Full Press Release Details
Therapeutics Announces Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update
Data from Phase 1a Portion of ADVANCED-1 Trial of TARA-002 for the Treatment of Non-Muscle Invasive Bladder Cancer Expected in 2Q23 -
Start up Activities for Phase 2 Trial of TARA-002 in Lymphatic Malformations Underway; Trial Initiation Expected 2H23 -
Cash, Cash Equivalents and Investments of $102.3M as of December 31, 2022 Expected to Fund Operations and Data Milestones into 2025 -
YORK, March 8, 2023 - Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative
therapies for the treatment of cancer and rare diseases, today announced financial results for the full year and fourth quarter ended
December 31, 2022, and provided a business update.
key data and milestones expected from our clinical programs for TARA-002, including data from the Phase 1a portion of the ADVANCED-1
trial in non-muscle invasive bladder cancer (NMIBC) in the second quarter of 2023, we believe this year will be a particularly exciting
time for Protara," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We look forward to leveraging
data from the ADVANCED-1 trial to inform the design of further clinical studies in our NIMBC program. For our program in lymphatic malformations
(LMs), a highly underserved pediatric population for which we believe TARA-002 could serve as a meaningful intervention, we have begun
Phase 2 clinical trial start up activities and anticipate initiating the trial in the second half of this year. We believe we are well
positioned to successfully execute on our pipeline programs and look forward to providing updates in due course."
Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)
Quarter and Full Year 2022 Financial Results
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.
Invasive Bladder Cancer (NMIBC)
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.
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.
Chloride and Intestinal Failure-associated Liver Disease (IFALD)
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,
Protara is committed
to advancing transformative therapies for people with cancer and rare diseases. 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; Protara's financial position; 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;
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 the COVID-19 pandemic on Protara's business and the global economy as well as the impact on Protara's
contract research organizations, study sites or other clinical partners; the impact of market volatility on cash reserves; the loss of
key members of management; the impact of general U.S. and foreign, economic, industry, market, regulatory or political conditions; 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
Protara Therapeutics,
except share and per share data)
| December 31, | ||||||||
| 2022 | 2021 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 24,127 | $ | 35,724 | ||||
| Marketable debt securities | 60,243 | 55,505 | ||||||
| Prepaid expenses and other current assets | 1,776 | 1,883 | ||||||
| Total current assets | 86,146 | 93,112 | ||||||
| Restricted cash, non-current | 745 | 745 | ||||||
| Marketable debt securities, non-current | 17,886 | 39,467 | ||||||
| Property and equipment, net | 1,592 | 1,719 | ||||||
| Operating lease right-of-use asset | 6,277 | 7,171 | ||||||
| Goodwill | - | 29,517 | ||||||
| Other assets | 644 | 865 | ||||||
| Total assets | $ | 113,290 | $ | 172,596 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,586 | $ | 954 | ||||
| Accrued expenses | 3,237 | 2,489 | ||||||
| Operating lease liability | 917 | 855 | ||||||
| Total current liabilities | 5,740 | 4,298 | ||||||
| Operating lease liability, non-current | 5,467 | 6,384 | ||||||
| Total liabilities | 11,207 | 10,682 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' Equity | ||||||||
| Preferred stock, $0.001 par value, authorized 10,000,000 shares: | ||||||||
| Series 1 convertible preferred stock, 8,028 shares authorized at December 31, 2022 and 2021, respectively 8,027 shares issued and outstanding as of December 31, 2022 and 2021, respectively. | - | - | ||||||
| Common stock, $0.001 par value, authorized 100,000,000 shares: | ||||||||
| Common stock, 11,267,389 and 11,235,731 shares issued and outstanding as of December 31, 2022 and 2021, respectively. | 11 | 11 | ||||||
| Additional paid in capital | 262,724 | 256,126 | ||||||
| Accumulated deficit | (159,964 | ) | (94,012 | ) | ||||
| Accumulated other comprehensive income (loss) | (688 | ) | (211 | ) | ||||
| Total stockholders' equity | 102,083 | 161,914 | ||||||
| Total liabilities and stockholders' equity | $ | 113,290 | $ | 172,596 |
Statements of Operations and Comprehensive Loss
thousands, except share and per share data)
| (Unaudited) | (Audited) | |||||||||||||||
| Three months ended | Years Ended December 31, | |||||||||||||||
| 12/31/2022 | 12/31/2021 | 2022 | 2021 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 4,989 | $ | 4,068 | $ | 16,808 | $ | 21,088 | ||||||||
| General and administrative | 5,003 | 6,220 | 20,737 | 26,401 | ||||||||||||
| Loss on impairment of goodwill | 29,517 | - | 29,517 | - | ||||||||||||
| Total operating expenses | 39,509 | 10,288 | 67,062 | 47,489 | ||||||||||||
| Loss from operations | (39,509 | ) | (10,288 | ) | (67,062 | ) | (47,489 | ) | ||||||||
| Interest and investment income | 543 | 59 | 1,110 | 237 | ||||||||||||
| Net loss | 38,966 | (10,229 | ) | (65,952 | ) | (47,252 | ) | |||||||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (3.46 | ) | $ | (0.91 | ) | $ | (5.86 | ) | $ | (4.21 | ) | ||||
| Weighted average shares outstanding, basic and diluted | 11,267,389 | 11,235,731 | 11,259,615 | 11,232,576 | ||||||||||||
| Other comprehensive income (loss): | ||||||||||||||||
| Net unrealized (loss) gain on marketable debt securities | 442 | (172 | ) | (477 | ) | (211 | ) | |||||||||
| Other comprehensive income (loss) | 442 | (172 | ) | (477 | ) | (211 | ) | |||||||||
| Comprehensive Loss | $ | (38,524 | ) | $ | (10,401 | ) | $ | (66,429 | ) | $ | (47,463 | ) |
Protara Therapeutics