- Dose Escalation Ongoing in Phase 1 ADVANCED-1 Study of TARA-002 for the Treatment of Non-Muscle Invasive Bladder Cancer -
- Following Recent Feedback from the U.S. Food and Drug Administration, Phase 2 Study of TARA-002 in Lymphatic Malformations Expected to Initiate in 2023 -
- Strong Cash, Cash Equivalents and Investments Position of $107.1M as of September 30, 2022 Now Expected to Fund Operations Into 2H2024 -
NEW YORK, Nov. 03, 2022 (GLOBE NEWSWIRE) -- 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 third quarter ended September 30, 2022 and provided a business update.
“We continue to make meaningful progress with dose escalation in the ongoing Phase 1a portion of our ADVANCED-1 study of TARA-002 in non-muscle invasive bladder cancer (NMIBC), and look forward to ultimately utilizing these data, as well as data from our ongoing preclinical work, to design effective later-stage clinical trials in this indication,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We are pleased to share that we have received initial feedback from the U.S. Food and Drug Administration (FDA) on our proposed Phase 2 study protocol evaluating TARA-002 in Lymphatic Malformations (LMs), and now expect to initiate the study in 2023. LMs represents a highly underserved rare pediatric indication for which we believe TARA-002 could serve as a much-needed treatment option.”
Recent Highlights
TARA-002 in NMIBC
TARA-002 in LMs
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)
Third Quarter 2022 Financial Results
About TARA-002
TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and 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 Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully demonstrated 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, tumor necrosis factor (TNF)-alpha, granulocyte colony-stimulating factor, and granulocyte-macrophage colony-stimulating factor are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.
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 identifying and 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 therapy for the treatment of intestinal failure-associated liver disease. 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, including potential alignment with the FDA on, and finalization of, clinical trial design for TARA-002 in pediatric LM patients; Protara’s financial footing; 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; the impact of the COVID-19 pandemic on Protara’s business and the global economy as well as the impact on Protara’s contract research organizations, study sites or other clinical partners; 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 or political 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.
Unaudited Condensed Consolidated Balance Sheets
(in thousands, except share and per share data) As of September 30,
2022 December 31,
2021 Assets Current assets: Cash and cash equivalents $47,498 $35,724 Marketable debt securities 48,819 55,505 Prepaid expenses and other current assets 2,350 1,883 Total current assets 98,667 93,112 Restricted cash, non-current 745 745 Marketable debt securities, non-current 10,760 39,467 Property and equipment, net 1,662 1,719 Operating lease right-of-use asset 6,506 7,171 Goodwill 29,517 29,517 Other assets 734 865 Total assets $148,591 $172,596 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $456 $954 Accrued expenses 2,354 2,489 Operating lease liability 901 855 Total current liabilities 3,711 4,298 Operating lease liability, non-current 5,702 6,384 Total liabilities 9,413 10,682 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 September 30, 2022 and December 31, 2021, 8,027 shares issued and outstanding as of September 30, 2022 and December 31, 2021. - - Common stock, $0.001 par value, authorized 100,000,000 shares: Common stock, 11,267,389 and 11,235,731 shares issued and outstanding as of September 30, 2022 and December 31, 2021, respectively. 11 11 Additional paid-in capital 261,294 256,126 Accumulated deficit (120,997) (94,012)Accumulated other comprehensive income (loss) (1,130) (211)Total stockholders’ equity 139,178 161,914 Total liabilities and stockholders’ equity $148,591 $172,596
PROTARA THERAPEUTICS, INC.
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, 2022 2021 2022 2021 Operating expenses: Research and development $3,466 $4,093 $11,819 $17,020 General and administrative 4,508 6,737 15,734 20,182 Total operating expenses 7,974 10,830 27,553 37,202 Loss from operations (7,974) (10,830) (27,553) (37,202)Other income (expense), net: Interest and investment income 283 53 568 178 Other income (expense), net 283 53 568 178 Net loss (7,691) (10,777) (26,985) (37,024) Net loss per share attributable to common stockholders, basic and diluted $(0.68) $(0.96) $(2.40) $(3.30)Weighted-average shares outstanding, basic and diluted 11,265,475 11,235,507 11,256,995 11,231,513 Other comprehensive income (loss): Net unrealized (loss) gain on marketable debt securities (8) 62 (919) (39)Other comprehensive income (loss) (8) 62 (919) (39)Comprehensive Loss $(7,699) $(10,715) $(27,904) $(37,063)
Company Contact:
Justine O'Malley
Protara Therapeutics
Justine.OMalley@protaratx.com
646-817-2836