | (225 | ) | ||||||||
| Total other income, net | (10 | ) | (126 | ) | (125 | ) | (225 | ) | ||||||||
| Net loss | (12,782 | ) | (7,140 | ) | (26,247 | ) | (17,200 | ) | ||||||||
| Other comprehensive gain (loss): | ||||||||||||||||
| Unrealized gains (losses) on available-for-sale marketable debt securities | 63 | - | (101 | ) | - | |||||||||||
| Total other comprehensive gain (loss) | 63 | - | (101 | ) | - | |||||||||||
| Comprehensive Loss | $ | (12,719 | ) | $ | (7,140 | ) | $ | (26,348 | ) | $ | (17,200 | ) | ||||
| Weighted Average Shares Outstanding, basic and diluted | 11,232,010 | 5,843,203 | 11,229,484 | 5,701,855 | ||||||||||||
| Net loss per share, basic and diluted | $ | (1.14 | ) | $ | (1.22 | ) | $ | (2.34 | ) | $ | (3.02 | ) |
Protara Therapeutics