NEW YORK, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today provided a business update and announced financial results for the third quarter ended September 30, 2024.
“We continue to make significant progress advancing all of our ongoing programs as we strive toward our goal of bringing life-changing therapies to patients impacted by cancer and rare diseases,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “Notably, we remain on track to report interim data in the fourth quarter of 2024 from our ADVANCED-2 study of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC). Given its ease of use, favorable safety profile, and encouraging early clinical data, we believe TARA-002 could be an impactful addition to the NMIBC treatment landscape.”
Mr. Shefferman added, “In addition to our NMIBC program, we remain on track to commence the pivotal THRIVE-3 study of intravenous (IV) Choline Chloride, for which we recently received Fast Track designation from the U.S. Food and Drug Administration (FDA), in the first quarter of 2025. Finally, we continue to expect interim data in the first half of 2025 from our ongoing Phase 2 STARBORN-1 trial of TARA-002 in lymphatic malformations (LMs), a rare condition primarily impacting children for which there are no FDA approved therapies.”
Recent Progress and Highlights
TARA-002 in NMIBC
IV Choline Chloride for Patients on Parenteral Support (PS)
TARA-002 in LMs
Third Quarter 2024 Financial Results
About TARA-002
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 80% of patients dependent on PS are choline-deficient and have some degree of liver damage, 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 and has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PS patients. The U.S. Patent and Trademark Office has issued the Company 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.
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 naïve to treatment with Bacillus Calmette-Guérin (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.
References
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. AND SUBSIDIARIES Unaudited Condensed Consolidated Balance Sheets (in thousands, except share and per share data) | ||||||||
| As of | ||||||||
| September 30, 2024 | December 31, 2023 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 51,757 | $ | 39,586 | ||||
| Marketable debt securities | 29,742 | 25,994 | ||||||
| Prepaid expenses and other current assets | 3,583 | 3,125 | ||||||
| Total current assets | 85,082 | 68,705 | ||||||
| Restricted cash, non-current | 745 | 745 | ||||||
| Property and equipment, net | 1,109 | 1,296 | ||||||
| Operating lease right-of-use asset | 4,514 | 5,264 | ||||||
| Other assets | 2,640 | 2,944 | ||||||
| Total assets | $ | 94,090 | $ | 78,954 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,295 | $ | 2,434 | ||||
| Accrued expenses and other current liabilities | 5,268 | 2,732 | ||||||
| Operating lease liability | 1,079 | 983 | ||||||
| Total current liabilities | 8,642 | 6,149 | ||||||
| Operating lease liability, non-current | 3,657 | 4,484 | ||||||
| Total liabilities | 12,299 | 10,633 | ||||||
| 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 September 30, 2024 and December 31, 2023, 7,991 shares issued and outstanding as of September 30, 2024 and December 31, 2023. | - | - | ||||||
| Common stock, $0.001 par value, authorized 100,000,000 shares: Common stock, 20,629,772 and 11,364,903 shares issued and outstanding as of September 30, 2024 and December 31, 2023, respectively. | 21 | 11 | ||||||
| Additional paid-in capital | 313,952 | 268,725 | ||||||
| Accumulated deficit | (232,211 | ) | (200,384 | ) | ||||
| Accumulated other comprehensive income (loss) | 29 | (31 | ) | |||||
| Total stockholders’ equity | 81,791 | 68,321 | ||||||
| Total liabilities and stockholders’ equity | $ | 94,090 | $ | 78,954 | ||||
| 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 September 30, | For the Nine Months Ended September 30, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 8,070 | $ | 6,218 | $ | 22,205 | $ | 18,608 | ||||||||
| General and administrative | 4,260 | 4,482 | 12,637 | 13,964 | ||||||||||||
| Total operating expenses | 12,330 | 10,700 | 34,842 | 32,572 | ||||||||||||
| Loss from operations | (12,330 | ) | (10,700 | ) | (34,842 | ) | (32,572 | ) | ||||||||
| Other income (expense), net: | ||||||||||||||||
| Interest and investment income | 1,111 | 840 | 3,015 | 2,373 | ||||||||||||
| Other income (expense), net | 1,111 | 840 | 3,015 | 2,373 | ||||||||||||
| Net income (loss) | $ | (11,219 | ) | $ | (9,860 | ) | $ | (31,827 | ) | $ | (30,199 | ) | ||||
| Net income (loss) per share attributable to common stockholders, basic and diluted | $ | (0.50 | ) | $ | (0.87 | ) | $ | (1.74 | ) | $ | (2.67 | ) | ||||
| Weighted-average shares outstanding, basic and diluted | 22,329,772 | 11,347,887 | 18,342,566 | 11,320,027 | ||||||||||||
| Other comprehensive income (loss): | ||||||||||||||||
| Net unrealized gain (loss) on marketable debt securities | 29 | 171 | 60 | 523 | ||||||||||||
| Other comprehensive income (loss) | 29 | 171 | 60 | 523 | ||||||||||||
| Comprehensive income (loss) | $ | (11,190 | ) | $ | (9,689 | ) | $ | (31,767 | ) | $ | (29,676 | ) | ||||
Company Contact:
Justine O'Malley
Protara Therapeutics
This email address is being protected from spambots. You need JavaScript enabled to view it.
646-817-2836
| Last Trade: | US$5.37 |
| Daily Change: | 0.19 3.67 |
| Daily Volume: | 1,150,166 |
| Market Cap: | US$184.300M |
December 11, 2024 December 09, 2024 December 05, 2024 | |
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