Full Press Release Details
Protara Therapeutics Announces First Quarter
2025 Financial Results and Provides Business Update
NEW YORK, May 8, 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 first quarter ended March 31, 2025, and provided a business update.
"We have made significant progress thus
far in 2025, notably with the recent presentation of positive interim results from our ADVANCED-2 trial of TARA-002 in BCG-Unresponsive
and BCG-Na ve patients which demonstrated durable 12-month landmark responses," said Jesse Shefferman, Chief Executive Officer
of Protara Therapeutics. "We believe TARA-002 is well positioned to make a meaningful difference
in the lives of patients with non-muscle invasive bladder cancer (NMIBC). In addition to our NMIBC program, we are pleased with
the continued progress we have made across our rare disease programs and look forward to several exciting data milestones in the coming
Recent Progress and Highlights
IV Choline Chloride for Patients on Parenteral
First Quarter 2025 Financial Results
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.
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
Non-Muscle Invasive Bladder Cancer (NMIBC)
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
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 80% 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 2041.
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.
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 reporting data from approximately 25 6-month evaluable
BCG-Unresponsive patients by the end of 2025); 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 law.
PROTARA THERAPEUTICS, INC. AND SUBSIDIARIES
Unaudited Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
| As of | ||||||||
| March 31, 2025 | December 31, 2024 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 91,461 | $ | 162,798 | ||||
| Marketable debt securities | 32,902 | 7,494 | ||||||
| Prepaid expenses and other current assets | 2,169 | 1,863 | ||||||
| Total current assets | 126,532 | 172,155 | ||||||
| Restricted cash, non-current | 745 | 745 | ||||||
| Marketable debt securities, non-current | 33,154 | - | ||||||
| Property and equipment, net | 988 | 1,027 | ||||||
| Operating lease right-of-use asset | 3,992 | 4,255 | ||||||
| Other assets | 3,148 | 3,272 | ||||||
| Total assets | $ | 168,559 | $ | 181,454 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,310 | $ | 4,429 | ||||
| Accrued expenses and other current liabilities | 3,538 | 5,408 | ||||||
| Operating lease liability | 1,170 | 1,124 | ||||||
| Total current liabilities | 7,018 | 10,961 | ||||||
| Operating lease liability, non-current | 3,057 | 3,359 | ||||||
| Total liabilities | 10,075 | 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 March 31, 2025 and December 31, 2024, 5,615 and 7,991 shares issued and outstanding as of March 31, 2025 and December 31, 2024, respectively | - | - | ||||||
| Common stock, $0.001 par value, authorized 100,000,000 shares: | ||||||||
| Common stock, 38,577,813 and 35,044,772 shares issued and outstanding as of March 31, 2025 and December 31, 2024, respectively | 39 | 35 | ||||||
| Additional paid-in capital | 415,250 | 412,077 | ||||||
| Accumulated deficit | (256,894 | ) | (244,980 | ) | ||||
| Accumulated other comprehensive income (loss) | 89 | 2 | ||||||
| Total stockholders' equity | 158,484 | 167,134 | ||||||
| Total liabilities and stockholders' equity | $ | 168,559 | $ | 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 March 31, | ||||||||
| 2025 | 2024 | |||||||
| Operating expenses: | ||||||||
| Research and development | $ | 9,148 | $ | 7,748 | ||||
| General and administrative | 4,976 | 4,103 | ||||||
| Total operating expenses | 14,124 | 11,851 | ||||||
| Income (Loss) from operations | (14,124 | ) | (11,851 | ) | ||||
| Other income (expense), net: | ||||||||
| Interest and investment income (expenses) | 1,729 | 756 | ||||||
| Other income (expense) | 481 | - | ||||||
| Other income (expense), net | 2,210 | 756 | ||||||
| Net income (loss) | $ | (11,914 | ) | $ | (11,095 | ) | ||
| Other comprehensive income (loss): | ||||||||
| Net unrealized gain (loss) on marketable debt securities | 87 | 30 | ||||||
| Other comprehensive income (loss) | 87 | 30 | ||||||
| Comprehensive income (loss) | $ | (11,827 | ) | $ | (11,065 | ) | ||
| Net income (loss) per share attributable to common stockholders, basic and diluted | $ | (0.29 | ) | $ | (0.97 | ) | ||
| Weighted-average shares outstanding, basic and diluted | 40,707,937 | 11,420,948 |
Protara Therapeutics