Company Overview - Protara Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases [2] - 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) [2] - Protara is conducting an ongoing Phase 2 trial for TARA-002 in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to Bacillus Calmette-Guérin (BCG) treatment, as well as a Phase 2 trial in pediatric patients with LMs [2] Upcoming Events - Protara management will participate in the H.C. Wainwright & Co. 3 Annual BioConnect Investor Conference with a fireside chat scheduled for May 20, 2025, at 12:30 pm ET in New York [3] - The company will also take part in the TD Cowen 6 Annual Oncology Innovation Summit, which will be held virtually on May 27, 2025, at 1:30 pm ET [3] Additional Information - Protara is also developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral nutrition who cannot meet their choline needs through oral or enteral routes [2] - Live webcasts of the investor conferences will be available on the company's website and archived for a limited time [1]

PART I – FINANCIAL INFORMATION [Item 1. Condensed Consolidated Financial Statements](index=6&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements) Q1 2025 unaudited condensed consolidated financial statements report a $11.9 million net loss, increased cash usage, and decreased total assets [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of March 31, 2025, total assets decreased to $168.6 million, primarily due to a significant reduction in cash and cash equivalents, while liabilities and equity also declined Condensed Consolidated Balance Sheets (in thousands) | | As of March 31, 2025 | As of December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $91,461 | $162,798 | | Total current assets | $126,532 | $172,155 | | **Total assets** | **$168,559** | **$181,454** | | **Liabilities and Stockholders' Equity** | | | | Total current liabilities | $7,018 | $10,961 | | **Total liabilities** | **$10,075** | **$14,320** | | **Total stockholders' equity** | **$158,484** | **$167,134** | | **Total liabilities and stockholders' equity** | **$168,559** | **$181,454** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For Q1 2025, the net loss increased to $11.9 million due to higher operating expenses, primarily from increased research and development activities, resulting in a net loss per share of $(0.29) Condensed Consolidated Statements of Operations (in thousands) | | For the Three Months Ended March 31, | | :--- | :--- | :--- | | | **2025** | **2024** | | 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 | $2,210 | $756 | | **Net income (loss)** | **$(11,914)** | **$(11,095)** | | **Net income (loss) per share, basic and diluted** | **$(0.29)** | **$(0.97)** | | Weighted-average shares outstanding, basic and diluted | 40,707,937 | 11,420,948 | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to $14.7 million in Q1 2025, with a significant shift to cash used in investing activities, resulting in a net decrease of $71.3 million in cash and equivalents Condensed Consolidated Statements of Cash Flows (in thousands) | | For the Three Months Ended March 31, | | :--- | :--- | :--- | | | **2025** | **2024** | | Net cash provided by (used in) operating activities | $(14,713) | $(10,379) | | Net cash provided by (used in) investing activities | $(58,354) | $23,100 | | Net cash provided by (used in) financing activities | $1,730 | $(76) | | **Net increase (decrease) in cash and cash equivalents and restricted cash** | **$(71,337)** | **$12,645** | | Cash and cash equivalents and restricted cash - beginning of period | $163,543 | $40,331 | | **Cash and cash equivalents and restricted cash - end of period** | **$92,206** | **$52,976** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) The notes describe the company as a clinical-stage biopharmaceutical firm with no revenue, detailing recent capital raises and R&D expense drivers, while affirming sufficient financial resources for the next twelve months - The company is a clinical-stage biopharmaceutical company focused on therapies for cancer and rare diseases, including TARA-002 for NMIBC and LMs, and IV Choline Chloride for patients on parenteral support[26](index=26&type=chunk) - The company believes its current financial resources are **sufficient to fund operations for at least twelve months** from the financial statement issuance date[28](index=28&type=chunk) - In April 2024, the company completed a private placement, resulting in net proceeds of **approximately $42.0 million**[68](index=68&type=chunk) - In December 2024, the company closed a public offering with net proceeds of **approximately $93.4 million**. In January 2025, the underwriters partially exercised their option, providing additional net proceeds of **approximately $2.5 million**[79](index=79&type=chunk)[83](index=83&type=chunk) R&D Expenses by Program (in thousands) | Program | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | TARA-002 in NMIBC | $3,557 | $2,789 | | TARA-002 in LMs | $549 | $933 | | IV Choline Chloride | $2,515 | $258 | | Other research and development | $2,335 | $3,394 | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=29&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses clinical program advancements, increased R&D expenses, a $11.9 million net loss, and $157.5 million in cash, deemed sufficient for the next year [Overview and Clinical Program Updates](index=29&type=section&id=Overview%20and%20Clinical%20Program%20Updates) Protara, a clinical-stage biopharmaceutical company, is advancing TARA-002 for NMIBC and LMs, and IV Choline Chloride, with positive interim Phase 2 data and plans for a registrational Phase 3 trial in Q3 2025 - The lead oncology program is TARA-002 for NMIBC. The ADVANCED-2 Phase 2 trial is ongoing, with Cohort B expected to be registrational[117](index=117&type=chunk)[119](index=119&type=chunk) - Interim data from the ADVANCED-2 trial (BCG-Unresponsive cohort) showed a **100% complete response (CR) rate** at six months (5/5 patients) and **67% at 12 months** (2/3 patients)[120](index=120&type=chunk) - The company plans to initiate THRIVE-3, a registrational Phase 3 trial for IV Choline Chloride, in the third quarter of 2025[130](index=130&type=chunk) - The STARBORN-1 Phase 2 trial for TARA-002 in LMs is ongoing, with interim data showing **two of three patients achieved a complete response** after one injection[132](index=132&type=chunk)[133](index=133&type=chunk) [Results of Operations](index=34&type=section&id=Results%20of%20Operations) Total operating expenses increased to $14.1 million in Q1 2025, driven by higher R&D costs for the IV Choline Chloride program and increased G&A expenses, while other income also rose Comparison of Operations (in thousands) | | Q1 2025 | Q1 2024 | Change | | :--- | :--- | :--- | :--- | | Research and development | $9,148 | $7,748 | $1,400 | | General and administrative | $4,976 | $4,103 | $873 | | **Total operating expenses** | **$14,124** | **$11,851** | **$2,273** | | **Net income (loss)** | **$(11,914)** | **$(11,095)** | **$(819)** | - The increase in R&D expenses was primarily due to a **$2.6 million** increase in direct expenses for product candidates, mainly a **$2.3 million** increase for the IV Choline Chloride program[146](index=146&type=chunk) - The increase in G&A expenses was primarily due to a **$0.4 million** increase in personnel-related expenses and a combined **$0.4 million** increase in market development and investor relations activities[147](index=147&type=chunk) [Liquidity and Capital Resources](index=35&type=section&id=Liquidity%20and%20Capital%20Resources) As of March 31, 2025, the company held $157.5 million in unrestricted cash and marketable securities, with $14.7 million used in operations, and believes current resources are sufficient for the next twelve months - As of March 31, 2025, the company had **$157.5 million** in unrestricted cash and cash equivalents and marketable debt securities[149](index=149&type=chunk) - The company believes its current financial resources are **sufficient to satisfy estimated liquidity needs for at least twelve months**[151](index=151&type=chunk) Cash Flow Summary (in thousands) | | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(14,713) | $(10,379) | | Net cash (used in) provided by investing activities | $(58,354) | $23,100 | | Net cash provided by (used in) financing activities | $1,730 | $(76) | - The **$81.5 million** change in investing cash flow was primarily due to a **$65.8 million** increase in purchases of marketable debt securities and a **$15.6 million** decrease in proceeds from maturities[155](index=155&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=39&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section is not applicable to the company for this reporting period - **Not applicable**[164](index=164&type=chunk) [Item 4. Controls and Procedures](index=39&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting during the quarter - Management concluded that as of March 31, 2025, the company's disclosure controls and procedures were **effective at the reasonable assurance level**[166](index=166&type=chunk) - There were **no changes in internal control over financial reporting** during the quarter ended March 31, 2025, that have materially affected, or are reasonably likely to materially affect, internal controls[168](index=168&type=chunk) PART II – OTHER INFORMATION [Item 1. Legal Proceedings](index=40&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any legal proceedings that are expected to have a material adverse effect on its business - The company is **not currently a party to any legal proceedings that management believes would likely have a material adverse effect** on the business[170](index=170&type=chunk) [Item 1A. Risk Factors](index=40&type=section&id=Item%201A.%20Risk%20Factors) There have been no material changes to the risk factors previously disclosed in the company's Annual Report on Form 10-K for the year ended December 31, 2024 - There were **no material changes to the risk factors** previously disclosed in the Annual Report on Form 10-K for the year ended December 31, 2024[171](index=171&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=40&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) During Q1 2025, 1,191 shares of Series 1 Convertible Preferred Stock were converted into 1,190,996 shares of common stock, stemming from a January 2020 private placement - During the three months ended March 31, 2025, **1,191 shares** of Series 1 Convertible Preferred Stock were converted into **1,190,996 shares** of common stock[172](index=172&type=chunk) [Item 5. Other Information](index=40&type=section&id=Item%205.%20Other%20Information) During the first quarter of 2025, no director or officer of the company adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement - **No director or officer adopted or terminated** a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement during the quarter[176](index=176&type=chunk) [Item 6. Exhibits](index=41&type=section&id=Item%206.%20Exhibits) This section provides an index of all exhibits filed as part of the Quarterly Report on Form 10-Q, including corporate governance documents, agreements related to securities, and officer certifications

[Recent Progress and Highlights](index=2&type=section&id=Recent%20Progress%20and%20Highlights) Protara reported significant Q1 2025 clinical progress, including positive interim ADVANCED-2 trial results for TARA-002 in NMIBC and plans to initiate the THRIVE-3 trial for IV Choline Chloride in Q3 2025 [TARA-002 in NMIBC](index=2&type=section&id=TARA-002%20in%20NMIBC) - At the AUA 2025 Annual Meeting, updated interim results from the ADVANCED-2 trial were presented, showing promising complete response (CR) rates for TARA-002[8](index=8&type=chunk) ADVANCED-2 Trial Interim Results (as of April 16, 2025) | Patient Cohort | CR Rate (Any Time) | 12-Month CR Rate | | :--- | :--- | :--- | | BCG-Unresponsive | 100% (5/5) | 67% (2/3) | | BCG-Naïve | 76% (16/21) | 43% (3/7) | - TARA-002 was generally well-tolerated, with the majority of adverse events being Grade 1 and transient, and no Grade 3 or greater treatment-related adverse events reported[8](index=8&type=chunk) - Future milestones include announcing interim results from approximately 25 six-month evaluable BCG-Unresponsive patients by the end of 2025 and providing an update on the planned BCG-Naïve registrational trial design in H2 2025[8](index=8&type=chunk) [IV Choline Chloride for Patients on Parenteral Support (PS)](index=2&type=section&id=IV%20Choline%20Chloride%20for%20Patients%20on%20Parenteral%20Support%20%28PS%29) - The company plans to initiate THRIVE-3, a registrational Phase 3 clinical trial, in the **third quarter of 2025**[6](index=6&type=chunk) - THRIVE-3 is a seamless Phase 2b/3 trial to assess the efficacy and safety of IV Choline Chloride in adolescents and adults on long-term parenteral support[6](index=6&type=chunk) - IV Choline Chloride has previously been granted Fast Track designation by the FDA[6](index=6&type=chunk) [TARA-002 in Lymphatic Malformations (LMs)](index=2&type=section&id=TARA-002%20in%20Lymphatic%20Malformations%20%28LMs%29) - Dosing is progressing in the Phase 2 STARBORN-1 trial for pediatric patients with macrocystic and mixed cystic LMs[7](index=7&type=chunk) - An interim update from the STARBORN-1 trial is planned for the **second half of 2025**[7](index=7&type=chunk) [Corporate Update](index=3&type=section&id=Corporate%20Update) In April 2025, Protara significantly strengthened its leadership team with the strategic appointments of a new Chief Medical Officer and a new Chief People Officer, bringing extensive experience in oncology, drug development, and life sciences organizational growth - Appointed Leonardo Viana Nicacio, M.D., as Chief Medical Officer. Dr. Nicacio has nearly **20 years of experience** in oncology, drug development, and regulatory affairs, previously serving at Stemline Therapeutics[9](index=9&type=chunk) - Appointed Shane Williams, Ph.D., as VP, Head of Human Resources, Chief People Officer. Dr. Williams has a strong background in driving growth and building high-performing teams, most recently at Century Therapeutics[9](index=9&type=chunk) [First Quarter 2025 Financial Results](index=3&type=section&id=First%20Quarter%202025%20Financial%20Results) Protara reported a net loss of **$11.9 million** for Q1 2025, up from **$11.1 million** in Q1 2024 due to increased clinical trial and personnel costs, while maintaining a strong financial position with **$157.5 million** in cash, cash equivalents, and investments, projected to fund operations into 2027 Q1 2025 vs. Q1 2024 Financial Performance (in millions, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Research & Development Expenses | $9.1 | $7.7 | | General & Administrative Expenses | $5.0 | $4.1 | | Net Loss | $11.9 | $11.1 | | Net Loss Per Share | $0.29 | $0.97 | - The increase in R&D expenses was mainly due to a **$2.6 million** rise in clinical trial activities for TARA-002 and IV Choline[13](index=13&type=chunk) - The increase in G&A expenses was primarily driven by higher personnel-related costs and professional fees[13](index=13&type=chunk) [Financial Statements](index=6&type=section&id=Financial%20Statements) This section includes the unaudited condensed consolidated balance sheets as of March 31, 2025, compared to December 31, 2024, and the unaudited condensed consolidated statements of operations for the three-month periods ending March 31, 2025, and 2024 [Unaudited Condensed Consolidated Balance Sheets](index=6&type=section&id=Unaudited%20Condensed%20Consolidated%20Balance%20Sheets) Balance Sheet Summary (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $91,461 | $162,798 | | Total current assets | $126,532 | $172,155 | | Total assets | $168,559 | $181,454 | | Total current liabilities | $7,018 | $10,961 | | Total liabilities | $10,075 | $14,320 | | Total stockholders' equity | $158,484 | $167,134 | [Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Unaudited%20Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations Summary (in thousands, except per share data) | Account | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Total operating expenses | $14,124 | $11,851 | | Income (Loss) from operations | $(14,124) | $(11,851) | | Net income (loss) | $(11,914) | $(11,095) | | Net income (loss) per share | $(0.29) | $(0.97) | | Weighted-average shares outstanding | 40,707,937 | 11,420,948 |

Core Insights - Protara Therapeutics reported significant progress in 2025, particularly with positive interim results from the ADVANCED-2 trial of TARA-002 for non-muscle invasive bladder cancer (NMIBC) [2][5] - The company plans to initiate the THRIVE-3 Phase 3 clinical trial for IV Choline Chloride in Q3 2025, targeting patients on parenteral support [4][5] - Protara's financial results for Q1 2025 show a net loss of $11.9 million, with cash reserves expected to support operations into 2027 [14][24] Recent Progress and Highlights - TARA-002 demonstrated a complete response (CR) rate of 100% in BCG-Unresponsive patients and 76% in BCG-Naïve patients during the ADVANCED-2 trial [6] - The company expects to announce interim results from approximately 25 six-month evaluable BCG-Unresponsive patients by the end of 2025 [5][6] - Leadership team strengthened with key appointments, including Dr. Leonardo Viana Nicacio as Chief Medical Officer [9] Financial Overview - As of March 31, 2025, Protara had cash, cash equivalents, and investments totaling $158 million, sufficient to fund operations into 2027 [5][14] - Research and development expenses increased to $9.1 million in Q1 2025 from $7.7 million in the prior year, primarily due to clinical trial activities [14][24] - General and administrative expenses rose to $5.0 million in Q1 2025 from $4.1 million in the prior year [14][24] Product Development - TARA-002 is being evaluated for NMIBC and lymphatic malformations (LMs), with ongoing trials expected to yield interim updates in the second half of 2025 [8][11] - IV Choline Chloride is positioned as a potential first FDA-approved IV choline formulation for patients on parenteral nutrition [16] - The company continues to explore combination treatments and dosing strategies for TARA-002 in NMIBC patients [6][12]

TARA-002 Product Characteristics - TARA-002 is a lyophilized, inactivated Group A Streptococcus pyogenes [8] - TARA-002 is an investigational, genetically distinct strain of Streptococcus pyogenes that is inactivated while retaining its immune-stimulating properties [10] - TARA-002 activates Th1 Immune Cascade [12] Clinical Trial Results in BCG-Unresponsive Patients - TARA-002 monotherapy demonstrates a 100% complete response rate at any time in BCG-unresponsive participants [14] - Specifically, 5 out of 5 (100%) BCG-unresponsive patients with CIS only achieved a high-grade complete response at any time [15] - At Month 6, 5 out of 5 (100%) BCG-unresponsive patients with CIS only achieved a high-grade complete response [15] - At Month 12, 2 out of 3 (66.7%) BCG-unresponsive patients with CIS only achieved a high-grade complete response [15] Clinical Trial Results in BCG-Naïve Patients - TARA-002 monotherapy demonstrates a 76.2% complete response rate at any time in BCG-naïve participants [22, 23] - Specifically, 10 out of 11 (90.9%) BCG-naïve patients with CIS only achieved a high-grade complete response at any time [23] - At Month 6, 5 out of 6 (83.3%) BCG-naïve patients with CIS only achieved a high-grade complete response [23] - At Month 12, 3 out of 5 (60.0%) BCG-naïve patients with CIS only achieved a high-grade complete response [23] Safety Profile - Across both BCG-unresponsive and BCG-naïve patients (N=43), 74% experienced any grade of treatment-emergent adverse events (TEAEs) [25] - 30% of participants experienced related TEAEs [25] - No Grade 4/5 treatment-related adverse events were observed [7, 25]

Core Insights - Protara Therapeutics, Inc. has granted inducement non-qualified stock options and restricted stock units (RSUs) to six newly-hired employees as part of its 2020 Inducement Plan [1][2][3] Employee Compensation - Shane Williams, the new Vice President, Head of Human Resources and Chief People Officer, received 41,000 stock option awards and 20,500 RSU awards with an exercise price of $4.32 per share on April 25, 2025 [2] - An aggregate of 59,100 stock option awards and 29,550 RSU awards were granted to five new employees on May 1, 2025, with an exercise price of $3.46 per share [3] Vesting Schedule - All option awards vest over four years, with 25% vesting on the one-year anniversary and the remainder vesting monthly thereafter [4] - RSU awards vest over three years, with 33 1/3% vesting on each of the three consecutive anniversaries of the grant date [4] Company Overview - Protara is a clinical-stage biotechnology company focused on developing therapies for cancer and rare diseases, including its lead candidate TARA-002 for non-muscle invasive bladder cancer and lymphatic malformations [5] - The company is conducting ongoing Phase 2 trials for TARA-002 in patients with carcinoma in situ and pediatric patients with lymphatic malformations [5] - Protara is also developing IV Choline Chloride for patients on parenteral nutrition who cannot meet their choline needs through oral or enteral routes [5]

Summary of Protara Therapeutics (TARA) Update / Briefing April 28, 2025 Company Overview - **Company**: Protara Therapeutics - **Focus**: Development of TARA-two, an investigational treatment for non-muscle invasive bladder cancer (NMIBC) Key Industry Insights - **Clinical Trial**: Ongoing Phase II ADVANCE-two clinical trial of TARA-two in NMIBC patients - **Treatment Landscape**: TARA-two is positioned as a competitive option in the evolving NMIBC treatment landscape, particularly for BCG unresponsive patients Core Findings and Arguments 1. **Positive Interim Results**: TARA-two demonstrated a complete response (CR) rate of 100% at any time and 100% at six months in the BCG unresponsive cohort, with rates of 80% at nine months and 67% at twelve months [12][15] 2. **Durable Responses**: The treatment shows durable responses at twelve months in both BCG unresponsive and naive cohorts, with 76% CR at any time in the naive cohort [15] 3. **Safety Profile**: TARA-two exhibited a favorable safety profile, with most adverse events being grade one and transient, and no patients discontinued due to adverse events [16] 4. **Mechanism of Action**: TARA-two activates a broader immunologic profile compared to other therapies, potentially leading to durable antitumor responses [9][14] 5. **Regulatory Engagement**: The company is in discussions with the FDA regarding the design of registrational studies for both BCG unresponsive and naive patient populations [15][81] Additional Important Insights 1. **Patient Enrollment**: The company has seen an increase in trial enrollment due to site expansion efforts, with 17 patients currently enrolled [12][28] 2. **Future Milestones**: Protara anticipates additional data updates and milestones throughout the year, including a preplanned analysis of 25 BCG unresponsive patients by the end of 2025 [19][29] 3. **Combination Therapy Potential**: The company is exploring systemic priming and combination therapies to enhance treatment efficacy and patient quality of life [45][47] 4. **Market Positioning**: TARA-two is viewed as a potential best-in-class treatment for NMIBC, with characteristics that drive adoption among healthcare providers [32][33] Conclusion - Protara Therapeutics is making significant strides in the development of TARA-two for NMIBC, with promising interim results and a strong safety profile. The company is well-positioned to capitalize on upcoming milestones and regulatory discussions, potentially transforming the treatment landscape for patients with NMIBC.

Core Insights - Protara Therapeutics, Inc. announced updated results from its Phase 2 ADVANCED-2 trial for TARA-002, a cell-based therapy for high-risk Non-Muscle Invasive Bladder Cancer (NMIBC) patients, which will be presented at the American Urological Association 2025 Annual Meeting [1][2] Interim Results - The BCG-Unresponsive cohort included five patients, all evaluable at six and nine months, with three evaluable at 12 months as of April 16, 2025 [3] - The BCG-Naïve cohort included 21 patients, with 16 evaluable at six months, eight at nine months, and seven at 12 months as of April 16, 2025 [4] Safety Profile - Most adverse events were Grade 1 and transient, with no Grade 3 or greater treatment-related adverse events reported. Common adverse events included flu-like symptoms and urinary tract issues, which typically resolved shortly after administration [5] Efficacy Data - TARA-002 demonstrated a 100% complete response (CR) rate at any time in BCG-Unresponsive patients, with 67% CR rate at 12 months. In BCG-Naïve patients, the CR rate was 76% at any time and 43% at 12 months [6][7] Future Plans - The company is on track to present updated interim data from approximately 25 six-month evaluable BCG-Unresponsive patients by the end of 2025 [6][8] - A conference call and webcast to discuss the data will be held on April 28, 2025 [9] About the Trial - The ADVANCED-2 trial is assessing TARA-002 in NMIBC patients with carcinoma in situ who are BCG-Unresponsive or BCG-Naïve, with a total of approximately 100 patients in the BCG-Unresponsive cohort [10] About TARA-002 - TARA-002 is an investigational cell therapy for NMIBC and has received Rare Pediatric Disease Designation from the FDA. It is developed from a master cell bank of genetically distinct group A Streptococcus pyogenes [11] About NMIBC - NMIBC accounts for approximately 80% of bladder cancer diagnoses in the U.S., with around 65,000 new cases diagnosed annually [13] About Protara Therapeutics - Protara is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases, including TARA-002 for NMIBC and lymphatic malformations [14]

Core Viewpoint - Protara Therapeutics is set to present updated safety and efficacy data from the Phase 2 ADVANCED-2 trial of TARA-002 for non-muscle invasive bladder cancer (NMIBC) on April 28, 2025, with data showcased at the American Urological Association 2025 Annual Meeting on April 26, 2025 [1][2] Group 1: Company Overview - Protara Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases [7] - The company's lead candidate, TARA-002, is an investigational cell-based therapy aimed at treating NMIBC and lymphatic malformations (LMs) [7] - Protara is also developing IV Choline Chloride for patients on parenteral nutrition [7] Group 2: Clinical Trial Details - The ADVANCED-2 trial (NCT05951179) is a Phase 2 open-label study assessing TARA-002 in NMIBC patients who are BCG-unresponsive or BCG-naïve [3] - The trial includes approximately 100 BCG-unresponsive patients and 31 BCG-naïve patients, with the BCG-unresponsive cohort designed to be registrational [3] Group 3: Product Information - TARA-002 is derived from a master cell bank of genetically distinct group A Streptococcus pyogenes, similar to OK-432, which is marketed in Japan [4] - The therapy is hypothesized to activate immune cells and induce a pro-inflammatory response, enhancing the antitumor immune response [5] Group 4: Disease Context - NMIBC accounts for about 80% of bladder cancer diagnoses, with approximately 65,000 new cases diagnosed annually in the United States [6]

Core Viewpoint - Protara Therapeutics has appointed Dr. Leonardo Viana Nicacio as Chief Medical Officer, bringing nearly 20 years of experience in oncology and drug development to the company [1][2]. 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 an investigational cell-based therapy for non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) [5]. - Protara is 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 [5]. Leadership Appointment - Dr. Nicacio previously served as Head of Clinical Development and Global Medical Affairs at Stemline Therapeutics, where he established global solid tumor development strategies [2]. - His experience includes significant roles at Seagen, AstraZeneca, Flatiron Health, Sanofi, and YM Biosciences, focusing on oncology and drug development [3]. - Dr. Nicacio holds a Medical Degree from Faculdade de Ciencias Medicas de Minas Gerais and is board-certified in internal medicine and medical oncology [4]. Future Prospects - Dr. Nicacio expressed optimism about Protara's pipeline, highlighting the potential to impact patients with underserved cancers and rare diseases [4]. - Important milestones are anticipated, including the presentation of interim data from the ADVANCED-2 clinical trial of TARA-002 in NMIBC patients later this month [4]. Compensation Details - As part of his employment, Dr. Nicacio received an inducement grant of options to purchase 150,000 shares at an exercise price of $4.17, along with a restricted stock unit (RSU) award for 50,000 shares [6][7].