Full Press Release Details
Protara Therapeutics Announces First Quarter
2023 Financial Results and Business Update
NEW YORK, May 4, 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 first quarter ended March 31, 2023 and provided a business update.
"We are off to a strong start in 2023,
notably with the recent presentation of positive preliminary data from the dose escalation portion of the ADVANCED-1 trial of
TARA-002 in patients with high-grade non-muscle invasive bladder cancer (NMIBC) and expect to sustain this productive momentum in
the months ahead," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Supported by these exciting
preliminary findings, we are keenly focused on continued execution across the NMIBC program, including the recently announced Phase
1b/2 ADVANCED-2 trial of TARA-002 in patients with high-grade carcinoma in situ (CIS), which we expect to commence in the second
half of this year. With final regulatory clearance from the U.S. Food and Drug Administration (FDA) on our Phase 2 trial in
lymphatic malformations (LMs), we look forward to initiating STARBORN-1 in the near future and progressing our program to bring a
much-needed therapy to these pediatric patients who have no approved treatments today. We remain committed to unlocking the full
potential of TARA-002 and look forward to sharing our progress throughout the coming quarters."
TARA-002 in Lymphatic Malformations (LMs)
IV Choline Chloride in Intestinal Failure Associated
Liver Disease (IFALD)
First Quarter 2023 Financial Results
TARA-002 is an investigational cell therapy in
development for the treatment of NMIBC and of 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 approved in Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully
shown 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, and tumor necrosis factor (TNF)-alpha are secreted by immune cells to induce a strong inflammatory reaction and destroy the
About Non-Muscle Invasive Bladder Cancer (NMIBC)
Bladder cancer is 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.
About Lymphatic Malformations (LMs)
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 in PN patients.
About Protara Therapeutics, Inc.
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 for patients dependent on parenteral nutrition. 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 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 market volatility on cash reserves; the loss of key members of management; the impact of general U.S.
and foreign, economic, industry, market, regulatory, political or public health 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 as required by law.
PROTARA THERAPEUTICS, INC. AND SUBSIDIARIES
Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
| As of | ||||||||
| March 31, 2023 | December 31, 2022 | |||||||
| Assets | (unaudited) | |||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 21,035 | $ | 24,127 | ||||
| Marketable debt securities | 65,575 | 60,243 | ||||||
| Prepaid expenses and other current assets | 8,006 | 1,776 | ||||||
| Total current assets | 94,616 | 86,146 | ||||||
| Restricted cash, non-current | 745 | 745 | ||||||
| Marketable debt securities, non-current | 2,893 | 17,886 | ||||||
| Property and equipment, net | 1,521 | 1,592 | ||||||
| Operating lease right-of-use asset | 6,044 | 6,277 | ||||||
| Other assets | 586 | 644 | ||||||
| Total assets | $ | 106,405 | $ | 113,290 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 4,081 | $ | 1,586 | ||||
| Accrued expenses | 1,396 | 3,237 | ||||||
| Operating lease liability | 933 | 917 | ||||||
| Total current liabilities | 6,410 | 5,740 | ||||||
| Operating lease liability, non-current | 5,227 | 5,467 | ||||||
| Total liabilities | 11,637 | 11,207 | ||||||
| Commitments and contingencies (Note 8) | ||||||||
| Stockholders' Equity: | ||||||||
| Preferred stock, $0.001 par value, authorized 10,000,000 shares: Series 1 Convertible Preferred Stock, 8,028 shares authorized at March 31, 2023 and December 31, 2022, 8,027 shares issued and outstanding as of March 31, 2023 and December 31, 2022. | - | - | ||||||
| Common stock, $0.001 par value, authorized 100,000,000 shares: Common stock, 11,306,753 and 11,267,389 shares issued and outstanding as of March 31, 2023 and December 31, 2022, respectively. | 11 | 11 | ||||||
| Additional paid-in capital | 264,235 | 262,724 | ||||||
| Accumulated deficit | (169,009 | ) | (159,964 | ) | ||||
| Accumulated other comprehensive income (loss) | (469 | ) | (688 | ) | ||||
| Total stockholders' equity | 94,768 | 102,083 | ||||||
| Total liabilities and stockholders' equity | $ | 106,405 | $ | 113,290 |
PROTARA THERAPEUTICS, INC. AND SUBSIDIARIES
Unaudited Condensed Consolidated Statements
of Operations and Comprehensive Loss
(in thousands, except share and per share data)
| For the Three Months Ended March 31, | ||||||||
| 2023 | 2022 | |||||||
| Operating expenses: | ||||||||
| Research and development | $ | 5,143 | $ | 5,269 | ||||
| General and administrative | 4,589 | 5,605 | ||||||
| Total operating expenses | 9,732 | 10,874 | ||||||
| Loss from operations | (9,732 | ) | (10,874 | ) | ||||
| Other income (expense), net: | ||||||||
| Interest and investment income | 687 | 119 | ||||||
| Other income (expense), net | 687 | 119 | ||||||
| Net loss | (9,045 | ) | (10,755 | ) | ||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.80 | ) | $ | (0.96 | ) | ||
| Weighted-average shares outstanding, basic and diluted | 11,303,869 | 11,250,127 | ||||||
| Other comprehensive income (loss): | ||||||||
| Net unrealized (loss) gain on marketable debt securities | 219 | (731 | ) | |||||
| Other comprehensive income (loss) | 219 | (731 | ) | |||||
| Comprehensive Loss | $ | (8,826 | ) | $ | (11,486 | ) |
Protara Therapeutics