Full Press Release Details
Protara Therapeutics Announces Fourth Quarter
Financial Results and Provides Business Update
NEW YORK, March 13, 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, 2023 and provided a business update.
"We expect 2024 to be a milestone-rich year
for Protara and believe we are well positioned to continue to execute on advancing our pipeline to deliver meaningful new therapies to
patients with cancer and rare diseases," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Notably,
we anticipate sharing Phase 1b proof of concept (POC) and preliminary Phase 2 data for our lead TARA-002 indication, non-muscle invasive
bladder cancer (NMIBC), later this year, which we believe will continue to support its potential to play an important role in the evolving
NMIBC treatment landscape. Progress also continues for our program of TARA-002 in lymphatic malformations (LMs), a highly underserved
pediatric population, with dosing in our Phase 2 study now underway."
Recent Progress and Highlights
IV Choline Chloride for Patients on Parenteral
Fourth Quarter and Full Year 2023 Financial
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 pro-inflammatory response with release of cytokines
such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma IL-6, IL-10, IL-12. TARA-002 also directly kills tumor cells and triggers
a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.
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 Intravenous (IV) Choline Chloride
IV Choline Chloride is an investigational, IV
phospholipid substrate replacement therapy initially in development for patients receiving PN. Choline is a known important substrate
for phospholipids that are critical for healthy liver function and also plays an important role in modulating gene expression, cell membrane
signaling, brain development and neurotransmission, muscle function, and bone health. PN patients are unable to synthesize choline from
enteral nutrition sources, and there are currently no available PN formulations containing choline. Approximately 80 percent of PN-dependent
patients are choline-deficient and have some degree of liver damage, which can lead to hepatic failure. IV Choline Chloride has been granted
Orphan Drug Designation by the FDA for the prevention of choline deficiency in PN patients. The Company was issued a patent claiming a
choline composition with a term expiring in 2041.
About Protara Therapeutics, Inc.
Protara is a clinical-stage biotechnology company committed to advancing
transformative therapies for people with cancer and rare diseases. Protara's portfolio includes its lead candidate, TARA-002, an
investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations
(LMs). The Company is evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients with carcinoma in situ (CIS) who are unresponsive
or na ve to treatment with Bacillus Calmette-Gu rin (BCG), as well as a Phase 2 trial in pediatric patients with LMs. Additionally,
Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral nutrition
who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit www.protaratx.com.
1. Klinische Einheit, or KE, is a German term indicating a specified
weight of dried cells in a vial.
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; failure to attract and retain management and key personnel; 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.
Consolidated Balance Sheets
(in thousands, except share and per share data)
| December 31, | ||||||||
| 2023 | 2022 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 39,586 | $ | 24,127 | ||||
| Marketable debt securities | 25,994 | 60,243 | ||||||
| Prepaid expenses and other current assets | 3,125 | 1,776 | ||||||
| Total current assets | 68,705 | 86,146 | ||||||
| Restricted cash, non-current | 745 | 745 | ||||||
| Marketable debt securities, non-current | - | 17,886 | ||||||
| Property and equipment, net | 1,296 | 1,592 | ||||||
| Operating lease right-of-use asset | 5,264 | 6,277 | ||||||
| Other assets | 2,944 | 644 | ||||||
| Total assets | $ | 78,954 | $ | 113,290 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,434 | $ | 1,586 | ||||
| Accrued expenses | 2,732 | 3,237 | ||||||
| Operating lease liability | 983 | 917 | ||||||
| Total current liabilities | 6,149 | 5,740 | ||||||
| Operating lease liability, non-current | 4,484 | 5,467 | ||||||
| Total liabilities | 10,633 | 11,207 | ||||||
| Commitments and contingencies (Note 10) | ||||||||
| 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, 2023 and 2022, 7,991 and 8,027 shares issued and outstanding as of December 31, 2023 and 2022, respectively. | - | - | ||||||
| Common stock, $0.001 par value, authorized 100,000,000 shares: | ||||||||
| Common stock, 11,364,903 and 11,267,389 shares issued and outstanding as of December 31, 2023 and 2022, respectively. | 11 | 11 | ||||||
| Additional paid in capital | 268,725 | 262,724 | ||||||
| Accumulated deficit | (200,384 | ) | (159,964 | ) | ||||
| Accumulated other comprehensive income (loss) | (31 | ) | (688 | ) | ||||
| Total stockholders' equity | 68,321 | 102,083 | ||||||
| Total liabilities and stockholders' equity | $ | 78,954 | $ | 113,290 |
PROTARA THERAPEUTICS, INC. AND SUBSIDIARIES
Consolidated Statements of Operations and Comprehensive
(in thousands, except share and per share data)
| (Unaudited) | (Audited) | |||||||||||||||
| For the Three Months Ended December 31, | For the Years Ended December 31, | |||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 6,381 | $ | 4,989 | $ | 24,989 | $ | 16,808 | ||||||||
| General and administrative | 4,660 | 5,003 | 18,624 | 20,737 | ||||||||||||
| Loss on impairment of goodwill | - | 29,517 | - | 29,517 | ||||||||||||
| Total operating expenses | 11,041 | 39,509 | 43,613 | 67,062 | ||||||||||||
| Loss from operations | (11,041 | ) | (39,509 | ) | (43,613 | ) | (67,062 | ) | ||||||||
| Other income (expense), net: | ||||||||||||||||
| Interest and investment income | 820 | 543 | 3,193 | 1,110 | ||||||||||||
| Other income (expense), net | 820 | 543 | 3,193 | 1,110 | ||||||||||||
| Net loss | $ | (10,221 | ) | $ | (38,966 | ) | $ | (40,420 | ) | $ | (65,952 | ) | ||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.90 | ) | $ | (3.46 | ) | $ | (3.57 | ) | $ | (5.86 | ) | ||||
| Weighted-average shares outstanding, basic and diluted | 11,364,903 | 11,267,389 | 11,331,338 | 11,259,615 | ||||||||||||
| Other comprehensive income (loss): | ||||||||||||||||
| Net unrealized gain (loss) on marketable debt securities | 134 | (442 | ) | 657 | (477 | ) | ||||||||||
| Other comprehensive income (loss) | 134 | (443 | ) | 657 | (477 | ) | ||||||||||
| Comprehensive Loss | $ | (10,087 | ) | $ | (38,524 | ) | $ | (39,763 | ) | $ | (66,429 | ) |
Protara Therapeutics