NEW YORK, June 02, 2025 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced the appointment of William “Bill” Conkling as Chief Commercial Officer. He brings more than two decades of experience developing and commercializing novel cancer and rare disease therapeutics to Protara. “It is a pleasure to welcome Bill, a deeply experienced and proven commercial leader,” said Jesse ...

Protara Therapeutics (TARA) FY Conference Summary Company Overview - **Company**: Protara Therapeutics - **Focus**: Development of innovative therapies for oncology, specifically targeting non-muscle invasive bladder cancer (NMIBC) with their lead program, TAR-002 Key Points and Arguments Product Development and Mechanism of Action - **TAR-002**: An investigational, genetically distinct strain of *Streptococcus pyogenes* designed to retain immune-stimulating properties through a proprietary manufacturing process [3][4] - **Comparison with BCG**: - Both TAR-002 and BCG are bacterial immune potentiators that drive a TH1 pro-inflammatory response, critical for antitumor immunity [5] - TAR-002 acts as a TLR2 agonist, while BCG is a TLR4 agonist, leading to significantly stronger tumor cell killing and higher upregulation of pro-inflammatory cytokines like TNF-alpha and interferon-gamma [6] - TAR-002 downregulates IL-8, which is associated with bladder cancer recurrence, contrasting with BCG's upregulation of IL-8 [7] Clinical Data and Efficacy Expectations - **Durable Responses**: Nonclinical studies show around 70% of mice remain cancer-free at the 60-day mark, indicating potential for durable clinical responses [7] - **Efficacy Benchmarks**: - A competitive complete response rate (CRR) of 30% is needed to be considered viable, with 40% being competitive and 50% considered best-in-class [12][13] - Initial data suggests a CRR of 63% at six months and 43% at twelve months in BCG-naive patients, with a total CRR of 76% [41] Enrollment and Market Strategy - **Enrollment Progress**: Enrollment is progressing well, with 30 sites in the US and approvals in Japan and China, aiming for full enrollment by spring 2026 [19][20] - **Patient Population**: The study is expected to have a similar split of 75% CIS and 25% concomitant papillary patients, consistent with other studies in this patient population [23][22] Competitive Landscape and Market Opportunity - **Market Size**: The addressable market is estimated at around $5 billion, with a significant number of patients seeking to avoid cystectomy [26] - **Treatment Sequencing**: Future treatment strategies may involve sequencing immune potentiators and cytotoxic drugs, enhancing the potential for TAR-002 in the treatment landscape [26] Regulatory and Development Plans - **FDA Engagement**: Plans to engage with the FDA regarding registrational studies for TAR-002, particularly in the BCG-naive setting, acknowledging the need for alternative treatment options [43][44] - **Comparative Studies**: Proposals to use chemotherapy as a comparator in studies, reflecting real-world treatment practices [46] Upcoming Catalysts - **Key Data Releases**: Anticipated data on 25 BCG unresponsive patients by the end of the year, along with updates on other ongoing studies [50][51] Additional Important Insights - **Tolerability and Administration**: TAR-002 is noted for its favorable tolerability profile, which may facilitate its adoption in community settings where treatment disruptions are less feasible [37][38] - **Long-term Development**: The company aims to position TAR-002 as a viable alternative to BCG, especially for patients who cannot access or tolerate standard BCG therapy [42][44] This summary encapsulates the critical insights from the Protara Therapeutics conference, highlighting the company's strategic direction, product differentiation, and market potential in the oncology space.

Financial Data and Key Metrics Changes - The company reported a 67% complete response (CR) rate in the pivotal BCG unresponsive cohort as of April, indicating strong performance in this segment [13][21]. - The target for the unresponsive dataset is a CR rate in the sixties, potentially reaching the seventies, which would be competitive with existing therapies [21]. Business Line Data and Key Metrics Changes - The lead asset, Tara 002, is positioned as a broad-spectrum immunopotentiator, with a focus on muscle invasive bladder cancer (MIBC) and non-muscle invasive bladder cancer (NMIBC) [10][12]. - The company is in late-stage development across three programs, including Tara 002 and IV choline chloride phospholipid substrate replacement therapy, indicating a diversified pipeline [9]. Market Data and Key Metrics Changes - The unmet need in NMIBC is characterized by a strong motivation to reduce cystectomies, with the company aiming to address this through innovative treatment options [10][11]. - The competitive landscape includes various mechanisms of action, with the company’s product being the only broad-spectrum immunopotentiator in late-stage development [12]. Company Strategy and Development Direction - The company aims to redefine risk in its portfolio by understanding and containing risks associated with each asset, particularly in the context of evolving regulatory and manufacturing landscapes [5][9]. - The strategy includes engaging with the FDA to demonstrate that standard of care for BCG eligible patients extends beyond just BCG, incorporating other chemotherapeutics [24][26]. Management's Comments on Operating Environment and Future Outlook - Management highlighted the importance of real-world evidence in shaping trial designs and regulatory interactions, particularly in the context of BCG shortages [24][26]. - The company anticipates that as the dataset broadens, fewer patients will require reinduction, which could enhance the overall treatment profile [30]. Other Important Information - The company is conducting a single-arm open-label study for Tara 002 in pediatric nonmalignant diseases, which has garnered interest due to its potential and existing standard of care in Japan [9]. - The company emphasizes the importance of tolerability and ease of use in community settings, which could drive adoption among urologists [36][38]. Q&A Session Summary Question: What is the unmet need in NMIBC and how does Tara 002 address it? - The unmet need is categorized into three buckets, with a focus on reducing cystectomies and addressing the heterogeneous nature of the disease through various treatment modalities [10][11]. Question: What benchmarks should be set for the six-month data in the BCG unresponsive cohort? - The company targets a CR rate of around 75% in CIS only patients, with expectations that the overall CR rate will be in the sixties or seventies [21]. Question: How does the company plan to approach the FDA regarding registrational studies? - The company plans to present real-world evidence to the FDA, arguing that the standard of care includes both BCG and other chemotherapeutics due to BCG shortages [24][26]. Question: What are the key differentiating factors for Tara 002 in the NMIBC space? - Key differentiators include the drug's tolerability, safety profile, and the ability to be integrated into existing workflows without causing disruptions [36][38].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-36694 10010 (Zip Code) (646) 844-0337 (Registrant's telephone number, including area code) Securities registered pursuant to Sect ...

Exhibit 99.1 Protara Therapeutics Announces First Quarter 2025 Financial Results and Provides Business Update NEW YORK, May 8, 2025 – 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 first quarter ended March 31, 2025, and provided a business update. "We have made significant progress thus far in 2025, notably with the recent presentation of positive interim results ...

Reported positive interim results demonstrating durable responses in the ongoing Phase 2 ADVANCED-2 trial of TARA-002 in NMIBC Results from planned interim analysis of approximately 25 six-month evaluable BCG-Unresponsive patients expected by the end of 2025 Dosing of first patient in THRIVE-3 registrational trial of IV Choline Chloride in patients dependent on parenteral support expected in Q3 2025Strengthened leadership team with key appointments of Leonardo Viana Nicacio, M.D., as Chief Medical Officer, ...

Interim Data Presentation from the American Urological Association Annual Meeting April 2025 Forward Looking Statements Statements contained in this presentation 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," ...

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]