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Protara Therapeutics (NasdaqGM:TARA) Update Summary Company Overview - Protara Therapeutics is a clinical-stage company focused on developing transformative therapies for cancer and rare diseases, with its lead asset being TARA-002, aimed at treating various conditions including lymphatic malformations (LMs) and non-muscle invasive bladder cancer (NMIBC) [5][6][10]. Industry Context - Lymphatic malformations are rare congenital conditions characterized by abnormal lymphatic vessel development, often diagnosed in early childhood. Current treatment options are limited, with no FDA-approved therapies available for LMs, leading to significant unmet medical needs [6][8][24]. Key Points from the Call STARBORN-1 Trial Results - The ongoing phase two open-label STARBORN-1 trial is assessing TARA-002 in pediatric patients with macrocystic and mixed cystic LMs. Interim results show that 80% of patients achieved clinical success, defined as a 90%-100% reduction in total LM volume or a substantial response of 60%-90% [4][16][18]. - As of the data cutoff on November 12, 2025, 100% of patients who completed the eight-week response assessment achieved clinical success [16]. - The trial has enrolled 12 patients, with a focus on safety and efficacy across different age cohorts [14][16]. Mechanism of Action - TARA-002 is a genetically distinct strain of Streptococcus pyogenes that activates the immune system to target and eliminate mutated cells in lymphatic cysts. This mechanism promotes tissue remodeling and long-term resolution of cysts [12][13][19]. Market Opportunity - Approximately 1,400-1,800 new LM cases are diagnosed annually in the U.S., with an estimated 20,000 patients living with macro and mixed cystic LMs. The majority of these cases are diagnosed before the age of three [7][8][70]. - Current treatment options include surgical interventions and off-label sclerosing agents, which have high complication and recurrence rates, with 40%-70% of interventions being unsuccessful [8][24]. Competitive Landscape - TARA-002 is positioned as a promising alternative to existing treatments, leveraging a differentiated immune potentiator mechanism that minimizes tissue destruction compared to traditional sclerosing agents [24][34]. - The historical efficacy of OK-432, a predecessor to TARA-002, supports the potential success of TARA-002 in treating LMs and other maxillofacial cysts [9][78]. Regulatory Path and Future Steps - Protara plans to engage with the FDA to discuss the path to approval for TARA-002 based on the positive interim data from the STARBORN-1 trial. The company aims to complete the trial by next year and is optimistic about the regulatory process [38][68][78]. - The potential for TARA-002 to treat other types of cystic malformations beyond LMs is being explored, with encouraging results from initial patient experiences [27][78]. Additional Insights - The safety profile of TARA-002 has been consistent with historical data from OK-432, with most adverse events being mild to moderate [18][19]. - The emotional and physical impact of LMs on pediatric patients is significant, highlighting the need for effective treatment options that can improve quality of life [24][25]. This summary encapsulates the key points discussed during the Protara Therapeutics update, focusing on the company's ongoing clinical trials, market potential, and the innovative approach of TARA-002 in addressing unmet medical needs in pediatric patients with lymphatic malformations.

TARA-002 in Lymphatic Malformations (LMs) - LMs represent a significant pediatric rare disease opportunity with no currently approved therapies[8] - TARA-002 has the potential to treat macrocystic and mixed cystic LMs, which most often are present in the head and neck region[11] - The incidence of LMs is approximately 1,400-1,800 cases per year, with a prevalence of approximately 20,000 patients seeking treatment[14] - Macrocystic LMs account for approximately 47%, microcystic LMs for approximately 21%, and mixed LMs for approximately 32% of cases[14] STARBORN-1 Interim Data - In the STARBORN-1 trial, TARA-002 demonstrated clinical success in 80% of patients that completed treatment[30] - Among evaluable patients in STARBORN-1, TARA-002 showed a 100% clinical success rate at 8-weeks post-treatment[30] - In evaluable patients, 83% with macrocystic LMs, 100% with mixed-cystic LMs and 100% with Ranula achieved complete or substantial response[35] - In the STARBORN-1 trial, 66.7% of patients experienced any grade TEAEs, with 8.3% experiencing Grade 3 TEAEs and no Grade 4/5 TEAEs[39] OK-432 Data Review - OK-432, the predecessor compound to TARA-002, has shown strong safety and efficacy results in over 500 U S pediatric LMs patients in a University of Iowa-led study[18] - OK-432 demonstrated a 69% clinical success rate in the immediate treatment group 6 months after enrollment[46] - OK-432 showed an 84% clinical success rate in patients with macrocystic lesion types[46]

Core Insights - Protara Therapeutics announced positive interim results from the Phase 2 STARBORN-1 trial for TARA-002, a cell-based therapy for pediatric patients with lymphatic malformations (LMs) [1][2] STARBORN-1 Interim Results - The interim analysis included 12 patients, with 8 evaluable at the eight-week post-treatment assessment; 7 out of 8 achieved clinical success [3][5] - The majority of patients (80%) who completed treatment and all patients (100%) who completed the eight-week assessment achieved clinical success [5][6] - 83% of macrocystic patients achieved a complete response, with one patient achieving a substantial response [6] Safety Profile - Adverse events were mostly mild to moderate, with no serious adverse events reported; common adverse events included swelling and fatigue [4][5] - One patient discontinued treatment due to a Grade 2 adverse event of fatigue [4] About TARA-002 - TARA-002 is a genetically distinct strain of streptococcus pyogenes, developed from the same master cell bank as OK-432, which has been the standard of care for LMs in Japan for 30 years [9] - TARA-002 has received Rare Pediatric Disease designation from the FDA for the treatment of LMs [9] About Lymphatic Malformations - Lymphatic malformations are rare congenital conditions, primarily affecting the head and neck, with significant morbidity including airway obstruction and recurrent infections [10] Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases, including TARA-002 for LMs and non-muscle invasive bladder cancer [11]

Core Insights - Protara Therapeutics, Inc. is hosting a conference call on November 19, 2025, to discuss new data from the interim analysis of the Phase 2 STARBORN-1 trial for TARA-002, a cell-based therapy for pediatric patients with lymphatic malformations [1][2] Group 1: Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases [5] - The company's lead candidate, TARA-002, is in development for non-muscle invasive bladder cancer and lymphatic malformations [5] - TARA-002 has received Rare Pediatric Disease designation from the U.S. FDA for the treatment of lymphatic malformations [3] Group 2: Product Details - TARA-002 is a genetically distinct strain of streptococcus pyogenes that retains immune-stimulating properties while being inactivated [3] - The therapy was developed from the same master cell banks as OK-432, which has been the standard of care in Japan for 30 years [3] - A large Phase 2 trial of OK-432 demonstrated significant clinical success in over 500 patients with lymphatic malformations [3] Group 3: Lymphatic Malformations - Lymphatic malformations are rare congenital conditions affecting lymphatic vessels, often diagnosed in early childhood [4] - More than 50% of lymphatic malformations are detected at birth, with 90% diagnosed before the age of three [4] - Common complications include airway obstruction, intralesional bleeding, and recurrent infections, leading to significant morbidity [4]

Core Insights - Protara Therapeutics, Inc. announced interim data from the Phase 2 ADVANCED-2 trial of TARA-002 for treating non-muscle invasive bladder cancer (NMIBC) [1][2] - The trial data will be presented at the 26th Annual Meeting of the Society of Urologic Oncology from December 2 to December 5, 2025 [1] - TARA-002 is an investigational cell therapy that has received Rare Pediatric Disease Designation from the FDA [3][6] Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing therapies for cancer and rare diseases [6] - The company's lead candidate, TARA-002, is being evaluated in ongoing Phase 2 trials for NMIBC and lymphatic malformations [6] - Protara is also developing IV Choline Chloride for patients on parenteral nutrition [6] Trial Details - The ADVANCED-2 trial includes BCG-Naïve patients with NMIBC, specifically those with carcinoma in situ or CIS [2] - The trial involves an induction course of six weekly intravesical instillations of TARA-002, followed by maintenance treatments [2] - The upcoming poster presentation will include updated safety and efficacy data from 31 enrolled patients [1][3] Disease Context - NMIBC accounts for approximately 80% of bladder cancer diagnoses in the U.S., with around 65,000 new cases each year [5] - Bladder cancer is the sixth most common cancer in the United States [5] Mechanism of Action - TARA-002 is hypothesized to activate immune cells in the tumor, leading to a pro-inflammatory response and the release of cytokines [4] - The therapy aims to directly kill tumor cells and enhance the antitumor immune response through immunogenic cell death [4]

Financial Performance - As of September 30, 2025, the company had an accumulated deficit of approximately $285.1 million[132]. - The company expects to continue incurring significant and increasing expenses and operating losses for at least the next few years as it develops and seeks marketing approvals for its product candidates[132]. - Net income (loss) for Q3 2025 was $(13.3) million, compared to $(11.2) million in Q3 2024, representing an increase in loss of $2.1 million[140]. - Total operating expenses for the nine months ended September 30, 2025, were $45.5 million, an increase of $10.6 million compared to the same period in 2024[146]. - Cash used in operating activities for the nine months ended September 30, 2025, was approximately $(39.4) million, an increase of $12.9 million compared to $(26.5) million in the same period of 2024[156]. - The company incurred a net loss of $40.1 million for the nine months ended September 30, 2025, compared to a net loss of $31.8 million for the same period in 2024[151]. - Net cash provided by financing activities was $1.8 million for the nine months ended September 30, 2025, a decrease from $42.0 million for the same period in 2024[158]. Research and Development - The ADVANCED-2 clinical trial for TARA-002 in NMIBC has enrolled 31 patients in Cohort A, with a complete response rate of 100% in BCG-Unresponsive patients at six months[117]. - The CR rate in BCG-Naïve patients was 76% at any time, with 63% at six months[118]. - The company plans to initiate the THRIVE-3 Phase 3 clinical trial for IV Choline Chloride by the end of 2025, with a primary endpoint measuring plasma choline concentration[126]. - In the THRIVE-1 study, 78% of patients dependent on parenteral support were found to be choline deficient, with 63% of those having liver dysfunction[125]. - The STARBORN-1 trial for TARA-002 in lymphatic malformations has enrolled approximately 30 patients, with interim data showing two patients achieving complete response after one injection[129]. - TARA-002 is being developed for both NMIBC and lymphatic malformations, with ongoing studies to explore its potential in combination therapies[121][127]. - IV Choline Chloride has received Orphan Drug Designation from the FDA for the prevention and/or treatment of choline deficiency in patients on long-term parenteral nutrition[123]. - Total research and development expenses for the nine months ended September 30, 2025, were $29.5 million, an increase of approximately $7.3 million compared to the same period in 2024[148]. Cash and Liquidity - The company has approximately $133.6 million in unrestricted cash and cash equivalents as of September 30, 2025[133]. - Unrestricted cash and cash equivalents and marketable debt securities as of September 30, 2025, were $133.6 million, down from $170.3 million as of December 31, 2024[151]. - Cash used in investing activities for the nine months ended September 30, 2025, was approximately $(112.6) million, a significant increase from $(3.3) million in the same period of 2024[157]. - Other income (expense), net for Q3 2025 was $1.5 million, an increase of approximately $0.4 million compared to Q3 2024, driven by higher returns on invested cash[145]. - Cash provided during the nine months ended September 30, 2025, included $2.5 million from the exercise of the Underwriters' Option from the December 2024 Public Offering[158]. - The cash provided during the nine months ended September 30, 2024, was primarily from $42.0 million in proceeds from the April 2024 Private Placement[158]. Obligations and Contingencies - The company has operating lease obligations for its corporate headquarters and manufacturing facilities located in North America[159]. - Certain contracts require milestone payments upon achieving specific development, regulatory, or commercial milestones, but these are not recorded as liabilities until claims are asserted[162]. - Future payments to third parties are contingent on the achievement of certain milestones and are not included on the balance sheet[163]. - The company currently has no off-balance sheet arrangements as defined by SEC regulations[164].

Financial Performance - Protara reported a net loss of $13.3 million, or $0.31 per share, for Q3 2025, compared to a net loss of $11.2 million, or $0.50 per share, in Q3 2024[11]. - Net income (loss) for Q3 2025 was $(13,258) million, compared to $(11,219) million in Q3 2024, reflecting a decline of 18.2%[22]. - Net income (loss) per share attributable to common stockholders for Q3 2025 was $(0.31), an improvement from $(0.50) in Q3 2024[22]. - Comprehensive income (loss) for the nine months ended September 30, 2025, was $(39,991) million, compared to $(31,767) million for the same period in 2024, indicating a decline of 25.8%[22]. - Total net income (loss) for the nine months ended September 30, 2025, was $(40,132) million, compared to $(31,827) million for the same period in 2024, a decline of 26.0%[22]. Expenses - Research and development expenses increased to $9.6 million in Q3 2025 from $8.1 million in the prior year, primarily due to a $0.9 million increase in startup costs for the IV Choline Chloride THRIVE-3 clinical trial[11]. - General and administrative expenses rose to $5.2 million in Q3 2025 from $4.3 million in the prior year, mainly due to a $0.7 million increase in personnel-related expenses[11]. - Total operating expenses for Q3 2025 were $14,760 million, up from $12,330 million in Q3 2024, representing an increase of 19.7%[22]. - Research and development expenses increased to $9,591 million in Q3 2025 from $8,070 million in Q3 2024, a rise of 18.8%[22]. - General and administrative expenses rose to $5,169 million in Q3 2025 from $4,260 million in Q3 2024, an increase of 21.3%[22]. Assets and Cash Position - As of September 30, 2025, Protara had cash, cash equivalents, and investments totaling approximately $134 million, expected to support operations into mid-2027[4]. - Protara's total assets decreased to $144.6 million as of September 30, 2025, down from $181.5 million at the end of 2024[20]. Clinical Trials and Research - The company anticipates reporting interim results from approximately 25 six-month evaluable NMIBC patients in Q1 2026[5]. - Protara expects to dose the first patient in the THRIVE-3 trial by year-end 2025, which is a seamless Phase 2b/3 trial involving 105 patients[10]. - The Phase 2 STARBORN-1 trial of TARA-002 in pediatric patients is on track to provide an interim update in Q4 2025[4]. - The company continues to evaluate subcutaneous dosing combined with intravesical dosing for TARA-002 in NMIBC patients[5]. Recognition and Culture - Protara was recognized among BioSpace's Best Places to Work in November 2025, highlighting its strong culture and innovation[8]. Shareholder Information - Weighted-average shares outstanding increased to 42,272,104 in Q3 2025 from 22,329,772 in Q3 2024, showing a significant increase in share count[22]. - Interest and investment income for Q3 2025 was $1,502 million, up from $1,111 million in Q3 2024, marking an increase of 35.2%[22]. Other Comprehensive Income - Other comprehensive income for Q3 2025 included a net unrealized gain of $66 million on marketable debt securities, compared to a gain of $29 million in Q3 2024[22].

Core Insights - Protara Therapeutics reported significant advancements in its clinical programs, particularly in the development of TARA-002 for non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) [2][5][10] - The company is on track to report interim results from its ADVANCED-2 trial in NMIBC patients in Q1 2026 and expects to provide updates on its STARBORN-1 trial for pediatric LMs in Q4 2025 [6][7] Clinical Development Updates - TARA-002 is being evaluated in the ADVANCED-2 trial for NMIBC, with interim results expected from approximately 25 BCG-Unresponsive patients in early 2026 [6][7] - The STARBORN-1 trial for TARA-002 in pediatric patients with LMs is progressing, with an interim update anticipated in Q4 2025 [5][6] - The THRIVE-3 trial for IV Choline Chloride is set to dose its first patient by the end of 2025, focusing on patients dependent on parenteral support [6][7] Financial Performance - As of September 30, 2025, Protara had approximately $134 million in cash and investments, expected to fund operations into mid-2027 [6][15] - Research and development expenses increased to $9.6 million in Q3 2025 from $8.1 million in the same period of 2024, primarily due to startup costs for the THRIVE-3 trial [15][23] - The company reported a net loss of $13.3 million, or $0.31 per share, for Q3 2025, compared to a net loss of $11.2 million, or $0.50 per share, in Q3 2024 [15][23] Corporate Recognition - Protara was recognized as one of BioSpace's Best Places to Work in November 2025, highlighting its strong culture and commitment to innovation [8]