Full Press Release Details
Therapeutics Announces Second Quarter 2025 Financial Results and Provides Business Update
NEW YORK, July 31, 2025
-- 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 second quarter ended June 30, 2025, and provided a business update.
"As we look toward the
second half of the year, we believe we are well positioned to continue to advance our pipeline of potentially transformative therapies
for the treatment of patients with cancer and rare diseases," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics.
"We continue to execute on our ADVANCED-2 trial of TARA-002 in non-muscle invasive bladder cancer (NMIBC) and look forward to presenting
interim results in the first quarter of 2026. In addition, we remain on track to dose the first patient in our THRIVE-3 registrational
trial of IV Choline Chloride in patients dependent on parenteral support (PS) by the end of this quarter and expect to provide an interim
update from the STARBORN-1 trial in pediatric patients with lymphatic malformations (LMs) in the fourth quarter of this year."
Recent Progress and Highlights
IV Choline Chloride for Patients on Parenteral Support (PS)
Second Quarter 2025 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 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 three 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
IV Choline Chloride is an investigational, intravenous phospholipid
substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for
phospholipids that are critical for healthy liver function that also play an important role in modulating gene expression, cell membrane
signaling, brain development and neurotransmission, muscle function, and bone health. PS patients are unable to synthesize choline from
enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 78% of patients dependent
on PS are choline-deficient and of those approximately 63% have some degree of liver dysfunction, which can lead to hepatic failure. Every
year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term
PS. IV Choline Chloride has the potential to become the first U.S. Food and Drug Administration (FDA) approved IV choline formulation
for PS patients. It has been granted Orphan Drug Designation by the FDA for the prevention and/or treatment of choline deficiency in patients
on long-term PN and been granted Fast Track Designation as a source of choline when oral or enteral nutrition is not possible, insufficient,
or contraindicated. The U.S. Patent and Trademark Office has issued us a U.S. patent claiming a choline composition
and a U.S. patent claiming a method for treating choline deficiency with a choline composition, each with a term expiring in
About Protara Therapeutics,
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.
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 (including the timing of any particular phases of such trials and the timing of the announcement
of any data produced during such trials or phases thereof); statements related to expectations regarding interactions with the U.S.
Food and Drug Administration (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 and future periods. 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
PROTARA THERAPEUTICS, INC. AND SUBSIDIARIES
Unaudited Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
| As of | ||||||||
| June 30, 2025 | December 31, 2024 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 31,496 | $ | 162,798 | ||||
| Marketable debt securities | 90,720 | 7,494 | ||||||
| Prepaid expenses and other current assets | 2,875 | 1,863 | ||||||
| Total current assets | 125,091 | 172,155 | ||||||
| Restricted cash, non-current | 745 | 745 | ||||||
| Marketable debt securities, non-current | 23,392 | - | ||||||
| Property and equipment, net | 912 | 1,027 | ||||||
| Operating lease right-of-use asset | 3,725 | 4,255 | ||||||
| Other assets | 3,068 | 3,272 | ||||||
| Total assets | $ | 156,933 | $ | 181,454 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 5,299 | $ | 4,429 | ||||
| Accrued expenses and other current liabilities | 3,263 | 5,408 | ||||||
| Operating lease liability | 1,199 | 1,124 | ||||||
| Total current liabilities | 9,761 | 10,961 | ||||||
| Operating lease liability, non-current | 2,749 | 3,359 | ||||||
| Total liabilities | 12,510 | 14,320 | ||||||
| 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 June 30, 2025 and December 31, 2024, 5,615 and 7,991 shares issued and outstanding as of June 30, 2025 and December 31, 2024, respectively | - | - | ||||||
| Common stock, $0.001 par value, authorized 100,000,000 shares: | ||||||||
| Common stock, 38,581,863 and 35,044,772 shares issued and outstanding as of June 30, 2025 and December 31, 2024, respectively | 39 | 35 | ||||||
| Additional paid-in capital | 416,161 | 412,077 | ||||||
| Accumulated deficit | (271,854 | ) | (244,980 | ) | ||||
| Accumulated other comprehensive income (loss) | 77 | 2 | ||||||
| Total stockholders' equity | 144,423 | 167,134 | ||||||
| Total liabilities and stockholders' equity | $ | 156,933 | $ | 181,454 |
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 June 30, | For the Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 10,770 | $ | 6,387 | $ | 19,918 | $ | 14,135 | ||||||||
| General and administrative | 5,816 | 4,274 | 10,792 | 8,377 | ||||||||||||
| Total operating expenses | 16,586 | 10,661 | 30,710 | 22,512 | ||||||||||||
| Income (Loss) from operations | (16,586 | ) | (10,661 | ) | (30,710 | ) | (22,512 | ) | ||||||||
| Other income (expense), net: | ||||||||||||||||
| Interest and investment income (expense) | 1,626 | 1,148 | 3,355 | 1,904 | ||||||||||||
| Other income (expense) | - | - | 481 | - | ||||||||||||
| Other income (expense), net | 1,626 | 1,148 | 3,836 | 1,904 | ||||||||||||
| Net income (loss) | $ | (14,960 | ) | $ | (9,513 | ) | $ | (26,874 | ) | $ | (20,608 | ) | ||||
| Other comprehensive income (loss): | ||||||||||||||||
| Net unrealized gain (loss) on marketable debt securities | (12 | ) | 1 | 75 | 31 | |||||||||||
| Other comprehensive income (loss): | (12 | ) | 1 | 75 | 31 | |||||||||||
| Comprehensive income (loss) | $ | (14,972 | ) | $ | (9,512 | ) | $ | (26,799 | ) | $ | (20,577 | ) | ||||
| Net income (loss) per share attributable to common stockholders, basic and diluted | $ | (0.35 | ) | $ | (0.45 | ) | $ | (0.65 | ) | $ | (1.26 | ) | ||||
| Weighted-average shares outstanding, basic and diluted | 42,270,855 | 21,233,163 | 41,493,714 | 16,327,056 |
Protara Therapeutics