Protara Therapeutics Update Summary Company Overview - **Company**: Protara Therapeutics (NasdaqGM:TARA) - **Focus**: Development of transformative therapies for cancer and rare diseases, with a lead asset TARA-002 targeting non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations. Industry Context - **Industry**: Oncology, specifically treatments for NMIBC. - **Current Landscape**: BCG (Bacillus Calmette-Guérin) is the standard treatment for NMIBC, but there are significant supply issues and limitations in its use, leading to a high demand for alternative therapies. Key Points from the Call TARA-002 Clinical Trial Results - **Trial**: Ongoing Phase 2 open-label ADVANCED-2 trial assessing TARA-002 in NMIBC patients. - **Patient Cohorts**: Focus on BCG naive and BCG unresponsive patients. - **Efficacy Results**: - 50% complete response (CR) rate at 12 months for BCG naive patients [12][14] - 72% CR rate at any time, 69% at six months, and 50% at 12 months for BCG naive patients [13] - 80% of reinduced patients converted to CR at six months, with 100% maintaining CR at 12 months [14] - **Safety Profile**: Favorable safety and tolerability, with most adverse events being grade 1 and transient [15][16] Mechanism of Action - **TARA-002**: A TLR2 NOD2 agonist that engages both innate and adaptive immune pathways, leading to a localized anti-tumor immune response [8][9] - **Comparison to BCG**: TARA-002 has a more favorable safety profile, is easier to administer, and does not require special handling like live bacteria [10][11] Regulatory Pathway - **FDA Engagement**: Protara has received positive feedback from the FDA regarding the registrational path for TARA-002 in BCG naive patients, with intravesical chemotherapy as an acceptable comparator [22][23] - **Trial Design**: The upcoming registrational trial will include fewer than 500 patients, focusing on landmark CR rates at six months and duration of response [23][24] Market Opportunity - **Unmet Need**: Up to 40% of high-grade NMIBC patients do not receive BCG due to supply issues, creating a significant market for alternatives like TARA-002 [22] - **Potential Adoption**: TARA-002's ease of use and favorable safety profile position it as a compelling alternative to BCG, especially in community settings where BCG supply is inconsistent [20][21] Competitive Landscape - **Current Alternatives**: Gemcitabine and other intravesical chemotherapies are used when BCG is unavailable, but they have limitations in terms of administration complexity and safety concerns [46][47] - **Positioning Against BCG**: TARA-002 is viewed as a potential first-line choice due to its similar administration protocol to BCG, which minimizes retraining for healthcare providers [56][58] Future Directions - **Ongoing Trials**: Protara is focused on advancing the development of TARA-002 in both BCG naive and exposed populations, with plans for further studies to explore its efficacy and safety [24][37] - **Community Feedback**: There is a growing enthusiasm among urologists for non-BCG alternatives, particularly given the challenges associated with BCG supply and administration [20][22] Conclusion - Protara Therapeutics is positioned to address significant unmet needs in the NMIBC treatment landscape with TARA-002, which demonstrates promising efficacy and safety profiles. The company is actively engaging with regulatory bodies to advance its clinical programs and capitalize on the growing demand for alternative therapies in the face of BCG supply challenges.

Core Insights - Protara Therapeutics announced updated interim data from the Phase 2 ADVANCED-2 trial of TARA-002 for treating non-muscle invasive bladder cancer (NMIBC) [1][2] - The results indicate a promising potential for TARA-002 in the NMIBC treatment landscape, particularly for Bacillus Calmette-Guérin (BCG)-Naïve patients [2] Updated Interim Results - The dataset includes 31 BCG-Naïve patients, with 29 evaluable for efficacy as of November 7, 2025 [3] - TARA-002 demonstrated a 72% complete response (CR) rate at any time, a 69% CR rate at six months, and a 50% CR rate at 12 months [4][5] - Among initial responders, 88% maintained their response through six months and 100% through 12 months [5] - Re-induction therapy showed high conversion rates, with 80% of re-induced patients achieving CR at six months [5] Safety Profile - The treatment exhibited a favorable safety and tolerability profile, with no Grade 3 or greater treatment-related adverse events reported [4][6] - Common treatment-related adverse events included dysuria (13%), fatigue (13%), and hematuria (6%) [6] Regulatory Update - The company is in dialogue with the FDA regarding the registrational path for TARA-002, with written feedback supporting a controlled trial design for BCG-Naïve patients [7] - The FDA has agreed that BCG is not required as a comparator, and intravesical chemotherapy is acceptable [7] About ADVANCED-2 Trial - ADVANCED-2 is a Phase 2 open-label trial assessing TARA-002 in NMIBC patients with carcinoma in situ or CIS [8] - The trial includes both BCG-Unresponsive and BCG-Naïve cohorts, with ongoing enrollment expected to complete in the second half of 2026 [9] Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing therapies for cancer and rare diseases, with TARA-002 as its lead candidate [14]

Core Insights - Several biotechnology and pharmaceutical companies experienced significant gains in after-hours trading due to corporate updates and upcoming event announcements [1] Company Updates - Jasper Therapeutics, Inc. (JSPR) saw a 21.51% increase to $2.09 after hours, following the announcement of a webinar on December 2 to present findings from the BEACON study and preliminary data from the ETESIAN study [2] - NRx Pharmaceuticals, Inc. (NRXP) rose 13.39% to $2.71 after hours, with the CEO scheduled to present at NobleCon21 on December 3, discussing the company's expanded focus and clinical revenue progress [3] - Unicycive Therapeutics, Inc. (UNCY) increased by 5.42% to $6.33, with the CEO participating in two upcoming events, although no new announcements were made on Monday [4] - Spruce Biosciences, Inc. (SPRB) shares rose 4.68% to $87.00 after reporting a net loss of $8.2 million for the quarter ending September 30, 2025, an improvement from a net loss of $8.7 million in the previous year [5] - Evaxion A/S (EVAX) added 3.96% to $5.65, announcing progress in its CMV vaccine program with new protective data and ongoing preclinical development [6] - Solana Company (HSDT) gained 3.48% to $3.87, reporting a net loss of $352.8 million for the third quarter, a significant increase from a net loss of $3.7 million in the prior-year period, with revenue totaling $697,000 [7] - Protara Therapeutics, Inc. (TARA) rose 5.04% to $7.08, planning a conference call on December 3 to review interim data from its Phase 2 trial of TARA-002 in bladder cancer patients [8]

NEW YORK, Dec. 01, 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 that it will host a conference call and live webcast at 8:30 am ET on Wednesday, December 3, 2025, to review new data from an interim analysis of the ongoing Phase 2 open-label ADVANCED-2 trial of TARA-002 in BCG-Naïve patients with non-muscle invasive bladder cancer (NMIBC). The data will be feature ...

Core Points - The call is hosted by Protara, focusing on updates relevant to investors and corporate affairs [1] Group 1 - The conference is being recorded, and participants are reminded to hold questions until the formal remarks are completed [1] - Justine O'Malley, Senior Vice President, is leading the call and will provide insights into the company's current status [1]

The U.S. Food and Drug Administration on Wednesday approved Bayer's drug for patients with a type of lung cancer. ...

Group 1 - The article does not provide any relevant content regarding company or industry insights [1]

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]