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]

Core Insights - Protara Therapeutics, Inc. has been recognized as one of the Best Places to Work by BioSpace for 2026, highlighting its status as a desirable employer in the biotech sector [1][2] - The company is particularly noted for its commitment to fostering a vibrant workplace culture that attracts top talent and inspires employees [2][3] Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases [4] - The company's lead candidate, TARA-002, is an investigational cell-based therapy aimed at treating non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) [4] - Protara is currently conducting a Phase 2 trial for TARA-002 in NMIBC patients who are unresponsive or naïve to Bacillus Calmette-Guérin (BCG) treatment, as well as a Phase 2 trial in pediatric patients with LMs [4] - Additionally, the company is developing IV Choline Chloride, a phospholipid substrate replacement for patients on parenteral nutrition [4] Workplace Environment - Protara is located in the Flatiron District of New York City and emphasizes a modern, collaborative working environment that values integrity, tenacity, and teamwork [3] - The company offers competitive benefits and prioritizes professional development, inclusivity, and work-life balance [3] - The Best Places to Work nominations were based on votes from over 7,500 life sciences professionals, who evaluated companies on various attributes including compensation, innovation, and culture [3]

Core Insights - Protara Therapeutics is a clinical-stage biotech company focused on developing treatments for cancer and rare diseases, with its lead program TARA-002 targeting non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) [2][8] - TARA-002 utilizes a novel approach derived from a genetically distinct cell bank, functioning through dual mechanisms of directly killing tumor cells and stimulating an immune response [1][8] - The company is also advancing IV Choline Chloride for patients requiring parenteral nutrition, addressing a significant unmet need [10] TARA-002 Development - TARA-002 is currently being evaluated in a mid-stage study called ADVANCED-2 for NMIBC, showing promising early results with a 67% complete remission rate in BCG-unresponsive patients at six months [8][12] - In the Phase 2 STARBORN-1 trial for lymphatic malformations, two out of three pediatric patients achieved complete response after one injection, indicating a favorable safety profile [9][12] - Protara plans to deliver interim results for both trials in 1Q26 and 4Q25, respectively [8][9] Market Potential and Analyst Sentiment - Analysts project TARA-002 represents a potential market opportunity exceeding $500 million, with a strong buy consensus rating and a price target suggesting an 840% upside [12][13] - The average price target from analysts for TARA shares is $24.20, indicating a potential upside of approximately 659% [13] Financial Performance of Alpha Teknova - Alpha Teknova reported $10.3 million in revenue for 2Q25, a 7.3% year-over-year increase, with a reaffirmed full-year revenue guidance of $39-42 million [17] - The company is positioned to benefit from growth in the bioprocessing market, with expectations of over 20% revenue growth and 30%+ adjusted EBITDA margins in the long term [19][20] Analyst Ratings for Alpha Teknova - Alpha Teknova holds a strong buy consensus rating, with a price target of $12 indicating a potential one-year upside of approximately 159% [20]

Summary of Protara Therapeutics FY Conference Call Company Overview - **Company**: Protara Therapeutics (NasdaqGM:TARA) - **Date of Conference**: September 09, 2025 Key Industry and Company Insights Lymphatic Malformations - The largest data set on lymphatic malformations was generated by the University of Iowa, involving over 550 patients across 27 centers [1] - Protara is preparing a registration package for FDA submission, leveraging this data set and experience from Japan to seek accelerated approval [1] - TARA-002 is positioned as a treatment for BCG-unresponsive patients, with a focus on its unique mechanism of action compared to BCG [2][3] Mechanism of Action - TARA-002 acts as a TLR2/NOD2 activator, differing significantly from BCG, which is a TLR4 activator [2] - TARA-002 has demonstrated three-year stability at refrigerated temperatures and can be administered easily without special handling [2] - The drug upregulates key cytokines such as TNF-alpha, interferon-gamma, and IL-12, which are important for immune activation, while down-regulating IL-8, associated with recurrence [3][4] Clinical Trials and Data - Protara is currently enrolling patients in an Advanced II study for NMIBC (non-muscle invasive bladder cancer) and plans to publish interim data in February [4] - The company has reported a complete response rate of over 70% at six months and over 40% at twelve months in BCG-naive patients [6] - A significant portion of BCG-eligible patients (approximately 35%) do not receive BCG treatment, presenting an opportunity for TARA-002 [5] Safety and Efficacy - TARA-002 has shown no treatment-related grade 3 adverse events, with common side effects being mild and related to administration [7] - The administration process is straightforward, with efficacy comparable to other products in development [8] IV Choline Chloride Program - Protara is also developing IV Choline Chloride as a therapy for patients on parenteral support, addressing metabolic deficiencies [9][10] - The pivotal study aims to demonstrate elevated choline levels in serum as a primary endpoint, with interim data expected in mid-2026 [10][11] Market Potential and Future Directions - The incidence of lymphatic malformations is estimated at 1 in 2,000 to 1 in 4,000 live births, with a significant portion being macrocystic [15][16] - Protara aims to expand TARA-002's application to other maxillofacial cysts, potentially addressing a larger patient population [17] - The company holds $146 million in cash, sufficient to fund operations through 2027 and multiple catalysts in 2026 [18] Additional Important Points - Protara is actively engaging with the FDA regarding study designs for BCG alternative settings [5] - The company is focused on differentiating its product profile to drive adoption in a competitive market [7] - The ongoing studies and data collection are critical for establishing TARA-002's efficacy and safety in various patient populations [12][14]

Protara Therapeutics (TARA) Conference Call Summary Company Overview - Protara Therapeutics is a development-stage company focused on oncology and rare diseases, with two main franchises: oncology (specifically bladder cancer) and rare diseases [2][3] Oncology Segment TARA-002 Program - TARA-002 is a genetically distinct strain of Streptococcus pyogenes, currently in registrational studies for non-muscle invasive bladder cancer (NMIBC) [2] - The program aims to leverage established biological mechanisms and evolving science to accelerate development and commercialization [2] - Previous data releases showed a 100% complete response rate in a small cohort of BCG-unresponsive carcinoma in situ (CIS) patients, with 80% at six months and over 60% at 12 months [5][6] - Upcoming data release will include 18-month data for both BCG-unresponsive and BCG-naive patients [7][8] Competitive Landscape - Competitive response rates are discussed, with a threshold of 50% complete response at six months to be part of the conversation, and 75% or higher considered a "category killer" [9][10] - Protara aims to differentiate TARA-002 by its ease of administration, similar to BCG, and a favorable safety profile [29][30] Enrollment and Regulatory Pathway - Enrollment is proceeding as expected, with a focus on the right patient population [31] - The company is actively adding international sites to enhance enrollment, particularly in the BCG-unresponsive cohort [31] - Discussions with the FDA regarding the regulatory pathway for BCG-naive patients are ongoing, with updates expected by the end of the year [44] Rare Disease Segment IV Choline Chloride Program - The Thrive 3 trial is a phase 2B3 seamless design study, currently facing enrollment challenges in the U.S. due to competition for hospital beds [46][47] - European sites are expected to enroll significantly more patients, with one site in France having 500 patients on parenteral support [49][50] - Initial data from the Thrive 3 trial is anticipated by the summer of 2026 [51] Key Priorities - The primary focus for the upcoming year is on patient enrollment across both oncology and rare disease programs [52][54] Additional Insights - The management team has extensive experience in biotech and drug development, which informs their strategic approach [3] - Protara's strategy includes a focus on manufacturing capabilities and cost of goods sold, which allows for competitive pricing and distribution flexibility [30]