Protara Therapeutics (NasdaqGM:TARA) FY Conference Transcript

Protara Therapeutics Conference Call Summary Company Overview - Company: Protara Therapeutics - Ticker: TARA - Industry: Biotechnology Key Assets and Development Programs TARA-002 - Indications: - Non-muscle invasive bladder cancer (NMIBC) - Lymphatic malformations - Development Stage: Late-stage development for both indications [3][4] Mechanism of Action - TARA-002 is a genetically distinct strain of inactivated Streptococcus pyogenes, engineered to retain its immune-stimulating properties [4] - It has been used in Japan for decades as a standard of care for lymphatic malformations and adjunctively with chemotherapy for various cancers [5] Clinical Trials and Data - ADVANCED-2 Study: - Focuses on BCG-unresponsive NMIBC patients - Reported a 68% complete response rate at 6 months from 22 patients [9] - Preliminary 12-month complete response rate of 33% from 15 evaluable patients [9] - ADVANCED-3 Study: - Aims to utilize TARA-002 in the frontline setting as an alternative to BCG, targeting a significant population due to BCG shortages [6][16] - STARBORN-1 Study: - Focused on macrocystic and mixed cystic lymphatic malformations, with ongoing patient enrollment and upcoming FDA engagement for a registrational path [41] Market Opportunity - Unmet Need: - Approximately 35% of BCG-eligible patients do not receive BCG due to shortages or intolerance, translating to an annual population of 11,000 to 15,000 patients [25][26] - Potentially addressing tens of thousands of patients annually, including those exposed to BCG [27] - Competitive Landscape: - TARA-002 is positioned as a viable alternative in the NMIBC setting, with a focus on ease of use and reliability compared to existing treatments [32] Regulatory Insights - The FDA has indicated that a head-to-head trial against BCG is not necessary, which may facilitate a smoother path to approval [19] Other Programs IV Choline Chloride - Target Population: Approximately 35,000 patients in the U.S. on long-term chronic TPN [56] - Clinical Need: High prevalence of choline deficiency and liver dysfunction in patients on parenteral nutrition [56] - Intellectual Property: Orange Book listed product claim patent extending to 2041, providing exclusivity [62] Financial Considerations - The company is optimistic about pricing strategies based on established benchmarks in the NMIBC setting, allowing flexibility in both NMIBC and lymphatic malformations markets [46] Conclusion - Protara Therapeutics is actively advancing its clinical programs, particularly TARA-002, which addresses significant unmet needs in oncology. The company is well-positioned to capture market share in both NMIBC and lymphatic malformations, with a favorable regulatory outlook and a strong pipeline of assets.