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.

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]