Prothena(US:PRTA)2025-05-08 20:15Clinical Trials and Investigational Therapies - Prothena's investigational therapeutic birtamimab has shown a statistically significant survival benefit of 74% for Mayo Stage IV AL amyloidosis patients treated with birtamimab plus standard of care compared to 49% for placebo plus standard of care at 9 months [165]. - The Phase 3 AFFIRM-AL clinical trial for birtamimab is expected to enroll up to 220 newly diagnosed, treatment-naïve patients with Mayo Stage IV AL amyloidosis, with topline results anticipated in Q2 2025 [164]. - Prasinezumab, an investigational antibody for Parkinson's disease, demonstrated a potential clinical effect with a hazard ratio of 0.84 in the primary endpoint of time to confirmed motor progression in the Phase 2b PADOVA trial [169]. - In the PADOVA trial, prasinezumab showed a more pronounced effect in the levodopa-treated subgroup with a hazard ratio of 0.79, indicating a significant reduction in motor progression [169]. - Coramitug, designed for the treatment of ATTR amyloidosis, targets non-native TTR to improve organ function and has shown promise in preclinical studies for inhibiting amyloid fibril formation [172][177]. - The Phase 3 VITAL clinical trial results indicated that birtamimab-treated patients had a mean decrease of 0.75 in quality of life compared to a mean decrease of 5.40 for placebo, demonstrating a significant quality of life advantage [166]. - A Phase 2 clinical trial for BMS-986446 is being conducted with approximately 475 participants with early Alzheimer's disease, initiated in Q1 2024 [186]. - PRX123, a dual Aβ-tau vaccine, is in development for the potential treatment and prevention of Alzheimer's disease, with preclinical studies showing promising results [190]. - The Phase 1 clinical trial for PRX019 was initiated in November 2024 to evaluate its safety and pharmacokinetics in healthy adults [195]. Financial Performance - Total revenue for the three months ended March 31, 2025, was $2.8 million, a significant increase from $0.1 million in the same period of 2024, representing a 5,556% growth [200]. - Collaboration revenue for the three months ended March 31, 2025, was $2.8 million, compared to nil for the same period in 2024, attributed to the PRX019 Phase 1 Clinical Trial [201]. - Total operating expenses for the three months ended March 31, 2025, were $81.6 million, a decrease of 16% from $68.4 million in the same period of 2024 [205]. - Research and development expenses decreased by $13.3 million for the three months ended March 31, 2025, primarily due to lower clinical trial expenses related to PRX012 [208]. - R&D expenses totaled $50.8 million for the three months ended March 31, 2025, a decrease of 20.6% from $64.1 million in the same period of 2024 [209]. - Interest income decreased by $2.8 million to $4.3 million, a decline of 39% compared to $7.2 million in the prior year, primarily due to lower yields and cash balances [210]. - The benefit from income taxes decreased by $1.0 million or 46%, from $2.2 million in 2024 to $1.2 million in 2025, mainly due to a decrease in capitalized deferred tax assets [212]. Cash and Liabilities - Working capital decreased by $49.2 million to $387.7 million as of March 31, 2025, primarily due to cash used in operating activities of $53.4 million [214]. - Cash and cash equivalents were $417.9 million as of March 31, 2025, sufficient to meet obligations for at least the next twelve months [215]. - Net cash used in operating activities was $53.4 million for the three months ended March 31, 2025, with total operating expenses of $68.4 million [221]. - Total liabilities were $57.7 million as of March 31, 2025, a slight decrease from $60.2 million as of December 31, 2024 [214]. - Contractual obligations included minimum cash payments under operating leases of $11.3 million and purchase obligations of $15.6 million as of March 31, 2025 [226]. - The company expects full year 2025 net cash used in operating and investing activities to be approximately $168 million to $175 million [229]. Regulatory Designations and Collaborations - The company has received Fast Track Designation and Orphan Drug Designation from the FDA for birtamimab, emphasizing its potential as a critical treatment option for AL amyloidosis [159]. - Prothena's partnered programs include prasinezumab for Parkinson's disease in collaboration with Roche, and BMS-986446 for Alzheimer's disease in collaboration with Bristol Myers Squibb [156]. - The FDA granted Fast Track designation for PRX012 in April 2022, aimed at expediting its development for Alzheimer's disease [189]. - The FDA cleared the IND application for PRX019 in December 2023, with potential milestone payments of up to $617.5 million [194]. Investment and Credit Risk - The company places cash and cash equivalents with high credit quality financial institutions [234]. - The investment policy limits credit exposure with any one financial institution [234]. - Deposits held with banks exceed federally insured limits [234]. - The company has not experienced any losses on deposits of cash and cash equivalents [234]. - Credit risk exposure is recorded on the Condensed Consolidated Balance Sheets [234].